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{ "eu_designation_number": "EU/3/01/038", "medicine_name": "", "active_substance": "retroviral gamma c cDNA containing vector", "intended_use": "Treatment of severe combined immunodeficiency (SCID)-Xl Disease", "status": "Positive", "designation_date": "2001-05-30", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-01-038", "last_updated": "2009-05-15" }, { "eu_designation_number": "EU/3/01/039", "medicine_name": "", "active_substance": "Ecteinascidin 743 (trabectedin)", "intended_use": "Treatment of soft tissue sarcoma", "status": "Expired", "designation_date": "2001-05-30", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-01-039", "last_updated": "2017-10-03" }, { "eu_designation_number": "EU/3/01/040", "medicine_name": "", "active_substance": "Fomepizole", "intended_use": "Treatment of methanol poisoning", "status": "Withdrawn", "designation_date": 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"medicine_name": "", "active_substance": "muramyl tripeptide phosphatidyl ethanolamine", "intended_use": "Treatment of osteosarcoma", "status": "Expired", "designation_date": "2004-06-21", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-04-206", "last_updated": "2019-07-11" }, { "eu_designation_number": "EU/3/04/207", "medicine_name": "", "active_substance": "sorafenib tosilate", "intended_use": "Treatment of renal-cell carcinoma", "status": "Expired", "designation_date": "2004-07-29", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-04-207", "last_updated": "2016-08-10" }, { "eu_designation_number": "EU/3/04/208", "medicine_name": "", "active_substance": "acetylsalicylic acid", "intended_use": "Treatment of polycythaemia vera", "status": "Withdrawn", "designation_date": "2004-07-29", "related_ema_product_number": "", "url": 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"last_updated": "2020-10-23" }, { "eu_designation_number": "EU/3/04/214", "medicine_name": "Rydapt", "active_substance": "Midostaurin", "intended_use": "Treatment of acute myeloid leukaemia", "status": "Positive", "designation_date": "2004-07-29", "related_ema_product_number": "EMEA/H/C/004095", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-04-214", "last_updated": "2018-05-29" }, { "eu_designation_number": "EU/3/04/215", "medicine_name": "", "active_substance": "porfimer sodium", "intended_use": "Treatment of cholangiocarcinoma", "status": "Withdrawn", "designation_date": "2004-07-29", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-04-215", "last_updated": "2013-04-09" }, { "eu_designation_number": "EU/3/04/216", "medicine_name": "Surfaxin; Surfaxin", "active_substance": "sinapultide;Dipalmitoylphosphatidylcholine;palmitoyloleoyl phosphatidylglycerol;palmitic acid", "intended_use": 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"eu_designation_number": "EU/3/04/223", "medicine_name": "", "active_substance": "heparin sodium", "intended_use": "Treatment of idiopathic pulmonary fibrosis", "status": "Withdrawn", "designation_date": "2004-09-02", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-04-223", "last_updated": "2017-02-10" }, { "eu_designation_number": "EU/3/04/224", "medicine_name": "", "active_substance": "Homoharringtonine", "intended_use": "Treatment of chronic myeloid leukaemia", "status": "Withdrawn", "designation_date": "2004-09-02", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-04-224", "last_updated": "2016-08-10" }, { "eu_designation_number": "EU/3/04/225", "medicine_name": "", "active_substance": "sodium dichloroacetate", "intended_use": "Treatment of systemic monochloroacetate poisoning", "status": "Withdrawn", "designation_date": "2004-09-02", 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"eu_designation_number": "EU/3/04/254", "medicine_name": "", "active_substance": "Val-Leu-Gln-Glu-Leu-Asn-Val-Thr-Val (Pr1 nanopeptide, sequence 169-177, of proteinase 3)", "intended_use": "Treatment of myelodysplastic syndromes", "status": "Withdrawn", "designation_date": "2004-12-21", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-04-254", "last_updated": "2006-01-06" }, { "eu_designation_number": "EU/3/04/255", "medicine_name": "", "active_substance": "sabarubicin", "intended_use": "Treatment of small cell lung cancer", "status": "Withdrawn", "designation_date": "2004-12-21", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-04-255", "last_updated": "2009-10-15" }, { "eu_designation_number": "EU/3/04/256", "medicine_name": "", "active_substance": "17-allylamino-17-demethoxygeldanamycin", "intended_use": "Treatment of chronic myeloid leukaemia", "status": 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"medicine_name": "Nurzigma", "active_substance": "4-[3-(Methylsulfonyl)phenyl]-1-propylpiperidine x HC1 (pridopidine hydrochloride)", "intended_use": "Treatment of Huntington’s disease", "status": "Positive", "designation_date": "2005-06-20", "related_ema_product_number": "EMEA/H/C/006261", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-05-288", "last_updated": "2026-05-11" }, { "eu_designation_number": "EU/3/05/289", "medicine_name": "", "active_substance": "pegylated arginine deiminase", "intended_use": "Treatment of hepatocellular carcinoma", "status": "Positive", "designation_date": "2005-06-20", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-05-289", "last_updated": "2005-07-26" }, { "eu_designation_number": "EU/3/05/290", "medicine_name": "", "active_substance": "Humanised antibody fragment (EP-CAM)-truncated Pseudomonas exotoxin A fusion protein", "intended_use": "Treatment of 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"active_substance": "adeno-associated viral vector containing a modified U7 snRNA gene", "intended_use": "Treatment of Duchenne muscular dystrophy", "status": "Withdrawn", "designation_date": "2005-07-27", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-05-297", "last_updated": "2025-09-04" }, { "eu_designation_number": "EU/3/05/298", "medicine_name": "", "active_substance": "mifepristone", "intended_use": "Treatment of Cushing's syndrome secondary to ectopic ACTH secretion", "status": "Withdrawn", "designation_date": "2005-07-27", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-05-298", "last_updated": "2017-02-10" }, { "eu_designation_number": "EU/3/05/299", "medicine_name": "", "active_substance": "4-Imino-1, 3-diazobicyclo-[3.1.0]-hexan-2-one", "intended_use": "Treatment of pancreatic cancer", "status": "Positive", "designation_date": "2005-07-27", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-05-299", "last_updated": "2005-11-29" }, { "eu_designation_number": "EU/3/05/300", "medicine_name": "", "active_substance": "nemorubicin hydrochloride", "intended_use": "Treatment of hepatocellular carcinoma", "status": "Positive", "designation_date": "2005-07-27", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-05-300", "last_updated": "2005-10-27" }, { "eu_designation_number": "EU/3/05/301", "medicine_name": "", "active_substance": "chimeric monoclonal antibody to shiga toxin 1 and 2", "intended_use": "Treatment of shiga-toxin-producing bacterial infection", "status": "Positive", "designation_date": "2005-08-26", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-05-301", "last_updated": "2019-04-12" }, { "eu_designation_number": "EU/3/05/302", "medicine_name": "", "active_substance": "Extract of Sorghum bicolour leaf, Pterocarpus osun stem, Piper guineense seed and Caryophylli flower", "intended_use": "Treatment of sickle cell disease", "status": "Positive", "designation_date": "2005-08-26", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-05-302", "last_updated": "2005-10-27" }, { "eu_designation_number": "EU/3/05/303", "medicine_name": "", "active_substance": "human autologous mesenchymal adult stem cells extracted from adipose tissue", "intended_use": "Treatment of anal fistula", "status": "Withdrawn", "designation_date": "2005-08-26", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-05-303", "last_updated": "2022-07-01" }, { "eu_designation_number": "EU/3/05/304", "medicine_name": "", "active_substance": "imatinib mesilate", "intended_use": "Treatment of acute lymphoblastic leukaemia", "status": 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"active_substance": "Mecasermin", "intended_use": "Treatment of primary growth hormone insensitivity syndrome", "status": "Positive", "designation_date": "2005-08-26", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-05-307", "last_updated": "2007-07-04" }, { "eu_designation_number": "EU/3/05/308", "medicine_name": "", "active_substance": "sapropterin", "intended_use": "Treatment of hyperphenylalaninaemia", "status": "Withdrawn", "designation_date": "2005-08-26", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-05-308", "last_updated": "2011-03-29" }, { "eu_designation_number": "EU/3/05/309", "medicine_name": "", "active_substance": "sodium valproate", "intended_use": "Treatment of 5q spinal muscular atrophy", "status": "Withdrawn", "designation_date": "2005-08-26", "related_ema_product_number": "", "url": 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"active_substance": "2-{4-[(5,6-Diphenylpyrazin-2-yl)(isopropyl)amino]butoxy}-N-(methylsulfonyl)acetamide", "intended_use": "Treatment of pulmonary arterial hypertension and chronic thromboembolic pulmonary hypertension", "status": "Withdrawn", "designation_date": "2005-08-26", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-05-316", "last_updated": "2018-02-12" }, { "eu_designation_number": "EU/3/05/317", "medicine_name": "", "active_substance": "anti-CD3 mAb (SPV-T3a)-ricin A chain fusion protein;anti-CD7 mAb (WT1)-ricin A chain fusion protein", "intended_use": "Treatment of graft-versus-host disease", "status": "Positive", "designation_date": "2005-08-26", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-05-317", "last_updated": "2024-04-11" }, { "eu_designation_number": "EU/3/05/318", "medicine_name": "", "active_substance": "recombinant modified vaccinia 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"eu_designation_number": "EU/3/06/351", "medicine_name": "", "active_substance": "miglustat", "intended_use": "Treatment of Niemann-Pick's disease, type C", "status": "Expired", "designation_date": "2006-02-16", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-06-351", "last_updated": "2019-02-20" }, { "eu_designation_number": "EU/3/06/352", "medicine_name": "", "active_substance": "26 base single stranded phosphodiester DNA oligonucleotide", "intended_use": "Treatment of pancreatic cancer", "status": "Withdrawn", "designation_date": "2006-02-16", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-06-352", "last_updated": "2011-05-18" }, { "eu_designation_number": "EU/3/06/353", "medicine_name": "", "active_substance": "26 base single stranded phosphodiester DNA oligonucleotide", "intended_use": "Treatment of renal-cell carcinoma", "status": "Withdrawn", 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"https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-07-475", "last_updated": "2009-09-17" }, { "eu_designation_number": "EU/3/07/476", "medicine_name": "", "active_substance": "5'-O-(trans-9\"-octadecenoyl)-1-ß-D-arabinofuranosyl cytosine", "intended_use": "Treatment of acute myeloid leukaemia", "status": "Positive", "designation_date": "2007-09-14", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-07-476", "last_updated": "2014-05-08" }, { "eu_designation_number": "EU/3/07/477", "medicine_name": "", "active_substance": "4-Amino-1-[5-O-[(2R,4S)-2-oxido-4-(4-pyridinyl)-1,3,2-dioxaphosphorinan-2-yl]-ß-D-arabinofuranosyl]-2(1H)-pyrimidinone", "intended_use": "Treatment of hepatocellular carcinoma", "status": "Positive", "designation_date": "2007-09-14", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-07-477", "last_updated": "2008-01-22" }, { "eu_designation_number": "EU/3/07/478", "medicine_name": "", "active_substance": "N-(2-amino-phenyl)-4-[(4-pyridin-3-yl-pyrimidin-2-ylamino)-methyl] benzamide (mocetinostat)", "intended_use": "Treatment of Hodgkin's lymphoma", "status": "Withdrawn", "designation_date": "2007-09-14", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-07-478", "last_updated": "2022-05-10" }, { "eu_designation_number": "EU/3/07/479", "medicine_name": "", "active_substance": "N-adamantanyl-N'-Geranyl-ethylenediamine", "intended_use": "Treatment of tuberculosis", "status": "Positive", "designation_date": "2007-09-14", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-07-479", "last_updated": "2008-07-29" }, { "eu_designation_number": "EU/3/07/480", "medicine_name": "", "active_substance": "naptumomab estafenatox", "intended_use": "Treatment of renal-cell carcinoma", "status": "Positive", "designation_date": "2007-09-14", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-07-480", "last_updated": "2011-11-11" }, { "eu_designation_number": "EU/3/07/481", "medicine_name": "", "active_substance": "R-salbutamol sulfate", "intended_use": "Treatment of cutaneus forms of lupus erythromatosus", "status": "Positive", "designation_date": "2007-09-14", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-07-481", "last_updated": "2008-07-02" }, { "eu_designation_number": "EU/3/07/482", "medicine_name": "", "active_substance": "sulfonated monophosphorylated mannose oligosaccharide", "intended_use": "Treatment of hepatocellular carcinoma", "status": "Withdrawn", "designation_date": "2007-09-14", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-07-482", "last_updated": "2009-10-15" }, { "eu_designation_number": "EU/3/07/483", "medicine_name": "", "active_substance": "amonafide L-malate", "intended_use": "Treatment of acute myeloid leukaemia", "status": "Withdrawn", "designation_date": "2007-10-22", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-07-483", "last_updated": "2011-05-18" }, { "eu_designation_number": "EU/3/07/484", "medicine_name": "", "active_substance": "adenovirus associated viral vector serotype 4 containing the human RPE65 gene", "intended_use": "Treatment of Leber's congenital amaurosis", "status": "Positive", "designation_date": "2007-10-22", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-07-484", "last_updated": "2022-08-09" }, { "eu_designation_number": "EU/3/07/485", "medicine_name": "", "active_substance": "alvocidib", "intended_use": "Treatment of chronic lymphocytic leukaemia", "status": "Positive", "designation_date": "2007-10-23", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-07-485", "last_updated": "2022-03-07" }, { "eu_designation_number": "EU/3/07/486", "medicine_name": "", "active_substance": "adenovirus associated viral vector serotype 4 containing the human RPE65 gene", "intended_use": "Treatment of retinitis pigmentosa", "status": "Positive", "designation_date": "2007-11-14", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-07-486", "last_updated": "2022-08-09" }, { "eu_designation_number": "EU/3/07/487", "medicine_name": "", "active_substance": "4-Ethoxy-2-(piperazin-1-yl)-7-(pyridin-4-yl)-5H-pyrimido[5,4-b]indol", "intended_use": "Treatment of chronic lymphocytic leukaemia", "status": "Positive", "designation_date": "2007-11-14", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-07-487", "last_updated": "2008-07-29" }, { "eu_designation_number": "EU/3/07/488", "medicine_name": "", "active_substance": "everolimus", "intended_use": "Treatment of gastro-entero-pancreatic neuroendocrine tumours", "status": "Withdrawn", "designation_date": "2007-11-14", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-07-488", "last_updated": "2015-02-09" }, { "eu_designation_number": "EU/3/07/489", "medicine_name": "", "active_substance": "ciclosporin", "intended_use": "Treatment of herpes simplex virus stromal keratitis", "status": "Withdrawn", "designation_date": "2007-10-29", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-07-489", "last_updated": "2015-05-13" }, { "eu_designation_number": "EU/3/07/490", "medicine_name": "", "active_substance": "human autologous bone-forming cells derived from bone marrow stem cells", "intended_use": "Treatment of non-traumatic osteonecrosis", "status": "Withdrawn", "designation_date": "2007-10-29", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-07-490", "last_updated": "2019-11-20" }, { "eu_designation_number": "EU/3/07/491", "medicine_name": "", "active_substance": "Interferon gamma", "intended_use": "Treatment of idiopathic pulmonary fibrosis", "status": "Positive", "designation_date": "2007-10-29", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-07-491", "last_updated": "2013-03-01" }, { "eu_designation_number": "EU/3/07/492", "medicine_name": "", "active_substance": "Iodine (131I) chlorotoxin", "intended_use": "Treatment of glioma", "status": "Withdrawn", "designation_date": "2007-10-22", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-07-492", "last_updated": "2019-02-21" }, { "eu_designation_number": "EU/3/07/493", "medicine_name": "", "active_substance": "Isofagomine tartrate", "intended_use": "Treatment of Gaucher disease", "status": "Withdrawn", "designation_date": "2007-10-23", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-07-493", "last_updated": "2019-02-21" }, { "eu_designation_number": "EU/3/07/494", "medicine_name": "", "active_substance": "lenalidomide", "intended_use": "Treatment of chronic lymphocytic leukaemia", "status": "Withdrawn", "designation_date": "2007-11-19", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-07-494", "last_updated": "2020-01-13" }, { "eu_designation_number": "EU/3/07/495", "medicine_name": "", "active_substance": "methotrexate", "intended_use": "Treatment of acute lymphoblastic leukaemia", "status": "Positive", "designation_date": "2007-10-24", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-07-495", "last_updated": "2014-07-11" }, { "eu_designation_number": "EU/3/07/496", "medicine_name": "Loulla", "active_substance": "Mercaptopurine", "intended_use": "Treatment of acute lymphoblastic leukaemia", "status": "Positive", "designation_date": "2007-10-22", "related_ema_product_number": "EMEA/H/C/002501", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-07-496", "last_updated": "2014-01-22" }, { "eu_designation_number": "EU/3/07/497", "medicine_name": "", "active_substance": "4-[3,5-Bis(trimethylsilyl)benzamido] benzoic acid", "intended_use": "Treatment of hepatocellular carcinoma", "status": "Withdrawn", "designation_date": "2007-10-22", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-07-497", "last_updated": "2011-05-18" }, { "eu_designation_number": "EU/3/07/498", "medicine_name": "Akantior", "active_substance": "Polihexanide", "intended_use": "Treatment of acanthamoeba keratitis", "status": "Positive", "designation_date": "2007-11-14", "related_ema_product_number": "EMEA/H/C/005858", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-07-498", "last_updated": "2008-07-02" }, { "eu_designation_number": "EU/3/07/499", "medicine_name": "", "active_substance": "terguride", "intended_use": "Treatment of pulmonary arterial hypertension and chronic thromboembolic pulmonary hypertension", "status": "Positive", "designation_date": "2007-11-29", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-07-499", "last_updated": "2008-07-29" }, { "eu_designation_number": "EU/3/07/500", "medicine_name": "", "active_substance": "recombinant human rod-derived cone viability factor", "intended_use": "Treatment of retinitis pigmentosa", "status": "Withdrawn", "designation_date": "2007-11-29", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-07-500", "last_updated": "2015-03-06" }, { "eu_designation_number": "EU/3/07/501", "medicine_name": "", "active_substance": "olaparib", "intended_use": "Treatment of ovarian cancer", "status": "Withdrawn", "designation_date": "2007-12-06", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-07-501", "last_updated": "2018-05-28" }, { "eu_designation_number": "EU/3/07/502", "medicine_name": "", "active_substance": "Picoplatin", "intended_use": "Treatment of small cell lung cancer", "status": "Withdrawn", "designation_date": "2007-12-06", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-07-502", "last_updated": "2014-04-01" }, { "eu_designation_number": "EU/3/07/503", "medicine_name": "", "active_substance": "recombinant human hepatitis C monoclonal antibody against C4 region of E1", "intended_use": "Prevention of recurrent hepatitis C virus induced liver disease in liver transplant recipients", "status": "Positive", "designation_date": "2007-12-06", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-07-503", "last_updated": "2008-08-18" }, { "eu_designation_number": "EU/3/07/504", "medicine_name": "", "active_substance": "irinotecan hydrochloride", "intended_use": "Treatment of glioma", "status": "Withdrawn", "designation_date": "2007-11-30", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-07-504", "last_updated": "2017-04-25" }, { "eu_designation_number": "EU/3/07/505", "medicine_name": "", "active_substance": "Interferon beta", "intended_use": "Treatment of acute respiratory distress syndrome (ARDS)", "status": "Withdrawn", "designation_date": "2007-11-29", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-07-505", "last_updated": "2024-04-11" }, { "eu_designation_number": "EU/3/07/506", "medicine_name": "", "active_substance": "heterologous human adult liver-derived stem cells", "intended_use": "Treatment of Crigler-Najjar syndrome", "status": "Positive", "designation_date": "2007-11-29", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-07-506", "last_updated": "2024-10-15" }, { "eu_designation_number": "EU/3/07/507", "medicine_name": "", "active_substance": "doxorubicin hydrochloride", "intended_use": "Treatment of glioma", "status": "Withdrawn", "designation_date": "2007-11-30", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-07-507", "last_updated": "2017-04-25" }, { "eu_designation_number": "EU/3/07/508", "medicine_name": "", "active_substance": "chimeric anti-interleukin-6 monoclonal antibody (siltuximab)", "intended_use": "Treatment of Castleman's disease", "status": "Expired", "designation_date": "2007-11-30", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-07-508", "last_updated": "2024-06-07" }, { "eu_designation_number": "EU/3/07/509", "medicine_name": "", "active_substance": "azacitidine", "intended_use": "Treatment of acute myeloid leukaemia", "status": "Expired", "designation_date": "2007-11-29", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-07-509", "last_updated": "2019-02-20" }, { "eu_designation_number": "EU/3/07/510", "medicine_name": "", "active_substance": "artesunate", "intended_use": "Treatment of malaria", "status": "Positive", "designation_date": "2007-12-07", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-07-510", "last_updated": "2013-10-17" }, { "eu_designation_number": "EU/3/07/511", "medicine_name": "", "active_substance": "3-methoxy-pregnenolone", "intended_use": "Treatment of spinal cord injury", "status": "Positive", "designation_date": "2007-12-04", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-07-511", "last_updated": "2008-07-01" }, { "eu_designation_number": "EU/3/07/512", "medicine_name": "", "active_substance": "17-(Allylamino)-17-demethoxygeldanamycin, hydroquinone hydrochloride", "intended_use": "Treatment of malignant gastrointestinal stromal tumours", "status": "Withdrawn", "designation_date": "2007-11-29", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-07-512", "last_updated": "2011-03-25" }, { "eu_designation_number": "EU/3/07/513", "medicine_name": "Dovprela (previously Pretomanid FGK)", "active_substance": "(S)-2-nitro-6-(4-trifluoromethoxy)benzyloxy)-6,7-dihydro-5H-imidazo[2,1-b] [1,3] oxazine (pretomanid)", "intended_use": "Treatment of tuberculosis", "status": "Positive", "designation_date": "2007-11-29", "related_ema_product_number": "EMEA/H/C/005167", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-07-513", "last_updated": "2020-12-09" }, { "eu_designation_number": "EU/3/07/514", "medicine_name": "Cerdelga", "active_substance": "", "intended_use": "Treatment of Gaucher disease", "status": "Positive", "designation_date": "2007-12-04", "related_ema_product_number": "EMEA/H/C/003724", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-07-514", "last_updated": "2023-07-18" }, { "eu_designation_number": "EU/3/07/515", "medicine_name": "", "active_substance": "tegafur;gimeracil;oteracil potassium", "intended_use": "Treatment of gastric cancer", "status": "Withdrawn", "designation_date": "2007-12-20", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-07-515", "last_updated": "2011-03-28" }, { "eu_designation_number": "EU/3/07/516", "medicine_name": "", "active_substance": "recombinant human histone H1.3 and recombinant human N-bis-met-histone H1.3", "intended_use": "Treatment of acute myeloid leukaemia", "status": "Positive", "designation_date": "2007-12-20", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-07-516", "last_updated": "2013-03-13" }, { "eu_designation_number": "EU/3/07/517", "medicine_name": "", "active_substance": "N-[4-(3-amino-1H-indazol-4 yl)phenyl]-N'-(2-fluoro-5-methylphenyl) urea", "intended_use": "Treatment of hepatocellular carcinoma", "status": "Withdrawn", "designation_date": "2007-12-20", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-07-517", "last_updated": "2016-08-19" }, { "eu_designation_number": "EU/3/07/518", "medicine_name": "", "active_substance": "Methyl 4,6-diamino-2-[1-(2-fluorobenzyl)-1H-pyrazolo[3,4-b]pyridine-3-yl]-5-pyrimidinyl(methyl)carbamate (riociguat)  ", "intended_use": "Treatment of pulmonary arterial hypertension", "status": "Withdrawn", "designation_date": "2007-12-20", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-07-518", "last_updated": "2022-06-14" }, { "eu_designation_number": "EU/3/07/519", "medicine_name": "Livtencity", "active_substance": "Maribavir", "intended_use": "Prevention of cytomegalovirus disease in patients with impaired cell-mediated immunity", "status": "Positive", "designation_date": "2007-12-18", "related_ema_product_number": "EMEA/H/C/005787", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-07-519", "last_updated": "2022-11-24" }, { "eu_designation_number": "EU/3/07/520", "medicine_name": "", "active_substance": "Human-papilloma-virus-type-16 E6;E7 synthetic long peptides", "intended_use": "Treatment of epithelial neoplasia of the vulva positive for human papilloma virus", "status": "Positive", "designation_date": "2007-12-20", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-07-520", "last_updated": "2013-06-26" }, { "eu_designation_number": "EU/3/07/521", "medicine_name": "", "active_substance": "H-Arg-Leu-Phe-Phe-Tyr-Arg-Lys-Ser-Val-OH, acetate salt& H-Tyr-Leu-Phe-Phe-Tyr-Arg-Lys-Ser-Val-OH, acetate salt", "intended_use": "Treatment of TERT positive non-small cell lung cancer in HLA-A2 positive patients", "status": "Positive", "designation_date": "2007-12-18", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-07-521", "last_updated": "2010-03-15" }, { "eu_designation_number": "EU/3/07/522", "medicine_name": "", "active_substance": "(Manganese, dichloro [(4aR, 13aR, 17aR, 21aR)-1, 2, 3, 4, 4a, 5, 6, 12, 13, 13a, 14, 15, 16, 17, 17a, 18, 19, 20, 21, 21a-eicosahydro-11, 7-nitrilo-7H-dibenzo[ b,h] [1,4,7,10] tetraazacycloheptadecine-κN5, κN13, κN18, κN21, κN22]-) (misopasem manganese)", "intended_use": "Prevention of oral mucositis in head and neck cancer patients undergoing radiation therapy", "status": "Positive", "designation_date": "2008-01-31", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-07-522", "last_updated": "2019-05-17" }, { "eu_designation_number": "EU/3/07/523", "medicine_name": "Lutathera", "active_substance": "Lutetium (177Lu)-N-[(4,7,10-Tricarboxymethyl-1,4,7,10-tetraazacyclododec-1-yl)acetyl]-D-phenylalanyl-L-cysteinyl-L-tyrosyl-D-tryptophanyl-L-lysyl-L-threoninyl-L-cysteinyl-L-threonine-cyclic(2-7)disulfide (lutetium (177Lu) oxodotreotide)", "intended_use": "Treatment of gastro-entero-pancreatic neuroendocrine tumours", "status": "Positive", "designation_date": "2008-01-31", "related_ema_product_number": "EMEA/H/C/004123", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-07-523", "last_updated": "2026-08-04" }, { "eu_designation_number": "EU/3/07/524", "medicine_name": "", "active_substance": "(R)-2-Methyl-6-nitro-2-{4-[4-(4-trifluoromethoxyphenoxy)piperidin-1-yl]phenoxymethyl}-2,3-dihydroimidazo[2,1-b]oxazole (delamanid)", "intended_use": "Treatment of tuberculosis", "status": "Expired", "designation_date": "2008-02-02", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-07-524", "last_updated": "2014-06-18" }, { "eu_designation_number": "EU/3/07/525", "medicine_name": "", "active_substance": "Iodine (131I) iobenguane", "intended_use": "Treatment of neuroblastoma", "status": "Positive", "designation_date": "2008-01-31", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-07-525", "last_updated": "2023-03-29" }, { "eu_designation_number": "EU/3/07/526", "medicine_name": "", "active_substance": "N- (2-Amino-phenyl)-4-[(4-pyridin-3-yl-pyrimidin-2-ylamino)-methyl] benzamide (mocetinostat)", "intended_use": "Treatment of acute myeloid leukaemia", "status": "Withdrawn", "designation_date": "2008-01-31", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-07-526", "last_updated": "2022-05-04" }, { "eu_designation_number": "EU/3/08/527", "medicine_name": "", "active_substance": "recombinant human monoclonal antibody to human IL-1beta of the IgG1/K class", "intended_use": "Treatment of systemic-onset juvenile idiopathic arthritis", "status": "Withdrawn", "designation_date": "2008-02-05", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-08-527", "last_updated": "2015-02-12" }, { "eu_designation_number": "EU/3/08/528", "medicine_name": "", "active_substance": "Lumiliximab", "intended_use": "Treatment chronic lymphocytic leukaemia", "status": "Withdrawn", "designation_date": "2008-02-03", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-08-528", "last_updated": "2010-06-23" }, { "eu_designation_number": "EU/3/08/529", "medicine_name": "", "active_substance": "tretazicar", "intended_use": "Treatment of visceral leishmaniasis", "status": "Positive", "designation_date": "2008-02-04", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-08-529", "last_updated": "2013-07-08" }, { "eu_designation_number": "EU/3/08/530", "medicine_name": "", "active_substance": "heterologous human adult liver derived stem cells", "intended_use": "Treatment of ornithinine transcarbamylase deficiency", "status": "Positive", "designation_date": "2008-02-04", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-08-530", "last_updated": "2024-10-15" }, { "eu_designation_number": "EU/3/08/531", "medicine_name": "", "active_substance": "ascorbic acid", "intended_use": "Treatment of Charcot-Marie-Tooth disease type 1A", "status": "Positive", "designation_date": "2008-04-01", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-08-531", "last_updated": "2014-04-01" }, { "eu_designation_number": "EU/3/08/532", "medicine_name": "", "active_substance": "filgrastim", "intended_use": "Treatment of amyotrophic lateral sclerosis", "status": "Positive", "designation_date": "2008-04-01", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-08-532", "last_updated": "2015-03-26" }, { "eu_designation_number": "EU/3/08/533", "medicine_name": "", "active_substance": "recombinant human monoclonal antibody against transforming growth factor beta-1, 2 and 3", "intended_use": "Treatment of idiopathic pulmonary fibrosis", "status": "Withdrawn", "designation_date": "2008-04-01", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-08-533", "last_updated": "2016-01-19" }, { "eu_designation_number": "EU/3/08/534", "medicine_name": "", "active_substance": "allogeneic human umbilical cord tissue-derived cells", "intended_use": "Treatment of retinitis pigmentosa", "status": "Withdrawn", "designation_date": "2008-04-01", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-08-534", "last_updated": "2010-06-23" }, { "eu_designation_number": "EU/3/08/535", "medicine_name": "", "active_substance": "Humanised monoclonal antibody to the folate 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"https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-08-537", "last_updated": "2012-05-24" }, { "eu_designation_number": "EU/3/08/538", "medicine_name": "", "active_substance": "amrubicin hydrochloride", "intended_use": "Treatment of small cell lung cancer", "status": "Withdrawn", "designation_date": "2008-04-02", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-08-538", "last_updated": "2016-08-11" }, { "eu_designation_number": "EU/3/08/539", "medicine_name": "", "active_substance": "ammonium tetrathiomolybdate", "intended_use": "Treatment of Wilson's disease", "status": "Positive", "designation_date": "2008-04-01", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-08-539", "last_updated": "2008-07-10" }, { "eu_designation_number": "EU/3/08/540", "medicine_name": "", "active_substance": "omigapil maleate", "intended_use": "Treatment of congenital muscular dystrophy with collagen VI deficiency", "status": "Withdrawn", "designation_date": "2008-05-08", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-08-540", "last_updated": "2025-01-15" }, { "eu_designation_number": "EU/3/08/541", "medicine_name": "", "active_substance": "[Nle4, D-Phe7]-alfa-melanocyte stimulating hormone (afamelanotide)", "intended_use": "Treatment of erythropoietic protoporphyria", "status": "Expired", "designation_date": "2008-05-08", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-08-541", "last_updated": "2025-01-09" }, { "eu_designation_number": "EU/3/08/542", "medicine_name": "", "active_substance": "ribonucleotide reductase R2 specific phosphorothioate oligonucleotide", "intended_use": "Treatment of acute myeloid leukaemia", "status": "Positive", "designation_date": "2008-05-08", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-08-542", "last_updated": "2022-03-18" }, { "eu_designation_number": "EU/3/08/543", "medicine_name": "", "active_substance": "sarsasapogenin", "intended_use": "Treatment of amyotrophic lateral sclerosis", "status": "Withdrawn", "designation_date": "2008-05-08", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-08-543", "last_updated": "2012-09-06" }, { "eu_designation_number": "EU/3/08/544", "medicine_name": "", "active_substance": "omigapil maleate", "intended_use": "Treatment of congenital muscular dystrophy with merosin (laminin alpha 2) deficiency", "status": "Withdrawn", "designation_date": "2008-05-08", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-08-544", "last_updated": "2025-01-15" }, { "eu_designation_number": "EU/3/08/545", "medicine_name": "", "active_substance": "", "intended_use": "Treatment of congenital erythropoietic porphyria", "status": "Positive", "designation_date": "2008-05-08", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-08-545", "last_updated": "2016-04-11" }, { "eu_designation_number": "EU/3/08/546", "medicine_name": "", "active_substance": "", "intended_use": "Treatment of congenital alpha-1 antitrypsin deficiency", "status": "Positive", "designation_date": "2008-06-03", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-08-546", "last_updated": "2013-10-17" }, { "eu_designation_number": "EU/3/08/547", "medicine_name": "", "active_substance": "anti-von Willebrand aptamer", "intended_use": "Treatment of thrombotic thrombocytopenic purpura", "status": "Withdrawn", "designation_date": "2008-06-03", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-08-547", "last_updated": "2010-11-18" }, { "eu_designation_number": "EU/3/08/548", "medicine_name": "", "active_substance": "carfilzomib", "intended_use": "Treatment of multiple myeloma", "status": "Expired", "designation_date": "2008-06-03", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-08-548", "last_updated": "2025-11-28" }, { "eu_designation_number": "EU/3/08/549", "medicine_name": "Zafiride", "active_substance": "NGR-human tumour necrosis factor", "intended_use": "Treatment of malignant mesothelioma", "status": "Positive", "designation_date": "2008-06-03", "related_ema_product_number": "EMEA/H/C/004455", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-08-549", "last_updated": "2008-07-29" }, { "eu_designation_number": "EU/3/08/550", "medicine_name": "", "active_substance": "nimotuzumab", "intended_use": "Treatment of pancreatic cancer", "status": "Positive", "designation_date": "2008-06-03", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-08-550", "last_updated": "2018-10-12" }, { "eu_designation_number": "EU/3/08/551", "medicine_name": "", "active_substance": "pegylated recombinant factor VIIa", "intended_use": "Treatment of haemophilia A", "status": "Withdrawn", "designation_date": "2008-06-04", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-08-551", "last_updated": "2015-03-06" }, { "eu_designation_number": "EU/3/08/552", "medicine_name": "", "active_substance": "pegylated recombinant factor VIIa", "intended_use": "Treatment of haemophilia B", "status": "Withdrawn", "designation_date": "2008-06-03", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-08-552", "last_updated": "2015-03-06" }, { "eu_designation_number": "EU/3/08/553", "medicine_name": "", "active_substance": "recombinant fusion protein of circulary-permuted IL-4 and pseudomonas exotoxin A, [IL-4(38-37)-PE38KDEL]", "intended_use": "Treatment of glioma", "status": "Positive", "designation_date": "2008-06-03", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-08-553", "last_updated": "2008-08-18" }, { "eu_designation_number": "EU/3/08/554", "medicine_name": "", "active_substance": "beraprost sodium", "intended_use": "Treatment of pulmonary arterial hypertension", "status": "Withdrawn", "designation_date": "2008-07-10", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-08-554", "last_updated": "2020-02-11" }, { "eu_designation_number": "EU/3/08/555", "medicine_name": "", "active_substance": "vincristine sulfate liposomes", "intended_use": "Treatment of acute lymphoblastic leukaemia", "status": "Withdrawn", "designation_date": "2008-07-08", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-08-555", "last_updated": "2026-05-26" }, { "eu_designation_number": "EU/3/08/556", "medicine_name": "", "active_substance": "N-(2,4-di-tert-butyl-5-hydroxyphenyl)-1,4-dihydro-4-oxoquinoline-3-carboxamide (ivacaftor)", "intended_use": "Treatment of cystic fibrosis", "status": "Expired", "designation_date": "2008-07-08", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-08-556", "last_updated": "2022-08-10" }, { "eu_designation_number": "EU/3/08/557", "medicine_name": "", "active_substance": "sapacitabine", "intended_use": "Treatment of myelodysplastic syndromes", "status": "Positive", "designation_date": "2008-07-08", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-08-557", "last_updated": "2009-05-15" }, { "eu_designation_number": "EU/3/08/557", "medicine_name": "", "active_substance": "sapacitabine", "intended_use": "Treatment of myelodysplastic syndromes", "status": "Positive", "designation_date": "2008-07-08", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-08-557-0", "last_updated": "2009-06-29" }, { "eu_designation_number": "EU/3/08/558", "medicine_name": "", "active_substance": "sapacitabine", "intended_use": "Treatment of acute myeloid leukaemia", "status": "Positive", "designation_date": "2008-07-10", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-08-558", "last_updated": "2009-05-15" }, { "eu_designation_number": "EU/3/08/559", "medicine_name": "", "active_substance": "Bosentan", "intended_use": "Treatment of idiopathic pulmonary fibrosis", "status": "Withdrawn", "designation_date": "2008-09-05", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-08-559", "last_updated": "2014-05-08" }, { "eu_designation_number": "EU/3/08/560", "medicine_name": "", "active_substance": "", "intended_use": "Treatment of moderate and severe closed traumatic brain injury", "status": "Positive", "designation_date": "2008-09-05", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-08-560", "last_updated": "2013-11-28" }, { "eu_designation_number": "EU/3/08/561", "medicine_name": "Luxceptar", "active_substance": "Donor lymphocyte preparation depleted of functional alloreactive T-cells", "intended_use": "Prevention of graft-versus-host disease", "status": "Withdrawn", "designation_date": "2008-09-05", "related_ema_product_number": "EMEA/H/C/002397", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-08-561", "last_updated": "2022-02-28" }, { "eu_designation_number": "EU/3/08/562", "medicine_name": "", "active_substance": "topotecan hydrochloride", "intended_use": "Treatment of glioma", "status": "Positive", "designation_date": "2008-09-05", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-08-562", "last_updated": "2009-04-24" }, { "eu_designation_number": "EU/3/08/563", "medicine_name": "", "active_substance": "recombinant derivative of C3 transferase", "intended_use": "Treatment of traumatic spinal cord injury", "status": "Positive", "designation_date": "2008-09-05", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-08-563", "last_updated": "2022-01-25" }, { "eu_designation_number": "EU/3/08/564", "medicine_name": "", "active_substance": "avian polyclonal IgY antibody", "intended_use": "Treatment of cystic fibrosis", "status": "Positive", "designation_date": "2008-09-23", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-08-564", "last_updated": "2009-04-24" }, { "eu_designation_number": "EU/3/08/565", "medicine_name": "", "active_substance": "drotrecogin alfa (activated)", "intended_use": "Treatment of acute respiratory distress syndrome (ARDS)", "status": "Withdrawn", "designation_date": "2008-09-22", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-08-565", "last_updated": "2021-09-21" }, { "eu_designation_number": "EU/3/08/566", "medicine_name": "", "active_substance": "Levofloxacin hemihydrate", "intended_use": "Treatment of cystic fibrosis", "status": "Withdrawn", "designation_date": "2008-09-23", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-08-566", "last_updated": "2018-02-09" }, { "eu_designation_number": "EU/3/08/567", "medicine_name": "", "active_substance": "Miltefosine", "intended_use": 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"https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-08-597", "last_updated": "2015-05-08" }, { "eu_designation_number": "EU/3/08/598", "medicine_name": "", "active_substance": "exon 44 specific phosphorothioate oligonucleotide", "intended_use": "Treatment of Duchenne muscular dystrophy", "status": "Positive", "designation_date": "2009-02-27", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-08-598", "last_updated": "2016-12-02" }, { "eu_designation_number": "EU/3/08/599", "medicine_name": "", "active_substance": "exon 51 specific phosphorothioate oligonucleotide", "intended_use": "Treatment of Duchenne muscular dystrophy", "status": "Positive", "designation_date": "2009-02-27", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-08-599", "last_updated": "2015-05-27" }, { "eu_designation_number": "EU/3/08/600", "medicine_name": "", "active_substance": "human anti-intercellular adhesion molecule-1 monoclonal antibody", "intended_use": "Treatment of multiple myeloma", "status": "Positive", "designation_date": "2009-01-20", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-08-600", "last_updated": "2009-06-15" }, { "eu_designation_number": "EU/3/08/601", "medicine_name": "", "active_substance": "Milatuzumab", "intended_use": "Treatment of multiple myeloma", "status": "Withdrawn", "designation_date": "2009-01-19", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-08-601", "last_updated": "2020-02-18" }, { "eu_designation_number": "EU/3/08/602", "medicine_name": "", "active_substance": "Milatuzumab", "intended_use": "Treatment of chronic lymphocytic leukaemia", "status": "Withdrawn", "designation_date": "2009-01-19", "related_ema_product_number": "", "url": 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"eu_designation_number": "EU/3/08/605", "medicine_name": "", "active_substance": "recombinant human monoclonal antibody to human Nogo-A protein of the IgG4/kappa class (atinumab)", "intended_use": "Treatment of spinal cord injury", "status": "Withdrawn", "designation_date": "2009-01-19", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-08-605", "last_updated": "2019-10-16" }, { "eu_designation_number": "EU/3/08/606", "medicine_name": "", "active_substance": "recombinant human residue 41 glutamic acid to glutamine variant of interferon alfa-2b", "intended_use": "Treatment of Behçet's Disease", "status": "Withdrawn", "designation_date": "2009-01-19", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-08-606", "last_updated": "2011-05-18" }, { "eu_designation_number": "EU/3/08/607", "medicine_name": "", "active_substance": "type I native bovine skin collagen", 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"active_substance": "2,2-Dimethylbutyric acid, sodium salt", "intended_use": "Treatment of sickle cell disease", "status": "Positive", "designation_date": "2009-03-18", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-09-621", "last_updated": "2015-03-20" }, { "eu_designation_number": "EU/3/09/622", "medicine_name": "", "active_substance": "N-(5-tert-Butylisoxazol-3-yl)-N'-{4-[7-(2-(morpholin-4-yl)ethoxy) imidazo[2,1-b][1,3]benzothiazol-2-yl]phenyl}urea di-hydrochloride salt", "intended_use": "Treatment of acute myeloid leukaemia", "status": "Withdrawn", "designation_date": "2009-03-23", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-09-622", "last_updated": "2026-05-26" }, { "eu_designation_number": "EU/3/09/623", "medicine_name": "", "active_substance": "autologous haematopoietic stem cells transduced with lentiviral vector encoding the human beta-globin 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"active_substance": "L-asparaginase encapsulated in erythrocytes", "intended_use": "Treatment of pancreatic cancer", "status": "Positive", "designation_date": "2009-05-15", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-09-633", "last_updated": "2009-06-09" }, { "eu_designation_number": "EU/3/09/634", "medicine_name": "", "active_substance": "S-[2,3-bispalmitoyloxy-(2R)-propyl]-cysteinyl-GNNDESNISFKEK", "intended_use": "Treatment of pancreatic cancer", "status": "Positive", "designation_date": "2009-05-15", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-09-634", "last_updated": "2009-06-09" }, { "eu_designation_number": "EU/3/09/635", "medicine_name": "", "active_substance": "treprostinil diethanolamine", "intended_use": "Treatment of systemic sclerosis", "status": "Positive", "designation_date": "2009-05-15", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-09-635", "last_updated": "2023-02-13" }, { "eu_designation_number": "EU/3/09/636", "medicine_name": "", "active_substance": "Dexamethasone phosphate", "intended_use": "Treatment of corneal graft rejection", "status": "Withdrawn", "designation_date": "2009-05-15", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-09-636", "last_updated": "2016-10-21" }, { "eu_designation_number": "EU/3/09/637", "medicine_name": "", "active_substance": "2',3',5'-Tri-O-acetyluridine", "intended_use": "Treatment of 5-fluorouracil overdose", "status": "Positive", "designation_date": "2009-05-15", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-09-637", "last_updated": "2023-09-11" }, { "eu_designation_number": "EU/3/09/638", "medicine_name": "", "active_substance": "Humanised IgG4 monoclonal antibody to the human 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"active_substance": "Desipramine chlorhydrate", "intended_use": "Treatment of Rett syndrome", "status": "Withdrawn", "designation_date": "2009-06-12", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-09-643", "last_updated": "2017-08-16" }, { "eu_designation_number": "EU/3/09/644", "medicine_name": "", "active_substance": "Murine monoclonal antibody to GD2", "intended_use": "Treatment of neuroblastoma", "status": "Withdrawn", "designation_date": "2009-06-12", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-09-644", "last_updated": "2011-03-28" }, { "eu_designation_number": "EU/3/09/645", "medicine_name": "", "active_substance": "octreotide hydrochloride", "intended_use": "Treatment of acromegaly", "status": "Withdrawn", "designation_date": "2009-06-12", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-09-645", "last_updated": "2025-06-30" }, { "eu_designation_number": "EU/3/09/646", "medicine_name": "", "active_substance": "talampanel", "intended_use": "Treatment of amyotrophic lateral sclerosis", "status": "Withdrawn", "designation_date": "2009-06-12", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-09-646", "last_updated": "2010-11-18" }, { "eu_designation_number": "EU/3/09/647", "medicine_name": "", "active_substance": "(S)-3'-(OH)-desazadesferrithiocin-polyether, magnesium salt (deferitazole)", "intended_use": "Treatment of chronic iron overload requiring chelation therapy", "status": "Withdrawn", "designation_date": "2009-07-24", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-09-647", "last_updated": "2018-03-26" }, { "eu_designation_number": "EU/3/09/648", "medicine_name": "", "active_substance": "afamelanotide", "intended_use": "Treatment of solar urticaria", "status": "Positive", "designation_date": "2009-07-24", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-09-648", "last_updated": "2016-04-11" }, { "eu_designation_number": "EU/3/09/649", "medicine_name": "", "active_substance": "allogeneic ex-vivo-expanded umbilical-cord blood cells", "intended_use": "Treatment of Hodgkin's lymphoma", "status": "Withdrawn", "designation_date": "2009-07-24", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-09-649", "last_updated": "2015-05-08" }, { "eu_designation_number": "EU/3/09/650", "medicine_name": "Blincyto", "active_substance": "blinatumomab", "intended_use": "Treatment of acute lymphoblastic leukaemia", "status": "Positive", "designation_date": "2009-07-24", "related_ema_product_number": "EMEA/H/C/003731", "url": 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"medicine_name": "", "active_substance": "recombinant histidine-tagged idiotype immunoglobulin Fab fragment of clonal B-cell receptors", "intended_use": "Treatment of diffuse large B-cell lymphoma", "status": "Positive", "designation_date": "2009-07-24", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-09-656", "last_updated": "2011-05-31" }, { "eu_designation_number": "EU/3/09/657", "medicine_name": "", "active_substance": "recombinant human N-acetylgalactosamine-6-sulfatase (elosulfase alfa)", "intended_use": "Treatment of mucopolysaccharidosis, type IVA (Morquio A syndrome)", "status": "Withdrawn", "designation_date": "2009-07-24", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-09-657", "last_updated": "2025-10-23" }, { "eu_designation_number": "EU/3/09/658", "medicine_name": "", "active_substance": "tamibarotene", "intended_use": "Treatment of acute 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"medicine_name": "Xermelo", "active_substance": "(S)-ethyl 2-amino-3-(4-(2-amino-6((R)-1-(4-chloro-2-(3-methyl-1H-pyrazol-1-yl)phenyl)-2,2,2-trifluoroethoxy)pyrimidin-4-yl)phenyl)propanoate", "intended_use": "Treatment of carcinoid syndrome", "status": "Positive", "designation_date": "2016-05-30", "related_ema_product_number": "EMEA/H/C/003937", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-09-661", "last_updated": "2022-07-28" }, { "eu_designation_number": "EU/3/09/662", "medicine_name": "", "active_substance": "26 base single stranded phosphodiester DNA oligonucleotide", "intended_use": "Treatment of acute myeloid leukaemia", "status": "Withdrawn", "designation_date": "2009-10-08", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-09-662", "last_updated": "2011-05-18" }, { "eu_designation_number": "EU/3/09/663", "medicine_name": "", "active_substance": "adeno-associated viral vector 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"https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-09-680", "last_updated": "2013-06-24" }, { "eu_designation_number": "EU/3/09/681", "medicine_name": "", "active_substance": "6-chloro-2,3,4,9-tetrahydro-1H-carbazole-1-carboxamide (selisistat)", "intended_use": "Treatment of Huntington’s disease", "status": "Positive", "designation_date": "2009-10-08", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-09-681", "last_updated": "2022-02-04" }, { "eu_designation_number": "EU/3/09/682", "medicine_name": "", "active_substance": "anti-EphA2 monoclonal antibody conjugated to maleimidocaproyl monomethylauristatin phenylalanine", "intended_use": "Treatment of ovarian cancer", "status": "Withdrawn", "designation_date": "2009-10-28", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-09-682", "last_updated": "2010-11-19" }, { "eu_designation_number": "EU/3/09/683", "medicine_name": "", "active_substance": "cholic acid", "intended_use": "Treatment of inborn errors in primary bile acid synthesis responsive to treatment with cholic acid", "status": "Withdrawn", "designation_date": "2009-10-29", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-09-683", "last_updated": "2020-07-29" }, { "eu_designation_number": "EU/3/09/684", "medicine_name": "Masican; Masiviera", "active_substance": "Masitinib mesilate", "intended_use": "Treatment of pancreatic cancer", "status": "Positive", "designation_date": "2009-10-28", "related_ema_product_number": "EMEA/H/C/002670; EMEA/H/C/002659", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-09-684", "last_updated": "2009-11-18" }, { "eu_designation_number": "EU/3/09/685", "medicine_name": "", "active_substance": "N-[(2S)-2,3-dihydroxypropyl]-3-[(2-fluoro-4-iodophenyl)amino]isonicotinamide hydrochloride", 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"2015-02-11" }, { "eu_designation_number": "EU/3/09/688", "medicine_name": "", "active_substance": "patupilone", "intended_use": "Treatment of fallopian tube cancer", "status": "Withdrawn", "designation_date": "2009-11-05", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-09-688", "last_updated": "2011-03-29" }, { "eu_designation_number": "EU/3/09/689", "medicine_name": "", "active_substance": "Peptides mimicking antigen receptors on autoimmune B cells and autoimmune T cells associated with myasthenia gravis", "intended_use": "Treatment of myasthenia gravis", "status": "Positive", "designation_date": "2009-11-09", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-09-689", "last_updated": "2014-10-07" }, { "eu_designation_number": "EU/3/09/690", "medicine_name": "", "active_substance": "human anthrax immunoglobulin", "intended_use": "Post-exposure prophylaxis of 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"https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-09-692", "last_updated": "2014-04-01" }, { "eu_designation_number": "EU/3/09/693", "medicine_name": "", "active_substance": "1-Cyclopropyl-3-[3-(5-morpholin-4-ylmethyl-1H-benzoimidazol-2-yl)-1H-pyrazol-4-yl]-urea", "intended_use": "Treatment of acute myeloid leukaemia", "status": "Withdrawn", "designation_date": "2009-11-26", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-09-693", "last_updated": "2019-02-21" }, { "eu_designation_number": "EU/3/09/694", "medicine_name": "", "active_substance": "6-Thioguanine", "intended_use": "Treatment of acute lymphoblastic leukaemia", "status": "Positive", "designation_date": "2009-11-26", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-09-694", "last_updated": "2013-11-28" }, { "eu_designation_number": "EU/3/09/695", "medicine_name": "", "active_substance": "8-[4-(1-aminocyclobutyl)phenyl]-9-phenyl-1,2,4-triazolo[3,4-f][1,6]naphthyridin-3(2H)-one mono-hydrochloride", "intended_use": "Treatment of ovarian cancer", "status": "Withdrawn", "designation_date": "2009-11-30", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-09-695", "last_updated": "2019-02-21" }, { "eu_designation_number": "EU/3/09/696", "medicine_name": "", "active_substance": "human MHC non-restricted cytotoxic T-cell line", "intended_use": "Treatment of ovarian cancer", "status": "Withdrawn", "designation_date": "2009-11-30", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-09-696", "last_updated": "2026-08-21" }, { "eu_designation_number": "EU/3/09/697", "medicine_name": "", "active_substance": "N-[6-(cis-2,6-dimethylmorpholin-4-yl)pyridine-3-yl]-2-methyl-4'-(trifluoromethoxy)[1,1'-biphenyl]-3-carboxamide", "intended_use": "Treatment of naevoid basal-cell carcinoma syndrome (Gorlin syndrome)", "status": "Withdrawn", "designation_date": "2009-11-25", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-09-697", "last_updated": "2015-02-10" }, { "eu_designation_number": "EU/3/09/698", "medicine_name": "", "active_substance": "pegylated carboxyhaemoglobin", "intended_use": "Treatment of sickle cell disease", "status": "Positive", "designation_date": "2009-11-26", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-09-698", "last_updated": "2021-10-13" }, { "eu_designation_number": "EU/3/09/699", "medicine_name": "", "active_substance": "recombinant chimeric monoclonal antibody against CD20 (ublituximab)", "intended_use": "Treatment of chronic lymphocytic leukaemia", "status": "Positive", "designation_date": "2009-11-26", "related_ema_product_number": "", "url": 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"beta-artemether;lumefantrine", "intended_use": "Treatment of malaria", "status": "Positive", "designation_date": "2010-01-28", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-09-702", "last_updated": "2010-02-25" }, { "eu_designation_number": "EU/3/09/703", "medicine_name": "", "active_substance": "brivudine", "intended_use": "Treatment of pancreatic cancer", "status": "Positive", "designation_date": "2010-01-28", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-09-703", "last_updated": "2010-02-25" }, { "eu_designation_number": "EU/3/09/704", "medicine_name": "", "active_substance": "givinostat", "intended_use": "Treatment of systemic-onset juvenile idiopathic arthritis", "status": "Withdrawn", "designation_date": "2010-01-28", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-09-704", 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"recombinant kallikrein inhibitor", "intended_use": "Treatment of Netherton syndrome", "status": "Positive", "designation_date": "2010-01-29", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-09-712", "last_updated": "2012-02-09" }, { "eu_designation_number": "EU/3/09/713", "medicine_name": "", "active_substance": "veltuzumab", "intended_use": "Treatment of chronic lymphocytic leukaemia", "status": "Withdrawn", "designation_date": "2010-01-29", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-09-713", "last_updated": "2020-03-06" }, { "eu_designation_number": "EU/3/09/714", "medicine_name": "", "active_substance": "streptococcus pyogenes Su strain cells treated with benzylpenicillin", "intended_use": "Treatment of congenital lymphatic malformations", "status": "Withdrawn", "designation_date": "2010-01-29", "related_ema_product_number": "", "url": 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"EU/3/09/722", "medicine_name": "", "active_substance": "pixantrone dimaleate", "intended_use": "Treatment of diffuse large B-cell lymphoma", "status": "Withdrawn", "designation_date": "2010-02-02", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-09-722", "last_updated": "2011-05-18" }, { "eu_designation_number": "EU/3/09/723", "medicine_name": "Idelvion", "active_substance": "recombinant fusion protein linking human coagulation factor IX with human albumin", "intended_use": "Treatment of haemophilia B", "status": "Positive", "designation_date": "2010-02-04", "related_ema_product_number": "EMEA/H/C/003955", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-09-723", "last_updated": "2018-03-28" }, { "eu_designation_number": "EU/3/09/724", "medicine_name": "", "active_substance": "recombinant human monoclonal antibody to human interleukin (IL)-17A of the IgG1/k class", "intended_use": "Treatment 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"Davunetide", "intended_use": "Treatment of progressive supranuclear palsy", "status": "Withdrawn", "designation_date": "2010-03-23", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-10-728", "last_updated": "2013-06-25" }, { "eu_designation_number": "EU/3/10/729", "medicine_name": "", "active_substance": "", "intended_use": "Treatment of acute myeloid leukaemia", "status": "Withdrawn", "designation_date": "2010-03-23", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-10-729", "last_updated": "2011-11-18" }, { "eu_designation_number": "EU/3/10/730", "medicine_name": "", "active_substance": "raloxifene hydrochloride", "intended_use": "Treatment of hereditary haemorrhagic telangiectasia", "status": "Positive", "designation_date": "2010-06-10", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-10-730", "last_updated": "2010-06-24" }, { "eu_designation_number": "EU/3/10/731", "medicine_name": "", "active_substance": "bafetinib", "intended_use": "Treatment of chronic myeloid leukaemia", "status": "Withdrawn", "designation_date": "2010-06-10", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-10-731", "last_updated": "2013-07-31" }, { "eu_designation_number": "EU/3/10/732", "medicine_name": "", "active_substance": "Entinostat", "intended_use": "Treatment of Hodgkin's lymphoma", "status": "Positive", "designation_date": "2010-06-10", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-10-732", "last_updated": "2023-09-13" }, { "eu_designation_number": "EU/3/10/733", "medicine_name": "Ravicti", "active_substance": "Glyceryl tri-(4-phenylbutyrate)", "intended_use": "Treatment of carbamoyl-phosphate synthase-1 deficiency", "status": "Positive", "designation_date": "2010-02-03", "related_ema_product_number": "EMEA/H/C/003822", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-10-733", "last_updated": "2022-11-18" }, { "eu_designation_number": "EU/3/10/734", "medicine_name": "Ravicti", "active_substance": "Glyceryl tri-(4-phenylbutyrate)", "intended_use": "Treatment of ornithine carbamoyltransferase deficiency", "status": "Positive", "designation_date": "2010-06-10", "related_ema_product_number": "EMEA/H/C/003822", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-10-734", "last_updated": "2022-11-18" }, { "eu_designation_number": "EU/3/10/735", "medicine_name": "Ravicti", "active_substance": "Glyceryl tri-(4-phenylbutyrate)", "intended_use": "Treatment of citrullinaemia type 1", "status": "Positive", "designation_date": "2010-06-10", "related_ema_product_number": "EMEA/H/C/003822", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-10-735", "last_updated": "2022-11-18" }, { "eu_designation_number": "EU/3/10/736", "medicine_name": "Ravicti", "active_substance": "Glyceryl tri-(4-phenylbutyrate)", "intended_use": "Treatment of argininosuccinic aciduria", "status": "Positive", "designation_date": "2010-06-10", "related_ema_product_number": "EMEA/H/C/003822", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-10-736", "last_updated": "2022-11-18" }, { "eu_designation_number": "EU/3/10/737", "medicine_name": "Ravicti", "active_substance": "Glyceryl tri-(4-phenylbutyrate)", "intended_use": "Treatment of hyperargininaemia", "status": "Positive", "designation_date": "2010-06-10", "related_ema_product_number": "EMEA/H/C/003822", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-10-737", "last_updated": "2022-11-18" }, { "eu_designation_number": "EU/3/10/738", "medicine_name": "Ravicti", "active_substance": "Glyceryl tri-(4-phenylbutyrate)", "intended_use": "Ornithine translocase deficiency (hyperornithinaemia-hyperammonaemia homocitrullinuria (HHH) syndrom", "status": "Positive", "designation_date": "2010-06-10", "related_ema_product_number": "EMEA/H/C/003822", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-10-738", "last_updated": "2022-11-18" }, { "eu_designation_number": "EU/3/10/739", "medicine_name": "", "active_substance": "Glyceryl tri-(4-phenylbutyrate)", "intended_use": "Treatment of citrullinaemia type 2", "status": "Positive", "designation_date": "2010-06-10", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-10-739", "last_updated": "2022-11-18" }, { "eu_designation_number": "EU/3/10/740", "medicine_name": "", "active_substance": "perifosine", "intended_use": "Treatment of multiple myeloma", "status": "Withdrawn", "designation_date": "2010-06-10", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-10-740", "last_updated": "2014-02-07" }, { "eu_designation_number": "EU/3/10/741", "medicine_name": "", "active_substance": "pralatrexate", "intended_use": "Treatment of cutaneous T-cell lymphoma", "status": "Positive", "designation_date": "2010-06-10", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-10-741", "last_updated": "2011-03-10" }, { "eu_designation_number": "EU/3/10/742", "medicine_name": "", "active_substance": "2-Methoxymethyl-2-hydroxymethyl-1-azabicyclo[2,2,2]octan-3-one", "intended_use": "Treatment of acute myeloid leukaemia", "status": "Positive", "designation_date": "2010-06-10", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-10-742", "last_updated": "2020-10-13" }, { "eu_designation_number": "EU/3/10/743", "medicine_name": "", "active_substance": "Pagibaximab", "intended_use": "Prevention of sepsis caused by Gram-positive pathogens in premature infants less than or equal to 34", "status": "Withdrawn", "designation_date": "2010-06-09", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-10-743", "last_updated": "2012-02-02" }, { "eu_designation_number": "EU/3/10/744", "medicine_name": "", "active_substance": "adrenomedullin", "intended_use": "Treatment of acute lung Injury", "status": "Positive", "designation_date": "2010-06-09", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-10-744", "last_updated": "2011-11-18" }, { "eu_designation_number": "EU/3/10/745", "medicine_name": "", "active_substance": "dexamethasone", "intended_use": "Treatment of multiple myeloma", "status": "Withdrawn", "designation_date": "2010-06-09", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-10-745", "last_updated": "2018-02-12" }, { "eu_designation_number": "EU/3/10/746", "medicine_name": "", "active_substance": "Maytansinoid-conjugated humanised monoclonal antibody against CD56", "intended_use": "Treatment of Merkel cell carcinoma", "status": "Withdrawn", "designation_date": "2010-06-09", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-10-746", "last_updated": "2015-03-06" }, { "eu_designation_number": "EU/3/10/747", "medicine_name": "", "active_substance": "N-{2-Chloro-4-[(6,7-dimethoxy-4-quinolyl)oxy]phenyl}-N'-(5-methyl-3-isoxazolyl) urea hydrochloride monohydrate", "intended_use": "Treatment of renal-cell carcinoma", "status": "Withdrawn", "designation_date": "2010-06-09", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-10-747", "last_updated": "2017-05-17" }, { "eu_designation_number": "EU/3/10/748", "medicine_name": "", "active_substance": "pravastatin;zoledronic acid", "intended_use": "Treatment of Hutchinson-Gilford progeria", "status": "Positive", "designation_date": "2010-06-09", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-10-748", "last_updated": "2010-06-23" }, { "eu_designation_number": "EU/3/10/749", "medicine_name": "Gamifant", "active_substance": "recombinant human anti-interferon gamma monoclonal antibody (emapalumab)", "intended_use": "Treatment of haemophagocytic lymphohistiocytosis", "status": "Positive", "designation_date": "2010-06-09", "related_ema_product_number": "EMEA/H/C/004386", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-10-749", "last_updated": "2019-06-19" }, { "eu_designation_number": "EU/3/10/750", "medicine_name": "", "active_substance": "rifapentine", "intended_use": "Treatment of tuberculosis", "status": "Positive", "designation_date": "2010-06-09", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-10-750", "last_updated": "2023-06-12" }, { "eu_designation_number": "EU/3/10/751", "medicine_name": "", "active_substance": "synthetic double-stranded siRNA oligonucleotide directed against p53 mRNA (teprasiran)", "intended_use": "Prevention of delayed graft function after renal transplantation", "status": "Withdrawn", "designation_date": "2010-06-06", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-10-751", "last_updated": "2022-02-28" }, { "eu_designation_number": "EU/3/10/752", "medicine_name": "", "active_substance": "velaglucerase alfa", "intended_use": "Treatment of Gaucher disease", "status": "Expired", "designation_date": "2010-06-06", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-10-752", "last_updated": "2022-09-09" }, { "eu_designation_number": "EU/3/10/753", "medicine_name": "", "active_substance": "6alpha-ethyl-chenodeoxycholic acid", "intended_use": "Treatment of primary biliary cirrhosis", "status": "Withdrawn", "designation_date": "2010-07-27", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-10-753", "last_updated": "2022-11-21" }, { "eu_designation_number": "EU/3/10/754", "medicine_name": "", "active_substance": "heparin-activated recombinant human fibroblast growth factor 1 (on a biodegradable device made from alpha-calcium sulfate hemihydrate)", "intended_use": "Treatment of traumatic spinal cord injury", "status": "Withdrawn", "designation_date": "2010-07-27", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-10-754", "last_updated": "2021-02-03" }, { "eu_designation_number": "EU/3/10/755", "medicine_name": "", "active_substance": "octenidine dihydrochloride", "intended_use": "Prevention of late-onset sepsis in premature infants of ≤ to 32 weeks of gestational age", "status": "Positive", "designation_date": "2010-07-27", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-10-755", "last_updated": "2010-08-05" }, { "eu_designation_number": "EU/3/10/756", "medicine_name": "", "active_substance": "tranilast", "intended_use": "Prevention of scarring post glaucoma filtration surgery", "status": "Positive", "designation_date": "2010-07-27", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-10-756", "last_updated": "2024-04-11" }, { "eu_designation_number": "EU/3/10/757", "medicine_name": "", "active_substance": "Pomalidomide", "intended_use": "Treatment of primary myelofibrosis", "status": "Withdrawn", "designation_date": "2010-07-27", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-10-757", "last_updated": "2017-02-10" }, { "eu_designation_number": "EU/3/10/758", "medicine_name": "", "active_substance": "Pomalidomide", "intended_use": "Treatment of post-polycythaemia vera myelofibrosis", "status": "Withdrawn", "designation_date": "2010-07-27", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-10-758", "last_updated": "2017-02-10" }, { "eu_designation_number": "EU/3/10/759", "medicine_name": "", "active_substance": "Pomalidomide", "intended_use": "Treatment of post-essential thrombocythaemia myelofibrosis", "status": "Withdrawn", "designation_date": "2010-07-27", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-10-759", "last_updated": "2017-02-10" }, { "eu_designation_number": "EU/3/10/760", "medicine_name": "Zejula", "active_substance": "(3S)-3-{4-[7-(aminocarbonyl)-2H-indazol-2-yl] phenyl} piperidine tosilate monohydrate salt (niraparib)", "intended_use": "Treatment of ovarian cancer", "status": "Positive", "designation_date": "2010-08-04", "related_ema_product_number": "EMEA/H/C/004249", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-10-760", "last_updated": "2020-11-06" }, { "eu_designation_number": "EU/3/10/761", "medicine_name": "", "active_substance": "3-(6-(1-(2,2-Difluorobenzo [d] [1,3] dioxol-5-yl)cyclopropanecarboxamido)-3-methylpyridin-2-yl)benzoic acid (lumacaftor)", "intended_use": "Treatment of cystic fibrosis", "status": "Withdrawn", "designation_date": "2010-08-04", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-10-761", "last_updated": "2017-07-17" }, { "eu_designation_number": "EU/3/10/762", "medicine_name": "", "active_substance": "Bosutinib", "intended_use": "Treatment of chronic myeloid leukaemia", "status": "Withdrawn", "designation_date": "2010-08-04", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-10-762", "last_updated": "2018-05-07" }, { "eu_designation_number": "EU/3/10/763", "medicine_name": "", "active_substance": "dexamethasone", "intended_use": "Treatment of non-infectious uveitis affecting the posterior segment of the eye", "status": "Withdrawn", "designation_date": "2010-08-04", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-10-763", "last_updated": "2010-11-19" }, { "eu_designation_number": "EU/3/10/764", "medicine_name": "", "active_substance": "everolimus", "intended_use": "Treatment of tuberous sclerosis", "status": "Expired", "designation_date": "2010-08-04", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-10-764", "last_updated": "2023-09-15" }, { "eu_designation_number": "EU/3/10/765", "medicine_name": "Rydapt", "active_substance": "Midostaurin", "intended_use": "Treatment of mastocytosis", "status": "Positive", "designation_date": "2010-08-04", "related_ema_product_number": "EMEA/H/C/004095", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-10-765", "last_updated": "2018-05-29" }, { "eu_designation_number": "EU/3/10/766", "medicine_name": "", "active_substance": "Pyr-His-Trp-Ser-Tyr-D-Lys(doxorubicinylglutarate)-Leu-Arg-Pro-Gly-NH2, acetate salt", "intended_use": "Treatment of ovarian cancer", "status": "Withdrawn", "designation_date": "2010-08-04", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-10-766", "last_updated": "2017-08-16" }, { "eu_designation_number": "EU/3/10/767", "medicine_name": "Enpaxiq; Epjevy", "active_substance": "11-(2-pyrrolidin-1-yl-ethoxy)-14,19-dioxa-5,7,26-triaza-tetracyclo[19.3.1.1(2,6).1(8,12)] heptacosa-1(25),2(26),3,5,8,10,12(27),16,21,23-decaene", "intended_use": "Treatment of post-essential thrombocythaemia 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"designation_date": "2010-09-20", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-10-771", "last_updated": "2013-10-17" }, { "eu_designation_number": "EU/3/10/772", "medicine_name": "", "active_substance": "adenovirus-associated viral vector serotype 10 carrying the human N-sulfoglucosamine sulfohydrolase and sulfatase modifying factor 1 cDNAs", "intended_use": "Treatment of mucopolysaccharidosis, type IIIA (Sanfilippo A syndrome)", "status": "Positive", "designation_date": "2010-09-20", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-10-772", "last_updated": "2015-05-13" }, { "eu_designation_number": "EU/3/10/773", "medicine_name": "", "active_substance": "allogeneic T cells encoding an exogenous TK gene", "intended_use": "Treatment of acute myeloid leukaemia", "status": "Positive", "designation_date": "2010-09-20", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-10-773", "last_updated": "2010-10-18" }, { "eu_designation_number": "EU/3/10/774", "medicine_name": "", "active_substance": "allogeneic human dermal fibroblasts", "intended_use": "Treatment of epidermolysis bullosa", "status": "Positive", "designation_date": "2010-09-20", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-10-774", "last_updated": "2012-02-09" }, { "eu_designation_number": "EU/3/10/775", "medicine_name": "", "active_substance": "autologous bone-marrow-derived mononuclear-cell fraction", "intended_use": "Treatment of thromboangiitis obliterans (Buerger's disease)", "status": "Positive", "designation_date": "2010-09-20", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-10-775", "last_updated": "2013-10-08" }, { "eu_designation_number": "EU/3/10/776", "medicine_name": "", "active_substance": "autologous dendritic cells pulsed with recombinant human-fusion protein (mucin 1 - glutathione S transferase) coupled to oxidised polymannose", "intended_use": "Treatment of ovarian cancer", "status": "Positive", "designation_date": "2010-09-20", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-10-776", "last_updated": "2013-10-17" }, { "eu_designation_number": "EU/3/10/777", "medicine_name": "Nulibry", "active_substance": "cyclic pyranopterin monophosphate (fosdenopterin)", "intended_use": "Treatment of molybdenum-cofactor deficiency type A", "status": "Positive", "designation_date": "2010-09-20", "related_ema_product_number": "EMEA/H/C/005378", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-10-777", "last_updated": "2023-07-17" }, { "eu_designation_number": "EU/3/10/778", "medicine_name": "", "active_substance": "cysteamine bitartrate (gastroresistant) (mercaptamine)", "intended_use": "Treatment of cystinosis", "status": "Positive", "designation_date": "2010-09-20", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-10-778", "last_updated": "2013-10-10" }, { "eu_designation_number": "EU/3/10/779", "medicine_name": "", "active_substance": "eflornithine", "intended_use": "Treatment of familial adenomatous polyposis (FAP)", "status": "Positive", "designation_date": "2010-09-20", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-10-779", "last_updated": "2019-04-29" }, { "eu_designation_number": "EU/3/10/780", "medicine_name": "", "active_substance": "Forodesine", "intended_use": "Treatment of chronic lymphocytic leukaemia", "status": "Positive", "designation_date": "2010-09-20", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-10-780", "last_updated": "2019-05-21" }, { "eu_designation_number": "EU/3/10/781", "medicine_name": "", "active_substance": "glutathione-pegylated liposomal doxorubicin hydrochloride", "intended_use": "Treatment of glioma", "status": "Withdrawn", "designation_date": "2010-09-20", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-10-781", "last_updated": "2015-05-08" }, { "eu_designation_number": "EU/3/10/782", "medicine_name": "", "active_substance": "nafamostat mesilate", "intended_use": "Treatment of cystic fibrosis", "status": "Positive", "designation_date": "2010-09-20", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-10-782", "last_updated": "2010-10-18" }, { "eu_designation_number": "EU/3/10/783", "medicine_name": "", "active_substance": "recombinant fusion protein consisting of human coagulation factor VIII attached to the Fc domain of human IgG1 (efmoroctocog alfa)", "intended_use": "Treatment of haemophilia A", "status": "Withdrawn", "designation_date": "2010-09-20", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-10-783", "last_updated": "2018-02-12" }, { "eu_designation_number": "EU/3/10/784", "medicine_name": "", "active_substance": "recombinant porcine factor VIII (B-domain-deleted)", "intended_use": "Treatment of haemophilia A", "status": "Withdrawn", "designation_date": "2010-09-20", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-10-784", "last_updated": "2016-01-19" }, { "eu_designation_number": "EU/3/10/785", "medicine_name": "", "active_substance": "vorinostat", "intended_use": "Treatment of malignant mesothelioma", "status": "Withdrawn", "designation_date": "2010-09-20", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-10-785", "last_updated": "2013-03-12" }, { "eu_designation_number": "EU/3/10/786", "medicine_name": "", "active_substance": "Heat-killed Mycobacterium vaccae (whole cell)", "intended_use": "Treatment of tuberculosis", "status": "Positive", "designation_date": "2010-09-20", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-10-786", "last_updated": "2022-03-08" }, { "eu_designation_number": "EU/3/10/787", "medicine_name": "", "active_substance": "(3S)-3-{4-[7-(aminocarbonyl)-2H-indazol-2-yl] phenyl} piperidine tosilate monohydrate salt", "intended_use": "Treatment of mantle cell lymphoma", "status": "Withdrawn", "designation_date": "2010-10-01", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-10-787", "last_updated": "2019-02-21" }, { "eu_designation_number": "EU/3/10/788", "medicine_name": "", "active_substance": "(S)-10-[(dimethylamino)methyl]-4-ethyl-9-hydroxy-4-O-[alpha-(2\", 4\", 5\", 7\"-tetranitro-9\"-fluorenylideneaminooxy)propionyl]-1H-pyrano[3', 4', 6', 7']indolizino[1,2-beta]-quinoline-3, 14-(4H, 12H)-dione, hydrochloride", "intended_use": "Treatment of hepatocellular carcinoma", "status": "Positive", "designation_date": "2010-10-01", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-10-788", "last_updated": "2010-10-14" }, { "eu_designation_number": "EU/3/10/789", "medicine_name": "", "active_substance": "16-base single-stranded peptide nucleic acid oligonucleotide linked to 7-amino acid peptide", "intended_use": "Treatment of medulloblastoma", "status": "Positive", "designation_date": "2010-10-01", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-10-789", "last_updated": "2014-04-01" }, { "eu_designation_number": "EU/3/10/790", "medicine_name": "", "active_substance": "ambrisentan", "intended_use": "Treatment idiopathic pulmonary fibrosis", "status": "Withdrawn", "designation_date": "2010-10-01", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-10-790", "last_updated": "2011-09-05" }, { "eu_designation_number": "EU/3/10/791", "medicine_name": "", "active_substance": "ciclosporin", "intended_use": "Treatment of moderate and severe closed traumatic brain injury", "status": "Positive", "designation_date": "2010-10-01", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-10-791", "last_updated": "2021-02-03" }, { "eu_designation_number": "EU/3/10/792", "medicine_name": "", "active_substance": "Maytansinoid-conjugated humanised monoclonal antibody against CD56", "intended_use": "Treatment of small cell lung cancer", "status": "Withdrawn", "designation_date": "2010-10-01", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-10-792", "last_updated": "2015-03-06" }, { "eu_designation_number": "EU/3/10/793", "medicine_name": "", "active_substance": "N-(6-(2-aminophenylamino)-6-oxohexyl)-4-methylbenzamide", "intended_use": "Treatment of Friedreich's ataxia", "status": "Positive", "designation_date": "2010-10-01", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-10-793", "last_updated": "2010-10-14" }, { "eu_designation_number": "EU/3/10/794", "medicine_name": "Inrebic", "active_substance": "N-tert-butyl-3-[(5-methyl-2-{[4-(2-pyrrolidin-1-ylethoxy)phenyl]amino}pyrimidin-4-yl)amino] benzenesulfonamide dihydrochloride monohydrate (fedratinib)", "intended_use": "Treatment of primary myelofibrosis", "status": "Positive", "designation_date": "2010-10-01", "related_ema_product_number": "EMEA/H/C/005026", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-10-794", "last_updated": "2021-09-17" }, { "eu_designation_number": "EU/3/10/795", "medicine_name": "", "active_substance": "pralatrexate", "intended_use": "Treatment of Hodgkin's lymphoma", "status": "Positive", "designation_date": "2010-10-01", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-10-795", "last_updated": "2010-10-14" }, { "eu_designation_number": "EU/3/10/796", "medicine_name": "", "active_substance": "recombinant humanised anti-human interleukin-1 beta monoclonal antibody", "intended_use": "Treatment of Behçet's Disease", "status": "Withdrawn", "designation_date": "2010-10-01", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-10-796", "last_updated": "2018-07-18" }, { "eu_designation_number": "EU/3/10/797", "medicine_name": "", "active_substance": "recombinant humanised monoclonal antibody to human Nogo-A protein of the IgG1/kappa class", "intended_use": "Treatment of amyotrophic lateral sclerosis", "status": "Withdrawn", "designation_date": "2010-10-01", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-10-797", "last_updated": "2016-01-19" }, { "eu_designation_number": "EU/3/10/798", "medicine_name": "", "active_substance": "synthetic double-stranded short interfering RNA oligonucleotide directed against proopiomelanocortin", "intended_use": "Treatment of adrenocorticotropin-dependent Cushing's syndrome", "status": "Positive", "designation_date": "2010-10-01", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-10-798", "last_updated": "2013-02-28" }, { "eu_designation_number": "EU/3/10/799", "medicine_name": "", "active_substance": "tecovirimat", "intended_use": "Treatment of cowpox infection", "status": "Withdrawn", "designation_date": "2010-10-01", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-10-799", "last_updated": "2012-03-28" }, { "eu_designation_number": "EU/3/10/802", "medicine_name": "", "active_substance": "2-(2-chlorophenyl)-4-[3-(dimethylamino)phenyl]-5-methyl-1H-pyrazolo[4,3-C]pyridine-3,6(2H,5H)-dione", "intended_use": "Treatment of idiopathic pulmonary fibrosis", "status": "Positive", "designation_date": "2010-11-26", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-10-802", "last_updated": "2026-08-21" }, { "eu_designation_number": "EU/3/10/803", "medicine_name": "Vyloy", "active_substance": "chimeric monoclonal antibody against claudin-18 splice variant 2", "intended_use": "Treatment of gastric cancer", "status": "Positive", "designation_date": "2010-11-26", "related_ema_product_number": "EMEA/H/C/005868", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-10-803", "last_updated": "2018-04-03" }, { "eu_designation_number": "EU/3/10/804", "medicine_name": "", "active_substance": 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"https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-10-806", "last_updated": "2019-03-28" }, { "eu_designation_number": "EU/3/10/807", "medicine_name": "", "active_substance": "Methylthioninium", "intended_use": "treatment of frontotemporal dementia with parkinsonism-17", "status": "Positive", "designation_date": "2010-11-26", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-10-807", "last_updated": "2019-03-28" }, { "eu_designation_number": "EU/3/10/808", "medicine_name": "Begedina", "active_substance": "murine monoclonal antibody against CD26", "intended_use": "Treatment of graft-versus-host disease", "status": "Positive", "designation_date": "2010-11-26", "related_ema_product_number": "EMEA/H/C/004144", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-10-808", "last_updated": "2014-01-22" }, { "eu_designation_number": "EU/3/10/809", "medicine_name": "", "active_substance": "nanoparticle albumin-bound paclitaxel", "intended_use": "Treatment of pancreatic cancer", "status": "Withdrawn", "designation_date": "2010-11-26", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-10-809", "last_updated": "2013-03-11" }, { "eu_designation_number": "EU/3/10/810", "medicine_name": "Inrebic", "active_substance": "N-tert-butyl-3-[(5-methyl-2-{[4-(2-pyrrolidin-1-ylethoxy)phenyl]amino}pyrimidin-4-yl)amino] benzenesulfonamide dihydrochloride monohydrate (fedratinib)", "intended_use": "Treatment of post-essential thrombocythaemia myelofibrosis", "status": "Positive", "designation_date": "2010-11-26", "related_ema_product_number": "EMEA/H/C/005026", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-10-810", "last_updated": "2021-09-17" }, { "eu_designation_number": "EU/3/10/811", "medicine_name": "Inrebic", "active_substance": 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"active_substance": "recombinant human arylsulfatase A", "intended_use": "treatment of metachromatic leukodystrophy", "status": "Positive", "designation_date": "2010-11-26", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-10-813", "last_updated": "2022-10-04" }, { "eu_designation_number": "EU/3/10/814", "medicine_name": "", "active_substance": "recombinant human von Willebrand factor", "intended_use": "Treatment of von Willebrand disease", "status": "Withdrawn", "designation_date": "2010-11-26", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-10-814", "last_updated": "2018-09-21" }, { "eu_designation_number": "EU/3/10/815", "medicine_name": "", "active_substance": "sildenafil citrate", "intended_use": "treatment of postcardiotomy right ventricular failure", "status": "Positive", "designation_date": "2010-11-26", "related_ema_product_number": "", "url": 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"eu_designation_number": "EU/3/10/818", "medicine_name": "Heparesc", "active_substance": "human heterologous liver cells", "intended_use": "Treatment of citrullinaemia type 1", "status": "Positive", "designation_date": "2009-10-08", "related_ema_product_number": "EMEA/H/C/003750", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-10-818", "last_updated": "2016-08-16" }, { "eu_designation_number": "EU/3/10/819", "medicine_name": "Heparesc", "active_substance": "human heterologous liver cells", "intended_use": "Treatment of hyperargininaemia", "status": "Positive", "designation_date": "2010-12-17", "related_ema_product_number": "EMEA/H/C/003750", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-10-819", "last_updated": "2016-08-16" }, { "eu_designation_number": "EU/3/10/820", "medicine_name": "Heparesc", "active_substance": "human heterologous liver cells", "intended_use": "Treatment of argininosuccinic aciduria", "status": 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"active_substance": "ovine anti-colchicine polyclonal antibody fragments", "intended_use": "Treatment of colchicine poisoning", "status": "Withdrawn", "designation_date": "2010-12-17", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-10-825", "last_updated": "2026-05-26" }, { "eu_designation_number": "EU/3/10/826", "medicine_name": "", "active_substance": "para-aminosalicylic acid", "intended_use": "Treatment of tuberculosis", "status": "Positive", "designation_date": "2010-12-17", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-10-826", "last_updated": "2018-06-08" }, { "eu_designation_number": "EU/3/10/827", "medicine_name": "Kanuma", "active_substance": "recombinant human lysosomal acid lipase", "intended_use": "Treatment of lysosomal acid lipase deficiency", "status": "Positive", "designation_date": "2010-12-17", "related_ema_product_number": 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"active_substance": "veliparib", "intended_use": "Treatment of ovarian cancer", "status": "Withdrawn", "designation_date": "2010-12-17", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-10-830", "last_updated": "2024-04-09" }, { "eu_designation_number": "EU/3/10/831", "medicine_name": "", "active_substance": "autologous tumour-derived immunoglobulin idiotype coupled to keyhole limpet hemocyanin", "intended_use": "Treatment of mantle cell lymphoma", "status": "Positive", "designation_date": "2011-02-23", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-10-831", "last_updated": "2011-03-04" }, { "eu_designation_number": "EU/3/10/832", "medicine_name": "", "active_substance": "Deferiprone", "intended_use": "Treatment of sickle cell disease", "status": "Positive", "designation_date": "2011-02-23", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-10-832", "last_updated": "2020-02-04" }, { "eu_designation_number": "EU/3/10/833", "medicine_name": "", "active_substance": "doxorubicin hydrochloride", "intended_use": "Treatment of hepatocellular carcinoma", "status": "Positive", "designation_date": "2011-02-23", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-10-833", "last_updated": "2019-01-22" }, { "eu_designation_number": "EU/3/10/834", "medicine_name": "", "active_substance": "Human plasmin", "intended_use": "Treatment of acute peripheral arterial occlusion", "status": "Withdrawn", "designation_date": "2011-02-23", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-10-834", "last_updated": "2018-03-12" }, { "eu_designation_number": "EU/3/10/835", "medicine_name": "", "active_substance": "Maytansinoid-conjugated humanised monoclonal 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}, { "eu_designation_number": "EU/3/10/838", "medicine_name": "", "active_substance": "plitidepsin", "intended_use": "Treatment of post-essential thrombocythaemia myelofibrosis", "status": "Withdrawn", "designation_date": "2011-02-23", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-10-838", "last_updated": "2011-09-06" }, { "eu_designation_number": "EU/3/10/839", "medicine_name": "", "active_substance": "plitidepsin", "intended_use": "Treatment of post-polycythaemia vera myelofibrosis", "status": "Withdrawn", "designation_date": "2011-02-23", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-10-839", "last_updated": "2011-09-06" }, { "eu_designation_number": "EU/3/10/840", "medicine_name": "", "active_substance": "recombinant human histone H1.3 and recombinant human N-bis-met-histone H1.3", "intended_use": "Treatment of acute lymphoblastic leukaemia", "status": "Positive", "designation_date": "2011-02-23", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-10-840", "last_updated": "2013-03-13" }, { "eu_designation_number": "EU/3/10/841", "medicine_name": "", "active_substance": "tasimelteon", "intended_use": "Treatment of non-24-hour sleep-wake disorders in blind people with no light perception", "status": "Expired", "designation_date": "2011-02-23", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-10-841", "last_updated": "2025-07-09" }, { "eu_designation_number": "EU/3/10/842", "medicine_name": "", "active_substance": "nimorazole", "intended_use": "Treatment of squamous-cell carcinoma of the head and neck in patients undergoing radiotherapy", "status": "Withdrawn", "designation_date": "2011-02-23", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-10-842", "last_updated": "2026-05-18" }, { "eu_designation_number": "EU/3/10/843", "medicine_name": "", "active_substance": "allogeneic aortic endothelial cells cultured in a porcine gelatin matrix", "intended_use": "Prevention of arteriovenous access failure in haemodialysis patients", "status": "Withdrawn", "designation_date": "2011-02-23", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-10-843", "last_updated": "2013-03-11" }, { "eu_designation_number": "EU/3/10/844", "medicine_name": "", "active_substance": "axitinib", "intended_use": "Treatment of renal-cell carcinoma", "status": "Withdrawn", "designation_date": "2011-02-23", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-10-844", "last_updated": "2017-02-10" }, { "eu_designation_number": "EU/3/10/845", "medicine_name": "Filsuvez", "active_substance": "dry extract from birch bark (DER 5-10 : 1), extraction solvent n-heptane 95% (w/w) (birch bark extract)", "intended_use": "Treatment of epidermolysis bullosa", "status": "Positive", "designation_date": "2011-02-23", "related_ema_product_number": "EMEA/H/C/005035", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-10-845", "last_updated": "2024-10-22" }, { "eu_designation_number": "EU/3/10/846", "medicine_name": "", "active_substance": "paclitaxel", "intended_use": "Treatment of oesophagus carcinoma", "status": "Positive", "designation_date": "2011-02-23", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-10-846", "last_updated": "2011-03-04" }, { "eu_designation_number": "EU/3/10/847", "medicine_name": "", "active_substance": "pegylated B-domain-deleted sequence-modified recombinant human factor VIII (damoctocog alfa pegol)", "intended_use": "Treatment of haemophilia A", "status": "Withdrawn", "designation_date": "2011-02-23", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-10-847", "last_updated": "2019-01-28" }, { "eu_designation_number": "EU/3/10/848", "medicine_name": "", "active_substance": "sodium thiosulfate", "intended_use": "Treatment of calciphylaxis", "status": "Withdrawn", "designation_date": "2011-02-23", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-10-848", "last_updated": "2021-02-03" }, { "eu_designation_number": "EU/3/11/849", "medicine_name": "Prevymis", "active_substance": "(S)-{8-Fluoro-2-2[4-(3-methoxyphenyl)-1-piperazinyl]-3-[2-methoxy-5-(trifluoromethyl)-phenyl]-3, 4-dihydro-4-quinazolinyl} acetic acid (letermovir)", "intended_use": "Prevention of cytomegalovirus disease in patients with impaired cell-mediated immunity", "status": "Positive", "designation_date": "2011-04-15", "related_ema_product_number": "EMEA/H/C/004536", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-11-849", "last_updated": "2018-06-28" }, { "eu_designation_number": "EU/3/11/850", "medicine_name": "", "active_substance": "Darinaparsin", "intended_use": "Treatment of peripheral T-cell lymphoma (nodal, other extranodal and leukaemic/disseminated)", "status": "Positive", "designation_date": "2011-04-15", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-11-850", "last_updated": "2022-07-14" }, { "eu_designation_number": "EU/3/11/851", "medicine_name": "", "active_substance": "Glufosfamide", "intended_use": "Treatment of pancreatic cancer", "status": "Positive", "designation_date": "2011-04-15", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-11-851", "last_updated": "2015-03-23" }, { "eu_designation_number": "EU/3/11/852", "medicine_name": "", "active_substance": "human anthrax monoclonal 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"eu_designation_number": "EU/3/11/855", "medicine_name": "", "active_substance": "recombinant fusion protein linking human coagulation factor VIIa with human albumin", "intended_use": "Treatment of haemophilia A", "status": "Positive", "designation_date": "2011-04-15", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-11-855", "last_updated": "2018-03-28" }, { "eu_designation_number": "EU/3/11/856", "medicine_name": "", "active_substance": "recombinant thymidine phosphorylase encapsulated in autologous erythrocytes", "intended_use": "Treatment of mitochondrial neurogastrointestinal encephalomyopathy (MNGIE)", "status": "Positive", "designation_date": "2011-04-15", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-11-856", "last_updated": "2011-04-26" }, { "eu_designation_number": "EU/3/11/857", "medicine_name": "Onpattro", "active_substance": "synthetic double-stranded siRNA oligonucleotide directed against transthyretin mRNA (also known as patisiran)", "intended_use": "Treatment of transthyretin-mediated amyloidosis", "status": "Positive", "designation_date": "2017-04-06", "related_ema_product_number": "EMEA/H/C/004699", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-11-857", "last_updated": "2018-05-30" }, { "eu_designation_number": "EU/3/11/858", "medicine_name": "", "active_substance": "R-baclofen", "intended_use": "Treatment of fragile X syndrome", "status": "Positive", "designation_date": "2011-04-15", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-11-858", "last_updated": "2011-04-26" }, { "eu_designation_number": "EU/3/11/859", "medicine_name": "", "active_substance": "[N-((2S,3R,3aS,3'R,4a'R,6S,6a'R,6b'S,7aR,12a'S,12b'S,Z)-3,6,11',12b'-tetramethyl-2',3a,3',4,4',4a',5,5',6,6',6a',6b',7,7a,7',8',10',12',12a',12b'-icosahydro-1'H,3H-spiro[furo[3,2-b]pyridine-2,9'-naphtho[2,1-a]azulene]-3'-yl)methanesulfonamide hydrochloride]", "intended_use": "Treatment of chondrosarcoma", "status": "Withdrawn", "designation_date": "2011-05-13", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-11-859", "last_updated": "2013-07-31" }, { "eu_designation_number": "EU/3/11/860", "medicine_name": "Lumevoq", "active_substance": "adeno-associated viral vector containing the human NADH-dehydrogenase-4 gene", "intended_use": "Treatment of Leber's hereditary optic neuropathy", "status": "Positive", "designation_date": "2011-05-13", "related_ema_product_number": "EMEA/H/C/005047", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-11-860", "last_updated": "2016-04-11" }, { "eu_designation_number": "EU/3/11/861", "medicine_name": "", "active_substance": "9-cis-Retinyl acetate", "intended_use": "Treatment of Leber's congenital amaurosis", "status": "Positive", "designation_date": "2011-05-13", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-11-861", "last_updated": "2019-05-13" }, { "eu_designation_number": "EU/3/11/862", "medicine_name": "", "active_substance": "apomorphine hydrochloride", "intended_use": "Treatment of moderate and severe traumatic brain injury", "status": "Positive", "designation_date": "2011-05-13", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-11-862", "last_updated": "2011-05-27" }, { "eu_designation_number": "EU/3/11/863", "medicine_name": "", "active_substance": "recombinant fusion protein linking human coagulation factor VIIa with human albumin", "intended_use": "Treatment of haemophilia B", "status": 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"medicine_name": "", "active_substance": "dextran sulfate", "intended_use": "Treatment for mobilisation of progenitor cells prior to stem cell transplantation", "status": "Positive", "designation_date": "2011-08-05", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-11-883", "last_updated": "2011-11-18" }, { "eu_designation_number": "EU/3/11/884", "medicine_name": "", "active_substance": "Methyl O-4-O-[2-[2-[2-[2-[[N-[(1R)-1-[[4-(aminoiminomethyl)phenyl]methyl]-2-oxo-2-(1-piperidinyl)ethyl]-N2-[(4-methoxy-2,3,6-trimethylphenyl)sulfonyl]-L-α-asparaginyl-4-aminobutanoyl-N6-[5-[(3aS,4S,6aR)-hexahydro-2-oxo-1H-thieno[3,4-d]imidazol-4-yl]-1-oxopentyl]-L-lysyl]amino]ethoxy]ethoxy]ethoxy]ethyl]-2,3-di-O-methyl-6-O-sulfo-α-D-glucopyranosyl....", "intended_use": "Prevention of ischaemia/reperfusion injury associated with solid organ transplantation", "status": "Positive", "designation_date": "2011-08-05", 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"https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-11-915", "last_updated": "2015-06-08" }, { "eu_designation_number": "EU/3/11/916", "medicine_name": "", "active_substance": "2-Hydroxyoleic acid", "intended_use": "Treatment of glioma", "status": "Positive", "designation_date": "2011-10-27", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-11-916", "last_updated": "2021-02-03" }, { "eu_designation_number": "EU/3/11/917", "medicine_name": "", "active_substance": "adeno-associated viral vector containing the human alpha-N-acetylglucosaminidase gene", "intended_use": "Treatment of mucopolysaccharidosis type IIIB (Sanfilippo B syndrome)", "status": "Positive", "designation_date": "2011-10-27", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-11-917", "last_updated": "2011-11-23" }, { "eu_designation_number": "EU/3/11/918", "medicine_name": "", 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"https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-11-922", "last_updated": "2026-05-26" }, { "eu_designation_number": "EU/3/11/923", "medicine_name": "", "active_substance": "L-cysteine, L-leucyl-L-alpha-glutamyl-L-alpha-glutamyl-L-lysyl-L-lysylglycyl-L-asparaginyl-L-tyrosyl-L-valyl-L-valyl-L-threonyl-L-alpha-aspartyl-L-histidyl-S-[1-[(4-carboxycyclohexyl)methyl]-2,5-dioxo-3-pyrrolidinyl]-complex with keyhole limpet haemocyanin", "intended_use": "Treatment of glioma", "status": "Withdrawn", "designation_date": "2011-10-27", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-11-923", "last_updated": "2020-12-04" }, { "eu_designation_number": "EU/3/11/924", "medicine_name": "", "active_substance": "lenalidomide", "intended_use": "Treatment of mantle cell lymphoma", "status": "Withdrawn", "designation_date": "2011-10-27", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-11-924", "last_updated": "2020-01-13" }, { "eu_designation_number": "EU/3/11/925", "medicine_name": "Corluxin", "active_substance": "mifepristone", "intended_use": "Treatment of hypercortisolism (Cushing's syndrome) of endogenous origin", "status": "Positive", "designation_date": "2011-10-27", "related_ema_product_number": "EMEA/H/C/002830", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-11-925", "last_updated": "2022-03-18" }, { "eu_designation_number": "EU/3/11/926", "medicine_name": "", "active_substance": "recombinant human minibody against complement component C5", "intended_use": "Treatment of primary membranoproliferative glomerulonephritis", "status": "Withdrawn", "designation_date": "2011-10-27", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-11-926", "last_updated": "2019-04-12" }, { "eu_designation_number": "EU/3/11/927", "medicine_name": "", "active_substance": "sinapultide;Dipalmitoylphosphatidylcholine;palmitoyl-oleoyl phosphatidylglycerol sodium salt;palmitic acid", "intended_use": "Treatment of cystic fibrosis", "status": "Positive", "designation_date": "2011-10-27", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-11-927", "last_updated": "2019-05-22" }, { "eu_designation_number": "EU/3/11/928", "medicine_name": "", "active_substance": "cysteamine", "intended_use": "Treatment of cystic fibrosis", "status": "Positive", "designation_date": "2011-12-09", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-11-928", "last_updated": "2019-05-21" }, { "eu_designation_number": "EU/3/11/929", "medicine_name": "", "active_substance": "adeno-associated viral vector serotype 8 containing the human AIPL1 gene", "intended_use": "Treatment of Leber's congenital amaurosis type 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"EU/3/11/932", "medicine_name": "", "active_substance": "pegylated proline-interferon alpha-2b (ropeginterferon alfa-2b)", "intended_use": "Treatment of polycythaemia vera", "status": "Withdrawn", "designation_date": "2011-12-09", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-11-932", "last_updated": "2019-05-08" }, { "eu_designation_number": "EU/3/11/933", "medicine_name": "Onivyde pegylated liposomal (previously Onivyde)", "active_substance": "nanoliposomal irinotecan", "intended_use": "Treatment of pancreatic cancer", "status": "Positive", "designation_date": "2011-12-09", "related_ema_product_number": "EMEA/H/C/004125", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-11-933", "last_updated": "2018-12-17" }, { "eu_designation_number": "EU/3/11/934", "medicine_name": "", "active_substance": "4-[[9-[(3S)-tetrahydro-3-furanyl]-8-[(2,4,6-trifluorophenyl)amino]-9H-purin-2-yl]amino]-trans-cyclohexanol (tanzisertib)", "intended_use": "Treatment of idiopathic pulmonary fibrosis", "status": "Withdrawn", "designation_date": "2011-12-09", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-11-934", "last_updated": "2018-02-22" }, { "eu_designation_number": "EU/3/11/935", "medicine_name": "", "active_substance": "Interferon gamma", "intended_use": "Treatment of Friedreich's ataxia", "status": "Withdrawn", "designation_date": "2011-12-09", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-11-935", "last_updated": "2025-09-04" }, { "eu_designation_number": "EU/3/11/936", "medicine_name": "", "active_substance": "human haptoglobin", "intended_use": "Treatment of sickle cell disease", "status": "Positive", "designation_date": "2011-12-09", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-11-936", "last_updated": "2019-03-18" }, { "eu_designation_number": "EU/3/11/937", "medicine_name": "", "active_substance": "alpha-tocotrienol quinone", "intended_use": "Treatment of Leigh syndrome", "status": "Withdrawn", "designation_date": "2011-12-09", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-11-937", "last_updated": "2026-05-18" }, { "eu_designation_number": "EU/3/11/938", "medicine_name": "", "active_substance": "adeno-associated viral vector containing the human factor IX gene", "intended_use": "Treatment of haemophilia B", "status": "Positive", "designation_date": "2012-01-11", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-11-938", "last_updated": "2021-08-06" }, { "eu_designation_number": "EU/3/11/939", "medicine_name": "", "active_substance": "Brentuximab vedotin", "intended_use": "Treatment of cutaneous T-cell lymphoma", "status": "Withdrawn", "designation_date": "2012-01-11", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-11-939", "last_updated": "2024-06-26" }, { "eu_designation_number": "EU/3/11/940", "medicine_name": "", "active_substance": "chimeric locked nucleic acid-deoxynucleoside phosphorothioate-linked oligonucleotide directed against microRNA-451", "intended_use": "Treatment of polycythaemia vera", "status": "Withdrawn", "designation_date": "2012-01-11", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-11-940", "last_updated": "2017-05-17" }, { "eu_designation_number": "EU/3/11/941", "medicine_name": "", "active_substance": "Lipopolysaccharide of Ochrobactrum intermedium", "intended_use": "Prevention of sepsis in at-risk premature infants of less than or equal to 32 weeks", "status": "Positive", "designation_date": "2012-01-11", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-11-941", "last_updated": "2014-10-08" }, { "eu_designation_number": "EU/3/11/942", "medicine_name": "Vyxeos liposomal (previously Vyxeos)", "active_substance": "cytarabine;daunorubicin", "intended_use": "Treatment of acute myeloid leukaemia", "status": "Positive", "designation_date": "2012-01-11", "related_ema_product_number": "EMEA/H/C/004282", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-11-942", "last_updated": "2014-10-08" }, { "eu_designation_number": "EU/3/11/943", "medicine_name": "", "active_substance": "mogamulizumab", "intended_use": "Treatment of peripheral T-cell lymphoma (nodal, other extranodal and leukaemic/disseminated)", "status": "Positive", "designation_date": "2012-01-11", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-11-943", "last_updated": "2018-10-18" }, { "eu_designation_number": "EU/3/11/944", "medicine_name": "", "active_substance": "N,N'-bis(2-mercaptoethyl)isophthalamide (emeramide)", "intended_use": "Treatment of mercury toxicity", "status": "Positive", "designation_date": "2012-01-11", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-11-944", "last_updated": "2017-08-16" }, { "eu_designation_number": "EU/3/11/945", "medicine_name": "", "active_substance": "ornithine phenylacetate", "intended_use": "Treatment of acute liver failure", "status": "Positive", "designation_date": "2025-02-20", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-11-945", "last_updated": "2025-03-12" }, { "eu_designation_number": "EU/3/11/946", "medicine_name": "", "active_substance": "recombinant homodimer of the human annexin V", "intended_use": "Prevention of ischaemia/reperfusion injury associated with solid organ transplantation", "status": "Withdrawn", "designation_date": "2012-01-11", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-11-946", "last_updated": "2014-04-01" }, { "eu_designation_number": "EU/3/11/947", "medicine_name": "", "active_substance": "recombinant protein consisting of modified human growth hormone releasing hormone and the translocation and endopeptidase domains of botulinum toxin serotype D", "intended_use": "Treatment of acromegaly", "status": "Withdrawn", "designation_date": "2012-01-11", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-11-947", "last_updated": "2019-05-23" }, { "eu_designation_number": "EU/3/11/948", "medicine_name": "", "active_substance": "sodium phenylbutyrate", "intended_use": "Treatment of 5q spinal muscular atrophy", "status": "Withdrawn", "designation_date": "2012-01-11", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-11-948", "last_updated": "2017-07-17" }, { "eu_designation_number": "EU/3/12/1000", "medicine_name": "", "active_substance": "polyinosine-polycytidylic acid coupled with the polycationic polyethyleneimine", "intended_use": "Treatment of pancreatic cancer", "status": "Withdrawn", "designation_date": "2012-06-06", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-12-1000", "last_updated": "2013-09-23" }, { "eu_designation_number": "EU/3/12/1001", "medicine_name": "", "active_substance": "1-(4-{4-Amino-7-[1-(2-hydroxyethyl)-1H-pyrazol-4-yl]thieno[3,2-c]pyridin-3-yl}phenyl)-3-(3-fluorophenyl)urea", "intended_use": "Treatment of ovarian cancer", "status": "Withdrawn", "designation_date": "2012-06-06", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-12-1001", "last_updated": "2015-06-08" }, { "eu_designation_number": "EU/3/12/1002", "medicine_name": "", "active_substance": "adenovirus-associated vector containing human Fas-c gene (ofranergene obadenovec)", "intended_use": "Treatment of glioma", "status": "Withdrawn", "designation_date": "2012-06-06", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-12-1002", "last_updated": "2022-09-26" }, { "eu_designation_number": "EU/3/12/1003", "medicine_name": "", "active_substance": "autologous haematopoietic stem cells transduced with lentiviral vector Lenti-D encoding the human ABCD1 cDNA (elivaldogene autotemcel)", "intended_use": "Treatment of adrenoleukodystrophy", "status": "Withdrawn", "designation_date": "2012-06-06", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-12-1003", "last_updated": "2022-04-04" }, { "eu_designation_number": "EU/3/12/1004", "medicine_name": "", "active_substance": "ramucirumab", "intended_use": "Treatment of gastric cancer", "status": "Withdrawn", "designation_date": "2012-07-04", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-12-1004", "last_updated": "2016-01-19" }, { "eu_designation_number": "EU/3/12/1005", "medicine_name": "", "active_substance": "talarozole", "intended_use": "Treatment of autosomal recessive congenital ichthyosis", "status": "Withdrawn", "designation_date": "2012-07-04", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-12-1005", "last_updated": "2016-01-19" }, { "eu_designation_number": "EU/3/12/1006", "medicine_name": "", "active_substance": "Doxorubicin", "intended_use": "Treatment of glioma", "status": "Withdrawn", "designation_date": "2012-11-28", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-12-1006", "last_updated": "2017-05-17" }, { "eu_designation_number": "EU/3/12/1007", "medicine_name": "", "active_substance": "17-(Dimethylaminoethylamino)-17-demethoxygeldanamycin", "intended_use": "Treatment of retinitis pigmentosa", "status": "Withdrawn", "designation_date": "2012-11-28", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-12-1007", "last_updated": "2016-08-11" }, { "eu_designation_number": "EU/3/12/1008", "medicine_name": "", "active_substance": "human erythrocytes encapsulating inositol hexaphosphate", "intended_use": "Treatment of sickle cell disease", "status": "Positive", "designation_date": "2012-07-04", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-12-1008", "last_updated": "2012-07-24" }, { "eu_designation_number": "EU/3/12/1009", "medicine_name": "Duvyzat", 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"Treatment of keratinopathic ichthyosis", "status": "Withdrawn", "designation_date": "2012-07-04", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-12-1014", "last_updated": "2016-01-19" }, { "eu_designation_number": "EU/3/12/1015", "medicine_name": "", "active_substance": "Eculizumab", "intended_use": "Treatment of infection-associated haemolytic uraemic syndrome", "status": "Withdrawn", "designation_date": "2012-07-04", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-12-1015", "last_updated": "2016-01-19" }, { "eu_designation_number": "EU/3/12/1016", "medicine_name": "", "active_substance": "16-base single-stranded peptide nucleic acid oligonucleotide linked to 7-amino acid peptide", "intended_use": "Treatment of neuroblastoma", "status": "Positive", "designation_date": "2012-07-04", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-12-1016", "last_updated": "2014-04-01" }, { "eu_designation_number": "EU/3/12/1017", "medicine_name": "", "active_substance": "talarozole", "intended_use": "Treatment of recessive X-linked ichthyosis", "status": "Withdrawn", "designation_date": "2012-07-04", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-12-1017", "last_updated": "2016-01-19" }, { "eu_designation_number": "EU/3/12/1018", "medicine_name": "", "active_substance": "recombinant adeno-associated viral vector containing human acid alfa-glucosidase-gene", "intended_use": "Treatment of glycogen storage disease type II (Pompe's disease)", "status": "Positive", "designation_date": "2012-07-04", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-12-1018", "last_updated": "2019-06-18" }, { "eu_designation_number": "EU/3/12/1019", "medicine_name": "", "active_substance": "ramucirumab", "intended_use": "Treatment of hepatocellular carcinoma", "status": "Withdrawn", "designation_date": "2012-07-04", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-12-1019", "last_updated": "2016-08-11" }, { "eu_designation_number": "EU/3/12/1020", "medicine_name": "", "active_substance": "recombinant human pentraxin-2", "intended_use": "Treatment of idiopathic pulmonary fibrosis", "status": "Positive", "designation_date": "2012-07-17", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-12-1020", "last_updated": "2020-04-24" }, { "eu_designation_number": "EU/3/12/1021", "medicine_name": "", "active_substance": "1-[(2-Chloro-4-methoxyphenoxy)methyl]-4-[(2,6-dichlorophenoxy)methyl]benzene", "intended_use": "Prevention of poliomyelitis in patients with immunodeficiencies deemed at risk", "status": "Positive", 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"EU/3/12/1024", "medicine_name": "Myalepta", "active_substance": "Metreleptin", "intended_use": "Treatment of Lawrence syndrome", "status": "Positive", "designation_date": "2012-07-17", "related_ema_product_number": "EMEA/H/C/004218", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-12-1024", "last_updated": "2024-10-22" }, { "eu_designation_number": "EU/3/12/1025", "medicine_name": "Myalepta", "active_substance": "Metreleptin", "intended_use": "Treatment of Berardinelli-Seip syndrome", "status": "Positive", "designation_date": "2012-07-17", "related_ema_product_number": "EMEA/H/C/004218", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-12-1025", "last_updated": "2024-10-22" }, { "eu_designation_number": "EU/3/12/1026", "medicine_name": "", "active_substance": "Hexasodium phytate", "intended_use": "Treatment of calciphylaxis", "status": "Positive", "designation_date": "2012-07-17", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-12-1026", "last_updated": "2022-11-22" }, { "eu_designation_number": "EU/3/12/1027", "medicine_name": "", "active_substance": "human apotransferrin", "intended_use": "Treatment of congenital hypotransferrinaemia", "status": "Positive", "designation_date": "2012-07-17", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-12-1027", "last_updated": "2016-08-15" }, { "eu_designation_number": "EU/3/12/1028", "medicine_name": "Bylvay", "active_substance": "", "intended_use": "Treatment of progressive familial intrahepatic cholestasis", "status": "Positive", "designation_date": "2012-07-17", "related_ema_product_number": "EMEA/H/C/004691", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-12-1028", "last_updated": "2024-10-11" }, { "eu_designation_number": "EU/3/12/1029", "medicine_name": "", "active_substance": "Humanised monoclonal 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"https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-12-1031", "last_updated": "2012-09-26" }, { "eu_designation_number": "EU/3/12/1032", "medicine_name": "", "active_substance": "vatreptacog alfa (activated)", "intended_use": "Treatment of haemophilia B", "status": "Withdrawn", "designation_date": "2012-08-09", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-12-1032", "last_updated": "2015-03-06" }, { "eu_designation_number": "EU/3/12/1033", "medicine_name": "", "active_substance": "N-butyldeoxygalactonojirimycin", "intended_use": "Treatment of Fabry disease", "status": "Positive", "designation_date": "2012-08-09", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-12-1033", "last_updated": "2015-03-24" }, { "eu_designation_number": "EU/3/12/1034", "medicine_name": "", "active_substance": "Humanised monoclonal antibody against P-selectin (crizanlizumab)", "intended_use": "Treatment of sickle cell disease", "status": "Withdrawn", "designation_date": "2012-08-09", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-12-1034", "last_updated": "2026-05-26" }, { "eu_designation_number": "EU/3/12/1035", "medicine_name": "", "active_substance": "recombinant human monoclonal antibody against activin receptor type IIB", "intended_use": "Treatment of inclusion body myositis", "status": "Withdrawn", "designation_date": "2012-08-09", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-12-1035", "last_updated": "2018-10-24" }, { "eu_designation_number": "EU/3/12/1036", "medicine_name": "", "active_substance": "trans-4-[4-[5-[[6-(trifluoromethyl)-3-pyridinyl]amino]-2-pyridinyl]phenyl] cyclohexane acetic acid sodium salt", "intended_use": "Treatment of familial chylomicronaemia syndrome (type-I hyperlipoproteinaemia)", "status": "Withdrawn", "designation_date": "2012-09-14", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-12-1036", "last_updated": "2019-02-21" }, { "eu_designation_number": "EU/3/12/1037", "medicine_name": "", "active_substance": "Elotuzumab", "intended_use": "Treatment of multiple myeloma", "status": "Withdrawn", "designation_date": "2012-08-09", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-12-1037", "last_updated": "2016-05-23" }, { "eu_designation_number": "EU/3/12/1038", "medicine_name": "", "active_substance": "recombinant anti-CD3-bi-single-chain-Fv-diphtheria toxin fusion protein", "intended_use": "Treatment of peripheral T-cell lymphoma (nodal, other extranodal and leukaemic/disseminated)", "status": "Positive", "designation_date": "2012-08-09", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-12-1038", "last_updated": "2022-02-04" }, { "eu_designation_number": "EU/3/12/1039", "medicine_name": "", "active_substance": "recombinant anti-CD3-bi-single-chain-Fv-diphtheria toxin fusion protein", "intended_use": "Treatment of cutaneous T-cell lymphoma", "status": "Positive", "designation_date": "2012-08-09", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-12-1039", "last_updated": "2022-02-04" }, { "eu_designation_number": "EU/3/12/1040", "medicine_name": "Bylvay", "active_substance": "", "intended_use": "Treatment of Alagille syndrome", "status": "Withdrawn", "designation_date": "2012-08-09", "related_ema_product_number": "EMEA/H/C/004691", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-12-1040", "last_updated": "2026-05-26" }, { "eu_designation_number": "EU/3/12/1041", "medicine_name": "", "active_substance": "", "intended_use": "Treatment of primary biliary cirrhosis", "status": "Positive", "designation_date": "2012-08-09", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-12-1041", "last_updated": "2012-09-26" }, { "eu_designation_number": "EU/3/12/1042", "medicine_name": "", "active_substance": "Covalently closed DNA plasmids coding for cytomegalovirus phosphoprotein 65 and glycoprotein B genes", "intended_use": "Prevention of cytomegalovirus disease in patients with impaired cell-mediated immunity", "status": "Withdrawn", "designation_date": "2012-08-09", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-12-1042", "last_updated": "2019-02-21" }, { "eu_designation_number": "EU/3/12/1043", "medicine_name": "Folcepri", "active_substance": "N-[4-[[(2-amino-3,4-dihydro-4-oxo-6-pteridinyl)methyl]amino]benzoyl]-D-gamma-glutamyl-(2S)-2-amino-beta-alanyl-L-alpha-aspartyl-L-cysteine (etarfolatide)", "intended_use": "Diagnosis of positive folate-receptor status in ovarian cancer", "status": "Withdrawn", "designation_date": "2012-09-10", "related_ema_product_number": "EMEA/H/C/002570", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-12-1043", "last_updated": "2021-12-07" }, { "eu_designation_number": "EU/3/12/1044", "medicine_name": "", "active_substance": "folic acid", "intended_use": "Diagnosis of positive folate-receptor status in ovarian cancer", "status": "Withdrawn", "designation_date": "2012-09-10", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-12-1044", "last_updated": "2017-02-07" }, { "eu_designation_number": "EU/3/12/1045", "medicine_name": "", "active_substance": "alpha-1 proteinase inhibitor", "intended_use": "Treatment of cystic fibrosis", "status": "Positive", "designation_date": "2012-10-10", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-12-1045", "last_updated": "2012-11-16" }, { "eu_designation_number": "EU/3/12/1046", "medicine_name": "", "active_substance": "Mavoglurant ", "intended_use": "Treatment of fragile X syndrome", "status": "Withdrawn", "designation_date": "2012-10-10", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-12-1046", "last_updated": "2026-05-26" }, { "eu_designation_number": "EU/3/12/1047", "medicine_name": "", "active_substance": "Asp-Arg-Val-Tyr-Ile-His-Pro", "intended_use": "Treatment of acute lung Injury", "status": "Withdrawn", "designation_date": "2012-10-10", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-12-1047", "last_updated": "2016-01-19" }, { "eu_designation_number": "EU/3/12/1048", "medicine_name": "", "active_substance": "mixture of two allogeneic human pancreatic cancer cell lines stably transduced with a retroviral vector encoding the murine alpha-(1,3)-galactosyltransferase gene", "intended_use": "Treatment of pancreatic cancer", "status": "Withdrawn", "designation_date": "2012-10-10", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-12-1048", "last_updated": "2018-01-23" }, { "eu_designation_number": "EU/3/12/1049", "medicine_name": "", "active_substance": "rucaparib", "intended_use": "Treatment of ovarian cancer", "status": "Withdrawn", "designation_date": "2012-10-10", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-12-1049", "last_updated": "2019-02-19" }, { "eu_designation_number": "EU/3/12/1050", "medicine_name": "", "active_substance": "[2-Cyano-3-cyclopropyl-3-hydroxy-N-(3-methyl-4-trifluoromethylphenyl)prop-2-enamide]", "intended_use": "Treatment of traumatic spinal cord injury", "status": "Positive", "designation_date": "2012-10-10", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-12-1050", "last_updated": "2012-11-16" }, { "eu_designation_number": "EU/3/12/1051", "medicine_name": "", "active_substance": "recombinant human lecithin cholesterol acyltransferase", "intended_use": "Treatment of lecithin-cholesterol-acyltransferase deficiency", "status": "Withdrawn", "designation_date": "2012-10-10", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-12-1051", "last_updated": "2016-08-15" }, { "eu_designation_number": "EU/3/12/1052", "medicine_name": "", "active_substance": "Humanised monoclonal IgG4 antibody against tissue-factor-pathway inhibitor (concizumab)", "intended_use": "Treatment of haemophilia A", "status": "Withdrawn", "designation_date": "2012-10-10", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-12-1052", "last_updated": "2022-12-14" }, { "eu_designation_number": "EU/3/12/1053", "medicine_name": "", "active_substance": "lurbinectedin", "intended_use": "Treatment of ovarian cancer", "status": "Positive", "designation_date": "2012-10-10", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-12-1053", "last_updated": "2012-01-01" }, { "eu_designation_number": "EU/3/12/1054", "medicine_name": "", "active_substance": "obinutuzumab", "intended_use": "Treatment of chronic lymphocytic leukaemia", "status": "Positive", "designation_date": "2012-10-10", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-12-1054", "last_updated": "2018-02-21" }, { "eu_designation_number": "EU/3/12/1055", "medicine_name": "", "active_substance": "belinostat", "intended_use": "Treatment of peripheral T-cell lymphoma (nodal, other extranodal and leukaemic/disseminated)", "status": "Positive", "designation_date": "2012-10-10", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-12-1055", "last_updated": "2016-02-08" }, { "eu_designation_number": "EU/3/12/1056", "medicine_name": "", "active_substance": "liposomal daunorubicin", "intended_use": "Treatment of acute myeloid leukaemia", "status": "Withdrawn", "designation_date": "2012-10-10", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-12-1056", "last_updated": "2016-01-19" }, { "eu_designation_number": "EU/3/12/1057", "medicine_name": "", "active_substance": "naloxone hydrochloride dihydrate", "intended_use": "Treatment of cutaneous T-cell lymphoma", "status": "Positive", "designation_date": "2012-11-08", 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"EU/3/12/1077", "medicine_name": "", "active_substance": "exon 52 specific phosphorothioate oligonucleotide", "intended_use": "Treatment of Duchenne muscular dystrophy", "status": "Positive", "designation_date": "2012-12-06", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-12-1077", "last_updated": "2016-12-05" }, { "eu_designation_number": "EU/3/12/1078", "medicine_name": "", "active_substance": "exon 55 specific phosphorothioate oligonucleotide", "intended_use": "Treatment of Duchenne muscular dystrophy", "status": "Positive", "designation_date": "2012-12-06", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-12-1078", "last_updated": "2016-12-05" }, { "eu_designation_number": "EU/3/12/1079", "medicine_name": "", "active_substance": "artesunate", "intended_use": "Treatment of malaria", "status": "Positive", "designation_date": "2012-12-06", 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"medicine_name": "", "active_substance": "Erdosteine", "intended_use": "Treatment of lead toxicity", "status": "Positive", "designation_date": "2012-12-06", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-12-1084", "last_updated": "2013-01-25" }, { "eu_designation_number": "EU/3/12/1085", "medicine_name": "", "active_substance": "voclosporin", "intended_use": "Treatment of non-infectious uveitis", "status": "Positive", "designation_date": "2012-12-06", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-12-1085", "last_updated": "2013-01-25" }, { "eu_designation_number": "EU/3/12/1086", "medicine_name": "Flynpovi", "active_substance": "eflornithine", "intended_use": "Treatment of familial adenomatous polyposis (FAP)", "status": "Positive", "designation_date": "2013-01-24", "related_ema_product_number": "EMEA/H/C/005043", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-12-1086", "last_updated": "2019-04-29" }, { "eu_designation_number": "EU/3/12/1087", "medicine_name": "Ngenla", "active_substance": "recombinant modified human growth hormone", "intended_use": "Treatment of growth hormone deficiency", "status": "Positive", "designation_date": "2013-01-24", "related_ema_product_number": "EMEA/H/C/005633", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-12-1087", "last_updated": "2022-03-03" }, { "eu_designation_number": "EU/3/12/1088", "medicine_name": "", "active_substance": "allogeneic motor neuron progenitor cells derived from human embryonic stem cells", "intended_use": "Treatment of 5q spinal muscular atrophy", "status": "Positive", "designation_date": "2013-01-24", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-12-1088", "last_updated": "2013-03-01" }, { "eu_designation_number": "EU/3/12/1089", "medicine_name": "", "active_substance": "choline tetrathiomolybdate", "intended_use": "Treatment of Wilson's disease", "status": "Positive", "designation_date": "2013-01-24", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-12-1089", "last_updated": "2019-11-20" }, { "eu_designation_number": "EU/3/12/1090", "medicine_name": "", "active_substance": "recombinant human monoclonal antibody of the IgG1 kappa class against prostate stem-cell antigen", "intended_use": "Treatment of pancreatic cancer", "status": "Positive", "designation_date": "2013-01-24", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-12-1090", "last_updated": "2014-10-16" }, { "eu_designation_number": "EU/3/12/1091", "medicine_name": "", "active_substance": "autologous CD34+ haematopoietic stem cells transduced with lentiviral vector encoding the human betaA-T87Q-globin gene 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"eu_designation_number": "EU/3/12/951", "medicine_name": "", "active_substance": "sodium phenylbutyrate", "intended_use": "Treatment of carbamoyl-phosphate synthase-1 deficiency", "status": "Withdrawn", "designation_date": "2012-02-09", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-12-951", "last_updated": "2013-08-08" }, { "eu_designation_number": "EU/3/12/952", "medicine_name": "", "active_substance": "nimorazole maleate", "intended_use": "Treatment of squamous-cell carcinoma of the head and neck in patients undergoing radiotherapy", "status": "Withdrawn", "designation_date": "2012-02-09", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-12-952", "last_updated": "2021-03-19" }, { "eu_designation_number": "EU/3/12/953", "medicine_name": "", "active_substance": "", "intended_use": "Treatment of West syndrome", "status": "Withdrawn", "designation_date": 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"Withdrawn", "designation_date": "2012-05-22", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-12-962", "last_updated": "2015-06-08" }, { "eu_designation_number": "EU/3/12/963", "medicine_name": "Ledaga", "active_substance": "chlormethine", "intended_use": "Treatment of cutaneous T-cell lymphoma", "status": "Positive", "designation_date": "2012-05-22", "related_ema_product_number": "EMEA/H/C/002826", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-12-963", "last_updated": "2017-03-16" }, { "eu_designation_number": "EU/3/12/964", "medicine_name": "", "active_substance": "oleylphosphocholine", "intended_use": "Treatment of leishmaniasis", "status": "Positive", "designation_date": "2012-04-23", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-12-964", "last_updated": "2020-07-10" }, { "eu_designation_number": "EU/3/12/965", "medicine_name": "", "active_substance": "ketoconazole", "intended_use": "Treatment of Cushing's syndrome", "status": "Expired", "designation_date": "2012-04-23", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-12-965", "last_updated": "2024-11-28" }, { "eu_designation_number": "EU/3/12/966", "medicine_name": "", "active_substance": "(1-methyl-2-nitro-1H-imidazole-5-yl)methyl N,N'-bis(2-bromoethyl)diamidophosphate", "intended_use": "Treatment of soft tissue sarcoma", "status": "Withdrawn", "designation_date": "2012-03-05", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-12-966", "last_updated": "2016-10-21" }, { "eu_designation_number": "EU/3/12/967", "medicine_name": "", "active_substance": "sodium nitrite", "intended_use": "Treatment of pulmonary arterial hypertension", "status": "Positive", "designation_date": "2012-03-05", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-12-967", "last_updated": "2012-07-06" }, { "eu_designation_number": "EU/3/12/968", "medicine_name": "", "active_substance": "human monoclonal antibody targeting Staphylococcus aureus alpha-toxin (tosatoxumab)", "intended_use": "Treatment of pneumonia caused by Staphylococcus aureus", "status": "Positive", "designation_date": "2012-03-05", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-12-968", "last_updated": "2021-07-13" }, { "eu_designation_number": "EU/3/12/969", "medicine_name": "", "active_substance": "allogeneic human dendritic cells derived from a CD34+ progenitor cell line", "intended_use": "Treatment of acute myeloid leukaemia", "status": "Positive", "designation_date": "2012-05-22", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-12-969", "last_updated": "2022-09-15" }, { "eu_designation_number": "EU/3/12/970", "medicine_name": "", "active_substance": "6-ethynyl-1-(pentan-3-yl)-1H-imidazo[4,5-b]pyrazin-2(3H)-one (tirasemtiv)", "intended_use": "Treatment of amyotrophic lateral sclerosis", "status": "Positive", "designation_date": "2012-03-05", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-12-970", "last_updated": "2024-10-11" }, { "eu_designation_number": "EU/3/12/971", "medicine_name": "", "active_substance": "heterologous human adult liver-derived stem cells", "intended_use": "Treatment of carbamoyl-phosphate synthase-1 deficiency", "status": "Positive", "designation_date": "2012-03-05", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-12-971", "last_updated": "2021-08-26" }, { "eu_designation_number": "EU/3/12/972", "medicine_name": "", "active_substance": "sialic acid (also known as aceneuramic acid)", "intended_use": "Treatment of GNE myopathy", "status": "Withdrawn", "designation_date": "2015-10-16", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-12-972", "last_updated": "2018-06-13" }, { "eu_designation_number": "EU/3/12/973", "medicine_name": "Mepsevii", "active_substance": "recombinant human beta-glucuronidase (vestronidase alfa)", "intended_use": "Treatment of mucopolysaccharidosis type VII (Sly syndrome)", "status": "Positive", "designation_date": "2012-03-21", "related_ema_product_number": "EMEA/H/C/004438", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-12-973", "last_updated": "2020-11-11" }, { "eu_designation_number": "EU/3/12/974", "medicine_name": "", "active_substance": "adeno-associated viral vector of serotype 5 containing the human alanine-glyoxylate-aminotransferase gene", "intended_use": "Treatment of primary hyperoxaluria type 1", "status": "Withdrawn", "designation_date": "2012-03-21", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-12-974", "last_updated": "2024-04-09" }, { "eu_designation_number": "EU/3/12/975", "medicine_name": "", "active_substance": "carbetocin", "intended_use": "Treatment of Prader-Willi syndrome", "status": "Withdrawn", "designation_date": "2012-03-21", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-12-975", "last_updated": "2026-08-21" }, { "eu_designation_number": "EU/3/12/976", "medicine_name": "Spinraza", "active_substance": "antisense oligonucleotide targeted to the SMN2 gene (nusinersen)", "intended_use": "Treatment of 5q spinal muscular atrophy", "status": "Positive", "designation_date": "2012-04-02", "related_ema_product_number": "EMEA/H/C/004312", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-12-976", "last_updated": "2018-10-16" }, { "eu_designation_number": "EU/3/12/977", "medicine_name": "", "active_substance": "Linsitinib", "intended_use": "Treatment of adrenal cortical carcinoma", "status": "Withdrawn", "designation_date": "2012-04-02", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-12-977", "last_updated": "2013-03-11" }, { "eu_designation_number": "EU/3/12/978", "medicine_name": "", "active_substance": "melatonin", "intended_use": "Treatment of perinatal asphyxia", "status": "Positive", "designation_date": "2012-04-02", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-12-978", "last_updated": "2024-04-08" }, { "eu_designation_number": "EU/3/12/979", "medicine_name": "", "active_substance": "sodium thiosulfate", "intended_use": "Treatment of calciphylaxis", "status": "Positive", "designation_date": "2012-04-02", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-12-979", "last_updated": "2012-04-30" }, { "eu_designation_number": "EU/3/12/980", "medicine_name": "", "active_substance": "Genistein sodium salt dihydrate", "intended_use": "Treatment of mucopolysaccharidosis type III (Sanfilippo syndrome)", "status": "Positive", "designation_date": "2012-04-02", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-12-980", "last_updated": "2012-04-30" }, { "eu_designation_number": "EU/3/12/981", "medicine_name": "Luxturna", "active_substance": "adenovirus-associated viral vector serotype 2 containing the human RPE65 gene (voretigene neparvovec)", "intended_use": "Treatment of Leber's congenital amaurosis", "status": "Positive", "designation_date": "2012-04-02", "related_ema_product_number": "EMEA/H/C/004451", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-12-981", "last_updated": "2019-03-04" }, { "eu_designation_number": "EU/3/12/982", "medicine_name": "", "active_substance": "Dipalmitoylphosphatidylcholine, 1-palmitoyl-2-oleoyl-sn-glycero-3-phosphoglycerol, sodium salt, synthetic surfactant protein C analogue and synthetic surfactant protein B analogue", "intended_use": "Respiratory distress syndrome in premature neonates of less than 37 weeks of gestational age", "status": "Positive", "designation_date": "2012-04-02", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-12-982", "last_updated": "2012-04-30" }, { "eu_designation_number": "EU/3/12/983", "medicine_name": "", "active_substance": "heterologous human adult liver-derived stem cells", "intended_use": "Treatment of acute liver failure", "status": "Positive", "designation_date": "2012-04-26", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-12-983", "last_updated": "2021-08-26" }, { "eu_designation_number": "EU/3/12/984", "medicine_name": "", "active_substance": "1-[(3R)-3-[4-amino-3-(4-phenoxyphenyl)-1H-pyrazolo[3,4 d]pyrimidin-1-yl]-1-piperidinyl]-2-propen-1-one (ibrutinib)", "intended_use": "Treatment of chronic lymphocytic leukaemia", "status": "Withdrawn", "designation_date": "2012-04-26", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-12-984", "last_updated": "2021-10-21" }, { "eu_designation_number": "EU/3/12/985", "medicine_name": "", "active_substance": "recombinant human methionine proinsulin", "intended_use": "Treatment of retinitis pigmentosa", "status": "Positive", "designation_date": "2012-04-26", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-12-985", "last_updated": "2012-06-18" }, { "eu_designation_number": "EU/3/12/986", "medicine_name": "", "active_substance": "Pomalidomide", "intended_use": "Treatment of systemic sclerosis", "status": "Withdrawn", "designation_date": "2012-04-26", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-12-986", "last_updated": "2015-06-08" }, { "eu_designation_number": "EU/3/12/987", "medicine_name": "", "active_substance": "(E)-2,4,6-trimethoxystyryl-3-carboxymethylamino-4-methoxybenzyl-sulfone sodium salt (rigosertib sodium)", "intended_use": "Treatment of myelodysplastic syndromes", "status": "Withdrawn", "designation_date": "2012-04-26", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-12-987", "last_updated": "2021-10-27" }, { "eu_designation_number": "EU/3/12/988", "medicine_name": "", "active_substance": "Halofuginone hydrobromide", "intended_use": "Treatment of Duchenne muscular dystrophy", "status": "Withdrawn", "designation_date": "2012-04-26", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-12-988", "last_updated": "2019-02-21" }, { "eu_designation_number": "EU/3/12/989", "medicine_name": "", "active_substance": "2-Allyl-1-[6-(1-hydroxy-1-methylethyl)pyridin-2-yl]-6-{[4-(4-methylpiperazin-1-yl)phenyl]amino}-1,2-dihydro-3H-pyrazolo[3,4-d]pyrimidin-3-one", "intended_use": "Treatment of ovarian cancer", "status": "Withdrawn", "designation_date": "2012-04-26", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-12-989", "last_updated": "2019-02-21" }, { "eu_designation_number": "EU/3/12/990", "medicine_name": "", "active_substance": "vosaroxin", "intended_use": "Treatment of acute myeloid leukaemia", "status": "Positive", "designation_date": "2012-04-26", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-12-990", "last_updated": "2016-04-12" }, { "eu_designation_number": "EU/3/12/991", "medicine_name": "", "active_substance": "exon 45 specific phosphorothioate oligonucleotide", "intended_use": "Treatment of Duchenne muscular dystrophy", "status": "Positive", "designation_date": "2012-04-26", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-12-991", "last_updated": "2016-12-05" }, { "eu_designation_number": "EU/3/12/992", "medicine_name": "", "active_substance": "exon 53 specific phosphorothioate oligonucleotide", "intended_use": "Treatment of Duchenne muscular dystrophy", "status": "Positive", "designation_date": "2012-04-26", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-12-992", "last_updated": "2016-12-05" }, { "eu_designation_number": "EU/3/12/993", "medicine_name": "", "active_substance": "N-Hydroxy-4-(3-methyl-2-(S)-phenyl-butyrylamino) benzamide", "intended_use": "Treatment of neurofibromatosis type 2", "status": "Positive", "designation_date": "2012-04-26", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-12-993", "last_updated": "2022-07-05" }, { "eu_designation_number": "EU/3/12/994", "medicine_name": "", "active_substance": "yttrium (90Y)-DTPA-radiolabelled chimeric monoclonal antibody against frizzled homologue 10", "intended_use": "Treatment of soft tissue sarcoma", "status": "Positive", "designation_date": "2012-05-25", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-12-994", "last_updated": "2012-06-18" }, { "eu_designation_number": "EU/3/12/995", "medicine_name": "", "active_substance": "pegylated recombinant factor VIII (turoctocog alfa pegol)", "intended_use": "Treatment of haemophilia A", "status": "Withdrawn", "designation_date": "2012-04-26", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-12-995", "last_updated": "2019-06-28" }, { "eu_designation_number": "EU/3/12/996", "medicine_name": "", "active_substance": "N-Hydroxy-4-(3-methyl-2-(S)-phenyl-butyrylamino) benzamide", "intended_use": "Treatment of meningioma", "status": "Withdrawn", "designation_date": "2012-06-06", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-12-996", "last_updated": "2026-08-21" }, { "eu_designation_number": "EU/3/12/997", "medicine_name": "", "active_substance": "N-Hydroxy-4-(3-methyl-2-(S)-phenyl-butyrylamino) benzamide", "intended_use": "Treatment of schwannoma", "status": "Withdrawn", "designation_date": "2012-06-06", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-12-997", "last_updated": "2026-08-21" }, { "eu_designation_number": "EU/3/12/998", "medicine_name": "Waskyra", "active_substance": "autologous CD34+ cells transfected with lentiviral vector containing the Wiskott-Aldrich syndrome protein gene (etuvetidigene autotemcel)", "intended_use": "Treatment of Wiskott-Aldrich syndrome", "status": "Positive", "designation_date": "2012-06-06", "related_ema_product_number": "EMEA/H/C/006525", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-12-998", "last_updated": "2023-03-27" }, { "eu_designation_number": "EU/3/12/999", "medicine_name": "", "active_substance": "Letermovir", "intended_use": "Treatment of cytomegalovirus disease in patients with impaired cell-mediated immunity", "status": "Withdrawn", "designation_date": "2012-06-06", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-12-999", "last_updated": "2026-08-21" }, { "eu_designation_number": "EU/3/13/1099", "medicine_name": "", "active_substance": "recombinant adeno-associated viral vector containing the human CNGB3 gene", "intended_use": "Treatment of achromatopsia caused by mutations in the CNGB3 gene", "status": "Positive", "designation_date": "2013-02-08", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-13-1099", "last_updated": "2023-06-16" }, { "eu_designation_number": "EU/3/13/1100", "medicine_name": "", "active_substance": "Humanised IgG1 kappa antibody against serum amyloid A and AL amyloid", "intended_use": "Treatment of amyloid light-chain amyloidosis", "status": "Positive", "designation_date": "2013-02-08", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-13-1100", "last_updated": "2024-10-11" }, { "eu_designation_number": "EU/3/13/1101", "medicine_name": "", "active_substance": "progesterone", "intended_use": "Treatment of moderate and severe traumatic brain injury", "status": "Withdrawn", "designation_date": "2013-02-08", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-13-1101", "last_updated": "2014-10-16" }, { "eu_designation_number": "EU/3/13/1102", "medicine_name": "", "active_substance": "Cyclo-Cys-Gly-Gln-Arg-Glu-Thr-Pro-Glu-Gly-Ala-Glu-Ala-Lys-Pro-Trp-Tyr-Cys", "intended_use": "Treatment of high-altitude pulmonary oedema", "status": "Positive", "designation_date": "2013-02-08", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-13-1102", "last_updated": "2013-03-04" }, { "eu_designation_number": "EU/3/13/1103", "medicine_name": "Trepulmix", "active_substance": "treprostinil sodium", "intended_use": "Treatment of chronic thromboembolic pulmonary hypertension", "status": "Positive", "designation_date": "2013-02-08", "related_ema_product_number": "EMEA/H/C/005207", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-13-1103", "last_updated": "2020-04-08" }, { "eu_designation_number": "EU/3/13/1104", "medicine_name": "", "active_substance": "terguride", "intended_use": 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"https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-13-1106", "last_updated": "2013-03-04" }, { "eu_designation_number": "EU/3/13/1107", "medicine_name": "", "active_substance": "recombinant adeno-associated viral vector containing the human retinoschisin gene", "intended_use": "Treatment of X-linked juvenile retinoschisis", "status": "Positive", "designation_date": "2013-03-12", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-13-1107", "last_updated": "2022-10-06" }, { "eu_designation_number": "EU/3/13/1108", "medicine_name": "", "active_substance": "2-[4-Methoxy-3-(2-m-tolyl-ethoxy)-benzoylamino]-indan-2-carboxylic acid", "intended_use": "Treatment of systemic sclerosis", "status": "Withdrawn", "designation_date": "2013-03-12", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-13-1108", "last_updated": "2026-05-18" }, { "eu_designation_number": "EU/3/13/1109", "medicine_name": "", "active_substance": "4-[2-(6-Methylpyridin-2-yl)-5,6-dihydro-4H-pyrrolo[1,2-b]pyrazol-3-yl]-quinoline-6-carboxamide monohydrate", "intended_use": "Treatment of hepatocellular carcinoma", "status": "Withdrawn", "designation_date": "2013-03-12", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-13-1109", "last_updated": "2019-06-17" }, { "eu_designation_number": "EU/3/13/1110", "medicine_name": "", "active_substance": "recombinant human heat-shock protein 70", "intended_use": "Treatment of Niemann-Pick's disease, type C", "status": "Withdrawn", "designation_date": "2013-03-12", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-13-1110", "last_updated": "2017-08-14" }, { "eu_designation_number": "EU/3/13/1111", "medicine_name": "", "active_substance": "Gevokizumab", "intended_use": "Treatment of chronic 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"https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-13-1113", "last_updated": "2017-07-17" }, { "eu_designation_number": "EU/3/13/1114", "medicine_name": "", "active_substance": "Cyclo[L-alanyl-L-seryl-L-isoleucyl-L-prolyl-L-prolyl-L-glutaminyl-L-lysyl-L-tyrosyl-D-prolyl-L-prolyl-(2S)-2-aminodecanoyl-L-alpha-glutamyl-L-threonyl]acetate salt", "intended_use": "Treatment of congenital alpha-1 antitrypsin deficiency", "status": "Positive", "designation_date": "2013-03-20", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-13-1114", "last_updated": "2018-06-14" }, { "eu_designation_number": "EU/3/13/1115", "medicine_name": "", "active_substance": "1-[(3R)-3-[4-amino-3-(4-phenoxyphenyl)-1H-pyrazolo[3,4 d]pyrimidin-1-yl]-1-piperidinyl]-2-propen-1-one (ibrutinib)", "intended_use": "Treatment of mantle cell lymphoma", "status": "Withdrawn", "designation_date": "2013-03-12", "related_ema_product_number": "", "url": 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"https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-13-1120", "last_updated": "2019-06-17" }, { "eu_designation_number": "EU/3/13/1121", "medicine_name": "", "active_substance": "lenvatinib", "intended_use": "Treatment of papillary thyroid cancer", "status": "Withdrawn", "designation_date": "2013-04-26", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-13-1121", "last_updated": "2018-10-22" }, { "eu_designation_number": "EU/3/13/1122", "medicine_name": "", "active_substance": "R,S-O-(3-piperidino-2-hydroxy-1-propyl)-nicotinic acid amidoxime dihydrochloride", "intended_use": "Treatment of Duchenne muscular dystrophy", "status": "Positive", "designation_date": "2013-04-26", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-13-1122", "last_updated": "2015-03-24" }, { "eu_designation_number": "EU/3/13/1123", "medicine_name": "", "active_substance": 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"related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-13-1125", "last_updated": "2021-05-31" }, { "eu_designation_number": "EU/3/13/1126", "medicine_name": "", "active_substance": "Mexiletine hydrochloride", "intended_use": "Treatment of non-dystrophic myotonia", "status": "Positive", "designation_date": "2013-06-07", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-13-1126", "last_updated": "2021-04-30" }, { "eu_designation_number": "EU/3/13/1127", "medicine_name": "Besponsa", "active_substance": "Inotuzumab ozogamicin", "intended_use": "Treatment of B-cell acute lymphoblastic leukaemia", "status": "Positive", "designation_date": "2013-06-07", "related_ema_product_number": "EMEA/H/C/004119", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-13-1127", "last_updated": "2013-06-25" }, { "eu_designation_number": "EU/3/13/1128", "medicine_name": "", "active_substance": "N-[2,6-bis(1-methylethyl)phenyl]-N'-[[1-[4-(dimethylamino) phenyl]cyclopentyl]methyl]urea, hydrochloride salt", "intended_use": "Treatment of adrenocortical carcinoma", "status": "Positive", "designation_date": "2013-06-07", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-13-1128", "last_updated": "2017-11-13" }, { "eu_designation_number": "EU/3/13/1129", "medicine_name": "", "active_substance": "allogeneic bone-marrow-derived mesenchymal cells expanded ex vivo in synthetic media", "intended_use": "Treatment of graft-versus-host disease", "status": "Positive", "designation_date": "2013-06-07", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-13-1129", "last_updated": "2013-07-09" }, { "eu_designation_number": "EU/3/13/1130", "medicine_name": "", "active_substance": "recombinant human transglutaminase 1 encapsulated into liposomes", "intended_use": "Treatment of transglutaminase-1-deficient autosomal recessive congenital ichthyosis", "status": "Positive", "designation_date": "2013-06-07", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-13-1130", "last_updated": "2013-06-25" }, { "eu_designation_number": "EU/3/13/1131", "medicine_name": "", "active_substance": "recombinant human CXCL8 mutant", "intended_use": "Treatment of cystic fibrosis", "status": "Positive", "designation_date": "2013-06-07", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-13-1131", "last_updated": "2013-06-25" }, { "eu_designation_number": "EU/3/13/1132", "medicine_name": "", "active_substance": "N-methyl-4-({4-[({3-methyl(methylsulfonyl)aminopyrazin-2-yl}methyl)amino]-5-(trifluoromethyl)pyrimidin-2-yl}amino)benzamide hydrochloride (defactinib)", "intended_use": "Treatment of malignant mesothelioma", "status": "Withdrawn", "designation_date": "2013-06-07", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-13-1132", "last_updated": "2022-02-28" }, { "eu_designation_number": "EU/3/13/1133", "medicine_name": "Livtencity", "active_substance": "Maribavir", "intended_use": "Treatment of cytomegalovirus disease in patients with impaired cell-mediated immunity", "status": "Positive", "designation_date": "2013-06-07", "related_ema_product_number": "EMEA/H/C/005787", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-13-1133", "last_updated": "2022-11-24" }, { "eu_designation_number": "EU/3/13/1134", "medicine_name": "", "active_substance": "autologous CD34+ cells transduced with a lentiviral vector containing the human ADA gene", "intended_use": "Treatment of adenosine-deaminase-deficient severe combined immunodeficiency", "status": "Positive", "designation_date": "2013-06-07", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-13-1134", "last_updated": "2023-04-03" }, { "eu_designation_number": "EU/3/13/1135", "medicine_name": "", "active_substance": "recombinant human nerve growth factor", "intended_use": "Treatment of retinitis pigmentosa", "status": "Positive", "designation_date": "2013-06-07", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-13-1135", "last_updated": "2015-05-13" }, { "eu_designation_number": "EU/3/13/1136", "medicine_name": "", "active_substance": "5-[1-(2,6-Dichlorobenzyl)piperidin-4-ylmethoxy]quinazoline-2,4-diamine dihydrochloride", "intended_use": "Treatment of 5q spinal muscular atrophy", "status": "Positive", "designation_date": "2013-06-07", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-13-1136", "last_updated": "2013-10-07" }, { "eu_designation_number": "EU/3/13/1137", "medicine_name": "", "active_substance": "4,6,4'-trimethylangelicin", "intended_use": "Treatment of cystic fibrosis", "status": "Positive", "designation_date": "2013-06-19", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-13-1137", "last_updated": "2020-06-08" }, { "eu_designation_number": "EU/3/13/1138", "medicine_name": "", "active_substance": "copper meso-5,15-bis[3-[(1,2-dicarba-closo-dodecaboranyl)methoxy]phenyl]-meso-10,20-dinitroporphyrin", "intended_use": "Treatment of squamous-cell carcinoma of the head and neck in patients undergoing radiotherapy", "status": "Withdrawn", "designation_date": "2013-06-27", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-13-1138", "last_updated": "2020-12-10" }, { "eu_designation_number": "EU/3/13/1139", "medicine_name": "", "active_substance": "sodium chlorite", "intended_use": "Treatment of amyotrophic lateral sclerosis", "status": "Positive", "designation_date": "2013-06-19", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-13-1139", "last_updated": "2018-01-03" }, { "eu_designation_number": "EU/3/13/1140", "medicine_name": "", "active_substance": "expanded human allogeneic neural retinal progenitor cells extracted from neural retina", "intended_use": "Treatment of retinitis pigmentosa", "status": "Positive", "designation_date": "2013-06-19", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-13-1140", "last_updated": "2013-07-11" }, { "eu_designation_number": "EU/3/13/1141", "medicine_name": "", "active_substance": "synthetic double-stranded siRNA oligonucleotide directed against the keratin 6a N171K mutation", "intended_use": "Treatment of pachyonychia congenita", "status": "Positive", "designation_date": "2013-06-19", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-13-1141", "last_updated": "2013-07-11" }, { "eu_designation_number": "EU/3/13/1142", "medicine_name": "", "active_substance": "adenovirus associated viral vector serotype 5 containing the human pde6β gene", "intended_use": "Treatment of retinitis pigmentosa", "status": "Positive", "designation_date": "2013-06-19", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-13-1142", "last_updated": "2024-10-11" }, { "eu_designation_number": "EU/3/13/1143", "medicine_name": "", "active_substance": "immortalised human C3A hepatoblastoma cells", "intended_use": "Treatment of acute liver failure", "status": "Withdrawn", "designation_date": "2013-06-19", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-13-1143", "last_updated": "2013-07-11" }, { "eu_designation_number": "EU/3/13/1144", "medicine_name": "", "active_substance": "recombinant human alpha-N-acetylglucosaminidase", "intended_use": "Treatment of mucopolysaccharidosis type IIIB (Sanfilippo B syndrome)", "status": "Withdrawn", "designation_date": "2013-06-19", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-13-1144", "last_updated": "2017-10-19" }, { "eu_designation_number": "EU/3/13/1145", "medicine_name": "", "active_substance": "genetically modified serotype 5/3 adenovirus coding for granulocyte-macrophage colony-stimulating factor", "intended_use": "Treatment of soft tissue sarcoma", "status": "Withdrawn", "designation_date": "2013-06-19", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-13-1145", "last_updated": "2026-05-26" }, { "eu_designation_number": "EU/3/13/1146", "medicine_name": "", "active_substance": "unoprostone isopropyl", "intended_use": "Treatment of retinitis pigmentosa", "status": "Withdrawn", "designation_date": "2013-06-19", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-13-1146", "last_updated": "2016-08-11" }, { "eu_designation_number": "EU/3/13/1147", "medicine_name": "", "active_substance": "Granulocyte-macrophage colony-stimulating factor", "intended_use": "Treatment of pulmonary alveolar proteinosis", "status": "Positive", "designation_date": "2013-07-17", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-13-1147", "last_updated": "2022-04-06" }, { "eu_designation_number": "EU/3/13/1148", "medicine_name": "", "active_substance": "autologous bone-marrow-derived mesenchymal stromal cells secreting neurotrophic factors", "intended_use": "Treatment of amyotrophic lateral sclerosis", "status": "Positive", "designation_date": "2013-07-17", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-13-1148", "last_updated": "2013-08-16" }, { "eu_designation_number": "EU/3/13/1149", "medicine_name": "", "active_substance": "human haemin", "intended_use": "Prevention of ischaemia/reperfusion injury associated with solid organ transplantation", "status": "Positive", "designation_date": "2013-07-17", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-13-1149", "last_updated": "2013-08-22" }, { "eu_designation_number": "EU/3/13/1150", "medicine_name": "", "active_substance": "moxetumomab pasudotox", "intended_use": "Treatment of B-lymphoblastic leukaemia/lymphoma", "status": "Withdrawn", "designation_date": "2022-07-19", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-13-1150", "last_updated": "2022-07-25" }, { "eu_designation_number": "EU/3/13/1151", "medicine_name": "", 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"medicine_name": "", "active_substance": "sodium nitrite", "intended_use": "Treatment of aneurysmal subarachnoid haemorrhage", "status": "Positive", "designation_date": "2014-01-16", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-13-1224", "last_updated": "2019-08-19" }, { "eu_designation_number": "EU/3/13/1225", "medicine_name": "", "active_substance": "Lonafarnib", "intended_use": "Treatment of hepatitis delta virus infection", "status": "Positive", "designation_date": "2014-01-16", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-13-1225", "last_updated": "2019-08-19" }, { "eu_designation_number": "EU/3/13/1226", "medicine_name": "", "active_substance": "(6aS)-1,10-dimethoxy-6-methyl-5,6,6a,7-tetrahydro-4H-dibenzo[de,g]quinoline-2,9-diol", "intended_use": "Treatment of dystrophic myotonia", "status": "Positive", "designation_date": "2014-01-16", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-13-1226", "last_updated": "2014-03-21" }, { "eu_designation_number": "EU/3/13/1227", "medicine_name": "", "active_substance": "adenovirus-specific T-cells derived from allogeneic donor leukocytes, expanded ex vivo", "intended_use": "Treatment of adenovirus infection in allogeneic haematopoietic stem cell transplant recipients", "status": "Withdrawn", "designation_date": "2014-01-16", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-13-1227", "last_updated": "2020-02-12" }, { "eu_designation_number": "EU/3/13/1228", "medicine_name": "", "active_substance": "obeticholic acid", "intended_use": "Treatment of primary sclerosing cholangitis", "status": "Withdrawn", "designation_date": "2014-03-16", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-13-1228", "last_updated": "2022-05-10" }, { "eu_designation_number": "EU/3/13/1229", "medicine_name": "", "active_substance": "autologous dendritic cells pulsed with allogeneic tumour cell lysate", "intended_use": "Treatment of malignant mesothelioma", "status": "Positive", "designation_date": "2014-01-16", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-13-1229", "last_updated": "2015-03-23" }, { "eu_designation_number": "EU/3/13/1230", "medicine_name": "", "active_substance": "", "intended_use": "Treatment of acute myeloid leukaemia", "status": "Withdrawn", "designation_date": "2014-01-16", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-13-1230", "last_updated": "2024-04-11" }, { "eu_designation_number": "EU/3/13/1231", "medicine_name": "", "active_substance": "", "intended_use": "Treatment of acute lymphoblastic leukaemia", "status": "Positive", "designation_date": "2014-01-16", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-13-1231", "last_updated": "2024-04-11" }, { "eu_designation_number": "EU/3/13/1232", "medicine_name": "", "active_substance": "allantoin", "intended_use": "Treatment of epidermolysis bullosa", "status": "Withdrawn", "designation_date": "2014-01-16", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-13-1232", "last_updated": "2020-12-10" }, { "eu_designation_number": "EU/3/13/1233", "medicine_name": "", "active_substance": "allogeneic bone-marrow-derived ex-vivo-expanded multipotent adult progenitor cells", "intended_use": "Prevention of graft-versus-host disease", "status": "Positive", "designation_date": "2014-01-16", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-13-1233", "last_updated": "2014-03-24" }, { "eu_designation_number": "EU/3/13/1234", "medicine_name": "", "active_substance": "N-(3-(5-fluoro-2-(4-(2-methoxyethoxy)phenylamino)pyrimidin-4-ylamino)phenyl)acrylamide benzenesulfonic acid salt", "intended_use": "Treatment of chronic lymphocytic leukaemia / small lymphocytic lymphoma", "status": "Withdrawn", "designation_date": "2014-03-16", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-13-1234", "last_updated": "2015-03-06" }, { "eu_designation_number": "EU/3/14/1235", "medicine_name": "", "active_substance": "mixture of recombinant human IgG1 monoclonal antibodies against human cytomegalovirus envelope glycoproteins", "intended_use": "Prevention of congenital cytomegalovirus infection following primary cytomegalovirus infection", "status": "Withdrawn", "designation_date": "2014-02-20", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-14-1235", "last_updated": "2015-05-08" }, { "eu_designation_number": "EU/3/14/1236", "medicine_name": "", "active_substance": "Diacerein", "intended_use": "Treatment of epidermolysis bullosa", "status": "Positive", "designation_date": "2014-02-20", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-14-1236", "last_updated": "2024-04-11" }, { "eu_designation_number": "EU/3/14/1237", "medicine_name": "", "active_substance": "Gallium [Ga-68]-N-[(4,7,10-Tricarboxymethyl-1,4,7,10-tetraazacyclododec-1-yl)acetyl]-D-phenylalanyl-L-cysteinyl-L-tyrosyl-D-tryptophanyl-L-lysyl-L-threoninyl-Lcysteinyl-L-threonine-cyclic(2-7)disulfide", "intended_use": "Diagnosis of gastro-entero-pancreatic neuroendocrine tumours", "status": "Positive", "designation_date": "2014-02-19", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-14-1237", "last_updated": "2023-05-03" }, { "eu_designation_number": "EU/3/14/1238", "medicine_name": "", "active_substance": "Eculizumab", "intended_use": "Prevention of delayed graft function after solid organ transplantation", "status": "Withdrawn", "designation_date": "2014-02-19", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-14-1238", "last_updated": "2017-05-17" }, { "eu_designation_number": "EU/3/14/1239", "medicine_name": "", "active_substance": "11-(4-Dimethylamino-3-hydroxy-6-methyl-tetrahydro-pyran-2-yloxy)-2-ethyl-3,4,10-trihydroxy-3,5,6,8,10,12,14-heptamethyl-1-oxa-6-aza-cyclopentadecane-13,15-dione", "intended_use": "Treatment of cystic fibrosis", "status": "Positive", "designation_date": "2014-02-19", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-14-1239", "last_updated": "2014-04-01" }, { "eu_designation_number": "EU/3/14/1240", "medicine_name": "", "active_substance": "cysteamine", "intended_use": "Treatment of cystic fibrosis", "status": "Positive", "designation_date": "2014-02-19", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-14-1240", "last_updated": "2017-04-20" }, { "eu_designation_number": "EU/3/14/1241", "medicine_name": "", "active_substance": "Asp-Arg-Val-Tyr-Ile-His-Pro (Angiotensin 1-7);talfirastide", "intended_use": "Treatment of Duchenne muscular dystrophy", "status": "Positive", "designation_date": "2014-02-19", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-14-1241", "last_updated": "2024-10-22" }, { "eu_designation_number": "EU/3/14/1242", "medicine_name": "", "active_substance": "3-Chloro-4-fluorophenyl-[4-fluoro-4-{[(5-methylpyrimidin-2-ylmethyl) amino]methyl}piperidin-1-yl]methanone", "intended_use": "Treatment of Rett syndrome", "status": "Positive", "designation_date": "2014-02-19", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-14-1242", "last_updated": "2018-10-18" }, { "eu_designation_number": "EU/3/14/1243", "medicine_name": "", "active_substance": "recombinant human acid ceramidase", "intended_use": "Treatment of Farber disease", "status": "Positive", "designation_date": "2014-02-19", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-14-1243", "last_updated": "2021-06-25" }, { "eu_designation_number": "EU/3/14/1244", "medicine_name": "", "active_substance": "ruxolitinib", "intended_use": "Treatment of polycythaemia vera", "status": "Withdrawn", "designation_date": "2014-02-19", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-14-1244", "last_updated": "2015-05-08" }, { "eu_designation_number": "EU/3/14/1245", "medicine_name": "", "active_substance": "pioglitazone", "intended_use": "Treatment of adrenoleukodystrophy", "status": "Positive", "designation_date": "2014-02-19", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-14-1245", "last_updated": "2014-04-02" }, { "eu_designation_number": "EU/3/14/1246", "medicine_name": "", "active_substance": "satoreotide trizoxetan", "intended_use": "Diagnosis of gastro-entero-pancreatic neuroendocrine tumours", "status": "Positive", "designation_date": "2014-02-19", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-14-1246", "last_updated": "2025-07-17" }, { "eu_designation_number": "EU/3/14/1247", "medicine_name": "", "active_substance": "autologous dendritic cells pulsed with tumour antigen-derived synthetic peptides (MAGE-1, HER-2, AIM-2, TRP-2, gp-100, and interleukin-13 receptor alpha)", "intended_use": "Treatment of glioma", "status": "Positive", "designation_date": "2014-02-19", "related_ema_product_number": 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"https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-14-1265", "last_updated": "2024-04-09" }, { "eu_designation_number": "EU/3/14/1266", "medicine_name": "Kymriah", "active_substance": "autologous T cells transduced with lentiviral vector containing a chimeric antigen receptor directed against CD19 (tisagenlecleucel)", "intended_use": "Treatment of B-lymphoblastic leukaemia/lymphoma", "status": "Positive", "designation_date": "2014-04-29", "related_ema_product_number": "EMEA/H/C/004090", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-14-1266", "last_updated": "2018-06-13" }, { "eu_designation_number": "EU/3/14/1267", "medicine_name": "", "active_substance": "synthetic double-stranded siRNA oligonucleotide directed against transthyretin mRNA and covalently linked to a ligand containing three N-acetylgalactosamine residues for the treatment of ATTR amyloidosis", "intended_use": "Treatment of ATTR amyloidosis", "status": "Withdrawn", 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"https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-14-1269", "last_updated": "2017-09-05" }, { "eu_designation_number": "EU/3/14/1270", "medicine_name": "", "active_substance": "(5R,5aR,8aR,9S)-9-[[4,6-O-[(R)-Ethylidene]-β-D-glucopyranosyl]-oxy]-5-(4-({[(2,2-dimethyl-1,3-dioxolan-4-yl)methoxy]carbonyl}oxy)-3,5-dimethoxyphenyl)-5,8,8a,9-tetrahydroisobenzofuro[5,6-f][1,3]benzodioxol-6(5aH)-one (etoposide toniribate)", "intended_use": "Treatment of biliary tract cancer", "status": "Positive", "designation_date": "2014-06-04", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-14-1270", "last_updated": "2022-04-08" }, { "eu_designation_number": "EU/3/14/1271", "medicine_name": "", "active_substance": "177Lu-tetraxetan-tetulomab", "intended_use": "Treatment of follicular lymphoma", "status": "Positive", "designation_date": "2014-06-04", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-14-1271", "last_updated": "2014-07-10" }, { "eu_designation_number": "EU/3/14/1272", "medicine_name": "", "active_substance": "recombinant human alpha 1 chain homotrimer of type VII collagen", "intended_use": "Treatment of epidermolysis bullosa", "status": "Positive", "designation_date": "2014-06-04", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-14-1272", "last_updated": "2021-10-13" }, { "eu_designation_number": "EU/3/14/1273", "medicine_name": "", "active_substance": "autologous dendritic cells pulsed with RNA from glioma stem cells", "intended_use": "Treatment of glioma", "status": "Positive", "designation_date": "2014-06-04", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-14-1273", "last_updated": "2014-07-10" }, { "eu_designation_number": "EU/3/14/1274", "medicine_name": "", "active_substance": "Paclitaxel-succinate-Arg-Arg-Leu-Ser-Tyr-Ser-Arg-Arg-Arg-Phe", "intended_use": "Treatment of glioma", "status": "Positive", "designation_date": "2014-06-04", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-14-1274", "last_updated": "2015-05-27" }, { "eu_designation_number": "EU/3/14/1275", "medicine_name": "", "active_substance": "aganirsen", "intended_use": "Treatment of central retinal vein occlusion", "status": "Positive", "designation_date": "2014-06-10", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-14-1275", "last_updated": "2014-07-10" }, { "eu_designation_number": "EU/3/14/1276", "medicine_name": "Cresemba", "active_substance": "Isavuconazonium sulfate", "intended_use": "Treatment of mucormycosis", "status": "Positive", "designation_date": "2014-06-04", "related_ema_product_number": "EMEA/H/C/002734", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-14-1276", "last_updated": "2019-05-21" }, { "eu_designation_number": "EU/3/14/1277", "medicine_name": "", "active_substance": "Plasmid DNA encoding the human cystic fibrosis transmembrane conductance regulator gene complexed with a non-viral, cationic lipid based gene transfer agent", "intended_use": "Treatment of cystic fibrosis", "status": "Positive", "designation_date": "2014-06-04", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-14-1277", "last_updated": "2014-07-10" }, { "eu_designation_number": "EU/3/14/1278", "medicine_name": "", "active_substance": "adeno-associated viral vector serotype 2 containing the human CHM gene encoding human Rab escort protein 1", "intended_use": "Treatment of choroideraemia", "status": "Positive", "designation_date": "2014-06-04", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-14-1278", "last_updated": "2026-08-21" }, { "eu_designation_number": "EU/3/14/1279", "medicine_name": "", "active_substance": "4-(4-Methoxy-phenylamino)-6-methylcarbamyl-quinoline-3-carboxylic acid", "intended_use": "Prevention of scarring post glaucoma filtration surgery", "status": "Positive", "designation_date": "2014-06-04", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-14-1279", "last_updated": "2014-07-10" }, { "eu_designation_number": "EU/3/14/1280", "medicine_name": "", "active_substance": "autologous CD34+ cells transduced with a lentiviral vector containing the human SGSH gene", "intended_use": "Treatment of mucopolysaccharidosis, type IIIA (Sanfilippo A syndrome)", "status": "Positive", "designation_date": "2014-06-10", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-14-1280", "last_updated": "2022-07-14" }, { "eu_designation_number": "EU/3/14/1281", "medicine_name": "", "active_substance": "1-(2,2-difluoro-1,3-benzodioxol-5-yl)-N-{1-[(2R)-2,3-dihydroxypropyl]-6-fluoro-2-(1-hydroxy-2-methylpropan-2-yl)-1H-indol-5-yl}cyclopropanecarboxamide (tezacaftor)", "intended_use": "Treatment of cystic fibrosis", "status": "Positive", "designation_date": "2014-07-04", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-14-1281", "last_updated": "2022-01-25" }, { "eu_designation_number": "EU/3/14/1282", "medicine_name": "", "active_substance": "mixture of two adeno-associated viral vectors serotype 8 containing the 5'-half sequence of human MYO7A gene and the 3'-half sequence of human MYO7A gene", "intended_use": "Treatment of Usher syndrome", "status": "Positive", "designation_date": "2014-07-04", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-14-1282", "last_updated": "2022-11-07" }, { "eu_designation_number": "EU/3/14/1283", "medicine_name": "", "active_substance": "mixture of two adeno-associated viral vectors of serotye 8 containing the 5'-half sequence of human ABCA4 gene and the 3'-half sequence of human ABCA4 gene", "intended_use": "Treatment of Stargardt's disease", "status": "Positive", "designation_date": "2014-07-04", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-14-1283", "last_updated": "2022-11-07" }, { "eu_designation_number": "EU/3/14/1284", "medicine_name": "Cresemba", "active_substance": "Isavuconazonium sulfate", "intended_use": "Treatment of invasive aspergillosis", "status": "Positive", "designation_date": "2014-07-04", "related_ema_product_number": "EMEA/H/C/002734", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-14-1284", "last_updated": "2019-05-21" }, { "eu_designation_number": "EU/3/14/1285", "medicine_name": "", "active_substance": "afamelanotide", "intended_use": "Treatment of familial benign chronic pemphigus (Hailey-Hailey disease)", "status": "Positive", "designation_date": "2014-07-04", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-14-1285", "last_updated": "2016-04-11" }, { "eu_designation_number": "EU/3/14/1286", "medicine_name": "", "active_substance": "Humanised Fc engineered monoclonal antibody against CD19 (tafasitamab)", "intended_use": "Treatment of chronic lymphocytic leukaemia / small lymphocytic lymphoma", "status": "Positive", "designation_date": "2014-07-04", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-14-1286", "last_updated": "2021-11-18" }, { "eu_designation_number": "EU/3/14/1287", "medicine_name": "", "active_substance": "beloranib", "intended_use": "Treatment 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"2021-05-11" }, { "eu_designation_number": "EU/3/14/1290", "medicine_name": "", "active_substance": "adeno-associated viral vector serotype 2 containing the human REP1 gene", "intended_use": "Treatment of choroideraemia", "status": "Positive", "designation_date": "2014-07-04", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-14-1290", "last_updated": "2019-04-12" }, { "eu_designation_number": "EU/3/14/1291", "medicine_name": "", "active_substance": "rilotumumab", "intended_use": "Treatment of gastric cancer", "status": "Withdrawn", "designation_date": "2014-07-29", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-14-1291", "last_updated": "2014-09-01" }, { "eu_designation_number": "EU/3/14/1292", "medicine_name": "", "active_substance": "carboxy pyrrolidine hexanoyl pyrrolidine carboxylate (miridesap)", "intended_use": "Treatment of AL amyloidosis", "status": "Withdrawn", "designation_date": "2014-07-29", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-14-1292", "last_updated": "2019-07-09" }, { "eu_designation_number": "EU/3/14/1293", "medicine_name": "", "active_substance": "recombinant monoclonal antibody to human serum amyloid P component (dezamizumab)", "intended_use": "Treatment of AL amyloidosis", "status": "Withdrawn", "designation_date": "2014-07-29", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-14-1293", "last_updated": "2019-07-09" }, { "eu_designation_number": "EU/3/14/1294", "medicine_name": "", "active_substance": "sodium acetate salt of the synthetic peptide H-D-Ala-Ser-Pro-Met-Leu-Val-Ala-Tyr-Asp-D-Ala-OH", "intended_use": "Treatment of necrotising soft tissue infections", "status": "Positive", "designation_date": "2014-07-29", "related_ema_product_number": "", "url": 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"medicine_name": "", "active_substance": "synthetic double-stranded siRNA oligonucleotide directed against antithrombin mRNA and covalently linked to a ligand containing three N-acetylgalactosamine residues (fitusiran)", "intended_use": "Treatment of haemophilia A", "status": "Positive", "designation_date": "2014-07-29", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-14-1297", "last_updated": "2023-07-18" }, { "eu_designation_number": "EU/3/14/1298", "medicine_name": "", "active_substance": "synthetic double-stranded siRNA oligonucleotide directed against antithrombin mRNA and covalently linked to a ligand containing three N-acetylgalactosamine residues (fitusiran)", "intended_use": "Treatment of haemophilia B", "status": "Positive", "designation_date": "2014-07-29", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-14-1298", "last_updated": "2023-07-18" }, { "eu_designation_number": "EU/3/14/1299", "medicine_name": "", "active_substance": "riociguat", "intended_use": "Treatment of systemic sclerosis", "status": "Withdrawn", "designation_date": "2014-07-29", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-14-1299", "last_updated": "2019-02-21" }, { "eu_designation_number": "EU/3/14/1300", "medicine_name": "Reblozyl", "active_substance": "recombinant fusion protein consisting of a modified form of the extracellular domain of human activin receptor IIB linked to the human IgG1 Fc domain (luspatercept)", "intended_use": "Treatment of beta thalassaemia intermedia and major", "status": "Positive", "designation_date": "2014-07-29", "related_ema_product_number": "EMEA/H/C/004444", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-14-1300", "last_updated": "2023-03-21" }, { "eu_designation_number": "EU/3/14/1301", "medicine_name": "", "active_substance": "Humanised anti-alpha ν beta 6 monoclonal antibody", "intended_use": "Treatment of idiopathic pulmonary fibrosis", "status": "Positive", "designation_date": "2014-07-29", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-14-1301", "last_updated": "2023-04-03" }, { "eu_designation_number": "EU/3/14/1302", "medicine_name": "", "active_substance": "Oxytocin", "intended_use": "Treatment of Prader-Willi syndrome", "status": "Positive", "designation_date": "2014-07-29", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-14-1302", "last_updated": "2021-01-25" }, { "eu_designation_number": "EU/3/14/1303", "medicine_name": "", "active_substance": "cediranib", "intended_use": "Treatment of ovarian cancer", "status": "Positive", "designation_date": "2014-07-29", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-14-1303", "last_updated": "2014-09-01" }, { "eu_designation_number": "EU/3/14/1304", "medicine_name": "Soliris", "active_substance": "Eculizumab", "intended_use": "Treatment myasthenia gravis", "status": "Positive", "designation_date": "2014-07-29", "related_ema_product_number": "EMEA/H/C/000791", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-14-1304", "last_updated": "2019-11-20" }, { "eu_designation_number": "EU/3/14/1305", "medicine_name": "", "active_substance": "Humanised recombinant monoclonal antibody against epidermal growth factor receptor conjugated to maleimidocaproyl monomethylauristatin F(depatuxizumab mafodotin", "intended_use": "Treatment of glioma", "status": "Withdrawn", "designation_date": "2014-07-29", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-14-1305", "last_updated": "2019-10-15" }, { "eu_designation_number": "EU/3/14/1306", "medicine_name": "", "active_substance": "cysteamine bitartrate (gastroresistant) (mercaptamine)", "intended_use": "Treatment of Huntington’s disease", "status": "Positive", "designation_date": "2014-07-29", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-14-1306", "last_updated": "2014-09-01" }, { "eu_designation_number": "EU/3/14/1307", "medicine_name": "", "active_substance": "retinol", "intended_use": "Prevention of bronchopulmonary dysplasia", "status": "Positive", "designation_date": "2014-08-22", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-14-1307", "last_updated": "2014-09-29" }, { "eu_designation_number": "EU/3/14/1308", "medicine_name": "", "active_substance": "sodium ascorbate;menadione sodium bisulfite", "intended_use": "Treatment of autosomal dominant polycystic liver disease", "status": "Positive", "designation_date": "2014-08-22", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-14-1308", "last_updated": "2016-08-16" }, { "eu_designation_number": "EU/3/14/1309", "medicine_name": "Agamree", "active_substance": "17α,21-Dihydroxy-16α-methyl-pregna-1,4,9(11)-triene-3,20-dione (vamorolone)", "intended_use": "Treatment of Duchenne muscular dystrophy", "status": "Positive", "designation_date": "2014-08-22", "related_ema_product_number": "EMEA/H/C/005679", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-14-1309", "last_updated": "2022-09-14" }, { "eu_designation_number": "EU/3/14/1310", "medicine_name": "", "active_substance": "", "intended_use": "Treatment of Fabry disease", "status": "Positive", "designation_date": "2014-08-22", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-14-1310", "last_updated": "2023-07-18" }, { "eu_designation_number": "EU/3/14/1311", "medicine_name": "", "active_substance": "Gevokizumab", "intended_use": "Treatment of Schnitzler syndrome", "status": "Withdrawn", "designation_date": "2014-08-22", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-14-1311", "last_updated": "2018-07-18" }, { "eu_designation_number": "EU/3/14/1312", "medicine_name": "", "active_substance": "recombinant factor VIIa modified with three terminal repeats derived from the β chain of human chorionic gonadotropin", "intended_use": "Treatment of congenital factor VII deficiency", "status": "Positive", "designation_date": "2014-08-22", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-14-1312", "last_updated": "2021-09-16" }, { "eu_designation_number": "EU/3/14/1313", "medicine_name": "", "active_substance": "(Z)-3-(3-(3,5-bis(trifluoromethyl)phenyl)-1H-1,2,4-triazol-1-yl)-N'-(pyrazin-2-yl)acrylohydrazide", "intended_use": "Treatment of acute myeloid leukaemia", "status": "Positive", "designation_date": "2014-08-22", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-14-1313", "last_updated": "2026-05-11" }, { "eu_designation_number": "EU/3/14/1314", "medicine_name": "", "active_substance": "recombinant human apolipoprotein A-I in a complex with phospholipids", "intended_use": "Treatment of ATP-binding cassette transporter A1 deficiency", "status": "Positive", "designation_date": "2014-08-22", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-14-1314", "last_updated": "2014-09-30" }, { "eu_designation_number": "EU/3/14/1315", "medicine_name": "", "active_substance": "recombinant human apolipoprotein A-I in a complex with phospholipids", "intended_use": "Treatment of 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"2014-08-22", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-14-1317", "last_updated": "2026-05-11" }, { "eu_designation_number": "EU/3/14/1318", "medicine_name": "", "active_substance": "ulinastatin", "intended_use": "Treatment of acute pancreatitis", "status": "Positive", "designation_date": "2014-08-22", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-14-1318", "last_updated": "2014-10-01" }, { "eu_designation_number": "EU/3/14/1319", "medicine_name": "", "active_substance": "recombinant factor VIIa modified with three terminal repeats derived from the β chain of human chorionic gonadotropin", "intended_use": "Treatment of haemophilia B", "status": "Positive", "designation_date": "2014-08-22", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-14-1319", "last_updated": "2021-09-16" }, { "eu_designation_number": "EU/3/14/1320", "medicine_name": "", "active_substance": "recombinant human diamine oxidase", "intended_use": "Treatment of mastocytosis", "status": "Positive", "designation_date": "2014-08-22", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-14-1320", "last_updated": "2014-10-01" }, { "eu_designation_number": "EU/3/14/1321", "medicine_name": "", "active_substance": "adeno-associated viral vector serotype 8 containing the human UGT1A1 gene (volrubigene ralaparvovec)", "intended_use": "Treatment of Crigler-Najjar syndrome", "status": "Withdrawn", "designation_date": "2014-08-22", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-14-1321", "last_updated": "2021-03-19" }, { "eu_designation_number": "EU/3/14/1322", "medicine_name": "", "active_substance": "Humanised IgG1 monoclonal antibody against human KIR3DL2", "intended_use": "Treatment of cutaneous T-cell lymphoma", "status": "Positive", "designation_date": "2014-08-22", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-14-1322", "last_updated": "2014-10-01" }, { "eu_designation_number": "EU/3/14/1323", "medicine_name": "", "active_substance": "[5-Amino-1-(4-fluoro-phenyl)-1H-pyrazol-4-yl]-[3-(2,3-dihydroxy-propoxy)-phenyl]-methanone", "intended_use": "Treatment of pancreatic cancer", "status": "Positive", "designation_date": "2014-08-22", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-14-1323", "last_updated": "2014-10-02" }, { "eu_designation_number": "EU/3/14/1324", "medicine_name": "", "active_substance": "2-(2-Methyl-5-nitro-1H-imidazol-1-yl)ethylsulfamide", "intended_use": "Treatment of small cell lung cancer", "status": "Positive", "designation_date": "2014-08-22", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-14-1324", "last_updated": "2014-10-02" }, { "eu_designation_number": "EU/3/14/1325", "medicine_name": "", "active_substance": "obinutuzumab", "intended_use": "Treatment of diffuse large B-cell lymphoma", "status": "Withdrawn", "designation_date": "2014-08-22", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-14-1325", "last_updated": "2019-02-21" }, { "eu_designation_number": "EU/3/14/1325", "medicine_name": "", "active_substance": "obinutuzumab", "intended_use": "Treatment of diffuse large B-cell lymphoma", "status": "Positive", "designation_date": "2014-08-22", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-14-1325-0", "last_updated": "2015-01-01" }, { "eu_designation_number": "EU/3/14/1326", "medicine_name": "", "active_substance": "vector based on an adeno-associated virus serotype 2 backbone, pseudo-serotyped with a type 8 capsid, which carries the coding sequence of the human TYMP gene under the control of the human thyroxine binding globulin promoter", "intended_use": "Treatment of mitochondrial neurogastrointestinal encephalomyopathy (MNGIE)", "status": "Positive", "designation_date": "2014-08-22", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-14-1326", "last_updated": "2014-10-02" }, { "eu_designation_number": "EU/3/14/1327", "medicine_name": "", "active_substance": "S3,S13-cyclo(D-tyrolsyl-L-isoleucyl-L-cysteinyl-L-valyl-1-methyl-L-tryptophyl-L-glutaminyl-L-aspartyl-L-tryptophyl-N-methyl-L-glycyl-L-alanyl-L-histidyl-L-arginyl-L-cysteinyl-N-methyl-L-isoleucinamide)", "intended_use": "Treatment of paroxysmal nocturnal haemoglobinuria", "status": "Positive", "designation_date": "2014-10-22", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-14-1327", "last_updated": "2014-10-02" }, { "eu_designation_number": "EU/3/14/1328", "medicine_name": "", "active_substance": "4-{[(2R,3S,4R,5S)-4-(4-chloro-2-fluoro-phenyl)-3-(3-chloro-2-fluoro-phenyl)-4-cyano-5-(2,2-dimethyl-propyl)-pyrrolidine-2-carbonyl]-amino}-3-methoxy-benzoic acid ", "intended_use": "Treatment of acute myeloid leukaemia", "status": "Withdrawn", "designation_date": "2014-08-22", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-14-1328", "last_updated": "2025-01-15" }, { "eu_designation_number": "EU/3/14/1329", "medicine_name": "", "active_substance": "variant of recombinant human fibroblast growth factor 19", "intended_use": "Treatment of primary biliary cirrhosis", "status": "Withdrawn", "designation_date": "2014-08-22", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-14-1329", "last_updated": "2022-10-21" }, { "eu_designation_number": "EU/3/14/1330", "medicine_name": "", "active_substance": "Lentiviral vector containing the human liver and erythroid pyruvate kinase (PKLR) gene", "intended_use": "Treatment of pyruvate kinase deficiency", "status": "Positive", "designation_date": "2014-08-22", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-14-1330", "last_updated": "2022-11-11" }, { "eu_designation_number": "EU/3/14/1331", "medicine_name": "Reblozyl", "active_substance": "recombinant fusion protein consisting of a modified form of the extracellular domain of human activin receptor IIB linked to the human IgG1 Fc domain (luspatercept)", "intended_use": "Treatment of myelodysplastic syndromes", "status": "Positive", "designation_date": "2014-08-22", "related_ema_product_number": "EMEA/H/C/004444", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-14-1331", "last_updated": "2021-02-18" }, { "eu_designation_number": "EU/3/14/1332", "medicine_name": "", "active_substance": "Macromolecular conjugate of heparin sodium on a polymer backbone", "intended_use": "Prevention of ischaemia/reperfusion injury associated with solid organ transplantation", "status": "Positive", "designation_date": "2014-08-22", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-14-1332", "last_updated": "2016-03-29" }, { "eu_designation_number": "EU/3/14/1333", "medicine_name": "", "active_substance": "Lumacaftor;ivacaftor", "intended_use": "Treatment of cystic fibrosis", "status": "Withdrawn", "designation_date": "2014-08-22", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-14-1333", "last_updated": "2018-02-12" }, { "eu_designation_number": "EU/3/14/1334", "medicine_name": "", "active_substance": "", "intended_use": "Treatment of fragile X syndrome", "status": "Positive", "designation_date": "2014-10-15", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-14-1334", "last_updated": "2014-11-12" }, { "eu_designation_number": "EU/3/14/1335", "medicine_name": "", "active_substance": "(S)-2-(1-((6-amino-5-cyanopyrimidin-4-yl)amino)ethyl)-4-oxo-3-phenyl-3,4-dihydropyrrolo[2,1-f][1,2,4]triazine-5-carbonitrile (tanziertib)", "intended_use": "Treatment of pemphigus", "status": "Withdrawn", "designation_date": "2014-10-15", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-14-1335", "last_updated": "2018-02-22" }, { "eu_designation_number": "EU/3/14/1336", "medicine_name": "", "active_substance": "(S)-6-hydroxy-2,5,7,8-tetramethyl-N-((R)-piperidin-3-yl)chroman-2-carboxamide hydrochloride", "intended_use": "Treatment of Leigh syndrome", "status": "Withdrawn", "designation_date": "2014-10-15", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-14-1336", "last_updated": "2023-03-27" }, { "eu_designation_number": "EU/3/14/1337", "medicine_name": "", "active_substance": "acamprosate calcium", "intended_use": "Treatment of fragile X syndrome", "status": "Positive", "designation_date": "2014-10-15", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-14-1337", "last_updated": "2022-11-03" }, { "eu_designation_number": "EU/3/14/1338", "medicine_name": "", "active_substance": "adeno-associated viral vector serotype 8 containing the human UGT1A1 gene (volrubigene ralaparvovec)", "intended_use": "Treatment of Crigler-Najjar syndrome", "status": "Positive", "designation_date": "2014-10-15", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-14-1338", "last_updated": "2014-11-12" }, { "eu_designation_number": "EU/3/14/1339", "medicine_name": "Epidyolex", "active_substance": "cannabidiol", "intended_use": "Treatment of Dravet syndrome", "status": "Positive", "designation_date": "2014-10-15", "related_ema_product_number": "EMEA/H/C/004675", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-14-1339", "last_updated": "2023-06-14" }, { "eu_designation_number": "EU/3/14/1340", "medicine_name": "", "active_substance": "cultured allogeneic corneal limbal stem cells", "intended_use": "Treatment of limbal stem cell deficiency", "status": "Positive", "designation_date": "2014-10-15", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-14-1340", "last_updated": "2014-11-12" }, { "eu_designation_number": "EU/3/14/1341", "medicine_name": "Dropcys", "active_substance": "cysteamine hydrochloride", "intended_use": "Treatment of cystinosis", "status": "Positive", "designation_date": "2014-10-15", "related_ema_product_number": "EMEA/H/C/004038", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-14-1341", "last_updated": "2014-11-12" }, { "eu_designation_number": "EU/3/14/1342", "medicine_name": "", "active_substance": "Glucagon", "intended_use": "Treatment of congenital hyperinsulinism", "status": "Positive", "designation_date": "2009-10-08", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-14-1342", "last_updated": "2023-06-09" }, { "eu_designation_number": "EU/3/14/1343", "medicine_name": "", "active_substance": "Immunoglobulin G1, anti-(human tumour-associated calcium signal transducer 2)(human-Mus musculus monoclonal hRS7 heavy chain), disulfide with human-Mus musculus monoclonal hRS7 κ-chain, dimer, hexakis(thioether) with (4S)-4-[[[[4-[[(2S)-2-(4-aminobutyl)-2-[[2-[2-[[26-[4-[[[[4-[(3-mercapto-2,5-dioxo-1-pyrrolidinyl)methyl]cyclohexyl]carbonyl]amino]methyl]-1H-1,2,3-triazol-1-yl]-3,6,9,12,15,18,21,24-octaoxahexacos-1-yl]amino]-2-oxoethoxy]acetyl]amino]-1-oxoethyl]amino]phenyl]methoxy]carbonyl]oxy]-4,11-diethyl-9-hydroxy-1H-pyrano[3',4':6,7]indolizino[1,2-b]quinoline-3,14(4H,12H)-dione (sacituzumab govitecan)", "intended_use": "Treatment of pancreatic cancer", "status": "Withdrawn", "designation_date": "2014-10-15", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-14-1343", "last_updated": "2026-08-21" }, { "eu_designation_number": "EU/3/14/1344", "medicine_name": "", "active_substance": "nitric oxide", "intended_use": "Treatment of cystic fibrosis", "status": "Positive", "designation_date": "2014-10-15", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-14-1344", "last_updated": 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"designation_date": "2014-10-15", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-14-1347", "last_updated": "2014-11-12" }, { "eu_designation_number": "EU/3/14/1348", "medicine_name": "", "active_substance": "recombinant human bone morphogenetic protein 4", "intended_use": "Treatment of glioma", "status": "Positive", "designation_date": "2014-10-15", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-14-1348", "last_updated": "2020-11-30" }, { "eu_designation_number": "EU/3/14/1349", "medicine_name": "", "active_substance": "recombinant human insulin receptor monoclonal antibody-fused-α-L-iduronidase", "intended_use": "Treatment of mucopolysaccharidosis type I", "status": "Withdrawn", "designation_date": "2014-10-15", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-14-1349", "last_updated": 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"eu_designation_number": "EU/3/14/1352", "medicine_name": "", "active_substance": "raxibacumab", "intended_use": "Treatment of inhalation anthrax disease", "status": "Positive", "designation_date": "2014-10-15", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-14-1352", "last_updated": "" }, { "eu_designation_number": "EU/3/14/1353", "medicine_name": "Namuscla", "active_substance": "Mexiletine hydrochloride", "intended_use": "Treatment of myotonic disorders", "status": "Positive", "designation_date": "2014-11-19", "related_ema_product_number": "EMEA/H/C/004584", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-14-1353", "last_updated": "2018-06-13" }, { "eu_designation_number": "EU/3/14/1354", "medicine_name": "", "active_substance": "selinexor", "intended_use": "Treatment of chronic lymphocytic leukaemia / small lymphocytic lymphoma", "status": "Positive", "designation_date": "2014-11-19", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-14-1354", "last_updated": "2026-05-11" }, { "eu_designation_number": "EU/3/14/1355", "medicine_name": "", "active_substance": "selinexor", "intended_use": "Treatment of plasma cell myeloma", "status": "Positive", "designation_date": "2014-11-19", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-14-1355", "last_updated": "2021-06-07" }, { "eu_designation_number": "EU/3/14/1356", "medicine_name": "Luxceptar", "active_substance": "Donor T lymphocytes depleted ex-vivo of host alloreactive T cells using photodynamic treatment", "intended_use": "Treatment of acute myeloid leukaemia", "status": "Withdrawn", "designation_date": "2014-11-19", "related_ema_product_number": "EMEA/H/C/002397", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-14-1356", "last_updated": "2022-02-28" }, { "eu_designation_number": "EU/3/14/1357", "medicine_name": "", "active_substance": "imatinib", "intended_use": "Treatment of acute respiratory distress syndrome (ARDS)", "status": "Positive", "designation_date": "2014-11-19", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-14-1357", "last_updated": "2021-08-06" }, { "eu_designation_number": "EU/3/14/1358", "medicine_name": "", "active_substance": "recombinant human pentraxin-2", "intended_use": "Treatment of post-essential thrombocythaemia myelofibrosis", "status": "Positive", "designation_date": "2014-11-19", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-14-1358", "last_updated": "2020-06-08" }, { "eu_designation_number": "EU/3/14/1359", "medicine_name": "", "active_substance": "pentosan polysulfate sodium", "intended_use": "Treatment of mucopolysaccharidosis type I", "status": "Positive", "designation_date": "2014-11-19", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-14-1359", "last_updated": "2022-12-15" }, { "eu_designation_number": "EU/3/14/1360", "medicine_name": "", "active_substance": "A combination of H-Lys-Lys-Gly-Pro-Arg-Cys(SH)-Leu-Thr-Arg-Tyr-Tyr-Ser-Ser-Phe-Val-Asn-Met-Glu-Gly-Lys-Lys-OH and H-Lys-Lys-Gly-Asp-Asn-Ile-Met-Val-Thr-Phe-Arg-Asn-Gln-Ala-Ser-Arg-Pro-Tyr-Gly-Lys-Lys-OH", "intended_use": "Treatment of haemophilia A", "status": "Withdrawn", "designation_date": "2014-11-19", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-14-1360", "last_updated": "2024-11-19" }, { "eu_designation_number": "EU/3/14/1361", "medicine_name": "", "active_substance": "1-(6-benzothiazolylsulfonyl)-5-chloro-1H-indole-2-butanoic acid (lanifibranor)", "intended_use": "Treatment of systemic sclerosis", "status": "Withdrawn", "designation_date": "2014-11-19", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-14-1361", "last_updated": "2020-12-14" }, { "eu_designation_number": "EU/3/14/1362", "medicine_name": "", "active_substance": "1-(6-benzothiazolylsulfonyl)-5-chloro-1H-indole-2-butanoic acid (lanifibranor)", "intended_use": "Treatment of idiopathic pulmonary fibrosis", "status": "Positive", "designation_date": "2014-11-19", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-14-1362", "last_updated": "2015-01-15" }, { "eu_designation_number": "EU/3/14/1363", "medicine_name": "", "active_substance": "4-[[(1S,4S)-5-[[4-[4-(oxazol-2-yl)phenoxy]phenyl]methyl]-2,5-diazabicyclo[2.2.1]hept-2-yl]methyl]benzoic acid (acebilustat)", "intended_use": "Treatment of cystic fibrosis", "status": "Positive", "designation_date": "2014-11-19", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-14-1363", "last_updated": "2019-02-19" }, { "eu_designation_number": "EU/3/14/1364", "medicine_name": "", "active_substance": "olaptesed pegol", "intended_use": "Treatment of glioma", "status": "Positive", "designation_date": "2014-11-19", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-14-1364", "last_updated": "2015-01-15" }, { "eu_designation_number": "EU/3/14/1365", "medicine_name": "", "active_substance": "recombinant human pentraxin-2", "intended_use": "Treatment of post-polycythaemia vera myelofibrosis", "status": "Positive", "designation_date": "2014-11-19", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-14-1365", "last_updated": "2020-06-08" }, { "eu_designation_number": "EU/3/14/1366", "medicine_name": "", "active_substance": "recombinant human pentraxin-2", "intended_use": "Treatment of primary myelofibrosis", "status": "Positive", "designation_date": "2014-11-19", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-14-1366", "last_updated": "2020-06-08" }, { "eu_designation_number": "EU/3/14/1367", "medicine_name": "", "active_substance": "bazedoxifene acetate", "intended_use": "Treatment of hereditary haemorrhagic telangiectasia", "status": "Positive", "designation_date": "2014-11-19", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-14-1367", "last_updated": "2015-01-15" }, { "eu_designation_number": "EU/3/14/1368", "medicine_name": "Sohonos", "active_substance": "palovarotene", "intended_use": "Treatment of fibrodysplasia ossificans progressiva", "status": "Withdrawn", "designation_date": "2014-11-19", "related_ema_product_number": "EMEA/H/C/004867", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-14-1368", "last_updated": "2019-07-18" }, { "eu_designation_number": "EU/3/14/1369", "medicine_name": "", "active_substance": "siponimod", "intended_use": "Treatment of dermatomyositis", "status": "Withdrawn", "designation_date": "2014-11-19", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-14-1369", "last_updated": "2018-03-12" }, { "eu_designation_number": "EU/3/14/1370", "medicine_name": "", "active_substance": "siponimod", "intended_use": "Treatment of polymyositis", "status": "Withdrawn", "designation_date": "2014-11-19", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-14-1370", "last_updated": "2018-03-12" }, { "eu_designation_number": "EU/3/14/1371", "medicine_name": "", "active_substance": "Humanised IgG1 monoclonal antibody against human eotaxin-2", "intended_use": "Treatment of systemic sclerosis", "status": "Positive", "designation_date": "2014-11-19", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-14-1371", "last_updated": "2016-08-15" }, { "eu_designation_number": "EU/3/14/1372", "medicine_name": "Vynpenta (previously AvacopanChemoCentryx); Tavneos", "active_substance": "(2R,3S)-2-(4-cyclopentylaminophenyl)-1-(2-fluoro-6-methylbenzoyl)piperidine-3-carboxylic acid(4-methyl-3-trifluoromethylphenyl)amNide", "intended_use": "Treatment of microscopic polyangiitis (avacopan)", "status": "Positive", "designation_date": "2014-11-19", "related_ema_product_number": "EMEA/H/C/004487; EMEA/H/C/005523", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-14-1372", "last_updated": "2022-07-04" }, { "eu_designation_number": "EU/3/14/1373", "medicine_name": "Vynpenta (previously AvacopanChemoCentryx); Tavneos", "active_substance": "", "intended_use": "Treatment of granulomatosis with polyangiitis", "status": "Positive", "designation_date": "2014-11-19", "related_ema_product_number": "EMEA/H/C/004487; EMEA/H/C/005523", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-14-1373", "last_updated": "2022-07-04" }, { "eu_designation_number": "EU/3/14/1374", "medicine_name": "", "active_substance": "(3S)-1-azabicyclo[2.2.2]oct-3-yl{2-[2-(4-fluorophenyl)-1,3-thiazol-4-yl]propan-2-yl}carbaNmate (venglustat)", "intended_use": "Treatment of Gaucher disease", "status": "Positive", "designation_date": "2014-11-19", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-14-1374", "last_updated": "2023-07-18" }, { "eu_designation_number": "EU/3/14/1375", "medicine_name": "", "active_substance": "Pro-Pro-Thr-Val-Pro-Thr-Arg", "intended_use": "Treatment of xeroderma pigmentosum", "status": "Withdrawn", "designation_date": "2014-11-19", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-14-1375", "last_updated": "2022-02-25" }, { "eu_designation_number": "EU/3/14/1376", "medicine_name": "Miplyffa; Meplyffa", "active_substance": "arimoclomol citrate", "intended_use": "Treatment of Niemann-Pick's disease, type C", "status": "Positive", "designation_date": "2014-11-19", "related_ema_product_number": "EMEA/H/C/005203; EMEA/H/C/006736", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-14-1376", "last_updated": "2023-04-11" }, { "eu_designation_number": "EU/3/14/1377", "medicine_name": "", "active_substance": "chloroquine", "intended_use": "Treatment of glioma", "status": "Positive", "designation_date": "2014-11-19", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-14-1377", "last_updated": "2015-01-16" }, { "eu_designation_number": "EU/3/14/1378", "medicine_name": "", "active_substance": "Diaspirin cross-linked haemoglobin", "intended_use": "Treatment of hepatocellular carcinoma", "status": "Positive", "designation_date": "2014-11-19", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-14-1378", "last_updated": "2019-09-30" }, { "eu_designation_number": "EU/3/14/1379", "medicine_name": "", "active_substance": "Dantrolene sodium", "intended_use": "Treatment of malignant hyperthermia", "status": "Positive", "designation_date": "2014-11-19", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-14-1379", "last_updated": "2015-01-16" }, { "eu_designation_number": "EU/3/14/1380", "medicine_name": "", "active_substance": "ataluren", "intended_use": "Treatment of mucopolysaccharidosis type I", "status": "Withdrawn", "designation_date": "2014-11-19", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-14-1380", "last_updated": "2018-02-22" }, { "eu_designation_number": "EU/3/14/1381", "medicine_name": "", "active_substance": "adeno-associated viral vector serotype 8 containing the human MD1 gene", "intended_use": "Treatment of Duchenne muscular dystrophy", "status": "Positive", "designation_date": "2014-11-19", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-14-1381", "last_updated": "2015-01-16" }, { "eu_designation_number": "EU/3/14/1382", "medicine_name": "", "active_substance": "allogeneic CD34+ cells expanded ex vivo with an aryl hydrocarbon receptor antagonist (spanlecortemlocel)", "intended_use": "Treatment of acute lymphoblastic leukaemia", "status": "Withdrawn", "designation_date": "2014-12-16", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-14-1382", "last_updated": "2022-09-16" }, { "eu_designation_number": "EU/3/14/1383", "medicine_name": "", "active_substance": "single-chain urokinase plasminogen activator", "intended_use": "Treatment of pleural empyema", "status": "Positive", "designation_date": "2014-12-16", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-14-1383", "last_updated": "2021-09-27" }, { "eu_designation_number": "EU/3/14/1384", "medicine_name": "", "active_substance": "", "intended_use": "Treatment of WHIM syndrome", "status": "Positive", "designation_date": "2014-12-16", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-14-1384", "last_updated": "2015-02-26" }, { "eu_designation_number": "EU/3/14/1385", "medicine_name": "", "active_substance": "Heat-killed Mycobacterium obuense (whole cell)", "intended_use": "Treatment of pancreatic cancer", "status": "Positive", "designation_date": "2014-12-16", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-14-1385", "last_updated": "2022-03-08" }, { "eu_designation_number": "EU/3/14/1386", "medicine_name": "", "active_substance": "2-hydroxymethyl-2-methoxymethyl-1-azabicyclo[2,2,2]octan-3-one", "intended_use": "Treatment of ovarian cancer", "status": "Positive", "designation_date": "2014-12-16", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-14-1386", "last_updated": "2020-10-13" }, { "eu_designation_number": "EU/3/14/1387", "medicine_name": "", "active_substance": "Exisulind", "intended_use": "Treatment of familial cerebral cavernous malformations", "status": "Positive", "designation_date": "2014-12-16", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-14-1387", "last_updated": "2015-02-26" }, { "eu_designation_number": "EU/3/14/1388", "medicine_name": "", "active_substance": "allogeneic bone marrow derived mesenchymal cells expanded ex vivo in synthetic media", "intended_use": "Prevention of graft-versus-host disease", "status": "Positive", "designation_date": "2014-12-16", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-14-1388", "last_updated": "2015-02-26" }, { "eu_designation_number": "EU/3/14/1389", "medicine_name": "", "active_substance": "Adeno-associated viral vector serotype rh.rh.10 carrying the human N-sulfoglucosamine sulfohydrolase cDNA", "intended_use": "Treatment of mucopolysaccharidosis, type IIIA (Sanfilippo A syndrome)", "status": "Positive", "designation_date": "2014-12-16", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-14-1389", "last_updated": "2015-05-13" }, { "eu_designation_number": "EU/3/14/1390", "medicine_name": "Orblid", "active_substance": "bevacizumab", "intended_use": "Treatment of hereditary haemorrhagic 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"https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-14-1392", "last_updated": "2015-02-26" }, { "eu_designation_number": "EU/3/14/1393", "medicine_name": "Yescarta", "active_substance": "autologous T cells transduced with retroviral vector encoding an anti-CD19 CD28/CD3 zeta chimeric antigen receptor (axicabtagene ciloleucel)", "intended_use": "Treatment of diffuse large B-cell lymphoma", "status": "Positive", "designation_date": "2014-12-16", "related_ema_product_number": "EMEA/H/C/004480", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-14-1393", "last_updated": "2022-12-16" }, { "eu_designation_number": "EU/3/14/1394", "medicine_name": "", "active_substance": "pegylated recombinant human hyaluronidase PH20(pegvorhyaluronidase alfa)", "intended_use": "Treatment of pancreatic cancer", "status": "Withdrawn", "designation_date": "2014-12-16", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-14-1394", "last_updated": "2019-02-22" }, { "eu_designation_number": "EU/3/14/1395", "medicine_name": "", "active_substance": "allogeneic ex vivo-generated natural killer cells from CD34+ umbilical cord blood progenitor cells", "intended_use": "Treatment of acute myeloid leukaemia", "status": "Positive", "designation_date": "2014-12-16", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-14-1395", "last_updated": "2015-02-26" }, { "eu_designation_number": "EU/3/14/1396", "medicine_name": "", "active_substance": "adenovirus serotype 5 containing partial E1A deletion and an integrin-binding domain (tasadenoturev)", "intended_use": "Treatment of glioma", "status": "Positive", "designation_date": "2014-12-16", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-14-1396", "last_updated": "2021-09-16" }, { "eu_designation_number": "EU/3/14/1397", "medicine_name": "", "active_substance": "amikacin sulfate", "intended_use": "Treatment of Pseudomonas aeruginosa lung infection in cystic fibrosis", "status": "Positive", "designation_date": "2014-12-16", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-14-1397", "last_updated": "2015-02-26" }, { "eu_designation_number": "EU/3/14/1398", "medicine_name": "", "active_substance": "genetically modified serotype 5/3 adenovirus coding for granulocyte macrophage colony-stimulating factor", "intended_use": "Treatment of malignant mesothelioma", "status": "Withdrawn", "designation_date": "2014-12-16", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-14-1398", "last_updated": "2026-05-26" }, { "eu_designation_number": "EU/3/14/1399", "medicine_name": "", "active_substance": "edaravone", "intended_use": "Treatment of amyotrophic lateral sclerosis", "status": "Positive", "designation_date": "2014-12-16", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-14-1399", "last_updated": "2024-10-31" }, { "eu_designation_number": "EU/3/14/1400", "medicine_name": "", "active_substance": "(1S,4R,5R,7S)-3,4-dibenzyl-2-oxo-6,8-dioxa-3-azabyciclo[3.2.1]octane-7-carboxylic acid-L-lysine", "intended_use": "Treatment of neurotrophic keratitis", "status": "Positive", "designation_date": "2014-12-16", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-14-1400", "last_updated": "2018-06-08" }, { "eu_designation_number": "EU/3/14/1401", "medicine_name": "", "active_substance": "riluzole", "intended_use": "Treatment of traumatic spinal cord injury", "status": "Positive", "designation_date": "2014-12-16", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-14-1401", "last_updated": "2015-02-27" }, { "eu_designation_number": "EU/3/14/1402", "medicine_name": "", "active_substance": "benserazide hydrochloride", "intended_use": "Treatment of beta thalassaemia intermedia and major", "status": "Positive", "designation_date": "2014-12-16", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-14-1402", "last_updated": "2015-02-27" }, { "eu_designation_number": "EU/3/14/1403", "medicine_name": "", "active_substance": "Plerixafor", "intended_use": "Treatment of WHIM syndrome", "status": "Positive", "designation_date": "2014-12-16", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-14-1403", "last_updated": "2015-02-27" }, { "eu_designation_number": "EU/3/14/1404", "medicine_name": "", "active_substance": "1-(2-isopropoxyethyl)-2-thioxo-1,2,3,5-tetrahydro-pyrrolo[3,2-d] pyrimidin-4-one (verdiperstat)", "intended_use": "Treatment of multiple system atrophy", "status": "Positive", "designation_date": "2014-12-16", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-14-1404", "last_updated": "2021-09-21" }, { "eu_designation_number": "EU/3/14/1405", "medicine_name": "", "active_substance": "autologous collagen type II-specific regulatory T cells", "intended_use": "Treatment of non-infectious uveitis", "status": "Withdrawn", "designation_date": "2014-12-16", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-14-1405", "last_updated": "2019-11-19" }, { "eu_designation_number": "EU/3/14/1406", "medicine_name": "Chenodeoxycholic acid Leadiant (previously Chenodeoxycholic acid sigma-tau)", "active_substance": "chenodeoxycholic acid", "intended_use": "Treatment of inborn errors in primary bile acid synthesis", "status": "Positive", "designation_date": "2014-12-16", "related_ema_product_number": "EMEA/H/C/004061", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-14-1406", "last_updated": "2026-08-21" }, { "eu_designation_number": "EU/3/14/1407", "medicine_name": "", "active_substance": "allogeneic adipose-derived adult mesenchymal stem cells contained in a fibrin-based bioengineered dermis", "intended_use": "Treatment of epidermolysis bullosa", "status": "Positive", "designation_date": "2014-12-16", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-14-1407", "last_updated": "2015-02-27" }, { "eu_designation_number": "EU/3/14/1408", "medicine_name": "", "active_substance": "5,5'-(4-(trifluromethyl)benzylazanediyl)bis(methylene)diquinolin-8-ol", "intended_use": "Treatment of glioma", "status": "Positive", "designation_date": "2014-12-16", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-14-1408", "last_updated": "2015-02-27" }, { "eu_designation_number": "EU/3/14/1409", "medicine_name": "", "active_substance": "pegylated recombinant arginine deiminase (pegargiminase)", "intended_use": "Treatment of malignant mesothelioma", "status": "Positive", "designation_date": "2015-01-15", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-14-1409", "last_updated": "2022-08-02" }, { "eu_designation_number": "EU/3/14/1410", "medicine_name": "", "active_substance": "recombinant human aspartylglucosaminidase", "intended_use": "Treatment of aspartylglucosaminuria", "status": "Withdrawn", "designation_date": "2015-01-15", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-14-1410", "last_updated": "2021-06-25" }, { "eu_designation_number": "EU/3/14/1411", "medicine_name": "", "active_substance": "pentosan polysulfate sodium", "intended_use": "Treatment of interstitial cystitis", "status": "Withdrawn", "designation_date": "2015-01-15", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-14-1411", "last_updated": "2016-02-09" }, { "eu_designation_number": "EU/3/14/1412", "medicine_name": "", "active_substance": "Herpes simplex type 1 virus containing cellular B-myb gene as tumour-specific promoter", "intended_use": "Treatment of pancreatic cancer", "status": "Positive", "designation_date": "2015-01-15", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-14-1412", "last_updated": "2015-03-05" }, { "eu_designation_number": "EU/3/14/1413", "medicine_name": "", "active_substance": "allogeneic, umbilical cord blood-derived, ex vivo-expanded, haematopoietic CD133+ cells;allogeneic, umbilical cord blood-derived, non-expanded, haematopoietic CD133- cells", "intended_use": "Treatment of acute myeloid leukaemia", "status": "Withdrawn", "designation_date": "2015-01-15", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-14-1413", "last_updated": "2018-01-23" }, { "eu_designation_number": "EU/3/14/1414", "medicine_name": "", "active_substance": "sodium thiosulfate", "intended_use": "Treatment for calciphylaxis", "status": "Positive", "designation_date": "2015-01-15", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-14-1414", "last_updated": "2019-08-19" }, { "eu_designation_number": "EU/3/14/1415", "medicine_name": "", "active_substance": "adenoviral vector serotype 5 containing the vascular endothelial growth factor D isoform (preprocessed short form) from a CMV promoter", "intended_use": "Treatment of placental insufficiency", "status": "Positive", "designation_date": "2015-01-15", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-14-1415", "last_updated": "2015-03-05" }, { "eu_designation_number": "EU/3/14/1416", "medicine_name": "", "active_substance": "chimeric monoclonal antibody to O-acetyl-GD2 antigen", "intended_use": "Treatment of neuroblastoma", "status": "Positive", "designation_date": "2015-01-15", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-14-1416", "last_updated": "2016-08-15" }, { "eu_designation_number": "EU/3/14/1417", "medicine_name": "", "active_substance": "A lentiviral vector pseudotyped by the Indiana serotype of the vesicular stomatitis virus G protein encoding an antigen derived from the Tax, HBZ, p12I and p30II HTLV-1 proteins", "intended_use": "Treatment of adult T-cell leukaemia/lymphoma", "status": "Positive", "designation_date": "2015-01-15", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-14-1417", "last_updated": "2015-03-05" }, { "eu_designation_number": "EU/3/14/1418", "medicine_name": "", "active_substance": "A lentiviral vector pseudotyped by the New-Jersey serotype of the vesicular stomatitis virus G protein encoding an antigen derived from the Tax, HBZ, p12I and p30II HTLV-1 proteins", "intended_use": "Treatment of adult T-cell leukaemia/lymphoma", "status": "Positive", "designation_date": "2015-01-15", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-14-1418", "last_updated": "2015-03-05" }, { "eu_designation_number": "EU/3/14/1419", "medicine_name": "", "active_substance": "tenofovir disoproxil fumarate", "intended_use": "Treatment of Aicardi-Goutières syndrome", "status": "Positive", "designation_date": "2015-01-15", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-14-1419", "last_updated": "2015-03-05" }, { "eu_designation_number": "EU/3/14/1420", "medicine_name": "", "active_substance": "emtricitabine", "intended_use": "Treatment of Aicardi-Goutières syndrome", "status": "Positive", "designation_date": "2015-01-15", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-14-1420", "last_updated": "2015-03-06" }, { "eu_designation_number": "EU/3/14/1421", "medicine_name": "", "active_substance": "ponatinib hydrochloride", "intended_use": "Treatment of gastrointestinal stromal tumours", "status": "Withdrawn", "designation_date": "2015-01-15", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-14-1421", "last_updated": "2022-03-18" }, { "eu_designation_number": "EU/3/14/1422", "medicine_name": "", "active_substance": "chimeric fusion protein of recombinant human alpha-N-acetylglucosaminidase and human insulin-like growth factor 2 (tralesinidase alfa)", "intended_use": "Treatment of mucopolysaccharidosis type IIIB", "status": "Positive", "designation_date": "2015-01-15", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-14-1422", "last_updated": "2022-01-14" }, { "eu_designation_number": "EU/3/14/1423", "medicine_name": "", "active_substance": "synthetic signal peptide of human mucin-1 (amino acids 1-21)", "intended_use": "Treatment of plasma cell myeloma", "status": "Positive", "designation_date": "2015-01-15", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-14-1423", "last_updated": "2022-06-22" }, { "eu_designation_number": "EU/3/14/1424", "medicine_name": "Minjuvi", "active_substance": "Humanised Fc engineered monoclonal antibody against CD19 (tafasitamab)", "intended_use": "Treatment of diffuse large B-cell lymphoma", "status": "Positive", "designation_date": "2015-01-15", "related_ema_product_number": "EMEA/H/C/005436", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-14-1424", "last_updated": "2021-09-08" }, { "eu_designation_number": "EU/3/14/1425", "medicine_name": "", "active_substance": "ceftriaxone", "intended_use": "Treatment of spinocerebellar ataxia", "status": "Positive", "designation_date": "2015-01-15", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-14-1425", "last_updated": "2015-03-06" }, { "eu_designation_number": "EU/3/14/1426", "medicine_name": "", "active_substance": "allogeneic peripheral blood mononuclear cells induced to an early apoptotic state", "intended_use": "Prevention of graft-versus-host disease", "status": "Withdrawn", "designation_date": "2015-01-15", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-14-1426", "last_updated": "2022-02-28" }, { "eu_designation_number": "EU/3/14/1427", "medicine_name": "", "active_substance": "recombinant human alkaline phosphatase", "intended_use": "Treatment of hypophosphatasia", "status": "Positive", "designation_date": "2015-01-15", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-14-1427", "last_updated": "2015-03-06" }, { "eu_designation_number": "EU/3/14/1428", "medicine_name": "", "active_substance": "sodium valproate fNor", "intended_use": "Treatment of Wolfram syndrome", "status": "Positive", "designation_date": "2015-01-15", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-14-1428", "last_updated": "2021-09-16" }, { "eu_designation_number": "EU/3/14/1429", "medicine_name": "", "active_substance": "N-methyl-4-({4-[({3-methyl(methylsulfonyl)aminopyrazin-2-yl}methyl)amino]-5-(trifluoromethyl)pyrimidin-2-yl}amino)benzamide hydrochloride (defactinib)", "intended_use": "Treatment of ovarian cancer", "status": "Positive", "designation_date": "2015-01-15", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-14-1429", "last_updated": "2022-05-24" }, { "eu_designation_number": "EU/3/14/1430", "medicine_name": "", "active_substance": "adeno-associated viral vector serotype 8 containing the human factor-VII gene", "intended_use": "Treatment of congenital factor VII deficiency", "status": "Positive", "designation_date": "2015-01-15", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-14-1430", "last_updated": "2020-03-05" }, { "eu_designation_number": "EU/3/15/1431", "medicine_name": "", "active_substance": "3-[2-(4-carbamimidoyl-phenylcarbamoyl)-5-methoxy-4-vinyl-phenyl]-6-(cyclopropylmethyl-carbamoyl)-pyridine-2-carboxylic acid (avoralstat)", "intended_use": "Treatment of hereditary angioedema", "status": "Withdrawn", "designation_date": "2015-02-12", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-15-1431", "last_updated": "2017-04-25" }, { "eu_designation_number": "EU/3/15/1432", "medicine_name": "", "active_substance": "2'-O-methyl phosphorothioate RNA oligonucleotide, 5'-m5CUGm5CUGm5CUGm5CUGm5CUGm5CUGm5CUG-3'", "intended_use": "Treatment of Huntington’s disease", "status": "Positive", "designation_date": "2015-02-12", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-15-1432", "last_updated": "2020-06-23" }, { "eu_designation_number": "EU/3/15/1433", "medicine_name": "", "active_substance": "sevuparin sodium", "intended_use": "Treatment of sickle cell disease", "status": "Positive", "designation_date": "2015-02-12", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-15-1433", "last_updated": "2017-03-17" }, { "eu_designation_number": "EU/3/15/1434", "medicine_name": "", "active_substance": "chimeric group B adenovirus (11p/3) with deletions in the E3 and E4 regions (enadenotucirev)", "intended_use": "Treatment of ovarian cancer", "status": "Withdrawn", "designation_date": "2015-02-12", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-15-1434", "last_updated": "2021-06-25" }, { "eu_designation_number": "EU/3/15/1435", "medicine_name": "", "active_substance": "nitroglycerin", "intended_use": "Treatment of systemic sclerosis", "status": "Positive", "designation_date": "2015-02-12", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-15-1435", "last_updated": "2015-04-08" }, { "eu_designation_number": "EU/3/15/1436", "medicine_name": "", "active_substance": "Lactobacillus reuteri", "intended_use": "Prevention of necrotising enterocolitis", "status": "Positive", "designation_date": "2015-02-12", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-15-1436", "last_updated": "2015-04-09" }, { "eu_designation_number": "EU/3/15/1437", "medicine_name": "", "active_substance": "alvocidib", "intended_use": "Treatment of acute myeloid leukaemia", "status": "Positive", "designation_date": "2015-02-12", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-15-1437", "last_updated": "2022-03-07" }, { "eu_designation_number": "EU/3/15/1438", "medicine_name": "", "active_substance": "allogeneic CD4+ and CD8+ T lymphocytes ex vivo incubated with synthetic peptides of the viral antigens of cytomegalovirus, adenovirus and 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"EU/3/15/1445", "medicine_name": "", "active_substance": "ulocuplumab", "intended_use": "Treatment of acute myeloid leukaemia", "status": "Withdrawn", "designation_date": "2015-02-12", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-15-1445", "last_updated": "2020-01-13" }, { "eu_designation_number": "EU/3/15/1446", "medicine_name": "", "active_substance": "N-(3-(4-(3-(diisobutylamino)propyl)piperazin-1-yl)propyl)-1H-benzo[d]imidazol-2-amine disulfate salt", "intended_use": "Treatment of progressive supranuclear palsy", "status": "Positive", "designation_date": "2015-02-12", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-15-1446", "last_updated": "2019-07-10" }, { "eu_designation_number": "EU/3/15/1447", "medicine_name": "", "active_substance": "olaratumab", "intended_use": "Treatment of soft tissue sarcoma", "status": "Withdrawn", "designation_date": "2015-02-12", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-15-1447", "last_updated": "2020-01-15" }, { "eu_designation_number": "EU/3/15/1448", "medicine_name": "", "active_substance": "505 amino acid protein, corresponding to amino acids 2-506 of the wild type human histidyl-tRNA synthetase", "intended_use": "treatment of facioscapulohumeral muscular dystrophy", "status": "Withdrawn", "designation_date": "2015-02-12", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-15-1448", "last_updated": "2020-02-12" }, { "eu_designation_number": "EU/3/15/1449", "medicine_name": "", "active_substance": "myriocin", "intended_use": "Treatment of retinitis pigmentosa", "status": "Positive", "designation_date": "2015-02-12", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-15-1449", "last_updated": "2021-10-13" 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"Withdrawn", "designation_date": "2015-03-19", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-15-1452", "last_updated": "2019-02-21" }, { "eu_designation_number": "EU/3/15/1453", "medicine_name": "", "active_substance": "chimeric 2'-O-(2-methoxyethyl) modified oligonucleotide targeted to huntingtin RNA", "intended_use": "Treatment of Huntington’s disease", "status": "Positive", "designation_date": "2015-03-19", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-15-1453", "last_updated": "2018-05-30" }, { "eu_designation_number": "EU/3/15/1454", "medicine_name": "", "active_substance": "6-ethoxy-7-methoxy-2-(2-methylsulfanylphenyl)-3,1-benzoxazin-4-one", "intended_use": "Treatment of Netherton syndrome", "status": "Positive", "designation_date": "2015-03-19", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-15-1454", "last_updated": "2015-05-11" }, { "eu_designation_number": "EU/3/15/1455", "medicine_name": "", "active_substance": "Human plasma-derived alpha-1 proteinase inhibitor", "intended_use": "Treatment of graft-versus-host disease", "status": "Positive", "designation_date": "2015-03-19", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-15-1455", "last_updated": "2019-05-27" }, { "eu_designation_number": "EU/3/15/1456", "medicine_name": "", "active_substance": "recombinant human club cell 10 KDa protein", "intended_use": "Prevention of bronchopulmonary dysplasia", "status": "Positive", "designation_date": "2015-03-19", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-15-1456", "last_updated": "2015-05-11" }, { "eu_designation_number": "EU/3/15/1457", "medicine_name": "Turalio", 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"Enoxacin", "intended_use": "Treatment of amyotrophic lateral sclerosis", "status": "Positive", "designation_date": "2015-03-19", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-15-1459", "last_updated": "2021-09-02" }, { "eu_designation_number": "EU/3/15/1460", "medicine_name": "", "active_substance": "lenvatinib", "intended_use": "Treatment of hepatocellular carcinoma", "status": "Withdrawn", "designation_date": "2015-03-19", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-15-1460", "last_updated": "2018-10-22" }, { "eu_designation_number": "EU/3/15/1461", "medicine_name": "", "active_substance": "recombinant human monoclonal antibody binding to vascular adhesion protein-1", "intended_use": "Treatment of primary sclerosing cholangitis", "status": "Positive", "designation_date": "2015-03-19", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-15-1461", "last_updated": "2022-07-08" }, { "eu_designation_number": "EU/3/15/1462", "medicine_name": "", "active_substance": "sodium 3-[(4aR,6R,7R,7aS)-7-hydroxy-2-oxido-2-sulfanylidene-4a,6,7,7a-tetrahydro-4H-furo [3,2-d][1,3,2] dioxaphosphinin-6-yl]-2-bromo-6-phenyl-5H-imidazo[1,2-a]purin-9-one", "intended_use": "Treatment of retinitis pigmentosa", "status": "Positive", "designation_date": "2015-03-19", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-15-1462", "last_updated": "2015-05-11" }, { "eu_designation_number": "EU/3/15/1463", "medicine_name": "", "active_substance": "Melphalan flufenamide", "intended_use": "Treatment of plasma cell myeloma", "status": "Withdrawn", "designation_date": "2015-03-19", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-15-1463", "last_updated": "2022-08-22" }, { "eu_designation_number": "EU/3/15/1464", "medicine_name": "", "active_substance": "autologous adipose tissue-derived stromal vascular fraction cells", "intended_use": "Treatment of systemic sclerosis", "status": "Positive", "designation_date": "2015-03-19", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-15-1464", "last_updated": "2015-05-11" }, { "eu_designation_number": "EU/3/15/1465", "medicine_name": "", "active_substance": "ex-vivo-expanded autologous human keratinocytes containing epidermal stem cells transduced with a LAMB3-encoding retroviral vector", "intended_use": "Treatment of epidermolysis bullosa", "status": "Positive", "designation_date": "2015-03-19", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-15-1465", "last_updated": "2015-05-11" }, { "eu_designation_number": "EU/3/15/1466", "medicine_name": "", "active_substance": "ex-vivo-expanded autologous human keratinocytes containing epidermal stem cells transduced with a COL7A1-encoding retroviral vector", "intended_use": "Treatment of epidermolysis bullosa", "status": "Positive", "designation_date": "2015-03-19", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-15-1466", "last_updated": "2015-05-11" }, { "eu_designation_number": "EU/3/15/1467", "medicine_name": "", "active_substance": "ex-vivo-expanded autologous human keratinocytes containing epidermal stem cells transduced with a COL17A1-encoding retroviral vector", "intended_use": "Treatment of epidermolysis bullosa", "status": "Positive", "designation_date": "2015-03-19", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-15-1467", "last_updated": "2015-05-11" }, { "eu_designation_number": "EU/3/15/1468", "medicine_name": "", "active_substance": "adeno-associated viral vector 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"related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-15-1470", "last_updated": "2015-05-11" }, { "eu_designation_number": "EU/3/15/1471", "medicine_name": "", "active_substance": "trientine tetrahydrochloride", "intended_use": "Treatment of Wilson's disease", "status": "Withdrawn", "designation_date": "2015-03-19", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-15-1471", "last_updated": "2017-10-03" }, { "eu_designation_number": "EU/3/15/1472", "medicine_name": "", "active_substance": "fluciclovine (18F)", "intended_use": "Diagnosis of glioma", "status": "Positive", "designation_date": "2015-04-24", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-15-1472", "last_updated": "2015-05-21" }, { "eu_designation_number": "EU/3/15/1473", "medicine_name": "", "active_substance": "lenalidomide", "intended_use": "Treatment of marginal zone lymphoma", "status": "Withdrawn", "designation_date": "2015-04-24", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-15-1473", "last_updated": "2020-01-13" }, { "eu_designation_number": "EU/3/15/1474", "medicine_name": "", "active_substance": "Ecothiopate iodide", "intended_use": "Treatment of Stargardt's disease", "status": "Withdrawn", "designation_date": "2015-04-24", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-15-1474", "last_updated": "2020-12-10" }, { "eu_designation_number": "EU/3/15/1475", "medicine_name": "", "active_substance": "1-(4-(N-glycylamido)phenyl)-3-trifluoromethyl-5-(phenanthren-2-yl)-pyrazole-hydrochloride", "intended_use": "Treatment of cryptococcosis", "status": "Positive", "designation_date": "2015-04-24", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-15-1475", "last_updated": "2015-05-21" }, { "eu_designation_number": "EU/3/15/1476", "medicine_name": "", "active_substance": "1-(4-(N-glycylamido)phenyl)-3-trifluoromethyl-5-(phenanthren-2-yl)-pyrazole-hydrochloride", "intended_use": "Treatment of tularaemia", "status": "Positive", "designation_date": "2015-04-24", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-15-1476", "last_updated": "2015-05-21" }, { "eu_designation_number": "EU/3/15/1477", "medicine_name": "", "active_substance": "Human reovirus type 3 Dearing strain", "intended_use": "Treatment of pancreatic cancer", "status": "Positive", "designation_date": "2015-04-24", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-15-1477", "last_updated": "2015-05-21" }, { "eu_designation_number": "EU/3/15/1478", "medicine_name": "", "active_substance": "rimeporide", "intended_use": "Treatment of Duchenne muscular dystrophy", "status": "Positive", "designation_date": "2025-02-26", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-15-1478", "last_updated": "2025-03-11" }, { "eu_designation_number": "EU/3/15/1479", "medicine_name": "", "active_substance": "recombinant monoclonal IgG1 antibody against T-cell immune response cDNA 7", "intended_use": "Prevention of graft rejection following solid organ transplantation", "status": "Positive", "designation_date": "2015-04-24", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-15-1479", "last_updated": "2015-05-22" }, { "eu_designation_number": "EU/3/15/1480", "medicine_name": "", "active_substance": "rintatolimod", "intended_use": "Treatment of Ebola virus disease", "status": "Positive", "designation_date": "2015-04-24", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-15-1480", "last_updated": "2015-05-22" }, { "eu_designation_number": "EU/3/15/1481", "medicine_name": "", "active_substance": "phenol, 4-[2-(aminomethyl)-4-thiazolyl]-2,6-bis(1,1-dimethylethyl) monohydrochloride", "intended_use": "Treatment of Huntington’s disease", "status": "Withdrawn", "designation_date": "2015-04-24", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-15-1481", "last_updated": "2017-10-19" }, { "eu_designation_number": "EU/3/15/1482", "medicine_name": "", "active_substance": "adeno-associated viral vector serotype 5 containing the human CHM gene", "intended_use": "Treatment of choroideraemia", "status": "Positive", "designation_date": "2015-04-24", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-15-1482", "last_updated": "2017-08-16" }, { "eu_designation_number": "EU/3/15/1483", "medicine_name": "", "active_substance": "xenon", "intended_use": "Treatment of perinatal asphyxia", "status": "Positive", "designation_date": "2015-04-24", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-15-1483", "last_updated": "2015-05-22" }, { "eu_designation_number": "EU/3/15/1484", "medicine_name": "", "active_substance": "nitric oxide", "intended_use": "Treatment of cystic fibrosis", "status": "Withdrawn", "designation_date": "2015-04-24", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-15-1484", "last_updated": "2019-04-12" }, { "eu_designation_number": "EU/3/15/1485", "medicine_name": "", "active_substance": "sodium 2-hydroxylinoleate", "intended_use": "Treatment of neuroblastoma", "status": "Positive", "designation_date": "2015-04-24", "related_ema_product_number": "", "url": 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"https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-15-1538", "last_updated": "2015-10-02" }, { "eu_designation_number": "EU/3/15/1539", "medicine_name": "", "active_substance": "adeno-associated viral vector serotype 8 containing the human MTM1 gene (resamirigene bilparvovec)", "intended_use": "Treatment of X-linked myotubular myopathy", "status": "Positive", "designation_date": "2015-08-10", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-15-1539", "last_updated": "2020-09-18" }, { "eu_designation_number": "EU/3/15/1540", "medicine_name": "", "active_substance": "adeno-associated viral vector serotype 9 containing the human iduronate-2-sulfatase gene", "intended_use": "Treatment of mucopolysaccharidosis type II (Hunter's syndrome)", "status": "Withdrawn", "designation_date": "2015-08-10", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-15-1540", "last_updated": "2025-02-20" }, { "eu_designation_number": "EU/3/15/1541", "medicine_name": "", "active_substance": "Ibrutinib", "intended_use": "Treatment of marginal zone lymphoma", "status": "Withdrawn", "designation_date": "2015-08-10", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-15-1541", "last_updated": "2021-11-23" }, { "eu_designation_number": "EU/3/15/1542", "medicine_name": "", "active_substance": "2'-deoxyguanosylyl-(3',5'-phosphoryl)-2'-deoxythymidylyl-(3',5'-phosphoryl)- 2'-deoxyguanosylyl-(3',5'-phosphoryl)-2'-deoxycytidylyl-(3',5'-phosphoryl)-2'-deoxycytidylyl -(3',5'-phosphoryl)-2'-deoxycytidylyl-(3',5'-phosphoryl)-2'-deoxyguanosylyl...", "intended_use": "Treatment of diffuse large B-cell lymphoma", "status": "Withdrawn", "designation_date": "2015-08-10", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-15-1542", "last_updated": "2016-10-21" }, { "eu_designation_number": "EU/3/15/1543", "medicine_name": "", "active_substance": "(S)-6-hydroxy-2,5,7,8-tetramethyl-N-((R)-piperidin-3-yl)chroman-2-carboxamide hydrochloride", "intended_use": "Treatment of mitochondrial encephalomyopathy, lactic acidosis and stroke-like episodes", "status": "Withdrawn", "designation_date": "2015-08-10", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-15-1543", "last_updated": "2023-03-27" }, { "eu_designation_number": "EU/3/15/1544", "medicine_name": "", "active_substance": "2-(2-phenylvinyl)-4-[4- methylpiperazin-1-yl)]-6-(5-methyl-2H-pyrazol-3-yl-amino)-pyrimidine L(+) tartrate salt", "intended_use": "Treatment of hepatocellular carcinoma", "status": "Positive", "designation_date": "2015-08-10", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-15-1544", "last_updated": "2022-03-18" }, { "eu_designation_number": "EU/3/15/1545", "medicine_name": "", "active_substance": "CD33-directed antibody-drug conjugate consisting of an antibody conjugated to a DNA cross-linking pyrrolobenzodiazepine dimer drug (vadastuximab talirine)", "intended_use": "Treatment of acute myeloid leukaemia", "status": "Withdrawn", "designation_date": "2015-08-10", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-15-1545", "last_updated": "2019-07-09" }, { "eu_designation_number": "EU/3/15/1546", "medicine_name": "", "active_substance": "allogeneic umbilical cord blood cells treated ex vivo with 16,16-dimethyl prostaglandin E2", "intended_use": "Treatment of acute lymphoblastic leukaemia", "status": "Withdrawn", "designation_date": "2015-08-10", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-15-1546", "last_updated": "2017-02-07" }, { "eu_designation_number": "EU/3/15/1547", "medicine_name": "", "active_substance": "Mazindol", "intended_use": "Treatment of narcolepsy", "status": "Positive", "designation_date": "2015-10-09", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-15-1547", "last_updated": "2022-08-03" }, { "eu_designation_number": "EU/3/15/1548", "medicine_name": "", "active_substance": "ovine specific immunoglobulin (Fab) fragments raised against Vipera berus veno", "intended_use": "Treatment of snakebite envenomation", "status": "Positive", "designation_date": "2015-10-09", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-15-1548", "last_updated": "2022-10-21" }, { "eu_designation_number": "EU/3/15/1549", "medicine_name": "", "active_substance": "synthetic peptide L-cysteine, L-cysteinylglycyl-L-glutaminyl-L-arginyl-L-.alpha.-glutamyl-L-threonyl-L-prolyl-L-.alpha.-glutamylglycyl-L-alanyl-L-.alpha.-glutamyl-L-alanyl-L-lysyl-L-prolyl-L-tryptophyl-L-tyrosyl-, cyclic (1.fwdarw.17)-disulfide", "intended_use": "Treatment of primary graft dysfunction following lung transplantation", "status": "Positive", "designation_date": "2015-10-09", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-15-1549", "last_updated": "2015-11-16" }, { "eu_designation_number": "EU/3/15/1550", "medicine_name": "", "active_substance": "3-pentylbenzeneacetic acid sodium salt", "intended_use": "Treatment of idiopathic pulmonary fibrosis", "status": "Withdrawn", "designation_date": "2015-10-09", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-15-1550", "last_updated": "2022-02-28" }, { "eu_designation_number": "EU/3/15/1551", "medicine_name": "Takhzyro", "active_substance": "recombinant human IgG1 kappa light chain monoclonal antibody targeting plasma kallikrein (lanadelumab)", "intended_use": "Treatment of hereditary angioedema", "status": "Positive", "designation_date": "2015-10-09", "related_ema_product_number": "EMEA/H/C/004806", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-15-1551", "last_updated": "2022-10-04" }, { "eu_designation_number": "EU/3/15/1552", "medicine_name": "", "active_substance": "autologous T cells transduced with retroviral vector encoding an anti-CD19 CD28/CD3-zeta chimeric antigen receptor (axicabtagene ciloleucel)", "intended_use": "Treatment of mantle cell lymphoma", "status": "Withdrawn", "designation_date": "2015-10-09", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-15-1552", "last_updated": "2022-01-12" }, { "eu_designation_number": "EU/3/15/1553", "medicine_name": "Yescarta", "active_substance": "autologous T cells transduced with retroviral vector encoding an anti-CD19 CD28/CD3-zeta chimeric antigen receptor (axicabtagene ciloleucel)", "intended_use": "Treatment of primary mediastinal large B-cell lymphoma", "status": "Positive", "designation_date": "2015-10-09", "related_ema_product_number": "EMEA/H/C/004480", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-15-1553", "last_updated": "2021-09-15" }, { "eu_designation_number": "EU/3/15/1554", "medicine_name": "", "active_substance": "nimodipine", "intended_use": "Treatment of aneurysmal subarachnoid haemorrhage", "status": "Positive", "designation_date": "2015-10-09", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-15-1554", "last_updated": "2015-11-16" }, { "eu_designation_number": "EU/3/15/1555", "medicine_name": "", "active_substance": "synthetic hepcidin", "intended_use": "Treatment of beta thalassaemia intermedia and major", "status": "Withdrawn", "designation_date": "2015-10-09", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-15-1555", "last_updated": "2022-05-18" }, { "eu_designation_number": "EU/3/15/1556", "medicine_name": "", "active_substance": "recombinant adeno-associated viral vector expressing the human CNGA3 gene", "intended_use": "Treatment of achromatopsia caused by mutations in the CNGA3 gene", "status": "Positive", "designation_date": "2015-10-09", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-15-1556", "last_updated": "2023-06-27" }, { "eu_designation_number": "EU/3/15/1557", "medicine_name": "", "active_substance": "sirolimus", "intended_use": "Treatment of tuberous sclerosis", "status": "Positive", "designation_date": "2015-10-09", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-15-1557", "last_updated": "2015-11-18" }, { "eu_designation_number": "EU/3/15/1558", "medicine_name": "", "active_substance": "three chimeric human/murine monoclonal antibodies against the Ebola (Zaire) surface glycoprotein (porgaviximab)", "intended_use": "Treatment for Ebola virus disease", "status": "Withdrawn", "designation_date": "2015-10-09", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-15-1558", "last_updated": "2021-03-19" }, { "eu_designation_number": "EU/3/15/1559", "medicine_name": "", "active_substance": "2-(2-chlorophenyl)-4-[3-(dimethylamino)phenyl]-5-methyl-1H-pyrazolo[4,3-C]pyridine-3,6(2H,5H)-dione", "intended_use": "Treatment of systemic sclerosis", "status": "Positive", "designation_date": "2015-10-09", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-15-1559", "last_updated": "2026-08-21" }, { "eu_designation_number": "EU/3/15/1560", "medicine_name": "", "active_substance": "N-(2-((4Z,7Z,10Z,13Z,16Z,19Z)-docosa-4,7,10,13,16,19-hexaenamido)ethyl)-2-hydroxybenzamide (edasalonexent)", "intended_use": "Treatment of Duchenne muscular dystrophy", "status": "Withdrawn", "designation_date": "2015-10-09", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-15-1560", "last_updated": "2021-09-02" }, { "eu_designation_number": "EU/3/15/1561", "medicine_name": "", "active_substance": "ataluren", "intended_use": "Treatment of aniridia", "status": "Withdrawn", "designation_date": "2015-10-09", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-15-1561", "last_updated": "2026-05-18" }, { "eu_designation_number": "EU/3/15/1562", "medicine_name": "", "active_substance": "staphylococcus aureus protein A", "intended_use": "Treatment of immune thrombocytopenia", "status": "Positive", "designation_date": "2015-10-09", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-15-1562", "last_updated": "2018-06-12" }, { "eu_designation_number": "EU/3/15/1563", "medicine_name": "", "active_substance": "recombinant human interleukin-3 truncated diphtheria toxin fusion protein", "intended_use": "Treatment of acute myeloid leukaemia", "status": "Positive", "designation_date": "2015-10-09", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-15-1563", "last_updated": "2022-04-25" }, { "eu_designation_number": "EU/3/15/1564", "medicine_name": "", "active_substance": "Dronabinol;cannabidiol", "intended_use": "Treatment of glioma", "status": "Withdrawn", "designation_date": "2015-10-09", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-15-1564", "last_updated": "2015-11-18" }, { "eu_designation_number": "EU/3/15/1565", "medicine_name": "", "active_substance": "2-Chloro-N6-(3-iodobenzyl)adenosine-5'-N-methyluronamide (namodenoson)", "intended_use": "Treatment of hepatocellular carcinoma", "status": "Positive", "designation_date": "2015-10-09", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-15-1565", "last_updated": "2022-08-10" }, { "eu_designation_number": "EU/3/15/1566", "medicine_name": "", "active_substance": "autologous human peripheral blood Vdelta1+ T lymphocytes activated in vitro by cytokine and monoclonal antibody treatment", "intended_use": "Treatment of chronic lymphocytic leukaemia / small lymphocytic lymphoma", "status": "Positive", "designation_date": "2015-10-09", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-15-1566", "last_updated": "2015-11-16" }, { "eu_designation_number": "EU/3/15/1567", "medicine_name": "Elzonris", "active_substance": "recombinant human interleukin-3 truncated diphtheria toxin fusion protein", "intended_use": "Treatment of blastic plasmacytoid dendritic cell neoplasm", "status": "Positive", "designation_date": "2015-11-11", "related_ema_product_number": "EMEA/H/C/005031", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-15-1567", "last_updated": "2021-01-25" }, { "eu_designation_number": "EU/3/15/1568", "medicine_name": "", "active_substance": "interferon alfa-n3", "intended_use": "Treatment of Middle East respiratory syndrome", "status": "Positive", "designation_date": "2015-11-11", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-15-1568", "last_updated": "2016-01-05" }, { "eu_designation_number": "EU/3/15/1569", "medicine_name": "", "active_substance": "pentetrazol", "intended_use": "Treatment of idiopathic hypersomnia", "status": "Withdrawn", "designation_date": "2015-11-11", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-15-1569", "last_updated": "2020-12-10" }, { "eu_designation_number": "EU/3/15/1570", "medicine_name": "", "active_substance": "azacitidine", "intended_use": "Treatment of nasopharyngeal carcinoma", "status": "Withdrawn", "designation_date": "2015-11-11", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-15-1570", "last_updated": "2017-02-08" }, { "eu_designation_number": "EU/3/15/1571", "medicine_name": "", "active_substance": "autologous T cells transduced with retroviral vector encoding an anti-CD19 CD28/CD3-zeta chimeric antigen receptor (axicabtagene ciloleucel)", "intended_use": "Treatment of acute lymphoblastic leukaemia", "status": "Withdrawn", "designation_date": "2015-11-11", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-15-1571", "last_updated": "2022-01-12" }, { "eu_designation_number": "EU/3/15/1572", "medicine_name": "", "active_substance": "autologous T cells transduced with retroviral vector encoding an anti-CD19 CD28/CD3-zeta chimeric antigen receptor (axicabtagene ciloleucel)", "intended_use": "Treatment of chronic lymphocytic leukaemia / small lymphocytic lymphoma", "status": "Withdrawn", "designation_date": "2015-11-11", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-15-1572", "last_updated": "2022-01-12" }, { "eu_designation_number": "EU/3/15/1573", "medicine_name": "", "active_substance": "adeno-associated viral vector serotype 8 encoding the human ATP7B gene under the control of the human alpha-1 antitrypsin promoter", "intended_use": "Treatment of Wilson's disease", "status": "Positive", "designation_date": "2015-11-11", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-15-1573", "last_updated": "2016-01-06" }, { "eu_designation_number": "EU/3/15/1574", "medicine_name": "", "active_substance": "4'-[(2-butyl-4-oxo-1,3-diazaspiro[4.4]non-1-en-3-yl)methyl]-N-(4,5-dimethyl-3-isoxazolyl)-2'-(ethoxymethyl)-[1,1'-biphenyl]-2-sulfonamide", "intended_use": "Treatment of focal segmental glomerulosclerosis", "status": "Positive", "designation_date": "2015-11-11", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-15-1574", "last_updated": "2022-01-12" }, { "eu_designation_number": "EU/3/15/1575", "medicine_name": "", "active_substance": "humanised fusion protein consisting of extracellular domain of CD24 linked to IgG1 Fc domain", "intended_use": "Prevention of graft-versus-host disease", "status": "Withdrawn", "designation_date": "2015-11-11", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-15-1575", "last_updated": "2026-05-26" }, { "eu_designation_number": "EU/3/15/1576", "medicine_name": "", "active_substance": "xevinapant", "intended_use": "Treatment of ovarian cancer", "status": "Withdrawn", "designation_date": "2015-11-11", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-15-1576", "last_updated": "2025-02-20" }, { "eu_designation_number": "EU/3/15/1577", "medicine_name": "", "active_substance": "adenovirus associated viral vector serotype 5 containing the human RPE65 gene", "intended_use": "Treatment of Leber's congenital amaurosis", "status": "Positive", "designation_date": "2015-11-11", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-15-1577", "last_updated": "2024-10-22" }, { "eu_designation_number": "EU/3/15/1578", "medicine_name": "", "active_substance": "adenovirus associated viral vector serotype 8 containing the human CNGB3 gene", "intended_use": "Treatment of achromatopsia caused by mutations in the CNGB3 gene", "status": "Positive", "designation_date": "2015-11-11", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-15-1578", "last_updated": "2024-10-22" }, { "eu_designation_number": "EU/3/15/1579", "medicine_name": "Yescarta", "active_substance": "autologous T cells transduced with retroviral vector encoding an anti-CD19 CD28/CD3-zeta chimeric antigen receptor (axicabtagene ciloleucel)", "intended_use": "Treatment of follicular lymphoma", "status": "Positive", "designation_date": "2015-11-11", "related_ema_product_number": "EMEA/H/C/004480", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-15-1579", "last_updated": "2022-07-12" }, { "eu_designation_number": "EU/3/15/1580", "medicine_name": "", "active_substance": "N-[5-(3,5-difluorobenzyl)-1H-indazol-3-yl]-4-(4 methylpiperazin-1-yl)-2-(tetrahydro-2H-pyran-4-ylamino) benzamide (entrectinib)", "intended_use": "Treatment of neuroblastoma", "status": "Withdrawn", "designation_date": "2015-11-11", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-15-1580", "last_updated": "2019-02-21" }, { "eu_designation_number": "EU/3/15/1581", "medicine_name": "", "active_substance": "sodium phenylbutyrate", "intended_use": "Treatment of pyruvate dehydrogenase complex deficiency", "status": "Positive", "designation_date": "2015-11-11", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-15-1581", "last_updated": "2022-11-07" }, { "eu_designation_number": "EU/3/15/1582", "medicine_name": "", "active_substance": "Magrolimab", "intended_use": "Treatment of acute myeloid leukaemia", "status": "Withdrawn", "designation_date": "2015-11-11", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-15-1582", "last_updated": "2025-03-12" }, { "eu_designation_number": "EU/3/15/1583", "medicine_name": "", "active_substance": "2-amino-2-[2-[2-chloro-4-[[3-(phenylmethoxy)phenyl]thio]phenyl]ethyl]-1,3-propanediol hydrochloride", "intended_use": "Prevention of graft-versus-host disease", "status": "Withdrawn", "designation_date": "2015-12-14", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-15-1583", "last_updated": "2018-01-22" }, { "eu_designation_number": "EU/3/15/1584", "medicine_name": "", "active_substance": "variant of recombinant human fibroblast growth factor 19", "intended_use": "Treatment of primary sclerosing cholangitis", "status": "Withdrawn", "designation_date": "2015-12-14", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-15-1584", "last_updated": "2022-09-28" }, { "eu_designation_number": "EU/3/15/1585", "medicine_name": "", "active_substance": "sirolimus", "intended_use": "Treatment of beta thalassaemia intermedia and major", "status": "Positive", "designation_date": "2015-12-14", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-15-1585", "last_updated": "2022-06-08" }, { "eu_designation_number": "EU/3/15/1586", "medicine_name": "Oxervate", "active_substance": "recombinant human nerve growth factor (cenegermin)", "intended_use": "Treatment of neurotrophic keratitis", "status": "Positive", "designation_date": "2015-12-14", "related_ema_product_number": "EMEA/H/C/004209", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-15-1586", "last_updated": "2016-02-02" }, { "eu_designation_number": "EU/3/15/1587", "medicine_name": "", "active_substance": "Combretastatin A1 diphosphate", "intended_use": "Treatment of acute myeloid leukaemia", "status": "Positive", "designation_date": "2015-12-14", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-15-1587", "last_updated": "2022-09-28" }, { "eu_designation_number": "EU/3/15/1588", "medicine_name": "", "active_substance": "(R)-1-[1-(4-acetoxy-3,3-dimethyl-2-oxo-butyl)-2-oxo-5-(pyridin-2-yl)-2,3-dihydro-1H-benzo[e][1,4]diazepin-3-yl]-3-(3-methylamino-phenyl)-urea (ceclazepide)", "intended_use": "Treatment of gastro-entero-pancreatic neuroendocrine tumours", "status": "Withdrawn", "designation_date": "2015-12-14", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-15-1588", "last_updated": "2023-04-04" }, { "eu_designation_number": "EU/3/15/1589", "medicine_name": "Amglidia", "active_substance": "glibenclamide", "intended_use": "Treatment of neonatal diabetes", "status": "Positive", "designation_date": "2016-01-15", "related_ema_product_number": "EMEA/H/C/004379", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-15-1589", "last_updated": "2019-01-04" }, { "eu_designation_number": "EU/3/15/1590", "medicine_name": "", "active_substance": "recombinant human monoclonal IgG1 antibody against programmed death ligand-1 (avelumab)", "intended_use": "Treatment of Merkel cell carcinoma", "status": "Withdrawn", "designation_date": "2015-12-14", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-15-1590", "last_updated": "2019-10-15" }, { "eu_designation_number": "EU/3/15/1591", "medicine_name": "", "active_substance": "synthetic peptide L-cysteine, L-cysteinylglycyl-L-glutaminyl-L-arginyl-L-.alpha.-glutamyl-L-threonyl-L-prolyl-L-.alpha.-glutamylglycyl-L-alanyl-L-.alpha.-glutamyl-L-alanyl-L-lysyl-L-prolyl-L-tryptophyl-L-tyrosyl-, cyclic (1.fwdarw.17)-disulfide", "intended_use": "Treatment of pseudohypoaldosteronism 1B", "status": "Positive", "designation_date": "2015-12-14", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-15-1591", "last_updated": "2016-02-03" }, { "eu_designation_number": "EU/3/15/1592", "medicine_name": "", "active_substance": "-[4-Aminobutanoic acid-glycyl-L-glutaminyl-L-arginyl-L-.alpha.-glutamyl-L-threonyl-L-prolyl-L-.alpha.-glutamylglycyl-L-alanyl-L-.alpha.-glutamyl-L-alanyl-L-lysyl-L-prolyl-L-tryptophyl-L-tyrosyl-L-aspartyl](cyclo 1-Dgamma17)", "intended_use": "Treatment of pseudohypoaldosteronism 1B", "status": "Positive", "designation_date": "2015-12-14", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-15-1592", "last_updated": "2016-02-03" }, { "eu_designation_number": "EU/3/15/1593", "medicine_name": "", "active_substance": "imetelstat sodium", "intended_use": "Treatment of myelofibrosis", "status": "Positive", "designation_date": "2015-12-14", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-15-1593", "last_updated": "2023-06-16" }, { "eu_designation_number": "EU/3/15/1594", "medicine_name": "", "active_substance": "Live attenuated Listeria monocytogenes delta actA/delta inlB strain expressing human mesothelin", "intended_use": "Treatment of malignant mesothelioma", "status": "Withdrawn", "designation_date": "2015-12-14", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-15-1594", "last_updated": "2020-01-13" }, { "eu_designation_number": "EU/3/15/1595", "medicine_name": "", "active_substance": "live attenuated Listeria monocytogenes bioengineered with a chimeric human epidermal growth factor receptor 2 fused to a truncated form of the Lm protein listeriolysin O", "intended_use": "Treatment of osteosarcoma", "status": "Positive", "designation_date": "2015-12-14", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-15-1595", "last_updated": "2018-05-03" }, { "eu_designation_number": "EU/3/15/1596", "medicine_name": "", "active_substance": "bilayer engineered collagen hydrogel-based skin graft composed of autologous keratinocytes and fibroblasts", "intended_use": "Treatment of partial deep dermal and full-thickness burns", "status": "Positive", "designation_date": "2015-12-14", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-15-1596", "last_updated": "2022-08-03" }, { "eu_designation_number": "EU/3/15/1597", "medicine_name": "", "active_substance": "sodium (2R,3S,5R)-5-(4-amino-2-oxo-1,3,5-triazin-1(2H)-yl)-2-(hydroxymethyl)tetrahydrofuran-3-yl ((2R,3S,5R)-5-(2-amino-6-oxo-1H-purin-9(6H)-yl)-3-hydroxytetrahydrofuran-2-yl)methyl phosphate (guadecitabine)", "intended_use": "Treatment of acute myeloid leukaemia", "status": "Withdrawn", "designation_date": "2015-12-14", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-15-1597", "last_updated": "2020-12-01" }, { "eu_designation_number": "EU/3/15/1598", "medicine_name": "", "active_substance": "2-(2-Chlorobenzylidene)hydrazinecarboximidamide acetate", "intended_use": "Treatment of Charcot-Marie-Tooth disease", "status": "Positive", "designation_date": "2015-12-14", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-15-1598", "last_updated": "2026-08-21" }, { "eu_designation_number": "EU/3/15/1599", "medicine_name": "", "active_substance": "Adeno-associated viral vector serotype rh10 containing the human factor IX gene for the treatment of haemophilia B-", "intended_use": "Treatment of haemophilia B", "status": "Withdrawn", "designation_date": "2015-12-14", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-15-1599", "last_updated": "2022-10-11" }, { "eu_designation_number": "EU/3/15/1600", "medicine_name": "", "active_substance": "sodium benzoate", "intended_use": "Treatment of hyperargininaemia", "status": "Positive", "designation_date": "2016-01-11", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-15-1600", "last_updated": "2019-08-19" }, { "eu_designation_number": "EU/3/15/1601", "medicine_name": "", "active_substance": "sodium benzoate", "intended_use": "Treatment of argininosuccinic aciduria", "status": "Positive", "designation_date": "2016-01-11", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-15-1601", "last_updated": "2019-08-19" }, { "eu_designation_number": "EU/3/15/1602", "medicine_name": "", "active_substance": "Live attenuated Listeria monocytogenes transfected with plasmids encoding the HPV-16E7 protein fused to a truncated fragment of the Lm protein listeriolysin O", "intended_use": "Treatment of anal cancer", "status": "Withdrawn", "designation_date": "2016-01-11", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-15-1602", "last_updated": "2024-11-19" }, { "eu_designation_number": "EU/3/15/1603", "medicine_name": "", "active_substance": "Live attenuated Listeria monocytogenes delta actA/delta inlB strain expressing human mesothelin", "intended_use": "Treatment of pancreatic cancer", "status": "Withdrawn", "designation_date": "2016-01-11", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-15-1603", "last_updated": "2020-01-13" }, { "eu_designation_number": "EU/3/15/1604", "medicine_name": "", "active_substance": "two allogeneic irradiated pancreatic tumour cell lines", "intended_use": "Treatment of pancreatic cancer", "status": "Withdrawn", "designation_date": "2016-01-11", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-15-1604", "last_updated": "2020-01-13" }, { "eu_designation_number": "EU/3/15/1605", "medicine_name": "", "active_substance": "synthetic double-stranded oligomer specific to the SERPINA1 gene and containing a cholesterol-conjugated, acyclic nucleobase analogue", "intended_use": "Treatment of congenital alpha-1 antitrypsin deficiency", "status": "Withdrawn", "designation_date": "2016-01-11", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-15-1605", "last_updated": "2017-05-17" }, { "eu_designation_number": "EU/3/15/1606", "medicine_name": "", "active_substance": "(S)-N-(5-((R)-2-(2,5-difluorophenyl)pyrrolidin-1-yl)pyrazolo[1,5-a]pyrimidin-3-yl)-3-hydroxypyrrolidine-1-carboxamide hydrogen sulfate (larotrectinib)", "intended_use": "Treatment of soft tissue sarcoma", "status": "Withdrawn", "designation_date": "2016-01-11", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-15-1606", "last_updated": "2019-10-24" }, { "eu_designation_number": "EU/3/15/1607", "medicine_name": "", "active_substance": "Entolimod", "intended_use": "Treatment of acute radiation syndrome", "status": "Withdrawn", "designation_date": "2016-01-11", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-15-1607", "last_updated": "2020-05-07" }, { "eu_designation_number": "EU/3/16/1608", "medicine_name": "", "active_substance": "S3,S13-cyclo(D-tyrolsyl-L-isoleucyl-L-cysteinyl-L-valyl-1-methyl-L-tryptophyl-L-glutaminyl-L-aspartyl-L-tryptophyl-N-methyl-L-glycyl-L-alanyl-L-histidyl-L-arginyl-L-cysteinyl-N-methyl-L-isoleucinamide)", "intended_use": "Treatment of C3 glomerulopathy", "status": "Positive", "designation_date": "2016-02-17", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-16-1608", "last_updated": "2016-04-06" }, { "eu_designation_number": "EU/3/16/1609", "medicine_name": "Enjaymo", "active_substance": "sutimlimab", "intended_use": "Treatment of autoimmune haemolytic anaemia", "status": "Positive", "designation_date": "2025-02-11", "related_ema_product_number": "EMEA/H/C/005776", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-16-1609", "last_updated": "2025-03-12" }, { "eu_designation_number": "EU/3/16/1610", "medicine_name": "", "active_substance": "arsenic trioxide", "intended_use": "Treatment of acute myeloid leukaemia", "status": "Positive", "designation_date": "2016-02-17", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-16-1610", "last_updated": "2016-04-06" }, { "eu_designation_number": "EU/3/16/1611", "medicine_name": "", "active_substance": "ex-vivo-expanded autologous fibroblasts transduced with lentiviral vector containing the COL7A1 gene", "intended_use": "Treatment of epidermolysis bullosa", "status": "Positive", "designation_date": "2016-02-17", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-16-1611", "last_updated": "2016-04-06" }, { "eu_designation_number": "EU/3/16/1612", "medicine_name": "", "active_substance": "Methyl 3-((2R)-2-hydroxy-4-(((((S)-1-methoxy-1-oxopropan-2-yl) amino)(phenoxy)phosphoryl)oxy)-3,3-dimethylbutanamido)propanoate", "intended_use": "Treatment of pantothenate-kinase-associated neurodegeneration", "status": "Withdrawn", "designation_date": "2016-02-17", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-16-1612", "last_updated": "2020-08-13" }, { "eu_designation_number": "EU/3/16/1613", "medicine_name": "", "active_substance": "tolfenamic acid", "intended_use": "Treatment of progressive supranuclear palsy", "status": "Positive", "designation_date": "2016-02-17", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-16-1613", "last_updated": "2016-04-07" }, { "eu_designation_number": "EU/3/16/1614", "medicine_name": "", "active_substance": "tolfenamic acid", "intended_use": "treatment of behavioural variant frontotemporal dementia", "status": "Positive", "designation_date": "2016-02-17", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-16-1614", "last_updated": "2016-04-07" }, { "eu_designation_number": "EU/3/16/1615", "medicine_name": "", "active_substance": "2-Ethylbutyl (2S)-2-{[(S)-{[(2R,3S,4R,5R)-5-(4-aminopyrrolo[2,1-f][1,2,4]triazin-7-yl)-5-cyano-3,4-dihydroxytetrahydrofuran-2-yl]methoxy}(phenoxy)phosphoryl]amino}propanoate (remdesivir)", "intended_use": "Treatment of Ebola virus disease", "status": "Positive", "designation_date": "2016-02-17", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-16-1615", "last_updated": "2018-06-13" }, { "eu_designation_number": "EU/3/16/1616", "medicine_name": "", "active_substance": "diclofenamide", "intended_use": "Treatment of periodic paralysis", "status": "Positive", "designation_date": "2016-02-17", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-16-1616", "last_updated": "2021-04-26" }, { "eu_designation_number": "EU/3/16/1617", "medicine_name": "", "active_substance": "venetoclax", "intended_use": "Treatment of acute myeloid leukaemia", "status": "Withdrawn", "designation_date": "2016-02-17", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-16-1617", "last_updated": "2019-04-12" }, { "eu_designation_number": "EU/3/16/1618", "medicine_name": "", "active_substance": "N-(4-Methoxyphenyl)-N,2,6-trimethylfuro[2,3-d]pyrimidin-4-amine", "intended_use": "Treatment of glioma", "status": "Positive", "designation_date": "2016-02-16", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-16-1618", "last_updated": "2016-04-07" }, { "eu_designation_number": "EU/3/16/1619", "medicine_name": "", "active_substance": "DNA plasmid encoding a recombinant fusion protein consisting of the extracellular domain of human TNFα p55 receptor linked to the human IgG1 Fc domain", "intended_use": "Treatment of non-infectious uveitis", "status": "Positive", "designation_date": "2016-02-17", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-16-1619", "last_updated": "2020-09-29" }, { "eu_designation_number": "EU/3/16/1620", "medicine_name": "", "active_substance": "allogeneic fetal human retinal progenitor cells expanded ex vivo", "intended_use": "Treatment of retinitis pigmentosa", "status": "Positive", "designation_date": "2016-02-17", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-16-1620", "last_updated": "2021-10-13" }, { "eu_designation_number": "EU/3/16/1621", "medicine_name": "", "active_substance": "Delta-9-tetrahydrocannabinol and cannabidiol from extracts of the Cannabis sativa L. plant", "intended_use": "Treatment of glioma", "status": "Positive", "designation_date": "2016-02-17", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-16-1621", "last_updated": "2023-06-15" }, { "eu_designation_number": "EU/3/16/1622", "medicine_name": "Roctavian; Roctavian", "active_substance": "adeno-associated viral vector serotype 5 containing a B-domain deleted variant of human coagulation factor VIII gene", "intended_use": "Treatment of haemophilia A", "status": "Positive", "designation_date": "2016-03-21", "related_ema_product_number": "EMEA/H/C/004749; EMEA/H/C/005830", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-16-1622", "last_updated": "2022-09-28" }, { "eu_designation_number": "EU/3/16/1623", "medicine_name": "", "active_substance": "adeno-associated viral vector serotype 8 encoding human ornithine transcarbamylase (avalotcagene ontaparvovec)", "intended_use": "Treatment of ornithine transcarbamylase deficiency", "status": "Positive", "designation_date": "2016-03-21", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-16-1623", "last_updated": "2020-11-11" }, { "eu_designation_number": "EU/3/16/1624", "medicine_name": "", "active_substance": "acalabrutinib", "intended_use": "Treatment of chronic lymphocytic leukaemia / small lymphocytic lymphoma", "status": "Withdrawn", "designation_date": "2016-03-21", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-16-1624", "last_updated": "2020-11-11" }, { "eu_designation_number": "EU/3/16/1625", "medicine_name": "", "active_substance": "acalabrutinib", "intended_use": "Treatment of mantle cell lymphoma", "status": "Withdrawn", "designation_date": "2016-03-21", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-16-1625", "last_updated": "2026-05-26" }, { "eu_designation_number": "EU/3/16/1626", "medicine_name": "", "active_substance": "acalabrutinib", "intended_use": "Treatment of lymphoplasmacytic lymphoma", "status": "Positive", "designation_date": "2016-03-21", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-16-1626", "last_updated": "2016-05-04" }, { "eu_designation_number": "EU/3/16/1627", "medicine_name": "Ebvallo", "active_substance": "allogeneic Epstein-Barr virus specific cytotoxic T lymphocytes (Tabelecleucel)", "intended_use": "Treatment of post-transplantation lymphoproliferative disorders", "status": "Positive", "designation_date": "2016-03-21", "related_ema_product_number": "EMEA/H/C/004577", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-16-1627", "last_updated": "2023-01-30" }, { "eu_designation_number": "EU/3/16/1628", "medicine_name": "", "active_substance": "Diaspirin cross-linked haemoglobin", "intended_use": "Treatment of oesophageal cancer", "status": "Positive", "designation_date": "2016-03-21", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-16-1628", "last_updated": "2019-09-30" }, { "eu_designation_number": "EU/3/16/1629", "medicine_name": "", "active_substance": "exenatide", "intended_use": "Treatment of idiopathic intracranial hypertension", "status": "Positive", "designation_date": "2016-03-21", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-16-1629", "last_updated": "2021-09-16" }, { "eu_designation_number": "EU/3/16/1630", "medicine_name": "", "active_substance": "Fenretinide", "intended_use": "Treatment of cutaneous T-cell lymphoma", "status": "Withdrawn", "designation_date": "2016-03-21", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-16-1630", "last_updated": "2022-01-19" }, { "eu_designation_number": "EU/3/16/1631", "medicine_name": "", "active_substance": "Florilglutamic acid (18F)", "intended_use": "Diagnosis of glioma", "status": "Withdrawn", "designation_date": "2016-03-21", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-16-1631", "last_updated": "2024-04-09" }, { "eu_designation_number": "EU/3/16/1632", "medicine_name": "", "active_substance": "Florilglutamic acid (18F)", "intended_use": "Diagnosis of 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"https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-16-1634", "last_updated": "2019-10-15" }, { "eu_designation_number": "EU/3/16/1635", "medicine_name": "", "active_substance": "N-acetyl-D-mannosamine monohydrate", "intended_use": "Treatment of GNE myopathy", "status": "Positive", "designation_date": "2016-03-21", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-16-1635", "last_updated": "2016-05-04" }, { "eu_designation_number": "EU/3/16/1636", "medicine_name": "", "active_substance": "sindbis virus envelope pseudotyped lentiviral vector encoding New York oesophageal squamous cell carcinoma-1 protein", "intended_use": "Treatment of soft tissue sarcoma", "status": "Withdrawn", "designation_date": "2016-03-21", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-16-1636", "last_updated": "2019-10-15" }, { "eu_designation_number": "EU/3/16/1637", "medicine_name": "Oxlumo", "active_substance": "synthetic double-stranded siRNA oligonucleotide directed against hydroxyacid oxidase 1 mRNA and covalently linked to a ligand containing three N-acetylgalactosamine residues (lumasiran)", "intended_use": "Treatment of primary hyperoxaluria", "status": "Positive", "designation_date": "2016-03-21", "related_ema_product_number": "EMEA/H/C/005040", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-16-1637", "last_updated": "2020-11-25" }, { "eu_designation_number": "EU/3/16/1638", "medicine_name": "", "active_substance": "ubenimex", "intended_use": "Treatment of pulmonary arterial hypertension", "status": "Positive", "designation_date": "2016-03-21", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-16-1638", "last_updated": "2019-08-19" }, { "eu_designation_number": "EU/3/16/1639", "medicine_name": "", "active_substance": "(1E,6E)-1,7-bis(3,4-dimethoxyphenyl)-4-cyclobutylmethyl-1,6-heptadiene-3,5-dione", "intended_use": "Treatment of X-linked spinal and bulbar muscular atrophy (Kennedy's disease)", "status": "Positive", "designation_date": "2016-04-28", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-16-1639", "last_updated": "2018-05-03" }, { "eu_designation_number": "EU/3/16/1640", "medicine_name": "", "active_substance": "2-methyl-1-[(4-[6-(trifluoromethyl)pyridin-2-yl]-6-{[2-(trifluoromethyl)pyridin-4-yl]amino}-1,3,5-triazin-2-yl)amino]propan-2-ol methanesulfonate (enasidenib)", "intended_use": "Treatment of acute myeloid leukaemia", "status": "Positive", "designation_date": "2016-04-28", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-16-1640", "last_updated": "2022-06-29" }, { "eu_designation_number": "EU/3/16/1641", "medicine_name": "", "active_substance": "antisense oligonucleotide complementary to the exonic splicer enhancer sequence at intron 26 of the centrosomal protein 290 pre-mRNA", "intended_use": "Treatment of Leber's congenital amaurosis", "status": "Positive", "designation_date": "2016-04-28", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-16-1641", "last_updated": "2024-10-02" }, { "eu_designation_number": "EU/3/16/1642", "medicine_name": "", "active_substance": "autologous dermal fibroblasts genetically modified ex vivo with a lentiviral vector containing the human COL7A1 gene", "intended_use": "Treatment of epidermolysis bullosa", "status": "Positive", "designation_date": "2016-04-28", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-16-1642", "last_updated": "2016-05-30" }, { "eu_designation_number": "EU/3/16/1643", "medicine_name": "", "active_substance": "autologous stromal vascular cell fraction from adipose tissue", "intended_use": "Treatment of systemic sclerosis", "status": "Withdrawn", "designation_date": "2016-04-28", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-16-1643", "last_updated": "2020-08-27" }, { "eu_designation_number": "EU/3/16/1644", "medicine_name": "", "active_substance": "brincidofovir", "intended_use": "Prevention of cytomegalovirus disease", "status": "Positive", "designation_date": "2016-04-28", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-16-1644", "last_updated": "2024-10-11" }, { "eu_designation_number": "EU/3/16/1645", "medicine_name": "", "active_substance": "cannabidiol", "intended_use": "Prevention of graft-versus-host disease", "status": "Positive", "designation_date": "2016-04-28", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-16-1645", "last_updated": "2016-05-30" }, { "eu_designation_number": "EU/3/16/1646", "medicine_name": "", "active_substance": "4-hydroxyandrostenedione;serenoa serrulata;alpha lipoic acid", "intended_use": "Treatment of multiple symmetric lipomatosis", "status": "Positive", "designation_date": "2016-04-28", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-16-1646", "last_updated": "2021-09-02" }, { "eu_designation_number": "EU/3/16/1647", "medicine_name": "", "active_substance": "Fluocinolone acetonide", "intended_use": "Treatment of non-infectious uveitis", "status": "Withdrawn", "designation_date": "2016-04-28", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-16-1647", "last_updated": "2017-08-16" }, { "eu_designation_number": "EU/3/16/1648", "medicine_name": "", "active_substance": "Human/murine chimeric monoclonal antibody against endoglin (carotuximab)", "intended_use": "Treatment of soft tissue sarcoma", "status": "Withdrawn", "designation_date": "2016-04-28", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-16-1648", "last_updated": "2019-06-17" }, { "eu_designation_number": "EU/3/16/1649", "medicine_name": "", "active_substance": "Humanised recombinant IgG4 anti-human tau antibody (tilavonemab)", "intended_use": "Treatment of progressive supranuclear palsy", "status": "Withdrawn", "designation_date": "2016-04-28", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-16-1649", "last_updated": "2020-04-01" }, { "eu_designation_number": "EU/3/16/1650", "medicine_name": "", "active_substance": "N-carboxymethyl-glycyl-L-threonyl-L-histidyl-L-3,3-diphenylalanyl-L-piperidincarboxy-3-yl-L-arginyl-L-S-methylthio-cystyl-L-arginyl-L-tryptophyl-aminohexanyl-N-carboxamidomethyl-glycine N-hexadecylamide", "intended_use": "Treatment of beta thalassaemia intermedia and major", "status": "Withdrawn", "designation_date": "2016-04-28", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-16-1650", "last_updated": "2017-08-10" }, { "eu_designation_number": "EU/3/16/1651", "medicine_name": "", "active_substance": "recombinant adeno-associated viral vector serotype 9 carrying the gene for the human E6-AP ubiquitin protein ligase", "intended_use": "Treatment of Angelman syndrome", "status": "Positive", "designation_date": "2016-04-28", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-16-1651", "last_updated": "2018-12-18" }, { "eu_designation_number": "EU/3/16/1652", "medicine_name": "", "active_substance": "recombinant human cerebral dopamine neurotrophic factor", "intended_use": "Treatment of amyotrophic lateral sclerosis", "status": "Positive", "designation_date": "2016-04-28", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-16-1652", "last_updated": "2016-05-30" }, { "eu_designation_number": "EU/3/16/1653", "medicine_name": "", "active_substance": "resiquimod", "intended_use": "Treatment of cutaneous T-cell lymphoma", "status": "Positive", "designation_date": "2016-04-28", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-16-1653", "last_updated": "2022-01-13" }, { "eu_designation_number": "EU/3/16/1654", "medicine_name": "", "active_substance": "S-acetyl-(S)-4'-phosphopantetheine, calcium salt", "intended_use": "Treatment of pantothenate-kinase-associated neurodegeneration", "status": "Positive", "designation_date": "2016-04-28", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-16-1654", "last_updated": "2019-12-02" }, { "eu_designation_number": "EU/3/16/1655", "medicine_name": "", "active_substance": "Tyr-Met-Phe-Pro-Asn-Ala-Pro-Tyr-Leu, Ser-Gly-Gln-Ala-Tyr-Met-Phe-Pro-Asn-Ala-Pro-Tyr-Leu-Pro-Ser-Cys-Leu-Glu-Ser, Arg-Ser-Asp-Glu-Leu-Val-Arg-His-His-Asn-Met-His-Gln-Arg-Asn-Met-Thr-Lys-Leu and Pro-Gly-Cys-Asn-Lys-Arg-Tyr-Phe-Lys-Leu-Ser-His-Leu-Gln-Met-His-Ser-Arg-Lys-His-Thr-Gly", "intended_use": "Treatment of acute myeloid leukaemia", "status": "Positive", "designation_date": "2016-04-28", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-16-1655", "last_updated": "2019-04-12" }, { "eu_designation_number": "EU/3/16/1656", "medicine_name": "", "active_substance": "Tyr-Met-Phe-Pro-Asn-Ala-Pro-Tyr-Leu, Ser-Gly-Gln-Ala-Tyr-Met-Phe-Pro-Asn-Ala-Pro-Tyr-Leu-Pro-Ser-Cys-Leu-Glu-Ser, Arg-Ser-Asp-Glu-Leu-Val-Arg-His-His-Asn-Met-His-Gln-Arg-Asn-Met-Thr-Lys-Leu and Pro-Gly-Cys-Asn-Lys-Arg-Tyr-Phe-Lys-Leu-Ser-His-Leu-Gln-Met-His-Ser-Arg-Lys-His-Thr-Gly", "intended_use": "Treatment of malignant mesothelioma", "status": "Positive", "designation_date": "2016-04-28", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-16-1656", "last_updated": "2019-04-12" }, { "eu_designation_number": "EU/3/16/1657", "medicine_name": "", "active_substance": "(R)-6-(2-fluorophenyl)-N-(3-(2-((2-methoxyethyl)amino)ethyl)phenyl)-5,6-dihydrobenzo[h]quinazolin-2-amine dihydrochloride (derazantinib)", "intended_use": "Treatment of biliary tract cancer", "status": "Positive", "designation_date": "2016-05-30", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-16-1657", "last_updated": "2019-05-13" }, { "eu_designation_number": "EU/3/16/1658", "medicine_name": "", "active_substance": "4-[(2E)-1-oxo-3-(2,6,6-trimethyl-1-cyclohexen-1-yl)-2-propen-1-yl]-1-piperazinecarboxamide", "intended_use": "Treatment of retinitis pigmentosa", "status": "Withdrawn", "designation_date": "2016-05-30", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-16-1658", "last_updated": "2017-08-16" }, { "eu_designation_number": "EU/3/16/1659", "medicine_name": "", "active_substance": "arimoclomol citrate", "intended_use": "Treatment of inclusion body myositis", "status": "Withdrawn", "designation_date": "2022-07-18", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-16-1659", "last_updated": "2022-07-19" }, { "eu_designation_number": "EU/3/16/1660", "medicine_name": "", "active_substance": "autologous CD34+ cells transduced with lentiviral vector encoding the human beta globin gene", "intended_use": "Treatment of beta thalassaemia intermedia and major", "status": "Positive", "designation_date": "2016-05-30", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-16-1660", "last_updated": "2022-09-26" }, { "eu_designation_number": "EU/3/16/1661", "medicine_name": "", "active_substance": "Fc- and CDR-modified humanised monoclonal antibody against C5 (ravulizumab)", "intended_use": "Treatment of paroxysmal nocturnal haemoglobinuria", "status": "Withdrawn", "designation_date": "2016-05-30", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-16-1661", "last_updated": "2019-07-11" }, { "eu_designation_number": "EU/3/16/1662", "medicine_name": "", "active_substance": "H-Phe-Ser-Arg-Tyr-Ala-Arg-OH acetate (Alirinetide)", "intended_use": "Treatment of amyotrophic lateral sclerosis", "status": "Positive", "designation_date": "2016-05-30", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-16-1662", "last_updated": "2018-12-18" }, { "eu_designation_number": "EU/3/16/1663", "medicine_name": "", "active_substance": "pentosan polysulfate sodium", "intended_use": "Treatment of interstitial cystitis", "status": "Positive", "designation_date": "2016-05-30", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-16-1663", "last_updated": "2022-05-16" }, { "eu_designation_number": "EU/3/16/1664", "medicine_name": "", "active_substance": "polyethylene glycol-modified human recombinant truncated cystathionine beta-synthase (pegtibatinase)", "intended_use": "Treatment of homocystinuria", "status": "Positive", "designation_date": "2016-05-30", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-16-1664", "last_updated": "2023-02-06" }, { "eu_designation_number": "EU/3/16/1665", "medicine_name": "", "active_substance": "recombinant adeno-associated viral vector containing the human RPGR gene", "intended_use": "Treatment of retinitis pigmentosa caused by mutations in the RPGR gene", "status": "Positive", "designation_date": "2016-05-30", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-16-1665", "last_updated": "2023-03-27" }, { "eu_designation_number": "EU/3/16/1666", "medicine_name": "", "active_substance": "rimiducid", "intended_use": "Treatment of graft-versus-host disease", "status": "Withdrawn", "designation_date": "2016-05-30", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-16-1666", "last_updated": "2022-04-07" }, { "eu_designation_number": "EU/3/16/1667", "medicine_name": "", "active_substance": "rovalpituzumab tesirine", "intended_use": "Treatment of small cell lung cancer", "status": "Withdrawn", "designation_date": "2016-05-30", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-16-1667", "last_updated": "2021-02-03" }, { "eu_designation_number": "EU/3/16/1668", "medicine_name": "", "active_substance": "sodium nitrite;ethylenediaminetetraacetic acid", "intended_use": "Treatment of cystic fibrosis", "status": "Positive", "designation_date": "2016-05-30", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-16-1668", "last_updated": "2016-06-22" }, { "eu_designation_number": "EU/3/16/1669", "medicine_name": "", "active_substance": "temsirolimus", "intended_use": "Treatment of adrenoleukodystrophy", "status": "Positive", "designation_date": "2016-05-30", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-16-1669", "last_updated": "2022-11-11" }, { "eu_designation_number": "EU/3/16/1670", "medicine_name": "", "active_substance": "vemurafenib", "intended_use": "Treatment of Langerhans' cell histiocytosis", "status": "Positive", "designation_date": "2016-05-30", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-16-1670", "last_updated": "2016-06-24" }, { 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"EU/3/16/1728", "medicine_name": "", "active_substance": "recombinant humanised monoclonal antibody against human complement component C5a", "intended_use": "Treatment of graft-versus-host disease", "status": "Withdrawn", "designation_date": "2016-08-29", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-16-1728", "last_updated": "2017-10-19" }, { "eu_designation_number": "EU/3/16/1729", "medicine_name": "Prohippur", "active_substance": "sodium benzoate", "intended_use": "Treatment of lysinuric protein intolerance", "status": "Positive", "designation_date": "2016-08-29", "related_ema_product_number": "EMEA/H/C/004150", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-16-1729", "last_updated": "2016-10-03" }, { "eu_designation_number": "EU/3/16/1730", "medicine_name": "Prohippur", "active_substance": "sodium benzoate", "intended_use": "Treatment of ornithine translocase deficiency", "status": 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myeloid leukaemia", "status": "Positive", "designation_date": "2016-10-14", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-16-1748", "last_updated": "2016-11-15" }, { "eu_designation_number": "EU/3/16/1749", "medicine_name": "", "active_substance": "crenolanib besilate", "intended_use": "Treatment of soft tissue sarcoma", "status": "Positive", "designation_date": "2016-09-20", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-16-1749", "last_updated": "2016-11-15" }, { "eu_designation_number": "EU/3/16/1750", "medicine_name": "", "active_substance": "Exendin (9-39)", "intended_use": "Treatment of noninsulinoma pancreatogenous hypoglycaemia syndrome", "status": "Positive", "designation_date": "2016-10-14", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-16-1750", "last_updated": "2019-08-19" }, { "eu_designation_number": "EU/3/16/1751", "medicine_name": "", "active_substance": "Fenretinide", "intended_use": "Treatment of peripheral T-cell lymphoma", "status": "Withdrawn", "designation_date": "2016-10-14", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-16-1751", "last_updated": "2022-01-19" }, { "eu_designation_number": "EU/3/16/1752", "medicine_name": "", "active_substance": "human monoclonal IgG1 antibody against tissue factor pathway inhibitor", "intended_use": "Treatment of haemophilia A", "status": "Withdrawn", "designation_date": "2016-10-14", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-16-1752", "last_updated": "2024-12-03" }, { "eu_designation_number": "EU/3/16/1753", "medicine_name": "", "active_substance": "Haematopoietic stem cells modified with a lentiviral vector containing the CD18 gene", "intended_use": "Treatment of leukocyte 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"EU/3/16/1795", "medicine_name": "", "active_substance": "adeno-associated viral vector serotype 8 containing the human CNGA3 gene under the control of a cone arrestin promoter", "intended_use": "Treatment of achromatopsia caused by mutations in the CNGA3 gene", "status": "Positive", "designation_date": "2016-12-12", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-16-1795", "last_updated": "2016-01-09" }, { "eu_designation_number": "EU/3/16/1796", "medicine_name": "", "active_substance": "adeno-associated viral vector serotype 8 encoding engineered rhodopsin DNA-binding repressor and human rhodopsin expression cassettes", "intended_use": "Treatment of retinitis pigmentosa", "status": "Positive", "designation_date": "2016-12-12", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-16-1796", "last_updated": "2022-11-07" }, { "eu_designation_number": "EU/3/16/1797", 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"https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-17-1867", "last_updated": "2017-05-15" }, { "eu_designation_number": "EU/3/17/1868", "medicine_name": "", "active_substance": "N-[(1R)-1-phenylethyl]-6-{1H-pyrazolo[3,4-d]pyrimidin-4-yl}quinazolin-2-amine", "intended_use": "Treatment of fragile X syndrome", "status": "Withdrawn", "designation_date": "2017-04-20", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-17-1868", "last_updated": "2024-11-19" }, { "eu_designation_number": "EU/3/17/1869", "medicine_name": "", "active_substance": "rituximab", "intended_use": "Treatment in solid organ transplantation", "status": "Positive", "designation_date": "2017-04-20", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-17-1869", "last_updated": "2017-05-15" }, { "eu_designation_number": "EU/3/17/1870", "medicine_name": "Kygevvi", "active_substance": "thymidine;deoxycytidine", "intended_use": "Treatment of mitochondrial DNA depletion syndrome, myopathic form", "status": "Positive", "designation_date": "2017-04-20", "related_ema_product_number": "EMEA/H/C/005119", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-17-1870", "last_updated": "2023-04-25" }, { "eu_designation_number": "EU/3/17/1871", "medicine_name": "", "active_substance": "225Ac-lintuzumab", "intended_use": "Treatment of acute myeloid leukaemia", "status": "Positive", "designation_date": "2017-05-22", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-17-1871", "last_updated": "2021-10-13" }, { "eu_designation_number": "EU/3/17/1872", "medicine_name": "", "active_substance": "chimeric locked nucleic acid deoxynucleoside phosphorothioate-linked oligonucleotide inhibitor directed against microRNA-155-5p", "intended_use": "Treatment of cutaneous T-cell lymphoma", "status": 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"last_updated": "2017-06-29" }, { "eu_designation_number": "EU/3/17/1876", "medicine_name": "", "active_substance": "sodium (1R,3R,4R,5S)-3-({2-N-acetylamino-2-deoxy-3-O-[(1S)-1-carboxylato-2-cyclohexylethyl]-beta-D-galactopyranosyl}oxy)-4-({6-deoxy-alpha-L-galactopyranosyl}oxy)-5-ethyl-cyclohexan-1-yl-(38-oxo-2,5,8,11,14,17,20,23,26,29,32,35-dodecaoxa-39-azahentetracontan-41-yl)carboxamide", "intended_use": "Treatment of acute myeloid leukaemia", "status": "Positive", "designation_date": "2017-05-22", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-17-1876", "last_updated": "2018-04-03" }, { "eu_designation_number": "EU/3/17/1877", "medicine_name": "", "active_substance": "tamoxifen citrate", "intended_use": "Treatment of cystic fibrosis", "status": "Positive", "designation_date": "2017-05-22", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-17-1877", "last_updated": "2017-06-29" }, { "eu_designation_number": "EU/3/17/1878", "medicine_name": "", "active_substance": "ursodeoxycholic acid", "intended_use": "Treatment of Niemann-Pick disease", "status": "Positive", "designation_date": "2017-05-22", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-17-1878", "last_updated": "2019-04-25" }, { "eu_designation_number": "EU/3/17/1879", "medicine_name": "", "active_substance": "Asp-Arg-Val-Tyr-Ile-His-Pro (Angiotensin 1-7);talfirastide", "intended_use": "Treatment of epidermolysis bullosa", "status": "Positive", "designation_date": "2017-06-20", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-17-1879", "last_updated": "2024-10-22" }, { "eu_designation_number": "EU/3/17/1880", "medicine_name": "", "active_substance": "avacopan", "intended_use": "Treatment of C3 glomerulopathy", "status": "Positive", "designation_date": 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"active_substance": "pentamer formyl thiophene acetic acid", "intended_use": "Treatment of Creutzfeldt-Jakob disease", "status": "Positive", "designation_date": "2017-06-20", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-17-1883", "last_updated": "2017-07-17" }, { "eu_designation_number": "EU/3/17/1884", "medicine_name": "", "active_substance": "recombinant human factor IX protein modified with three point mutations (dalcinonacog alfa)", "intended_use": "Treatment of haemophilia B", "status": "Withdrawn", "designation_date": "2017-06-20", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-17-1884", "last_updated": "2026-05-26" }, { "eu_designation_number": "EU/3/17/1885", "medicine_name": "", "active_substance": "sildenafil", "intended_use": "Treatment of congenital diaphragmatic hernia", "status": "Positive", "designation_date": "2017-06-20", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-17-1885", "last_updated": "2017-07-17" }, { "eu_designation_number": "EU/3/17/1886", "medicine_name": "", "active_substance": "sirolimus", "intended_use": "Treatment of tuberous sclerosis", "status": "Positive", "designation_date": "2017-06-20", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-17-1886", "last_updated": "2022-07-19" }, { "eu_designation_number": "EU/3/17/1887", "medicine_name": "", "active_substance": "synthetic glucagon analogue modified to contain 7 amino acid substitutions", "intended_use": "Treatment of congenital hyperinsulinism", "status": "Positive", "designation_date": "2017-06-20", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-17-1887", "last_updated": "2020-09-25" }, { "eu_designation_number": "EU/3/17/1888", "medicine_name": "", "active_substance": "tripotassium citrate monohydrate;potassium hydrogen carbonate", "intended_use": "Treatment of distal renal tubular acidosis", "status": "Withdrawn", "designation_date": "2017-06-20", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-17-1888", "last_updated": "2021-05-03" }, { "eu_designation_number": "EU/3/17/1889", "medicine_name": "Ayvakyt", "active_substance": "(S)-1-(4-fluorophenyl)-1-(2-(4-(6-(1-methyl-1H-pyrazol-4-yl)pyrrolo[2,1-f][1,2,4]triazin-4-yl)piperazin-yl)pyrimidin-5-yl)ethan-1-amine", "intended_use": "Treatment of gastrointestinal stromal tumours", "status": "Positive", "designation_date": "2017-07-17", "related_ema_product_number": "EMEA/H/C/005208", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-17-1889", "last_updated": "2019-05-22" }, { "eu_designation_number": "EU/3/17/1890", "medicine_name": "", "active_substance": "autologous CD4+ and CD8+ T cells expressing a CD19-specific chimeric antigen receptor (lisocabtagene maraleucel)", "intended_use": "Treatment of diffuse large B-cell lymphoma", "status": "Withdrawn", "designation_date": "2017-07-17", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-17-1890", "last_updated": "2022-05-04" }, { "eu_designation_number": "EU/3/17/1891", "medicine_name": "", "active_substance": "Bacillus subtilis oxalate decarboxylase", "intended_use": "Treatment of primary hyperoxaluria", "status": "Positive", "designation_date": "2017-07-17", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-17-1891", "last_updated": "2017-08-15" }, { "eu_designation_number": "EU/3/17/1892", "medicine_name": "", "active_substance": "Oxymetazoline hydrochloride", "intended_use": "Treatment of spinal cord injury", "status": "Positive", "designation_date": "2017-07-17", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-17-1892", "last_updated": "2017-08-15" }, { "eu_designation_number": "EU/3/17/1893", "medicine_name": "", "active_substance": "Polyphenyl(disodium 3-O-sulfo-beta-D-glucopyranuronate)-(1->3)-beta-D-galactopyranoside", "intended_use": "Treatment of anti-MAG neuropathy", "status": "Withdrawn", "designation_date": "2017-07-17", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-17-1893", "last_updated": "2021-11-15" }, { "eu_designation_number": "EU/3/17/1894", "medicine_name": "", "active_substance": "recombinant human antibody directed against misfolded human superoxide dismutase 1", "intended_use": "Treatment of amyotrophic lateral sclerosis", "status": "Positive", "designation_date": "2017-07-17", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-17-1894", "last_updated": "2017-08-14" }, { "eu_designation_number": "EU/3/17/1895", "medicine_name": "", "active_substance": "retinol", "intended_use": "Prevention of retinopathy of prematurity", "status": "Positive", "designation_date": "2017-07-17", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-17-1895", "last_updated": "2017-08-15" }, { "eu_designation_number": "EU/3/17/1896", "medicine_name": "", "active_substance": "sirolimus", "intended_use": "Treatment of pachyonychia congenita", "status": "Positive", "designation_date": "2017-07-17", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-17-1896", "last_updated": "2020-05-07" }, { "eu_designation_number": "EU/3/17/1897", "medicine_name": "", "active_substance": "tirapazamine", "intended_use": "Treatment of hepatocellular carcinoma", "status": "Positive", "designation_date": "2017-07-17", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-17-1897", "last_updated": "2017-08-14" }, { "eu_designation_number": "EU/3/17/1898", "medicine_name": "", "active_substance": "Adeno-associated viral vector serotype Anc80 containing the truncated human ATP7B gene under the control of the human alpha-1 antitrypsin promoter", "intended_use": "Treatment of Wilson's disease", "status": "Withdrawn", "designation_date": "2017-08-23", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-17-1898", "last_updated": "2020-12-03" }, { "eu_designation_number": "EU/3/17/1899", "medicine_name": "", "active_substance": "antisense oligonucleotide targeting exon 13 in the USH2A gene", "intended_use": "Treatment of retinitis pigmentosa", "status": "Positive", "designation_date": "2017-08-23", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-17-1899", "last_updated": "2017-10-17" }, { "eu_designation_number": "EU/3/17/1900", "medicine_name": "", "active_substance": "asunercept", "intended_use": "Treatment of myelodysplastic syndromes", "status": "Positive", "designation_date": "2017-08-23", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-17-1900", "last_updated": "2023-09-15" }, { "eu_designation_number": "EU/3/17/1901", "medicine_name": "", "active_substance": "Itraconazole", "intended_use": "Treatment of naevoid basal-cell carcinoma syndrome (Gorlin syndrome)", "status": "Withdrawn", "designation_date": "2017-08-23", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-17-1901", "last_updated": "2023-07-27" }, { "eu_designation_number": "EU/3/17/1902", "medicine_name": "", "active_substance": "N-{2-[(6-{[(2,6-dichloro-3,5-dimethoxyphenyl)carbamoyl](methyl)amino}pyrimidin-4-yl)amino]-5-(4-ethylpiperazin-1-yl)phenyl}prop-2-enamide", "intended_use": "Treatment of hepatocellular carcinoma", "status": "Withdrawn", "designation_date": "2017-08-23", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-17-1902", "last_updated": "2026-08-21" }, { "eu_designation_number": "EU/3/17/1903", "medicine_name": "", "active_substance": "odiparcil", "intended_use": "Treatment of mucopolysaccharidosis VI (Maroteaux-Lamy syndrome)", "status": "Positive", "designation_date": "2017-08-23", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-17-1903", "last_updated": "2017-10-17" }, { "eu_designation_number": "EU/3/17/1904", "medicine_name": "", "active_substance": "Picropodophyllin", "intended_use": "Treatment of glioma", "status": "Positive", "designation_date": "2017-08-23", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-17-1904", "last_updated": "2020-11-27" }, { "eu_designation_number": "EU/3/17/1905", "medicine_name": "", "active_substance": "purified pasteurised and freeze-dried cell-wall fragments from Mycobacterium tuberculosis strain RUTI", "intended_use": "Treatment of tuberculosis", "status": "Positive", "designation_date": "2017-08-23", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-17-1905", "last_updated": "2017-10-17" }, { "eu_designation_number": "EU/3/17/1906", "medicine_name": "", "active_substance": "recombinant adeno-associated viral vector serotype 5 carrying the gene for the human frataxin protein", "intended_use": "Treatment of Friedreich's ataxia", "status": "Positive", "designation_date": "2017-08-23", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-17-1906", "last_updated": "2018-12-18" }, { "eu_designation_number": "EU/3/17/1907", "medicine_name": "", "active_substance": "recombinant fragment of human surfactant protein-D", "intended_use": "Prevention of bronchopulmonary dysplasia", "status": "Positive", "designation_date": "2017-08-23", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-17-1907", "last_updated": "2019-08-19" }, { "eu_designation_number": "EU/3/17/1908", "medicine_name": "", "active_substance": "recombinant truncated N-terminal fragment of human lens epithelium-derived growth factor", "intended_use": "Treatment of retinitis pigmentosa", "status": "Withdrawn", "designation_date": "2017-08-23", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-17-1908", "last_updated": "2022-08-12" }, { "eu_designation_number": "EU/3/17/1909", "medicine_name": "", "active_substance": "Salmonella typhi Ty21a strain transfected with a plasmid vector encoding the human vascular endothelial growth factor receptor 2", "intended_use": "Treatment of glioma", "status": "Positive", "designation_date": "2017-08-23", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-17-1909", "last_updated": "2021-03-22" }, { "eu_designation_number": "EU/3/17/1910", "medicine_name": "Hyftor", "active_substance": "sirolimus", "intended_use": "Treatment of tuberous sclerosis", "status": "Positive", "designation_date": "2017-08-23", "related_ema_product_number": "EMEA/H/C/005896", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-17-1910", "last_updated": "2023-06-09" }, { "eu_designation_number": "EU/3/17/1911", "medicine_name": "", "active_substance": "sodium 2-hydroxylinoleate", "intended_use": "Treatment of pancreatic cancer", "status": "Positive", "designation_date": "2017-08-23", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-17-1911", "last_updated": "2017-10-17" }, { "eu_designation_number": "EU/3/17/1912", "medicine_name": "", "active_substance": "tacrolimus", "intended_use": "Treatment of pulmonary arterial hypertension", "status": "Positive", "designation_date": "2017-08-23", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-17-1912", "last_updated": "2017-10-17" }, { "eu_designation_number": "EU/3/17/1913", "medicine_name": "", "active_substance": "teicoplanin", "intended_use": "Treatment of cystic fibrosis", "status": "Positive", "designation_date": "2017-08-23", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-17-1913", "last_updated": "2021-05-06" }, { "eu_designation_number": "EU/3/17/1914", "medicine_name": "", "active_substance": "S)-3-((S)-2-(2-((2,6-difluorophenyl)amino)-2-oxoacetamido)propanamido)-4-oxo-5-(2,3,5,6-tetrafluorophenoxy)pentanoic acid", "intended_use": "Treatment of primary sclerosing cholangitis", "status": "Withdrawn", "designation_date": "2017-10-16", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-17-1914", "last_updated": "2026-05-26" }, { "eu_designation_number": "EU/3/17/1915", "medicine_name": "", "active_substance": "2-[N-(2-hydroxyethyl)]-N-(4-methoxybenzenesulfonyl)]amino-N-(4-chlorocinnamyl)-N-methylbenzylamine", "intended_use": "Treatment of Charcot-Marie-Tooth disease", "status": "Positive", "designation_date": "2017-10-16", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-17-1915", "last_updated": "2018-01-08" }, { "eu_designation_number": "EU/3/17/1916", "medicine_name": "", "active_substance": "5-amino-1-(2-methyl-1H-benzo[d]imidazol-5-yl)-1H-pyrazol-4-yl 1H-indol-2-yl ketone mono[(S)-2-hydroxysuccinate]", "intended_use": "Treatment of biliary tract cancer", "status": "Withdrawn", "designation_date": "2017-10-16", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-17-1916", "last_updated": "2020-12-11" }, { "eu_designation_number": "EU/3/17/1917", "medicine_name": "", "active_substance": "adenoviral vector of serotype 5 modified to contain a chimeric sequence consisting of a minimal urokinase-type plasminogen activator receptor promoter preceded by three Notch-responsive elements, and coated with oligopeptide end-modified poly (beta-amino) esters", "intended_use": "Treatment of pancreatic cancer", "status": "Positive", "designation_date": "2017-10-16", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-17-1917", "last_updated": "2018-01-09" }, { "eu_designation_number": "EU/3/17/1918", "medicine_name": "", "active_substance": "autologous ex-vivo-expanded peripheral polyclonal lymphocytes enriched in activated natural killer cells", "intended_use": "Treatment of multiple myeloma", "status": "Positive", "designation_date": "2017-10-16", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-17-1918", "last_updated": "2018-01-10" }, { "eu_designation_number": "EU/3/17/1919", "medicine_name": "", "active_substance": "bitopertin", "intended_use": "Treatment of beta thalassaemia intermedia and major", "status": "Withdrawn", "designation_date": "2017-10-16", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-17-1919", "last_updated": "2018-10-24" }, { "eu_designation_number": "EU/3/17/1920", "medicine_name": "", "active_substance": "cannabidiol", "intended_use": "Treatment of West syndrome", "status": "Positive", "designation_date": "2017-10-16", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-17-1920", "last_updated": "2023-06-15" }, { "eu_designation_number": "EU/3/17/1921", "medicine_name": "", "active_substance": "cannabidivarin", "intended_use": "Treatment of Rett syndrome", "status": "Positive", "designation_date": "2017-10-16", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-17-1921", "last_updated": "2023-06-15" }, { "eu_designation_number": "EU/3/17/1922", "medicine_name": "", "active_substance": "Entospletinib", "intended_use": "Treatment of acute myeloid leukaemia", "status": "Withdrawn", "designation_date": "2017-10-16", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-17-1922", "last_updated": "2025-06-10" }, { "eu_designation_number": "EU/3/17/1923", "medicine_name": "Daurismo", "active_substance": "Glasdegib maleate", "intended_use": "Treatment of acute myeloid leukaemia", "status": "Positive", "designation_date": "2017-10-16", "related_ema_product_number": "EMEA/H/C/004878", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-17-1923", "last_updated": "2018-01-09" }, { "eu_designation_number": "EU/3/17/1924", "medicine_name": "", "active_substance": "Glucopyranosyl lipid A", "intended_use": "Treatment of follicular lymphoma", "status": "Withdrawn", "designation_date": "2017-10-16", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-17-1924", "last_updated": "2019-10-15" }, { "eu_designation_number": "EU/3/17/1925", "medicine_name": "", "active_substance": "Humanised monoclonal antibody targeting B-cell maturation antigen conjugated with maleimidocaproyl monomethyl auristatin F", "intended_use": "Treatment of multiple myeloma", "status": "Withdrawn", "designation_date": "2017-10-16", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-17-1925", "last_updated": "2025-07-17" }, { "eu_designation_number": "EU/3/17/1926", "medicine_name": "", "active_substance": "ofranergene obadenovec", "intended_use": "Treatment of ovarian cancer", "status": "Withdrawn", "designation_date": "2017-10-16", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-17-1926", "last_updated": "2022-09-26" }, { "eu_designation_number": "EU/3/17/1927", "medicine_name": "", "active_substance": "pracinostat", "intended_use": "Treatment of acute myeloid leukaemia", "status": "Withdrawn", "designation_date": "2017-10-16", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-17-1927", "last_updated": "2022-02-18" }, { "eu_designation_number": "EU/3/17/1928", "medicine_name": "", "active_substance": "recombinant 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"https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-17-1932", "last_updated": "2018-01-08" }, { "eu_designation_number": "EU/3/17/1933", "medicine_name": "", "active_substance": "", "intended_use": "Treatment of systemic sclerosis", "status": "Positive", "designation_date": "2017-10-12", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-17-1933", "last_updated": "2018-12-07" }, { "eu_designation_number": "EU/3/17/1934", "medicine_name": "", "active_substance": "(R)-troloxamide quinone", "intended_use": "Treatment of amyotrophic lateral sclerosis", "status": "Positive", "designation_date": "2017-10-12", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-17-1934", "last_updated": "2019-12-03" }, { "eu_designation_number": "EU/3/17/1935", "medicine_name": "", "active_substance": "1,4-diamino-2,3-dicyano-1,4-bis[2-aminophenylthio] butadiene", 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"last_updated": "2018-03-28" }, { "eu_designation_number": "EU/3/17/1940", "medicine_name": "", "active_substance": "concizumab", "intended_use": "Treatment of haemophilia B", "status": "Withdrawn", "designation_date": "2017-10-12", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-17-1940", "last_updated": "2022-12-14" }, { "eu_designation_number": "EU/3/17/1941", "medicine_name": "Viokat", "active_substance": "diazoxide choline", "intended_use": "Treatment of Prader-Willi syndrome", "status": "Positive", "designation_date": "2017-10-12", "related_ema_product_number": "EMEA/H/C/006576", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-17-1941", "last_updated": "2026-04-24" }, { "eu_designation_number": "EU/3/17/1942", "medicine_name": "", "active_substance": "N-(2-aminophenyl)-4-(1-[(1,3-dimethyl-1H-pyrazol-4-yl)methyl]piperidin)benzamide", "intended_use": "Treatment of peripheral T-cell 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"https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-17-1944", "last_updated": "2019-07-30" }, { "eu_designation_number": "EU/3/17/1945", "medicine_name": "Emcitate", "active_substance": "tiratricol", "intended_use": "Treatment of Allan-Herndon-Dudley syndrome", "status": "Positive", "designation_date": "2017-10-12", "related_ema_product_number": "EMEA/H/C/005220", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-17-1945", "last_updated": "2024-12-13" }, { "eu_designation_number": "EU/3/17/1946", "medicine_name": "Voydeya", "active_substance": "(2S,4R)-1-(2-(3-acetyl-5-(2-methylpyrimidine-5-yl)-1H-indazol-1-yl)acetyl)-N-(6-bromopyridine-2-yl)-4-fluoropyrrolidine-2-carboxamide", "intended_use": "Treatment of paroxysmal nocturnal haemoglobinuria", "status": "Positive", "designation_date": "2017-12-12", "related_ema_product_number": "EMEA/H/C/005517", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-17-1946", "last_updated": "2020-06-08" }, { "eu_designation_number": "EU/3/17/1947", "medicine_name": "", "active_substance": "2-isopropyl-3H-naphtho[1,2-d]imidazole-4,5-dione", "intended_use": "Treatment of mitochondrial encephalomyopathy, lactic acidosis and stroke-like episodes", "status": "Positive", "designation_date": "2017-12-12", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-17-1947", "last_updated": "2021-02-03" }, { "eu_designation_number": "EU/3/17/1948", "medicine_name": "", "active_substance": "4-hydroxy-2,2,6,6-tetramethylpiperidine-N-oxyl", "intended_use": "Treatment of familial cerebral cavernous malformations", "status": "Positive", "designation_date": "2017-12-12", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-17-1948", "last_updated": "2022-10-11" }, { "eu_designation_number": "EU/3/17/1949", "medicine_name": "", "active_substance": "acetylleucine", "intended_use": "Treatment of GM2 gangliosidosis", "status": "Positive", "designation_date": "2017-12-12", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-17-1949", "last_updated": "2019-04-25" }, { "eu_designation_number": "EU/3/17/1950", "medicine_name": "", "active_substance": "adenovirus associated viral vector serotype 8 containing the human AIPL1 gene", "intended_use": "Treatment of Leber's congenital amaurosis", "status": "Positive", "designation_date": "2017-12-12", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-17-1950", "last_updated": "2024-10-22" }, { "eu_designation_number": "EU/3/17/1951", "medicine_name": "", "active_substance": "agammaglobulinaemia tyrosine kinase", "intended_use": "Treatment of pemphigus", "status": "Withdrawn", "designation_date": "2017-12-12", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-17-1951", "last_updated": "2021-11-15" }, { "eu_designation_number": "EU/3/17/1952", "medicine_name": "", "active_substance": "modified messenger ribonucleic acid encoding human argininosuccinate lyase enzyme encapsulated into lipid nanoparticles", "intended_use": "Treatment of argininosuccinic aciduria", "status": "Positive", "designation_date": "2017-12-12", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-17-1952", "last_updated": "2018-01-23" }, { "eu_designation_number": "EU/3/17/1953", "medicine_name": "", "active_substance": "pegunigalsidase alfa", "intended_use": "Treatment of Fabry disease", "status": "Withdrawn", "designation_date": "2017-12-12", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-17-1953", "last_updated": "2023-05-08" }, { "eu_designation_number": "EU/3/17/1954", "medicine_name": "", "active_substance": "venetoclax", "intended_use": "Treatment of mantle cell lymphoma", "status": "Withdrawn", "designation_date": "2017-12-12", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-17-1954", "last_updated": "2025-03-11" }, { "eu_designation_number": "EU/3/17/1955", "medicine_name": "", "active_substance": "adeno-associated viral vector serotype 2/6 encoding zinc-finger nucleases and the human alpha L-iduronidase gene (devafidugene civaparvovec)", "intended_use": "Treatment of mucopolysaccharidosis type I", "status": "Withdrawn", "designation_date": "2018-01-17", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-17-1955", "last_updated": "2023-09-12" }, { "eu_designation_number": "EU/3/17/1956", "medicine_name": "", "active_substance": "adeno-associated viral vector serotype 2/6 encoding zinc-finger nucleases and the human 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"active_substance": "genetically modified replication-incompetent herpes simplex virus-1 expressing collagen VII (beremagene geperpavec)", "intended_use": "Treatment of epidermolysis bullosa", "status": "Positive", "designation_date": "2018-04-16", "related_ema_product_number": "EMEA/H/C/006330", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-18-2012", "last_updated": "2025-06-10" }, { "eu_designation_number": "EU/3/18/2013", "medicine_name": "Polivy", "active_substance": "polatuzumab vedotin", "intended_use": "Treatment of diffuse large B-cell lymphoma", "status": "Positive", "designation_date": "2018-04-16", "related_ema_product_number": "EMEA/H/C/004870", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-18-2013", "last_updated": "2022-06-08" }, { "eu_designation_number": "EU/3/18/2014", "medicine_name": "", "active_substance": "1-(3-{4-[3,4-difluoro-2-(trifluoromethyl)phenyl]piperidine-1-carbonyl}-1H,4H,5H,6H,7H-pyrazolo[3,4- c]pyridin-6-yl)ethan-1-one (Tinlarebant)", "intended_use": "Treatment of Stargardt's disease", "status": "Positive", "designation_date": "2018-05-25", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-18-2014", "last_updated": "2022-12-01" }, { "eu_designation_number": "EU/3/18/2015", "medicine_name": "", "active_substance": "adeno-associated viral vector serotype 8 containing a functional copy of the codon-optimised F8 cDNA encoding the B-domain deleted human coagulation factor VIII", "intended_use": "Treatment of haemophilia A", "status": "Withdrawn", "designation_date": "2018-05-25", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-18-2015", "last_updated": "2024-04-09" }, { "eu_designation_number": "EU/3/18/2016", "medicine_name": "", "active_substance": "adeno-associated viral vector serotype 9 containing the human CLN1 gene", "intended_use": "Treatment of neuronal ceroid lipofuscinosis", "status": "Positive", "designation_date": "2018-05-25", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-18-2016", "last_updated": "2018-07-23" }, { "eu_designation_number": "EU/3/18/2017", "medicine_name": "", "active_substance": "ambroxol hydrochloride", "intended_use": "Treatment of amyotrophic lateral sclerosis", "status": "Positive", "designation_date": "2018-05-25", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-18-2017", "last_updated": "2018-07-24" }, { "eu_designation_number": "EU/3/18/2018", "medicine_name": "", "active_substance": "autologous CD4+ and CD8+ T cells expressing a CD19-specific chimeric antigen receptor (lisocabtagene maraleucel)", "intended_use": "Treatment of follicular lymphoma", "status": "Withdrawn", "designation_date": "2018-05-25", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-18-2018", "last_updated": "2022-05-04" }, { "eu_designation_number": "EU/3/18/2019", "medicine_name": "Imbarkyd", "active_substance": "Bardoxolone methyl", "intended_use": "Treatment of Alport syndrome", "status": "Positive", "designation_date": "2018-05-25", "related_ema_product_number": "EMEA/H/C/005869", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-18-2019", "last_updated": "2023-01-30" }, { "eu_designation_number": "EU/3/18/2020", "medicine_name": "", "active_substance": "daratumumab", "intended_use": "Treatment of AL amyloidosis", "status": "Withdrawn", "designation_date": "2018-05-25", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-18-2020", "last_updated": "2026-08-26" }, { "eu_designation_number": "EU/3/18/2021", "medicine_name": "", "active_substance": "Equine immunoglobulin F(ab')2 fragments targeting Shiga toxin", "intended_use": "Prevention of haemolytic uraemic syndrome", "status": "Positive", "designation_date": "2018-05-25", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-18-2021", "last_updated": "2018-07-24" }, { "eu_designation_number": "EU/3/18/2022", "medicine_name": "", "active_substance": "Glucagon analogue linked to a human immunoglobulin Fc fragment", "intended_use": "Treatment of congenital hyperinsulinism", "status": "Positive", "designation_date": "2018-05-25", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-18-2022", "last_updated": "2019-05-23" }, { "eu_designation_number": "EU/3/18/2023", "medicine_name": "", "active_substance": "H-Arg-Pro-Lys-Pro-Gln-Gln-Phe-2Thi-Gly-Leu-Met(O2)-NH2-DOTA-225-Actinium", "intended_use": "Treatment of glioma", "status": "Positive", "designation_date": "2018-05-25", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-18-2023", "last_updated": "2018-07-24" }, { "eu_designation_number": "EU/3/18/2024", "medicine_name": "", "active_substance": "Itraconazole", "intended_use": "Prevention of invasive aspergillosis", "status": "Positive", "designation_date": "2018-05-25", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-18-2024", "last_updated": "2020-05-15" }, { "eu_designation_number": "EU/3/18/2025", "medicine_name": "", "active_substance": "modified mRNA encoding human methylmalonyl-coenzyme A mutase encapsulated into lipid nanoparticles", "intended_use": "Treatment of methylmalonic acidaemia", "status": "Withdrawn", "designation_date": "2018-05-25", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-18-2025", "last_updated": "2023-06-26" }, { "eu_designation_number": "EU/3/18/2026", "medicine_name": "Amvuttra", "active_substance": "synthetic double-stranded siRNA oligonucleotide targeted against transthyretin mRNA, with six phosphorothioate linkages in the backbone, and nine 2'-fluoro and thirty-five 2'-O-methyl nucleoside residues in the sequence, which is covalently linked via a phosphodiester group to a ligand containing three N-acetylgalactosamine residues (Vutrisiran)", "intended_use": "Treatment of transthyretin-mediated amyloidosis (ATTR amyloidosis)", "status": "Positive", "designation_date": "2018-05-25", "related_ema_product_number": "EMEA/H/C/005852", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-18-2026", "last_updated": "2025-07-17" }, { "eu_designation_number": "EU/3/18/2027", "medicine_name": "", "active_substance": "three human monoclonal antibodies against the EBOV glycoprotein", "intended_use": "Treatment of Ebola virus disease", "status": 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"designation_date": "2018-06-27", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-18-2029", "last_updated": "2018-08-21" }, { "eu_designation_number": "EU/3/18/2030", "medicine_name": "", "active_substance": "20-hydroxyecdysone", "intended_use": "Treatment of Duchenne muscular dystrophy", "status": "Positive", "designation_date": "2018-06-27", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-18-2030", "last_updated": "2018-08-21" }, { "eu_designation_number": "EU/3/18/2031", "medicine_name": "", "active_substance": "argon", "intended_use": "Treatment of perinatal asphyxia", "status": "Positive", "designation_date": "2018-06-27", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-18-2031", "last_updated": "2018-08-22" }, { "eu_designation_number": "EU/3/18/2032", "medicine_name": "", "active_substance": "carmustine", "intended_use": "Treatment in haematopoietic stem cell transplantation", "status": "Positive", "designation_date": "2018-06-27", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-18-2032", "last_updated": "2018-08-21" }, { "eu_designation_number": "EU/3/18/2033", "medicine_name": "", "active_substance": "codon-optimised human ornithine transcarbamylase mRNA complexed with lipid-based nanoparticles", "intended_use": "Treatment of ornithine transcarbamylase deficiency", "status": "Withdrawn", "designation_date": "2018-06-27", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-18-2033", "last_updated": "2021-04-28" }, { "eu_designation_number": "EU/3/18/2034", "medicine_name": "Upkanz", "active_substance": "Deferiprone", "intended_use": "Treatment of neurodegeneration with brain iron accumulation", "status": "Positive", "designation_date": "2018-06-27", "related_ema_product_number": "EMEA/H/C/005004", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-18-2034", "last_updated": "2020-02-04" }, { "eu_designation_number": "EU/3/18/2035", "medicine_name": "", "active_substance": "Efpegsomatropin", "intended_use": "Treatment of growth hormone deficiency", "status": "Positive", "designation_date": "2018-06-27", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-18-2035", "last_updated": "2019-05-23" }, { "eu_designation_number": "EU/3/18/2036", "medicine_name": "", "active_substance": "L-cystine bis(N-methylpiperazide)", "intended_use": "Treatment of cystinuria", "status": "Positive", "designation_date": "2018-06-27", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-18-2036", "last_updated": "2021-04-30" }, { "eu_designation_number": "EU/3/18/2037", "medicine_name": "Skyclarys", "active_substance": "omaveloxolone", "intended_use": "Treatment of Friedreich’s ataxia", "status": "Positive", "designation_date": "2018-06-27", "related_ema_product_number": "EMEA/H/C/006084", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-18-2037", "last_updated": "2024-10-22" }, { "eu_designation_number": "EU/3/18/2038", "medicine_name": "", "active_substance": "palovarotene", "intended_use": "Treatment of multiple osteochondromas", "status": "Withdrawn", "designation_date": "2018-06-27", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-18-2038", "last_updated": "2021-10-19" }, { "eu_designation_number": "EU/3/18/2039", "medicine_name": "", "active_substance": "recombinant adeno-associated viral vector serotype 9 containing human iduronidase gene", "intended_use": "Treatment of mucopolysaccharidosis type I", "status": "Positive", "designation_date": "2018-06-27", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-18-2039", "last_updated": "2018-08-22" }, { "eu_designation_number": "EU/3/18/2040", "medicine_name": "", "active_substance": "recombinant human placental growth factor", "intended_use": "Treatment of pre-eclampsia", "status": "Positive", "designation_date": "2018-06-27", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-18-2040", "last_updated": "2018-08-22" }, { "eu_designation_number": "EU/3/18/2041", "medicine_name": "", "active_substance": "2ʹ-O-(2-methoxyethyl) antisense oligonucleotide targeting microtubule-associated protein tau pre-mRNA", "intended_use": "treatment of behavioural variant frontotemporal dementia", "status": "Positive", "designation_date": "2018-07-31", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-18-2041", "last_updated": "2020-10-13" }, { "eu_designation_number": "EU/3/18/2042", "medicine_name": "", "active_substance": "adenovirus associated viral vector serotype 2/8 containing the human CNGA3 gene (entacingene turiparvovec)", "intended_use": "Treatment of achromatopsia", "status": "Positive", "designation_date": "2018-07-31", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-18-2042", "last_updated": "2024-10-22" }, { "eu_designation_number": "EU/3/18/2043", "medicine_name": "", "active_substance": "carboplatin;sodium valproate", "intended_use": "Treatment of glioma", "status": "Positive", "designation_date": "2018-07-31", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-18-2043", "last_updated": "2022-03-18" }, { "eu_designation_number": "EU/3/18/2044", "medicine_name": "", "active_substance": "allogeneic bone marrow derived mesenchymal stromal cells, ex-vivo 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"https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-18-2046", "last_updated": "" }, { "eu_designation_number": "EU/3/18/2047", "medicine_name": "", "active_substance": "liposomal mannose-1-phosphate", "intended_use": "treatment of phosphomannomutase 2-congenital disorder of glycosylation", "status": "Positive", "designation_date": "2018-07-31", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-18-2047", "last_updated": "2020-05-07" }, { "eu_designation_number": "EU/3/18/2048", "medicine_name": "", "active_substance": "N-acetylgalactosamine-conjugated synthetic double-stranded oligomer specific to serpin family A member 1 gene", "intended_use": "Treatment of congenital alpha-1 antitrypsin deficiency", "status": "Positive", "designation_date": "2018-07-31", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-18-2048", "last_updated": "2021-03-02" }, { "eu_designation_number": "EU/3/18/2049", "medicine_name": "", "active_substance": "recombinant human ectonucleotide pyrophosphatase/phosphodiesterase 1 fused to the Fc fragment of IgG1", "intended_use": "treatment of ectonucleotide pyrophosphatase/phosphodiesterase 1 deficiency", "status": "Positive", "designation_date": "2018-07-31", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-18-2049", "last_updated": "" }, { "eu_designation_number": "EU/3/18/2050", "medicine_name": "Koselugo", "active_substance": "selumetinib", "intended_use": "Treatment of neurofibromatosis type 1", "status": "Positive", "designation_date": "2018-07-31", "related_ema_product_number": "EMEA/H/C/005244", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-18-2050", "last_updated": "2021-09-15" }, { "eu_designation_number": "EU/3/18/2051", "medicine_name": "", "active_substance": "synthetic antisense oligonucleotide directed against human dystrophin pre-mRNA", "intended_use": "Treatment of Duchenne muscular dystrophy", "status": "Positive", "designation_date": "2018-07-31", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-18-2051", "last_updated": "" }, { "eu_designation_number": "EU/3/18/2052", "medicine_name": "", "active_substance": "", "intended_use": "Treatment of primary hyperoxaluria", "status": "Positive", "designation_date": "2018-07-31", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-18-2052", "last_updated": "" }, { "eu_designation_number": "EU/3/18/2052", "medicine_name": "", "active_substance": "synthetic double-stranded siRNA oligonucleotide directed against lactate dehydrogenase A mRNA and containing four modified nucleosides which form a ligand cluster of four N-acetylgalactosamine residues", "intended_use": "Treatment of primary hyperoxaluria", "status": "Positive", "designation_date": "2018-07-31", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-18-2052-0", "last_updated": "2023-02-24" }, { "eu_designation_number": "EU/3/18/2053", "medicine_name": "", "active_substance": "tamibarotene", "intended_use": "Treatment of acute myeloid leukaemia", "status": "Positive", "designation_date": "2018-07-31", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-18-2053", "last_updated": "2019-05-21" }, { "eu_designation_number": "EU/3/18/2054", "medicine_name": "", "active_substance": "tetracosactide", "intended_use": "Treatment of Duchenne muscular dystrophy", "status": "Positive", "designation_date": "2018-07-31", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-18-2054", "last_updated": "" }, { "eu_designation_number": "EU/3/18/2055", "medicine_name": "", 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"EU/3/18/2085", "medicine_name": "", "active_substance": "autologous human adipose perivascular stromal cells genetically modified to secrete soluble tumour necrosis factor-related apoptosis-inducing ligand", "intended_use": "Treatment of pancreatic cancer", "status": "Positive", "designation_date": "2018-11-19", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-18-2085", "last_updated": "" }, { "eu_designation_number": "EU/3/18/2086", "medicine_name": "", "active_substance": "cyclo[L-alanyl-L-seryl-L-isoleucyl-L-prolyl-L-prolyl-L-glutaminyl-L-lysyl-L-tyrosyl-D-prolyl-L-prolyl-(2S)-2-aminodecanoyl-L-alpha-glutamyl-L-threonyl]acetate", "intended_use": "Treatment of cystic fibrosis", "status": "Positive", "designation_date": "2018-11-19", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-18-2086", "last_updated": "" }, { "eu_designation_number": "EU/3/18/2087", 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"last_updated": "2023-02-13" }, { "eu_designation_number": "EU/3/18/2131", "medicine_name": "", "active_substance": "rozanolixizumab", "intended_use": "Treatment of immune thrombocytopenia", "status": "Withdrawn", "designation_date": "2019-01-11", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-18-2131", "last_updated": "2023-05-08" }, { "eu_designation_number": "EU/3/18/2132", "medicine_name": "", "active_substance": "synthetic double-stranded siRNA oligonucleotide directed against TMPRSS6 mRNA and covalently linked to a ligand containing three N-acetylgalactosamine residues", "intended_use": "Treatment of beta thalassaemia intermedia and major", "status": "Withdrawn", "designation_date": "2019-01-11", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-18-2132", "last_updated": "" }, { "eu_designation_number": "EU/3/18/2133", "medicine_name": "", "active_substance": "vinorelbine tartrate", "intended_use": "Treatment of soft tissue sarcoma", "status": "Positive", "designation_date": "2019-01-11", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-18-2133", "last_updated": "" }, { "eu_designation_number": "EU/3/19/2134", "medicine_name": "", "active_substance": "9-cis, 12-cis-11,11-D2-linoleic acid ethyl ester", "intended_use": "Treatment of infantile neuroaxonal dystrophy", "status": "Positive", "designation_date": "2019-02-26", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-19-2134", "last_updated": "" }, { "eu_designation_number": "EU/3/19/2135", "medicine_name": "", "active_substance": "allogeneic cultured postnatal thymus-derived tissue", "intended_use": "Treatment of DiGeorge syndrome", "status": "Withdrawn", "designation_date": "2019-02-26", "related_ema_product_number": "", "url": 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spinal muscular atrophy", "status": "Withdrawn", "designation_date": "2019-02-26", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-19-2145", "last_updated": "2023-09-12" }, { "eu_designation_number": "EU/3/19/2146", "medicine_name": "", "active_substance": "1-[(3S)-3-{4-amino-3-[(3,5-dimethoxyphenyl)ethynyl]-1H-pyrazolo[3,4-d]pyrimidin-1-yl}pyrrolidin-1-yl]-2-propen-1-one (futibatinib)", "intended_use": "Treatment of biliary tract cancer", "status": "Withdrawn", "designation_date": "2019-04-01", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-19-2146", "last_updated": "2026-05-26" }, { "eu_designation_number": "EU/3/19/2147", "medicine_name": "", "active_substance": "2-[3-(2-chloro-4-{[5-cyclopropyl-3-(2,6-dichlorophenyl)-1,2-oxazol-4-yl]methoxy}phenyl)-3-hydroxyazetidin-1-yl]pyridine-4-carboxylic acid-2-amino-2-(hydroxymethyl)propane-1,3-diol (1/1)", "intended_use": "Treatment of primary sclerosing cholangitis", "status": "Withdrawn", "designation_date": "2019-04-01", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-19-2147", "last_updated": "2026-05-26" }, { "eu_designation_number": "EU/3/19/2148", "medicine_name": "", "active_substance": "4-hydroxy-6-{2-[4-(trifluoromethyl)phenyl]ethyl}pyridazin-3(2H)-one", "intended_use": "Treatment of Friedreich’s ataxia", "status": "Withdrawn", "designation_date": "2019-04-01", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-19-2148", "last_updated": "2019-11-20" }, { "eu_designation_number": "EU/3/19/2149", "medicine_name": "", "active_substance": "Adeno-associated viral vector serotype rh10 containing the human cholesterol 24-hydroxylase gene", "intended_use": "Treatment of Huntington’s disease", "status": "Withdrawn", "designation_date": "2019-04-01", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-19-2149", "last_updated": "2025-03-12" }, { "eu_designation_number": "EU/3/19/2150", "medicine_name": "", "active_substance": "codon-optimised human cystic fibrosis transmembrane conductance regulator messenger ribonucleic acid complexed with lipid-based nanoparticles;pomulmeran", "intended_use": "Treatment of cystic fibrosis", "status": "Withdrawn", "designation_date": "2019-04-01", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-19-2150", "last_updated": "2023-07-21" }, { "eu_designation_number": "EU/3/19/2151", "medicine_name": "", "active_substance": "Marzeptacog alfa (activated)", "intended_use": "Treatment of haemophilia B", "status": "Withdrawn", "designation_date": "2019-04-01", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-19-2151", 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"active_substance": "balipodect", "intended_use": "Treatment of fragile X syndrome", "status": "Positive", "designation_date": "2019-04-14", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-19-2154", "last_updated": "2020-08-26" }, { "eu_designation_number": "EU/3/19/2155", "medicine_name": "", "active_substance": "human culture expanded autologous mesenchymal stromal cells", "intended_use": "Treatment of amyotrophic lateral sclerosis", "status": "Positive", "designation_date": "2019-04-24", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-19-2155", "last_updated": "2022-05-03" }, { "eu_designation_number": "EU/3/19/2156", "medicine_name": "", "active_substance": "modified messenger ribonucleic acid encoding human propionyl-coenzyme A carboxylase alpha and beta subunits encapsulated into lipid nanoparticle", "intended_use": "Treatment of propionic acidaemia", 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"eu_designation_number": "EU/3/19/2161", "medicine_name": "", "active_substance": "Diacerein", "intended_use": "Treatment of epidermolysis bullosa", "status": "Positive", "designation_date": "2019-05-29", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-19-2161", "last_updated": "2019-08-19" }, { "eu_designation_number": "EU/3/19/2162", "medicine_name": "", "active_substance": "emixustat hydrochloride", "intended_use": "Treatment of Stargardt's disease", "status": "Positive", "designation_date": "2019-05-29", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-19-2162", "last_updated": "" }, { "eu_designation_number": "EU/3/19/2163", "medicine_name": "", "active_substance": "N-(trans-3-(5-((R)-1-hydroxyethyl)-1,3,4-oxadiazol-2-yl)cyclobutyl)-3-phenylisoxazole-5-carboxamide", "intended_use": "Treatment of cystic fibrosis", "status": "Withdrawn", "designation_date": "2019-05-29", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-19-2163", "last_updated": "2021-07-30" }, { "eu_designation_number": "EU/3/19/2164", "medicine_name": "", "active_substance": "relacorilant", "intended_use": "Treatment of Cushing's syndrome", "status": "Positive", "designation_date": "2019-05-29", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-19-2164", "last_updated": "2024-10-22" }, { "eu_designation_number": "EU/3/19/2165", "medicine_name": "", "active_substance": "sodium phenylacetate;sodium benzoate", "intended_use": "Treatment of citrullinaemia type 1", "status": "Positive", "designation_date": "2019-05-29", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-19-2165", "last_updated": "" }, { "eu_designation_number": "EU/3/19/2166", "medicine_name": "", "active_substance": "sodium 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"url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-19-2168", "last_updated": "2023-03-27" }, { "eu_designation_number": "EU/3/19/2169", "medicine_name": "", "active_substance": "reldesemtiv", "intended_use": "Treatment of spinal muscular atrophy", "status": "Positive", "designation_date": "2019-06-28", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-19-2169", "last_updated": "2024-10-11" }, { "eu_designation_number": "EU/3/19/2170", "medicine_name": "", "active_substance": "2-(2-{[2-(1H-benzimidazol-2-yl)ethyl]amino}ethyl)-N-[(3-fluoropyridine-2-yl)methyl]-1,3-oxazole-4-carboxamide trihydrochloride", "intended_use": "Treatment of beta thalassaemia intermedia and major", "status": "Positive", "designation_date": "2019-06-28", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-19-2170", "last_updated": "" }, { "eu_designation_number": "EU/3/19/2171", "medicine_name": "", "active_substance": "5'-cEtG-sp-cEt5MeU-sp-cEt5MeU-sp-dT-sp-dA-sp-dT-sp-dT-sp-dA-sp-dT-sp-dA-sp-dG-sp-dG-sp-dG-sp-cEt5MeC-sp-cEt5MeU-sp-cEt5MeU-3' (also known as DYN101)", "intended_use": "Treatment of centronuclear myopathies", "status": "Positive", "designation_date": "2019-06-28", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-19-2171", "last_updated": "" }, { "eu_designation_number": "EU/3/19/2172", "medicine_name": "", "active_substance": "Gaboxadol monohydrate", "intended_use": "Treatment of Angelman syndrome", "status": "Positive", "designation_date": "2019-06-28", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-19-2172", "last_updated": "" }, { "eu_designation_number": "EU/3/19/2173", "medicine_name": "", "active_substance": "Imidazolyl ethanamide pentandioic acid", "intended_use": "Treatment of acute radiation 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"designation_date": "2019-06-28", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-19-2175", "last_updated": "" }, { "eu_designation_number": "EU/3/19/2176", "medicine_name": "Altuvoct", "active_substance": "recombinant human coagulation factor VIII Fc - von Willebrand factor - XTEN fusion protein", "intended_use": "Treatment of haemophilia A", "status": "Positive", "designation_date": "2019-06-28", "related_ema_product_number": "EMEA/H/C/005968", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-19-2176", "last_updated": "" }, { "eu_designation_number": "EU/3/19/2177", "medicine_name": "", "active_substance": "regorafenib", "intended_use": "Treatment of glioma", "status": "Withdrawn", "designation_date": "2019-06-28", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-19-2177", "last_updated": "2022-12-20" }, { "eu_designation_number": "EU/3/19/2178", "medicine_name": "", "active_substance": "sodium benzoate;sodium phenylacetate", "intended_use": "Treatment of argininosuccinic aciduria", "status": "Positive", "designation_date": "2019-06-28", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-19-2178", "last_updated": "" }, { "eu_designation_number": "EU/3/19/2179", "medicine_name": "", "active_substance": "sodium benzoate;sodium phenylacetate", "intended_use": "Treatment of hyperargininaemia", "status": "Positive", "designation_date": "2019-06-28", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-19-2179", "last_updated": "" }, { "eu_designation_number": "EU/3/19/2180", "medicine_name": "", "active_substance": "2-(hydroxymethyl)-2-(methoxymethyl)-1-azabicyclo[2.2.2]octan-3-one", "intended_use": "Treatment of myelodysplastic syndromes", "status": "Positive", "designation_date": "2019-07-25", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-19-2180", "last_updated": "2020-10-13" }, { "eu_designation_number": "EU/3/19/2181", "medicine_name": "", "active_substance": "7-ethyl-10-hydroxy-camptothecin", "intended_use": "Treatment of soft tissue sarcoma", "status": "Positive", "designation_date": "2019-07-25", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-19-2181", "last_updated": "2023-06-09" }, { "eu_designation_number": "EU/3/19/2182", "medicine_name": "Iqirvo", "active_substance": "Elafibranor", "intended_use": "Treatment of primary biliary cholangitis", "status": "Positive", "designation_date": "2019-07-25", "related_ema_product_number": "EMEA/H/C/006231", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-19-2182", "last_updated": "2022-09-08" }, { "eu_designation_number": "EU/3/19/2183", "medicine_name": "Xolremdi", "active_substance": "mavorixafor", "intended_use": "Treatment of WHIM syndrome", "status": "Positive", "designation_date": "2019-07-25", "related_ema_product_number": "EMEA/H/C/006496", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-19-2183", "last_updated": "2026-08-04" }, { "eu_designation_number": "EU/3/19/2184", "medicine_name": "Ezmekly", "active_substance": "N-((R)-2,3-dihydroxypropoxyl)-3,4-difluro-2-(2-fluoro-4-iodo-phenylamino)-benzamide", "intended_use": "Treatment of neurofibromatosis type 1", "status": "Positive", "designation_date": "2019-07-25", "related_ema_product_number": "EMEA/H/C/006460", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-19-2184", "last_updated": "2024-04-11" }, { "eu_designation_number": "EU/3/19/2185", "medicine_name": "", "active_substance": "Parsaclisib", "intended_use": "Treatment of marginal zone lymphoma", "status": "Withdrawn", "designation_date": "2019-07-25", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-19-2185", "last_updated": "2022-12-13" }, { "eu_designation_number": "EU/3/19/2186", "medicine_name": "", "active_substance": "pevonedistat", "intended_use": "Treatment of acute myeloid leukaemia", "status": "Withdrawn", "designation_date": "2019-07-25", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-19-2186", "last_updated": "2022-02-18" }, { "eu_designation_number": "EU/3/19/2187", "medicine_name": "", "active_substance": "recombinant mutated extracellular domain of the human acetylcholine receptor subunit alpha1", "intended_use": "Treatment of myasthenia gravis", "status": "Positive", "designation_date": "2019-07-25", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-19-2187", "last_updated": "" }, { "eu_designation_number": "EU/3/19/2188", "medicine_name": "Kizfizo", "active_substance": "temozolomide", "intended_use": "Treatment of neuroblastoma", "status": "Positive", "designation_date": "2019-08-21", "related_ema_product_number": "EMEA/H/C/006169", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-19-2188", "last_updated": "2024-11-15" }, { "eu_designation_number": "EU/3/19/2189", "medicine_name": "", "active_substance": "gallium citrate", "intended_use": "Treatment of cystic fibrosis", "status": "Positive", "designation_date": "2019-08-21", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-19-2189", "last_updated": "2021-07-13" }, { "eu_designation_number": "EU/3/19/2190", "medicine_name": "", "active_substance": "peginterferon lambda-1a", "intended_use": "Treatment of hepatitis D virus infection", "status": "Positive", "designation_date": "2019-08-21", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-19-2190", "last_updated": "" }, { "eu_designation_number": "EU/3/19/2191", "medicine_name": "", "active_substance": "relacorilant", "intended_use": "Treatment of pancreatic cancer", "status": "Positive", "designation_date": "2019-08-21", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-19-2191", "last_updated": "2024-10-22" }, { "eu_designation_number": "EU/3/19/2192", "medicine_name": "Imcivree", "active_substance": "setmelanotide", "intended_use": "Treatment of Bardet-Biedl syndrome", "status": "Positive", "designation_date": "2019-08-21", "related_ema_product_number": "EMEA/H/C/005089", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-19-2192", "last_updated": "2022-09-30" }, { "eu_designation_number": "EU/3/19/2193", "medicine_name": "", "active_substance": "acetazolamide", "intended_use": "Treatment of periodic paralysis", "status": "Positive", "designation_date": "2019-08-21", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-19-2193", "last_updated": "" }, { "eu_designation_number": "EU/3/19/2194", "medicine_name": "", "active_substance": "4-(2-chloro-4-methoxy-5-methylphenyl)-N-[(1S)-2-cyclopropyl-1-(3-fluoro-4-methylphenyl)ethyl]-5-methyl-N-(2-propynyl)-1,3-thiazol-2-amine", "intended_use": "Treatment of congenital adrenal hyperplasia", "status": "Positive", "designation_date": "2019-08-21", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-19-2194", "last_updated": "" }, { "eu_designation_number": "EU/3/19/2195", "medicine_name": "", "active_substance": "1-(2,2-diphenyltetrahydrofuran-3-yl)-N,N-dimethylmethanamine hydrochloride", "intended_use": "Treatment of Rett syndrome", "status": "Positive", "designation_date": "2019-08-21", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-19-2195", "last_updated": "" }, { "eu_designation_number": "EU/3/19/2196", "medicine_name": "", "active_substance": "recombinant self-complementary adeno-associated viral vector serotype 9 containing the human CLN3 gene", "intended_use": "Treatment of neuronal ceroid lipofuscinosis", "status": "Positive", "designation_date": "2019-08-21", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-19-2196", "last_updated": "2024-10-22" }, { "eu_designation_number": "EU/3/19/2197", "medicine_name": "", "active_substance": "recombinant self-complementary adeno-associated viral vector serotype 9 containing the human CLN6 gene", "intended_use": "Treatment of neuronal ceroid lipofuscinosis", "status": "Positive", "designation_date": "2019-08-21", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-19-2197", "last_updated": "2024-10-22" }, { "eu_designation_number": "EU/3/19/2198", "medicine_name": "", "active_substance": "adenoviral vector serotype 5 encoding the human interleukin-12 p70 transgene under the control of activator ligand veledimex", "intended_use": "Treatment of glioma", "status": "Withdrawn", "designation_date": "2019-08-21", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-19-2198", "last_updated": "2022-03-21" }, { "eu_designation_number": "EU/3/19/2199", "medicine_name": "", "active_substance": "veledimex", "intended_use": "Treatment of glioma", "status": "Withdrawn", "designation_date": "2019-08-21", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-19-2199", "last_updated": "2022-03-21" }, { "eu_designation_number": "EU/3/19/2200", "medicine_name": "", "active_substance": "clofazimine", "intended_use": "Treatment of nontuberculous mycobacterial lung disease", "status": "Positive", "designation_date": "2019-08-21", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-19-2200", "last_updated": "2023-03-13" }, { "eu_designation_number": "EU/3/19/2201", "medicine_name": "", "active_substance": "Poly(oxy-1,2-ethanediyl), alpha-hydro-omega-hydroxy-,15,15'-diester with N-acetyl-L-isoleucyl-L-cysteinyl-L-valyl-1-methyl-L-tryptophyl-L-glutaminyl-L-alpha-aspartyl-L-tryptophylglycyl-L-alanyl-L-histidyl-L-arginyl-L-cysteinyl-L-threonyl-2-[2-(2-aminoethoxy)ethoxy]acetyl-N6-carboxy-L-lysinamide cyclic (2.fwdarw.12)-(disulfide); where two identical synthetic peptide domains are covalently linked at the ends of the polyethylene glycol chain (pegcetacoplan)", "intended_use": "Treatment of C3 glomerulopathy", "status": "Positive", "designation_date": "2019-08-21", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-19-2201", "last_updated": "2021-02-03" }, { "eu_designation_number": "EU/3/19/2202", "medicine_name": "", "active_substance": "(16E)-14-methyl-20-oxa-5,7,14,26-tetraaza-tetracyclo[19.3.1.1(2,6).1(8,12)]heptacosa-1(25),2(26),3,5,8(27),9,11,16,21,23-decaene–citric acid", "intended_use": "Treatment of glioma", "status": "Positive", "designation_date": "2019-10-17", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-19-2202", "last_updated": "2020-08-27" }, { "eu_designation_number": "EU/3/19/2203", "medicine_name": "", "active_substance": "(S)-2-isobutyrylamino-pentanedioic acid 5-amide 1-{[(2S,5S,8S,11R,12S,15S,18S,21R)-2,8-bis-((S)-sec-butyl)-21-hydroxy-5-(4-hydroxy-benzyl)-15-isobutyl-4,11-dimethyl-3,6,9,13,16,22-hexaoxo-10-oxa-1,4,7,14,17-pentaaza-bicyclo[16.3.1]docos-12-yl]-amide}", "intended_use": "Treatment of Netherton syndrome", "status": "Withdrawn", "designation_date": "2019-10-17", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-19-2203", "last_updated": "2025-06-30" }, { "eu_designation_number": "EU/3/19/2204", "medicine_name": "", "active_substance": "18-(p-(131I)-iodophenyl)octadecyl phosphocholine", "intended_use": "Treatment of multiple myeloma", "status": "Positive", "designation_date": "2019-10-17", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-19-2204", "last_updated": "2021-05-11" }, { "eu_designation_number": "EU/3/19/2205", "medicine_name": "Rezurock", "active_substance": "2-(3-(4-(1H-Indazol-5-ylamino)quinazolin-2-yl)phenoxy)-N-isopropylacetamide-methane sulfonic acid salt", "intended_use": "Treatment of graft-versus-host disease", "status": "Positive", "designation_date": "2019-10-17", "related_ema_product_number": "EMEA/H/C/006421", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-19-2205", "last_updated": "2025-10-17" }, { "eu_designation_number": "EU/3/19/2206", "medicine_name": "", "active_substance": "2'-O-(2-methoxyethyl)-D-ribose antisense oligonucleotide targeting glial fibrillary acidic protein messenger ribonucleic acid", "intended_use": "Treatment of Alexander disease", "status": "Positive", "designation_date": "2019-10-17", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-19-2206", "last_updated": "" }, { "eu_designation_number": "EU/3/19/2207", "medicine_name": "", "active_substance": "4-oxo-4H-chromene-2-carboxylic acid (2-(2-4-(2-(6,7-dimethoxy-3,4-dihydro-1H-isoquinolin-2-yl)-ethyl)-phenyl-2H-tetrazol-5-yl)-4,5-dimethoxy-phenyl)-amide (encequidar)", "intended_use": "Treatment of soft tissue sarcoma", "status": "Positive", "designation_date": "2019-10-17", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-19-2207", "last_updated": "2021-09-16" }, { "eu_designation_number": "EU/3/19/2208", "medicine_name": "", "active_substance": "Combination of two adeno-associated viral vectors of serotype 8 containing the 5'- and the 3'- half coding sequences of human ABCA4 fused to inteins", "intended_use": "Treatment of Stargardt's disease", "status": "Positive", "designation_date": "2019-10-17", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-19-2208", "last_updated": "2022-11-07" }, { "eu_designation_number": "EU/3/19/2209", "medicine_name": "", "active_substance": "anti-neonatal Fc receptor human monoclonal antibody", "intended_use": "Prevention of haemolytic disease of the foetus and newborn", "status": "Positive", "designation_date": "2019-10-17", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-19-2209", "last_updated": "2021-05-05" }, { "eu_designation_number": "EU/3/19/2210", "medicine_name": "Casgevy", "active_substance": "autologous CD34+ haematopoietic stem cells with a CRISPR-edited erythroid enhancer region of the BCL11A gene", "intended_use": "Treatment of beta thalassaemia intermedia and major", "status": "Positive", "designation_date": "2019-10-17", "related_ema_product_number": "EMEA/H/C/005763", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-19-2210", "last_updated": "2022-01-25" }, { "eu_designation_number": "EU/3/19/2211", "medicine_name": "", "active_substance": "besilesomab", "intended_use": "Treatment in haematopoietic stem cell transplantation", "status": "Positive", "designation_date": "2019-10-17", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-19-2211", "last_updated": "2022-12-14" }, { "eu_designation_number": "EU/3/19/2212", "medicine_name": "", "active_substance": "leriglitazone", "intended_use": "Treatment of Friedreich’s ataxia", "status": "Positive", "designation_date": "2019-10-17", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-19-2212", "last_updated": "" }, { "eu_designation_number": "EU/3/19/2213", "medicine_name": "Skytrofa (previously Lonapegsomatropin Ascendis Pharma)", "active_substance": "lonapegsomatropin", "intended_use": "Treatment of growth hormone deficiency", "status": "Positive", "designation_date": "2019-10-17", "related_ema_product_number": "EMEA/H/C/005367", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-19-2213", "last_updated": "2022-02-17" }, { "eu_designation_number": "EU/3/19/2214", "medicine_name": "Ogsiveo", "active_substance": "nirogacestat", "intended_use": "Treatment of soft tissue sarcoma", "status": "Positive", "designation_date": "2019-10-17", "related_ema_product_number": "EMEA/H/C/006071", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-19-2214", "last_updated": "2024-04-08" }, { "eu_designation_number": "EU/3/19/2215", "medicine_name": "", "active_substance": "paclitaxel", "intended_use": "Treatment of soft tissue sarcoma", "status": "Positive", "designation_date": "2019-10-17", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-19-2215", "last_updated": "2021-09-16" }, { "eu_designation_number": "EU/3/19/2216", "medicine_name": "Pemazyre", "active_substance": "pemigatinib", "intended_use": "Treatment of myeloid/lymphoid neoplasms with eosinophilia and rearrangement of PDGFRA, PDGFRB, or FGFR1, or with PCM1-JAK", "status": "Positive", "designation_date": "2019-10-17", "related_ema_product_number": "EMEA/H/C/005266", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-19-2216", "last_updated": "2021-02-01" }, { "eu_designation_number": "EU/3/19/2217", "medicine_name": "", "active_substance": "propranolol hydrochloride", "intended_use": "Treatment of retinopathy of prematurity", "status": "Positive", "designation_date": "2019-10-17", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-19-2217", "last_updated": "" }, { "eu_designation_number": "EU/3/19/2218", "medicine_name": "", "active_substance": "", "intended_use": "Treatment of GM2 gangliosidosis", "status": "Positive", "designation_date": "2019-11-13", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-19-2218", "last_updated": "" }, { "eu_designation_number": "EU/3/19/2219", "medicine_name": "", "active_substance": "4-((E)-(5-(2-(2-((S)-2-((S)-1-(L-threonyl-L-lysyl)pyrrolidine-2-carboxamido)-5-guanidinopentanamido)acetamido)-2-carboxyethyl)-2-hydroxyphenyl)diazenyl)phenyl (2-(trimethylammonio)ethyl) phosphate", "intended_use": "Treatment of non-infectious uveitis", "status": "Positive", "designation_date": "2019-11-13", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-19-2219", "last_updated": "" }, { "eu_designation_number": "EU/3/19/2220", "medicine_name": "Tecartus", "active_substance": "autologous peripheral blood T cells CD4 and CD8 selected and CD3 and CD28 activated transduced with retroviral vector expressing anti CD19 CD28/CD3-zeta chimeric antigen receptor and cultured (brexucabtagene autoleucel)", "intended_use": "Treatment of mantle cell lymphoma", "status": "Positive", "designation_date": "2019-11-13", "related_ema_product_number": "EMEA/H/C/005102", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-19-2220", "last_updated": "2021-09-15" }, { "eu_designation_number": "EU/3/19/2221", "medicine_name": "", "active_substance": "camsirubicin", "intended_use": "Treatment of soft tissue sarcoma", "status": "Positive", "designation_date": "2019-11-13", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-19-2221", "last_updated": "" }, { "eu_designation_number": "EU/3/19/2222", "medicine_name": "", "active_substance": "chimeric fibril-reactive IgG1k monoclonal antibody 11-1F4", "intended_use": "Treatment of AL amyloidosis", "status": "Positive", "designation_date": "2019-11-13", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-19-2222", "last_updated": "2022-01-28" }, { "eu_designation_number": "EU/3/19/2223", "medicine_name": "", "active_substance": "Exendin (9-39)", "intended_use": "Treatment of congenital hyperinsulinism", "status": "Positive", "designation_date": "2019-11-13", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-19-2223", "last_updated": "" }, { "eu_designation_number": "EU/3/19/2224", "medicine_name": "Ztalmy", "active_substance": "ganaxolone", "intended_use": "Treatment of CDKL5 deficiency disorder", "status": "Positive", "designation_date": "2019-11-13", "related_ema_product_number": "EMEA/H/C/005825", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-19-2224", "last_updated": "2025-06-05" }, { "eu_designation_number": "EU/3/19/2225", "medicine_name": "", "active_substance": "replication-incompetent, non-integrating, herpes simplex virus 1 vector expressing the human transglutaminase-1 enzyme (Inetagugene geperpavec)", "intended_use": "Treatment of autosomal recessive congenital ichthyosis", "status": "Positive", "designation_date": "2019-11-13", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-19-2225", "last_updated": "2023-01-13" }, { "eu_designation_number": "EU/3/19/2226", "medicine_name": "", "active_substance": "(E)-2-((2S,4S)-4-(((2R,4S,5S,6S)-4-amino-5-hydroxy-6-methyltetrahydro-2H-pyran-2-yl)oxy)-2,5,12-trihydroxy-7-methoxy-6,11-dioxo-1,2,3,4,6,11-hexahydrotetracen-2-yl)-10-(carboxymethyl)-1-hydroxy-13-(2-(2-(2-((E)-3-(3-((3-hydroxy-3,3 diphosphonopropyl)(methyl)amino)propoxy)benzylidene)hydrazine-1-carbonothioyl)hydrazineyl)-2-oxoethyl)-8-oxo-5-thioxo-3,4,6,7,10,13-hexaazapentadec-2-en-15-oic acid", "intended_use": "Treatment of osteosarcoma", "status": "Positive", "designation_date": "2019-12-16", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-19-2226", "last_updated": "" }, { "eu_designation_number": "EU/3/19/2227", "medicine_name": "Romvimza", "active_substance": "2-(isopropylamino)-3-methyl-5-(6-methyl-5-((2-(1-methyl-1H-pyrazol-4-yl)pyridin-4-yl)oxy)pyridin-2-yl)pyrimidin-4(3H)-one", "intended_use": "Treatment of tenosynovial giant cell tumour, localised and diffuse type", "status": "Positive", "designation_date": "2019-12-16", "related_ema_product_number": "EMEA/H/C/006363", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-19-2227", "last_updated": "2021-08-25" }, { "eu_designation_number": "EU/3/19/2228", "medicine_name": "", "active_substance": "5,7-dichloro-2-((ethylamino)methyl)-8-hydroxy-3-methylquinazolin-4(3H)-one mesilate", "intended_use": "Treatment of multiple system atrophy", "status": "Positive", "designation_date": "2019-12-16", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-19-2228", "last_updated": "2020-10-27" }, { "eu_designation_number": "EU/3/19/2229", "medicine_name": "", "active_substance": "adeno-associated virus vector encoding human phenylalanine hydroxylase", "intended_use": "Treatment of phenylalanine hydroxylase deficiency", "status": "Withdrawn", "designation_date": "2019-12-16", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-19-2229", "last_updated": "" }, { "eu_designation_number": "EU/3/19/2230", "medicine_name": "", "active_substance": "efgartigimod alfa", "intended_use": "Treatment of immune thrombocytopenia", "status": "Positive", "designation_date": "2019-12-16", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-19-2230", "last_updated": "2021-09-02" }, { "eu_designation_number": "EU/3/19/2231", "medicine_name": "", "active_substance": "H-Leu-Pro-Pro-Leu-Pro-Tyr-Pro-OH", "intended_use": "Treatment of amyotrophic lateral sclerosis", "status": "Positive", "designation_date": "2019-12-16", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-19-2231", "last_updated": "" }, { "eu_designation_number": "EU/3/19/2232", "medicine_name": "", "active_substance": "Lactobacillus plantarum", "intended_use": "Treatment of amyotrophic lateral sclerosis", "status": "Withdrawn", "designation_date": "2019-12-16", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-19-2232", "last_updated": "2025-04-09" }, { "eu_designation_number": "EU/3/19/2233", "medicine_name": "", "active_substance": "navitoclax", "intended_use": "Treatment of myelofibrosis", "status": "Withdrawn", "designation_date": "2019-12-16", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-19-2233", "last_updated": "2026-05-26" }, { "eu_designation_number": "EU/3/19/2234", "medicine_name": "", "active_substance": "pamrevlumab", "intended_use": "Treatment of Duchenne muscular dystrophy", "status": "Withdrawn", "designation_date": "2019-12-16", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-19-2234", "last_updated": "2024-11-19" }, { "eu_designation_number": "EU/3/19/2235", "medicine_name": "", "active_substance": "synthetic double-stranded siRNA oligonucleotide directed against SERPINA1 mRNA and containing four modified nucleosides which form a ligand cluster of four N-acetylgalactosamine residues", "intended_use": "Treatment of congenital alpha-1 antitrypsin deficiency", "status": "Withdrawn", "designation_date": "2019-12-16", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-19-2235", "last_updated": "2023-02-24" }, { "eu_designation_number": "EU/3/19/2236", "medicine_name": "", "active_substance": "dimethyl fumarate", "intended_use": "Treatment of adrenoleukodystrophy", "status": "Positive", "designation_date": "2020-01-09", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-19-2236", "last_updated": "2022-11-11" }, { "eu_designation_number": "EU/3/19/2237", "medicine_name": "", "active_substance": "2-(3,7-Dimethyl-octa-2, 6-dienyl)-6-ethylamino-3-hydroxy-5-pentyl-[1,4]benzoquinone", "intended_use": "Treatment of Huntington's disease", "status": "Positive", "designation_date": "2020-01-09", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-19-2237", "last_updated": "" }, { "eu_designation_number": "EU/3/19/2238", "medicine_name": "", "active_substance": "adeno-associated virus serotype 9 vector containing human n-acetylgalactosamine-6-sulfate sulfatase gene", "intended_use": "Treatment of mucopolysaccharidosis type IVA (Morquio A Syndrome)", "status": "Positive", "designation_date": "2020-01-09", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-19-2238", "last_updated": "2022-07-08" }, { "eu_designation_number": "EU/3/19/2239", "medicine_name": "", "active_substance": "tripotassium citrate monohydrate;potassium hydrogen carbonate", "intended_use": "Treatment of cystinuria", "status": "Positive", "designation_date": "2020-01-09", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-19-2239", "last_updated": "2023-04-13" }, { "eu_designation_number": "EU/3/19/2240", "medicine_name": "", "active_substance": "nicardipine", "intended_use": "Treatment of non-traumatic subarachnoid haemorrhage", "status": "Positive", "designation_date": "2020-01-09", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-19-2240", "last_updated": "" }, { "eu_designation_number": "EU/3/19/2241", "medicine_name": "", "active_substance": "adeno-associated virus serotype 2/6 encoding human alpha-galactosidase A cDNA", "intended_use": "Treatment of Fabry disease", "status": "Positive", "designation_date": "2020-01-09", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-19-2241", "last_updated": "2021-08-04" }, { "eu_designation_number": "EU/3/19/2242", "medicine_name": "Casgevy", "active_substance": "autologous CD34+ hematopoietic stem cells with a CRISPR-edited erythroid enhancer region of the BCL11A gene", "intended_use": "Treatment of sickle cell disease", "status": "Positive", "designation_date": "2020-01-09", "related_ema_product_number": "EMEA/H/C/005763", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-19-2242", "last_updated": "2022-01-25" }, { "eu_designation_number": "EU/3/19/2243", "medicine_name": "", "active_substance": "motixafortide", "intended_use": "Treatment of pancreatic cancer", "status": "Positive", "designation_date": "2020-01-09", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-19-2243", "last_updated": "" }, { "eu_designation_number": "EU/3/19/2244", "medicine_name": "", "active_substance": "ziritaxestat", "intended_use": "Treatment of systemic 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"medicine_name": "", "active_substance": "pelabresib monohydrate", "intended_use": "Treatment of myelofibrosis", "status": "Positive", "designation_date": "2025-02-17", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-20-2247", "last_updated": "2025-03-12" }, { "eu_designation_number": "EU/3/20/2248", "medicine_name": "", "active_substance": "Adeno-associated viral vector serotype S3 encoding human alpha-galactosidase A cDNA", "intended_use": "Treatment of Fabry disease", "status": "Positive", "designation_date": "2020-02-28", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-20-2248", "last_updated": "" }, { "eu_designation_number": "EU/3/20/2249", "medicine_name": "", "active_substance": "adeno-associated virus serotype 8 containing the human RdCVF sequence and the human RdCVFL sequence", "intended_use": "Treatment of inherited retinal dystrophies", "status": 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"eu_designation_number": "EU/3/20/2256", "medicine_name": "", "active_substance": "reldesemtiv", "intended_use": "Treatment of amyotrophic lateral sclerosis", "status": "Positive", "designation_date": "2020-02-28", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-20-2256", "last_updated": "2024-10-11" }, { "eu_designation_number": "EU/3/20/2257", "medicine_name": "", "active_substance": "sintilimab", "intended_use": "Treatment of peripheral T-cell lymphoma", "status": "Positive", "designation_date": "2020-02-28", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-20-2257", "last_updated": "" }, { "eu_designation_number": "EU/3/20/2258", "medicine_name": "", "active_substance": "sutimlimab", "intended_use": "Treatment of immune thrombocytopenia", "status": "Withdrawn", "designation_date": "2020-02-28", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-20-2258", "last_updated": "2026-05-26" }, { "eu_designation_number": "EU/3/20/2259", "medicine_name": "", "active_substance": "2-hydroxy-N,N,N-trimethylethan-1-aminium (Z)-4-(5-((3-benzyl-4-oxo-2-thioxothiazolidin-5-ylidene)methyl)furan-2-yl)benzoate", "intended_use": "Treatment of pancreatic cancer", "status": "Withdrawn", "designation_date": "2020-03-24", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-20-2259", "last_updated": "2023-02-16" }, { "eu_designation_number": "EU/3/20/2260", "medicine_name": "", "active_substance": "allogeneic multi-virus specific T lymphocytes targeting BK virus, cytomegalovirus, human herpesvirus-6, Epstein Barr virus and adenovirus", "intended_use": "Treatment in haematopoietic stem cell transplantation", "status": "Positive", "designation_date": "2020-03-24", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-20-2260", "last_updated": "2023-06-08" }, { "eu_designation_number": "EU/3/20/2261", "medicine_name": "Scemblix", "active_substance": "asciminib", "intended_use": "Treatment of chronic myeloid leukaemia", "status": "Positive", "designation_date": "2020-03-24", "related_ema_product_number": "EMEA/H/C/005605", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-20-2261", "last_updated": "2022-09-07" }, { "eu_designation_number": "EU/3/20/2262", "medicine_name": "", "active_substance": "fosgemcitabine palabenamide", "intended_use": "Treatment of biliary tract cancer", "status": "Withdrawn", "designation_date": "2020-03-24", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-20-2262", "last_updated": "" }, { "eu_designation_number": "EU/3/20/2263", "medicine_name": "", "active_substance": "Losmapimod", "intended_use": "treatment of 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{ "eu_designation_number": "EU/3/20/2266", "medicine_name": "", "active_substance": "DNA plasmid encoding IL-12 p35 and p40 genes", "intended_use": "Treatment of ovarian cancer", "status": "Positive", "designation_date": "2020-04-22", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-20-2266", "last_updated": "" }, { "eu_designation_number": "EU/3/20/2267", "medicine_name": "", "active_substance": "adeno-associated viral vector serotype 3B encoding human multidrug resistance protein 3A", "intended_use": "Treatment of progressive familial intrahepatic cholestasis", "status": "Withdrawn", "designation_date": "2020-04-22", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-20-2267", "last_updated": "2025-06-10" }, { "eu_designation_number": "EU/3/20/2268", "medicine_name": "", "active_substance": "florbetaben (18F)", "intended_use": "Diagnosis of AL amyloidosis", 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"EU/3/20/2271", "medicine_name": "Zemcelpro", "active_substance": "haematopoietic stem cells and blood progenitors umbilical cord-derived expanded with (1R, 4R)-N1-(2-benzyl-7-(2-methyl-2H-tetrazol-5-yl)-9H-pyrimido[4,5-b]indol-4-yl)cyclohexane-1,4-diamine dihydrobromide dihydrate", "intended_use": "Treatment in haematopoietic stem cell transplantation", "status": "Positive", "designation_date": "2020-04-20", "related_ema_product_number": "EMEA/H/C/005772", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-20-2271", "last_updated": "2025-06-20" }, { "eu_designation_number": "EU/3/20/2272", "medicine_name": "Rystiggo", "active_substance": "rozanolixizumab", "intended_use": "Treatment of myasthenia gravis", "status": "Positive", "designation_date": "2020-04-22", "related_ema_product_number": "EMEA/H/C/005824", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-20-2272", "last_updated": "" }, { "eu_designation_number": "EU/3/20/2273", "medicine_name": "", "active_substance": "anti-(integrin beta-3) human monoclonal antibody", "intended_use": "Prevention of fetal and neonatal alloimmune thrombocytopenia due to human platelet antigen-1a incompatibility", "status": "Positive", "designation_date": "2020-04-22", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-20-2273", "last_updated": "" }, { "eu_designation_number": "EU/3/20/2274", "medicine_name": "", "active_substance": "adeno-associated virus vector serotype hu37 encoding human factor VIII", "intended_use": "Treatment of haemophilia A", "status": "Withdrawn", "designation_date": "2020-04-22", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-20-2274", "last_updated": "2026-05-26" }, { "eu_designation_number": "EU/3/20/2275", "medicine_name": "", "active_substance": "Glucagon analogue linked to a human immunoglobulin Fc fragment", "intended_use": "Treatment of insulin autoimmune syndrome", "status": "Positive", "designation_date": "2020-04-22", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-20-2275", "last_updated": "" }, { "eu_designation_number": "EU/3/20/2276", "medicine_name": "", "active_substance": "methotrexate", "intended_use": "Treatment of retinal detachment", "status": "Positive", "designation_date": "2020-06-04", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-20-2276", "last_updated": "" }, { "eu_designation_number": "EU/3/20/2277", "medicine_name": "", "active_substance": "autologous T lymphocyte-enriched population of cells transduced with a lentiviral vector encoding a chimeric antigen receptor targeting human B cell maturation antigen with 4-1BB and CD3-zeta intracellular signalling domains", "intended_use": "Treatment of multiple myeloma", "status": "Withdrawn", "designation_date": "2020-06-04", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-20-2277", "last_updated": "2025-04-04" }, { "eu_designation_number": "EU/3/20/2278", "medicine_name": "", "active_substance": "rilzabrutinib", "intended_use": "Treatment of immune thrombocytopenia", "status": "Positive", "designation_date": "2020-06-04", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-20-2278", "last_updated": "2023-07-18" }, { "eu_designation_number": "EU/3/20/2279", "medicine_name": "", "active_substance": "Lumacaftor", "intended_use": "Treatment of non-traumatic subarachnoid haemorrhage", "status": "Positive", "designation_date": "2020-06-04", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-20-2279", "last_updated": "" }, { "eu_designation_number": "EU/3/20/2280", "medicine_name": "", "active_substance": "Lutetium (177Lu) lilotomab satetraxetan", "intended_use": "Treatment of marginal zone lymphoma", "status": "Positive", "designation_date": "2020-06-04", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-20-2280", "last_updated": "" }, { "eu_designation_number": "EU/3/20/2281", "medicine_name": "Fabhalta", "active_substance": "(4-{(2S,4S)-4-ethoxy-1-[(5-methoxy-7-methyl-1H-indol-4-yl)methyl]piperidin-2-yl}benzoic acid-hydrogen chloride(1/1))", "intended_use": "Treatment of paroxysmal nocturnal haemoglobinuria", "status": "Positive", "designation_date": "2020-06-04", "related_ema_product_number": "EMEA/H/C/005764", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-20-2281", "last_updated": "" }, { "eu_designation_number": "EU/3/20/2282", "medicine_name": "", "active_substance": "viltolarsen", "intended_use": "Treatment of Duchenne muscular dystrophy", "status": "Positive", "designation_date": "2020-06-04", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-20-2282", "last_updated": "2023-06-01" }, { "eu_designation_number": "EU/3/20/2283", "medicine_name": "", "active_substance": "Ile-Ala-Leu-Ile-Leu-Glu-Pro-Ile-Cys-Cys-Gln-Glu-Arg-Ala-Ala-(discrete-polyethylene glycol)24", "intended_use": "Treatment of neonatal encephalopathy", "status": "Positive", "designation_date": "2020-06-04", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-20-2283", "last_updated": "" }, { "eu_designation_number": "EU/3/20/2284", "medicine_name": "Albrioza", "active_substance": "sodium phenylbutyrate;tauroursodeoxycholic acid", "intended_use": "Treatment of amyotrophic lateral sclerosis", "status": "Positive", "designation_date": "2020-06-04", "related_ema_product_number": "EMEA/H/C/005901", "url": 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"url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-20-2286", "last_updated": "2026-08-04" }, { "eu_designation_number": "EU/3/20/2287", "medicine_name": "", "active_substance": "adeno-associated virus serotype 9 containing the human ASPA gene", "intended_use": "Treatment of Canavan disease", "status": "Positive", "designation_date": "2020-06-04", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-20-2287", "last_updated": "2021-04-06" }, { "eu_designation_number": "EU/3/20/2288", "medicine_name": "", "active_substance": "Magrolimab", "intended_use": "Treatment of myelodysplastic syndromes", "status": "Withdrawn", "designation_date": "2020-06-26", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-20-2288", "last_updated": "2025-03-12" }, { "eu_designation_number": "EU/3/20/2289", "medicine_name": "", "active_substance": "nomacopan", "intended_use": "Treatment of bullous pemphigoid", "status": "Positive", "designation_date": "2020-06-26", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-20-2289", "last_updated": "2022-01-26" }, { "eu_designation_number": "EU/3/20/2290", "medicine_name": "", "active_substance": "stiripentol", "intended_use": "Treatment of primary hyperoxaluria", "status": "Positive", "designation_date": "2020-06-26", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-20-2290", "last_updated": "" }, { "eu_designation_number": "EU/3/20/2291", "medicine_name": "", "active_substance": "onfekafusp alfa", "intended_use": "Treatment of glioma", "status": "Positive", "designation_date": "2020-06-26", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-20-2291", "last_updated": "" }, { "eu_designation_number": "EU/3/20/2292", 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"EU/3/20/2311", "medicine_name": "", "active_substance": "sodium (4-{(E)-3-(4-fluorophenyl)-3-[4-(3-morpholin-4-yl-prop1ynyl)phenyl]allyloxy}-2-methylphenoxy)acetate", "intended_use": "Treatment of mitochondrial encephalomyopathy, lactic acidosis and stroke-like episodes", "status": "Withdrawn", "designation_date": "2020-08-21", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-20-2311", "last_updated": "2024-04-11" }, { "eu_designation_number": "EU/3/20/2312", "medicine_name": "", "active_substance": "allogeneic hepatoblastoma cells encapsulated in alginate, ex vivo expanded", "intended_use": "Treatment of acute liver failure", "status": "Positive", "designation_date": "2020-08-21", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-20-2312", "last_updated": "2021-06-24" }, { "eu_designation_number": "EU/3/20/2313", "medicine_name": "", "active_substance": "2-(2-(18F)fluoropyridin-4-yl)-9H-pyrrolo[2,3-b:4,5-c']dipyridine", "intended_use": "Diagnosis of progressive supranuclear palsy", "status": "Positive", "designation_date": "2020-08-21", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-20-2313", "last_updated": "2026-08-21" }, { "eu_designation_number": "EU/3/20/2314", "medicine_name": "", "active_substance": "Humanised IgG1 monoclonal antibody against human eotaxin-2;nebokitug", "intended_use": "Treatment of primary sclerosing cholangitis", "status": "Positive", "designation_date": "2020-08-21", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-20-2314", "last_updated": "2025-07-17" }, { "eu_designation_number": "EU/3/20/2315", "medicine_name": "", "active_substance": "pentosan polysulfate sodium", "intended_use": "Treatment of mucopolysaccharidosis VI (Maroteaux-Lamy syndrome)", "status": "Positive", 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"related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-20-2317", "last_updated": "" }, { "eu_designation_number": "EU/3/20/2318", "medicine_name": "", "active_substance": "dextran sulfate low molecular weight", "intended_use": "Treatment of amyotrophic lateral sclerosis", "status": "Positive", "designation_date": "2020-08-21", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-20-2318", "last_updated": "" }, { "eu_designation_number": "EU/3/20/2319", "medicine_name": "", "active_substance": "sodium (4-{(E)-3-(4-fluorophenyl)-3-[4-(3-morpholin-4-yl-prop1ynyl)phenyl]allyloxy}-2-methylphenoxy)acetate", "intended_use": "Treatment of long-chain 3-hydroxyacyl-coenzyme A dehydrogenase deficiency", "status": "Withdrawn", "designation_date": "2020-08-21", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-20-2319", "last_updated": "2024-04-11" }, { "eu_designation_number": "EU/3/20/2320", "medicine_name": "", "active_substance": "copper histidinate", "intended_use": "Treatment of Menkes disease", "status": "Positive", "designation_date": "2020-08-21", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-20-2320", "last_updated": "" }, { "eu_designation_number": "EU/3/20/2321", "medicine_name": "", "active_substance": "adeno-associated viral vector serotype 3B encoding shortened human ATP7B", "intended_use": "Treatment of Wilson's disease", "status": "Withdrawn", "designation_date": "2020-08-21", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-20-2321", "last_updated": "" }, { "eu_designation_number": "EU/3/20/2322", "medicine_name": "", "active_substance": "6-((3S,4S)-4-methyl-1-(pyrimidin-2-yl-methyl)pyrrolidin-3-yl)-3-tetrahydropyran-4-yl-7H-imidazo(1,5-a)pyrazin-8-one 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"related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-20-2324", "last_updated": "2024-04-09" }, { "eu_designation_number": "EU/3/20/2325", "medicine_name": "", "active_substance": "venglustat", "intended_use": "Treatment of GM2 gangliosidosis", "status": "Positive", "designation_date": "2020-08-21", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-20-2325", "last_updated": "2023-07-18" }, { "eu_designation_number": "EU/3/20/2326", "medicine_name": "", "active_substance": "autologous CD34+ cells transduced with a lentiviral vector encoding glucosylceramidase beta", "intended_use": "Treatment of Gaucher disease", "status": "Withdrawn", "designation_date": "2020-08-21", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-20-2326", "last_updated": "2024-04-09" }, { "eu_designation_number": "EU/3/20/2327", "medicine_name": "", "active_substance": "autologous T cells transduced with lentiviral vector containing a tandem chimeric antigen receptor directed against CD20 and CD19", "intended_use": "Treatment of diffuse large B-cell lymphoma", "status": "Positive", "designation_date": "2020-08-21", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-20-2327", "last_updated": "" }, { "eu_designation_number": "EU/3/20/2328", "medicine_name": "", "active_substance": "human frataxin fused to TAT cell-penetrating peptide", "intended_use": "Treatment of Friedreich’s ataxia", "status": "Positive", "designation_date": "2020-08-21", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-20-2328", "last_updated": "" }, { "eu_designation_number": "EU/3/20/2329", "medicine_name": "Febseltiq", "active_substance": "Infigratinib", "intended_use": "Treatment of cholangiocarcinoma", "status": "Withdrawn", "designation_date": "2020-08-21", "related_ema_product_number": "EMEA/H/C/005361", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-20-2329", "last_updated": "2022-11-08" }, { "eu_designation_number": "EU/3/20/2330", "medicine_name": "", "active_substance": "1-(3-methylbutanoyl)-L-aspartyl-L-threonyl-L-histidyl-L-phenylalanyl-L-prolyl-(L-cystinyl-L-isoleucyl-[(N6-(S)-4-carboxy-4-palmitamidobutanoyl)-L-lysinyl]-L-phenylalanyl-L-glutamyl-L-prolyl-L-arginyl-L-serinyl-L-lysinyl-L-glycinyl-L-cystinyl)-L-lysinamide, disulfide, acetate salt", "intended_use": "Treatment of polycythaemia vera", "status": "Positive", "designation_date": "2020-10-19", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-20-2330", "last_updated": "" }, { "eu_designation_number": "EU/3/20/2331", "medicine_name": "", "active_substance": "teclistamab", "intended_use": "Treatment of multiple myeloma", "status": "Withdrawn", "designation_date": "2022-07-19", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-20-2331", "last_updated": "2022-10-13" }, { "eu_designation_number": "EU/3/20/2332", "medicine_name": "", "active_substance": "retigabine", "intended_use": "Treatment of KCNQ2 developmental and epileptic encephalopathy", "status": "Positive", "designation_date": "2020-10-19", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-20-2332", "last_updated": "" }, { "eu_designation_number": "EU/3/20/2333", "medicine_name": "", "active_substance": "tipifarnib", "intended_use": "Treatment of peripheral T-cell lymphoma", "status": "Withdrawn", "designation_date": "2020-10-19", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-20-2333", "last_updated": "2026-05-18" }, { "eu_designation_number": "EU/3/20/2334", "medicine_name": "", "active_substance": "adeno-associated viral vector serotype 9 encoding a codon-optimised human aspartylglucosaminidase transgene", "intended_use": "Treatment of aspartylglucosaminuria", "status": "Withdrawn", "designation_date": "2020-10-19", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-20-2334", "last_updated": "2024-04-09" }, { "eu_designation_number": "EU/3/20/2335", "medicine_name": "", "active_substance": "(S)-1-(5-((2,3-dihydro-[1,4]dioxino[2,3-b]pyridin-7-yl)sulfonyl)-3,4,5,6-tetrahydropyrrolo[3,4-c]pyrrol-2(1H)-yl)-3-hydroxy-2-phenylpropan-1-one", "intended_use": "Treatment of sickle cell disease", "status": "Positive", "designation_date": "2020-10-19", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-20-2335", "last_updated": "2023-09-22" }, { "eu_designation_number": "EU/3/20/2336", "medicine_name": "", "active_substance": "(4-{(2S,4S)-4-ethoxy-1-[(5-methoxy-7-methyl-1H-indol-4-yl)methyl]piperidin-2-yl}benzoic acid-hydrogen chloride(1/1))", "intended_use": "Treatment of primary IgA nephropathy", "status": "Positive", "designation_date": "2020-10-19", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-20-2336", "last_updated": "" }, { "eu_designation_number": "EU/3/20/2337", "medicine_name": "", "active_substance": "ribitol", "intended_use": "Treatment of limb-girdle muscular dystrophy", "status": "Positive", "designation_date": "2020-10-19", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-20-2337", "last_updated": "2023-02-27" }, { "eu_designation_number": "EU/3/20/2338", "medicine_name": "", "active_substance": "decitabine;tetrahydrouridine", "intended_use": "Treatment of sickle cell disease", "status": "Positive", "designation_date": "2020-10-19", "related_ema_product_number": 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"medicine_name": "", "active_substance": "poly(oxy-1,2-ethanediyl), alpha-(carboxymethyl)-omega-methoxy-, amide with cystathionine γ-lyase [Pyridoxal 5’-phosphate cofactor] (synthetic engineered human), tetramer", "intended_use": "Treatment of homocystinuria", "status": "Positive", "designation_date": "2020-10-19", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-20-2348", "last_updated": "" }, { "eu_designation_number": "EU/3/20/2349", "medicine_name": "", "active_substance": "miglustat", "intended_use": "Treatment of neuronal ceroid lipofuscinosis", "status": "Positive", "designation_date": "2020-10-19", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-20-2349", "last_updated": "" }, { "eu_designation_number": "EU/3/20/2350", "medicine_name": "Yorvipath", "active_substance": "Poly(oxy-1,2-ethanediyl), alpha-hydro-omega-methoxy, ether with 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"2020-11-13", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-20-2354", "last_updated": "2024-04-08" }, { "eu_designation_number": "EU/3/20/2355", "medicine_name": "", "active_substance": "anti-(pancreatic adenocarcinoma upregulated factor) IgG1 humanised monoclonal antibody", "intended_use": "Treatment of pancreatic cancer", "status": "Positive", "designation_date": "2020-11-13", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-20-2355", "last_updated": "" }, { "eu_designation_number": "EU/3/20/2356", "medicine_name": "", "active_substance": "autologous CD34+ cells transduced ex vivo with a lentiviral vector containing a modified gamma-globin gene", "intended_use": "Treatment of sickle cell disease", "status": "Withdrawn", "designation_date": "2020-11-13", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-20-2356", "last_updated": "2022-09-16" }, { "eu_designation_number": "EU/3/20/2357", "medicine_name": "", "active_substance": "tislelizumab", "intended_use": "Treatment of oesophageal cancer", "status": "Withdrawn", "designation_date": "2020-11-13", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-20-2357", "last_updated": "2021-12-14" }, { "eu_designation_number": "EU/3/20/2358", "medicine_name": "", "active_substance": "L-pyroglutamyl-L-asparaginyl-L-prolyl-D-tyrosyl-D-tryptophan amide", "intended_use": "Treatment of amyotrophic lateral sclerosis", "status": "Positive", "designation_date": "2020-11-13", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-20-2358", "last_updated": "" }, { "eu_designation_number": "EU/3/20/2359", "medicine_name": "", "active_substance": "adeno-associated viral 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"https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-20-2361", "last_updated": "2021-09-16" }, { "eu_designation_number": "EU/3/20/2362", "medicine_name": "", "active_substance": "autologous bone marrow derived CD34+ cells transduced ex vivo with a self-inactivating lentiviral vector containing a normal version of the coding region of the IL2RG gene", "intended_use": "Treatment of X-linked severe combined immunodeficiency", "status": "Withdrawn", "designation_date": "2020-11-13", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-20-2362", "last_updated": "2024-11-19" }, { "eu_designation_number": "EU/3/20/2363", "medicine_name": "", "active_substance": "3,5-diamino-6-chloro-N-(N-(4-(4-(2-(hexyl((2S,3R,4R,5R)-2,3,4,5,6-pentahydroxyhexyl)amino)ethoxy)phenyl)butyl)-carbamimidoyl)pyrazine-2-carboxamide, Sodium chloride solution 4.2% (w/v)", "intended_use": "Treatment of primary ciliary dyskinesia", "status": "Positive", "designation_date": "2020-11-16", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-20-2363", "last_updated": "2023-03-07" }, { "eu_designation_number": "EU/3/20/2364", "medicine_name": "", "active_substance": "DNA plasmid encoding human transferrin gene", "intended_use": "Treatment of retinitis pigmentosa", "status": "Positive", "designation_date": "2020-11-13", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-20-2364", "last_updated": "" }, { "eu_designation_number": "EU/3/20/2365", "medicine_name": "", "active_substance": "adeno-associated virus serotype rh74 containing the human sarcoglycan beta gene", "intended_use": "Treatment of limb-girdle muscular dystrophy", "status": "Positive", "designation_date": "2020-12-09", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-20-2365", "last_updated": "" }, { "eu_designation_number": "EU/3/20/2366", "medicine_name": "", "active_substance": "autologous CD4+ and CD8+ T cells genetically modified with a lentiviral vector encoding a B-cell maturation antigen-specific chimeric antigen receptor", "intended_use": "Treatment of multiple myeloma", "status": "Withdrawn", "designation_date": "2020-12-09", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-20-2366", "last_updated": "" }, { "eu_designation_number": "EU/3/20/2367", "medicine_name": "", "active_substance": "(R)-3-(1-(2,3-dichloro-4-(pyrazin-2-yl)phenyl)-2,2,2-trifluoroethyl)-1-methyl-1-(1-methylpiperidin-4-yl)urea fumarate", "intended_use": "Treatment of Prader-Willi syndrome", "status": "Positive", "designation_date": "2020-12-09", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-20-2367", "last_updated": "" }, { "eu_designation_number": "EU/3/20/2368", "medicine_name": "", "active_substance": "human interleukin 12 fused with immunoglobulin G4 C-terminal Fc fragment", "intended_use": "Treatment of glioma", "status": "Positive", "designation_date": "2020-12-09", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-20-2368", "last_updated": "" }, { "eu_designation_number": "EU/3/20/2369", "medicine_name": "Winrevair", "active_substance": "sotatercept", "intended_use": "Treatment of pulmonary arterial hypertension", "status": "Positive", "designation_date": "2020-12-09", "related_ema_product_number": "EMEA/H/C/005647", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-20-2369", "last_updated": "2024-06-28" }, { "eu_designation_number": "EU/3/20/2370", "medicine_name": "", "active_substance": "tremelimumab", "intended_use": "Treatment of hepatocellular carcinoma", "status": "Withdrawn", "designation_date": "2020-12-09", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-20-2370", "last_updated": "2022-12-09" }, { "eu_designation_number": "EU/3/20/2371", "medicine_name": "", "active_substance": "aspacytarabine", "intended_use": "Treatment of acute myeloid leukaemia", "status": "Positive", "designation_date": "2020-12-09", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-20-2371", "last_updated": "" }, { "eu_designation_number": "EU/3/20/2372", "medicine_name": "Finlee", "active_substance": "dabrafenib mesilate", "intended_use": "Treatment of glioma", "status": "Positive", "designation_date": "2020-12-09", "related_ema_product_number": "EMEA/H/C/005885", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-20-2372", "last_updated": "2023-12-05" }, { "eu_designation_number": "EU/3/20/2373", "medicine_name": "", "active_substance": "Humanised IgG1 monoclonal antibody against the extracellular domain of receptor tyrosine kinase-like orphan receptor 1 coupled via a proteolytically cleavable maleimidocaproyl-valine-citrulline-para-aminobenzoate linker to monomethyl auristatin E", "intended_use": "Treatment of mantle cell lymphoma", "status": "Positive", "designation_date": "2020-12-09", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-20-2373", "last_updated": "" }, { "eu_designation_number": "EU/3/20/2374", "medicine_name": "Spexotras", "active_substance": "trametinib dimethyl sulfoxide", "intended_use": "Treatment of glioma", "status": "Positive", "designation_date": "2020-12-09", "related_ema_product_number": "EMEA/H/C/005886", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-20-2374", "last_updated": "" }, { "eu_designation_number": "EU/3/20/2375", "medicine_name": "", "active_substance": "2'-O-(2-methoxyethyl) phosphorothioate antisense oligonucleotide targeting CD49d RNA", "intended_use": "Treatment of Duchenne muscular dystrophy", "status": "Positive", "designation_date": "2020-12-09", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-20-2375", "last_updated": "" }, { "eu_designation_number": "EU/3/20/2376", "medicine_name": "", "active_substance": "4-[(3S)-3-aminopyrrolidin-1-yl]-6-cyano-5-(3,5-difluorophenyl)-N-[(2S)-1,1,1-trifluoropropan-2-yl]pyridine-3-carboxamide", "intended_use": "Treatment of congenital hyperinsulinism", "status": "Withdrawn", "designation_date": "2020-12-09", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-20-2376", "last_updated": "2026-05-18" }, { "eu_designation_number": "EU/3/20/2377", "medicine_name": "", "active_substance": "2’-O-(2-methoxyethyl) modified antisense oligonucleotide targeting glycogen synthase 1 pre‑mRNA", "intended_use": "Treatment of progressive myoclonic epilepsy type 2 (Lafora disease)", "status": "Positive", "designation_date": "2020-12-09", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-20-2377", "last_updated": "" }, { "eu_designation_number": "EU/3/20/2378", "medicine_name": "", "active_substance": "adeno-associated viral vector serotype 9 encoding human ATP7B", "intended_use": "Treatment of Wilson's disease", "status": "Positive", "designation_date": "2020-12-09", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-20-2378", "last_updated": "" }, { "eu_designation_number": "EU/3/20/2379", "medicine_name": "", "active_substance": "synthetic oligonucleotide selectively targeting UBE3A antisense RNA transcripts", "intended_use": "Treatment of Angelman syndrome", "status": "Positive", "designation_date": "2020-12-09", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-20-2379", "last_updated": "2025-06-10" }, { "eu_designation_number": "EU/3/20/2380", "medicine_name": "", "active_substance": "allogeneic retinal pigment epithelial cells genetically modified with a non-viral vector to express beta-domain deleted human factor VIII", "intended_use": "Treatment of haemophilia A", "status": "Withdrawn", "designation_date": "2020-12-09", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-20-2380", "last_updated": "2022-12-05" }, { "eu_designation_number": "EU/3/20/2381", "medicine_name": "", "active_substance": "(S)-N-(5-(4-(1-(benzo[d][1,3]dioxol-5-yl)ethyl)piperazin-1-yl)-1,3,4-thiadiazol-2-yl)acetamide, hydrochloride salt", "intended_use": "Treatment of progressive supranuclear palsy", "status": "Positive", "designation_date": "2020-12-09", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-20-2381", "last_updated": "2023-06-27" }, { "eu_designation_number": "EU/3/20/2382", "medicine_name": "", "active_substance": "erlotinib", "intended_use": "Treatment of patients with Olmsted syndrome", "status": "Positive", "designation_date": "2020-12-09", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-20-2382", "last_updated": "2024-10-02" }, { "eu_designation_number": "EU/3/20/2383", "medicine_name": "", "active_substance": "perflubron", "intended_use": "Treatment of respiratory distress syndrome", "status": "Withdrawn", "designation_date": "2020-12-09", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-20-2383", "last_updated": "2026-05-13" }, { "eu_designation_number": "EU/3/20/2384", "medicine_name": "", "active_substance": "sulindac", "intended_use": "Treatment of fragile X syndrome", 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"eu_designation_number": "EU/3/22/2618", "medicine_name": "", "active_substance": "Icerguastat acetate", "intended_use": "Treatment of amyotrophic lateral sclerosis", "status": "Positive", "designation_date": "2022-05-16", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-22-2618", "last_updated": "2026-08-21" }, { "eu_designation_number": "EU/3/22/2619", "medicine_name": "", "active_substance": "chimeric peptide of human glucagon-like peptide-1, glucagon and gastric inhibitory polypeptide analogues linked to a human immunoglobulin Fc fragment", "intended_use": "Treatment of idiopathic pulmonary fibrosis", "status": "Positive", "designation_date": "2022-05-16", "related_ema_product_number": "", "url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-22-2619", "last_updated": "" }, { "eu_designation_number": "EU/3/22/2620", "medicine_name": "", "active_substance": "pasireotide", "intended_use": 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