Every medicine the European Commission has designated as an orphan medicine for a rare disease, on the EMA's recommendation, with its current status.
| Substance | Intended use | Status | Designated |
|---|---|---|---|
| Humanized anti-KSA monoclonal antibody - human interleukin-2-fusion protein EU/3/02/097 |
Treatment of renal-cell carcinoma | Withdrawn | 22 Mar 2002 |
| TGF-ร2-specific phosphorothioate antisense oligodeoxynucleotide EU/3/02/091 |
Treatment of high-grade glioma | Active (positive) | 22 Mar 2002 |
| Iodine (131I) chimeric IgG monoclonal antibody cG250 EU/3/02/095 |
Treatment of renal-cell carcinoma | Withdrawn | 19 Mar 2002 |
| chimeric IgG monoclonal antibody cG250 (girentuximab) EU/3/02/094 |
Treatment of renal-cell carcinoma | Withdrawn | 19 Mar 2002 |
| Human transferrin conjugated to mutant diptheria toxin EU/3/02/090 |
Treatment of glioma | Withdrawn | 19 Mar 2002 |
| GM-CSF receptor antagonist EU/3/02/089 |
Treatment of juvenile myelomonocytic leukaemia | Withdrawn | 18 Mar 2002 |
| nitisinone EU/3/02/096 |
Treatment of alkaptonuria | Withdrawn | 13 Mar 2002 |
| beclomethasone 17, 21-dipropionate EU/3/02/093 · now authorised as orBec |
Treatment of intestinal graft-versus-host disease | Active (positive) | 13 Mar 2002 |
| 4-(3,5-Bis(hydroxy-phenyl)-1,2,4) triazol-1-yl) benzoic acid EU/3/02/092 |
Treatment of chronic iron overload requiring chelation therapy | Expired | 13 Mar 2002 |
| porfimer sodium EU/3/02/086 |
Treatment of high-grade dysplasia in Barrett's oesophagus | Withdrawn | 6 Mar 2002 |
| carmustine EU/3/02/085 |
Treatment of glioma | Withdrawn | 5 Mar 2002 |
| Colistimethate sodium EU/3/02/088 3 clinical trials → |
Treatment of Pseudomonas aeruginosa lung infection (including colonisation) in cystic fibrosis | Withdrawn | 19 Feb 2002 |
| eflornithine hydrochloride EU/3/02/087 6 clinical trials → |
Treatment of familial adenomatous polyposis (FAP) | Withdrawn | 19 Feb 2002 |
| azacitidine EU/3/01/084 127 clinical trials → |
Treatment of myelodysplastic syndromes | Expired | 6 Feb 2002 |
| adenovirus-mediated herpes-simplex-virus thymidine-kinase (HKSV-tk) gene EU/3/01/083 · now authorised as Cerepro; Cerepro; Cerepro |
Treatment of high-grade glioma with subsequent use of ganciclovir sodium | Active (positive) | 6 Feb 2002 |
| 2-Chloro-9-[2-deoxy-2-fluoro-ร-D-arabinofuranosyl]adenine EU/3/01/082 |
Treatment of acute lymphoblastic leukaemia | Expired | 5 Feb 2002 |
| Fumagillin EU/3/01/081 |
Treatment of diarrhoea associated with intestinal microsporidial infection | Withdrawn | 5 Feb 2002 |
| human engineered monoclonal antibody specific for transforming growth factor ร1 EU/3/01/080 |
Treatment of systemic sclerosis | Withdrawn | 4 Feb 2002 |
| sinapultide;Dipalmitoylphosphatidylcholine;palmitoyloleoyl phosphatidylglycerol;palmitic acid EU/3/01/079 |
Treatment of acute lung Injury | Active (positive) | 4 Feb 2002 |
| ibritumomab tiuxetan - |
Treatment of B-cell non-Hodgkin`s lymphoma | Negative | 1 Feb 2002 |
| thalidomide EU/3/01/068 12 clinical trials → |
Treatment of multiple myeloma | Withdrawn | 19 Dec 2001 |
| [Gly2]-recombinant human glucagon-like peptide EU/3/01/077 |
Treatment of short bowel syndrome | Active (positive) | 12 Dec 2001 |
| Iduronate-2-sulfatase EU/3/01/078 |
Treatment of mucopolysaccharidosis type II (Hunter's syndrome) | Expired | 11 Dec 2001 |
| Octavalent Pseudomonas aeruginosa O-polysaccharide-toxin A conjugate EU/3/01/076 |
Prevention of Pseudomonas aeruginosa infections in patients with cystic fibrosis | Withdrawn | 11 Dec 2001 |
| Denileukin diftitox EU/3/01/075 |
Treatment of cutaneous T-cell lymphoma | Active (positive) | 11 Dec 2001 |
| Halofuginone hydrobromide EU/3/01/074 |
Treatment of systemic sclerosis | Withdrawn | 11 Dec 2001 |
| stiripentol EU/3/01/071 |
Treatment of severe myoclonic epilepsy in infancy | Expired | 6 Dec 2001 |
| recombinant human monoclonal antibody to hsp90 EU/3/01/073 |
Treatment of invasive fungal infections | Withdrawn | 5 Dec 2001 |
| apomorphine EU/3/01/072 |
Treatment of off-periods in Parkinson's disease not responding to other oral treatment | Withdrawn | 5 Dec 2001 |
| celecoxib EU/3/01/070 23 clinical trials → |
Treatment of familial adenomatous polyposis (FAP) | Withdrawn | 21 Nov 2001 |
| Deoxyribose phosphorothioate (5'-tct-ccc-agc-gtg-cgc-cat-3') EU/3/01/063 |
Treatment of chronic lymphocytic leukaemia | Withdrawn | 21 Nov 2001 |
| phenylephrine hydrochloride EU/3/01/069 12 clinical trials → |
Treatment of ileal pouch anal anastomosis (IPAA) related faecal incontinence | Active (positive) | 20 Nov 2001 |
| thalidomide EU/3/01/067 12 clinical trials → |
Treatment of multiple myeloma | Withdrawn | 20 Nov 2001 |
| thalidomide EU/3/01/066 12 clinical trials → |
Treatment of erythema nodosum leprosum (ENL) or type II lepra reactions | Withdrawn | 20 Nov 2001 |
| Deoxyribose phosphorothioate (5'-tct-ccc-agc-gtg-cgc-cat-3') EU/3/01/065 |
Treatment of multiple myeloma | Withdrawn | 20 Nov 2001 |
| abetimus sodium EU/3/01/064 |
Treatment of lupus nephritis | Withdrawn | 20 Nov 2001 |
| idebenone EU/3/01/062 2 clinical trials → |
Treatment of Friedreich's ataxia | Active (positive) | 20 Nov 2001 |
| imatinib mesilate EU/3/01/061 32 clinical trials → |
Treatment of malignant gastrointestinal stromal tumours | Expired | 20 Nov 2001 |
| dexrazoxane EU/3/01/059 8 clinical trials → |
Treatment of anthracycline extravasations | Expired | 19 Sep 2001 |
| repertaxin L-lysine salt EU/3/01/058 |
Prevention of delayed graft function in organ transplant | Active (positive) | 19 Sep 2001 |
| Porcine lung surfactant EU/3/01/057 |
Treatment of acute lung Injury | Withdrawn | 19 Sep 2001 |
| sinapultide;Dipalmitoylphosphatidylcholine;palmitoyloleoyl phosphatidylglycerol;palmitic acid EU/3/01/054 |
Treatment of meconium aspiration syndrome | Withdrawn | 19 Sep 2001 |
| cladribine EU/3/01/055 7 clinical trials → |
Treatment of indolent non-Hodgkin's lymphoma | Expired | 18 Sep 2001 |
| beraprost sodium EU/3/01/053 |
Treatment of pulmonary arterial hypertension and chronic thromboembolic pulmonary hypertension | Withdrawn | 18 Sep 2001 |
| pemetrexed disodium EU/3/01/060 228 clinical trials → |
Treatment of malignant mesothelioma | Withdrawn | 17 Sep 2001 |
| seocalcitol EU/3/01/052 |
Treatment of hepatocellular carcinoma | Withdrawn | 31 Jul 2001 |
| 1,3-Propanedisulfonic acid, disodium salt EU/3/01/051 · now authorised as Kiacta; Kiacta |
Treatment of systemic secondary amyloidosis | Active (positive) | 31 Jul 2001 |
| zinc acetate dihydrate EU/3/01/050 |
Treatment of Wilson's disease | Expired | 31 Jul 2001 |
| ziconotide EU/3/01/048 2 clinical trials → |
Treatment of chronic pain requiring intraspinal analgesia | Expired | 9 Jul 2001 |
| thalidomide EU/3/01/047 12 clinical trials → |
Treatment of multiple myeloma | Withdrawn | 9 Jul 2001 |
| thalidomide EU/3/01/046 12 clinical trials → |
Treatment of graft-versus-host disease | Withdrawn | 9 Jul 2001 |
| Betaine anhydrous EU/3/01/045 |
Treatment of homocystinuria | Withdrawn | 9 Jul 2001 |
| human alpha1-proteinase inhibitor EU/3/01/044 |
Treatment of emphysema secondary to congenital alpha-1 antitrypsin deficiency | Active (positive) | 9 Jul 2001 |
| human Milk Fat Globule 1;yttrium (90Y) human milk fat globule 1 - S p isothiocyanatobenzyl-diethylenetriaminepentaacetic acid EU/3/01/043 |
Treatment of ovarian cancer | Withdrawn | 30 May 2001 |
| human engineered monoclonal antibody specific for transforming growth factor ฮฒ2 EU/3/01/042 |
Prevention of scarring in glaucoma filtration surgical procedures | Withdrawn | 30 May 2001 |
| recombinant human alpha-1 antitrypsin EU/3/01/041 |
Treatment of emphysema secondary to congenital alpha-1 antitrypsin deficiency | Withdrawn | 30 May 2001 |
| Fomepizole EU/3/01/040 |
Treatment of methanol poisoning | Withdrawn | 30 May 2001 |
| Ecteinascidin 743 (trabectedin) EU/3/01/039 |
Treatment of soft tissue sarcoma | Expired | 30 May 2001 |
| retroviral gamma c cDNA containing vector EU/3/01/038 |
Treatment of severe combined immunodeficiency (SCID)-Xl Disease | Active (positive) | 30 May 2001 |
| recombinant human C1-inhibitor EU/3/01/036 |
Treatment of angioedema caused by C1 inhibitor deficiency | Withdrawn | 11 May 2001 |
| Humanised anti-HM1.24 monoclonal antibody EU/3/01/037 |
Treatment of multiple myeloma | Withdrawn | 10 May 2001 |
| levodopa;carbidopa EU/3/01/035 2 clinical trials → |
Treatment of advanced idiopathic Parkinson's disease with severe motor fluctuations | Withdrawn | 10 May 2001 |
| gusperimus trihydrochloride EU/3/01/034 · now authorised as Spanidin; Spanidin |
Treatment of Wegener's granulomatosis | Active (positive) | 29 Mar 2001 |
| ranpirnase EU/3/01/033 |
Treatment of malignant mesothelioma | Withdrawn | 29 Mar 2001 |
| arsenic trioxide EU/3/01/032 11 clinical trials → |
Treatment of multiple myeloma | Withdrawn | 29 Mar 2001 |
| arsenic trioxide EU/3/01/031 11 clinical trials → |
Treatment of myelodysplastic syndromes | Withdrawn | 29 Mar 2001 |
| 8-cyclopentyl-1, 3-dipropylxanthine EU/3/01/030 |
Treatment of cystic fibrosis | Withdrawn | 29 Mar 2001 |
| ribavirin EU/3/01/029 |
Treatment of adenovirus infection in immunocompromised patients | Withdrawn | 8 Mar 2001 |
| Ibuprofen EU/3/01/027 22 clinical trials → |
Patent ductus arteriosus in premature neonates of less than 34 weeks of gestational age | Withdrawn | 6 Mar 2001 |
| Inolimomab EU/3/01/028 |
Treatment of graft-versus-host disease | Active (positive) | 5 Mar 2001 |
| N-acetylgalactosamine 4-sulfatase EU/3/01/025 |
Treatment of mucopolysaccharidosis VI (Maroteaux-Lamy syndrome) | Expired | 15 Feb 2001 |
| pegvisomant EU/3/01/023 |
Treatment of acromegaly | Expired | 15 Feb 2001 |
| laronidase EU/3/01/022 |
Treatment of mucopolysaccharidosis type I | Expired | 15 Feb 2001 |
| imatinib mesilate EU/3/01/021 32 clinical trials → |
Treatment of chronic myeloid leukaemia | Expired | 15 Feb 2001 |
| Ibuprofen EU/3/01/020 22 clinical trials → |
Treatment of patent ductus arteriosus | Expired | 15 Feb 2001 |
| Bosentan EU/3/01/019 3 clinical trials → |
Treatment of pulmonary arterial hypertension and chronic thromboembolic pulmonary hypertension | Expired | 15 Feb 2001 |
| recombinant human acid alpha-glucosidase EU/3/01/018 |
Treatment of glycogen storage disease type II (Pompe's disease) | Expired | 15 Feb 2001 |
| L-Lysine-N-Acetyl-L-Cysteinate EU/3/01/026 |
Treatment of cystic fibrosis | Active (positive) | 14 Feb 2001 |
| ribavirin EU/3/01/024 |
Treatment of haemorrhagic fever with renal syndrome | Withdrawn | 14 Feb 2001 |
| arsenic trioxide EU/3/00/017 11 clinical trials → |
Treatment of acute promyelocytic leukaemia | Withdrawn | 17 Jan 2001 |
| Lusupultide EU/3/00/016 |
Treatment of acute respiratory distress syndrome (ARDS) | Withdrawn | 17 Jan 2001 |
| xaliproden hydrochloride EU/3/00/015 |
Treatment of amyotrophic lateral sclerosis | Withdrawn | 17 Jan 2001 |
| iloprost EU/3/00/014 4 clinical trials → |
Treatment of primary and of the following forms of secondary pulmonary hypertensionโฆ | Expired | 30 Dec 2000 |
| nitisinone EU/3/00/012 |
Treatment of tyrosinaemia type 1 | Expired | 30 Dec 2000 |
| busulfan EU/3/00/011 22 clinical trials → |
Conditioning treatment prior to haematopoietic-progenitor-cell transplantation | Expired | 30 Dec 2000 |
| anagrelide hydrochloride EU/3/00/010 2 clinical trials → |
Treatment of essential thrombocythaemia | Expired | 30 Dec 2000 |
| Ethyl Eicosapentaenoate EU/3/00/013 · now authorised as Ethyl Eicosapent soft gelatin capsules; Ethyl Eicosapent soft gelatin capsules |
Treatment of Huntingtonโs disease | Active (positive) | 29 Dec 2000 |
| thalidomide EU/3/00/009 12 clinical trials → |
Treatment of erythema nodosum leprosum (ENL) or type II lepra reactions | Withdrawn | 29 Dec 2000 |
| arsenic trioxide EU/3/00/008 11 clinical trials → |
Treatment of acute promyelocytic leukaemia | Expired | 18 Oct 2000 |
| N-carbamyl-L-glutamic acid EU/3/00/007 |
Treatment of N-acetylglutamate synthetase (NAGS) deficiency | Expired | 18 Oct 2000 |
| 1,5-(Butylimino)-1,5-dideoxy, D-glucitol EU/3/00/006 |
Treatment of Gaucher disease | Expired | 18 Oct 2000 |
| gemtuzumab ozogamicin EU/3/00/005 · now authorised as Mylotarg; Mylotarg 7 clinical trials → |
Treatment of acute myeloid leukaemia | Active (positive) | 18 Oct 2000 |
| Fluorouracil EU/3/00/004 94 clinical trials → |
Treatment of glioblastoma | Withdrawn | 18 Oct 2000 |
| alpha-galactosidase A EU/3/00/003 |
Treatment of Fabry disease | Expired | 8 Aug 2000 |
| alpha-galactosidase A EU/3/00/002 |
Treatment of Fabry disease | Expired | 8 Aug 2000 |
| somatropin EU/3/00/001 5 clinical trials → |
AIDS wasting | Withdrawn | 8 Aug 2000 |
| rinatabart sesutecan EU/3/26/3206 |
Treatment of ovarian cancer | Active (positive) | |
| 1-{[2-(3,6-Diazabicyclo[3.1.1]heptan-3-yl)-7-(1,3-thiazol-2-yl)-1,3-benzoxazol-4-yl]oxy}-1,1-difluoro-2-methylpropan-2-ol EU/3/26/3202 |
Treatment of palmoplantar pustulosis | Active (positive) | |
| orziloben EU/3/26/3198 |
Treatment of intestinal failure-associated liver disease | Active (positive) | |
| adeno-associated viral vector serotype ShH10 containing a codon-optimised human NDP gene EU/3/25/3133 |
Treatment of NDP gene-related disorders | Active (positive) |
Every row is copied from the EMA's orphan designations data, reproduced with acknowledgement of the source. Each substance links to its official EMA page.
A daily job downloads the EMA's orphan designations JSON report and lists every designation, newest first. No AI drafting and no editorial pass. See our editorial standards. The the EMA data on this page was last retrieved on 27 Sep 2026.