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 |
|---|---|---|---|
| N-(cyanomethyl)-4-(2-{[4-(morpholin-4-yl)phenyl]amino}pyrimidin-4-yl)benzamide, dihydrochloride salt (momelotinib) EU/3/11/887 · now authorised as Omjjara |
Treatment of post-essential thrombocythaemia myelofibrosis | Active (positive) | 5 Aug 2011 |
| N-(cyanomethyl)-4-(2-{[4-(morpholin-4-yl)phenyl]amino}pyrimidin-4-yl)benzamide, dihydrochloride salt (momelotinib) EU/3/11/886 · now authorised as Omjjara |
Treatment of post-polycythaemia vera myelofibrosis | Active (positive) | 5 Aug 2011 |
| mixture of seven synthetic fragments consisting of p21 RAS peptides EU/3/11/885 |
Treatment of pancreatic cancer | Active (positive) | 5 Aug 2011 |
| 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-ฮฑ-aspaโฆ EU/3/11/884 |
Prevention of ischaemia/reperfusion injury associated with solid organ transplantation | Active (positive) | 5 Aug 2011 |
| dextran sulfate EU/3/11/883 |
Treatment for mobilisation of progenitor cells prior to stem cell transplantation | Active (positive) | 5 Aug 2011 |
| Fresolimumab EU/3/11/882 |
Treatment of focal segmental glomerulosclerosis | Withdrawn | 5 Aug 2011 |
| acadesine EU/3/11/881 |
Treatment of multiple myeloma | Active (positive) | 5 Aug 2011 |
| genetically modified human adenovirus encoding human PH20 hyaluronidase EU/3/11/880 |
Treatment of pancreatic cancer | Active (positive) | 21 Jun 2011 |
| chimeric monoclonal antibody against GD2ย (dinutuximab) EU/3/11/879 |
Treatment of neuroblastoma | Withdrawn | 21 Jun 2011 |
| allogeneic T cells encoding an exogenous TK gene EU/3/11/878) |
Treatment of acute lymphoblastic leukaemia | Active (positive) | 21 Jun 2011 |
| adeno-associated viral vector serotype 9 containing the human sulfamidase gene EU/3/11/877 |
Treatment of mucopolysaccharidosis, type IIIA (Sanfilippo A syndrome) | Expired | 21 Jun 2011 |
| viral vector containing DNA encoding the human SMN protein EU/3/11/876 |
Treatment of 5q spinal muscular atrophy | Active (positive) | 21 Jun 2011 |
| Metronidazole EU/3/11/875 16 clinical trials → |
Treatment of pouchitis | Active (positive) | 21 Jun 2011 |
| human embryonic stem-cell-derived retinal pigment epithelial cells EU/3/11/874 |
Treatment of Stargardt's disease | Withdrawn | 21 Jun 2011 |
| human dermal fibroblasts cultured on a bioresorbable polyglactin mesh EU/3/11/873 |
Treatment of epidermolysis bullosa | Withdrawn | 21 Jun 2011 |
| sulfonated monophosphorylated mannose oligosaccharide EU/3/11/872 |
Treatment of hepatocellular carcinoma | Withdrawn | 21 Jun 2011 |
| salirasib EU/3/11/871 |
Treatment of pancreatic cancer | Withdrawn | 21 Jun 2011 |
| S-Nitrosoglutathione EU/3/11/870 |
Treatment of pre-eclampsia | Active (positive) | 13 May 2011 |
| Lisuride hydrogen maleate EU/3/11/869 |
Treatment of pulmonary arterial hypertension and chronic thromboembolic pulmonary hypertension | Active (positive) | 13 May 2011 |
| lenalidomide EU/3/11/868 167 clinical trials → |
Treatment of diffuse large B-cell lymphoma | Withdrawn | 13 May 2011 |
| allogeneic umbilical cord blood cells treated ex vivo with 16,16-dimethyl prostaglandin E2 EU/3/11/867 |
Treatment of acute myeloid leukaemia | Withdrawn | 13 May 2011 |
| allogeneic bone marrow stem cells treated ex vivo with 16,16-dimethyl prostaglandin E2 EU/3/11/866 |
Treatment of acute myeloid leukaemia | Withdrawn | 13 May 2011 |
| 9-cis-Retinyl acetate EU/3/11/865 |
Treatment of retinitis pigmentosa | Active (positive) | 13 May 2011 |
| adeno-associated viral vector containing the human ARSB gene EU/3/11/864 |
Treatment of mucopolysaccharidosis VI (Maroteaux-Lamy syndrome) | Active (positive) | 13 May 2011 |
| recombinant fusion protein linking human coagulation factor VIIa with human albumin EU/3/11/863 |
Treatment of haemophilia B | Active (positive) | 13 May 2011 |
| apomorphine hydrochloride EU/3/11/862 |
Treatment of moderate and severe traumatic brain injury | Active (positive) | 13 May 2011 |
| 9-cis-Retinyl acetate EU/3/11/861 |
Treatment of Leber's congenital amaurosis | Active (positive) | 13 May 2011 |
| adeno-associated viral vector containing the human NADH-dehydrogenase-4 gene EU/3/11/860 · now authorised as Lumevoq |
Treatment of Leber's hereditary optic neuropathy | Active (positive) | 13 May 2011 |
| [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'โฆ EU/3/11/859 |
Treatment of chondrosarcoma | Withdrawn | 13 May 2011 |
| R-baclofen EU/3/11/858 |
Treatment of fragile X syndrome | Active (positive) | 15 Apr 2011 |
| recombinant thymidine phosphorylase encapsulated in autologous erythrocytes EU/3/11/856 |
Treatment of mitochondrial neurogastrointestinal encephalomyopathy (MNGIE) | Active (positive) | 15 Apr 2011 |
| recombinant fusion protein linking human coagulation factor VIIa with human albumin EU/3/11/855 |
Treatment of haemophilia A | Active (positive) | 15 Apr 2011 |
| vorinostat EU/3/11/854 5 clinical trials → |
Treatment of multiple myeloma | Withdrawn | 15 Apr 2011 |
| ombrabulin EU/3/11/853 |
Treatment of soft tissue sarcoma | Withdrawn | 15 Apr 2011 |
| human anthrax monoclonal antibody EU/3/11/852 |
Treatment of inhalation anthrax disease | Withdrawn | 15 Apr 2011 |
| Glufosfamide EU/3/11/851 |
Treatment of pancreatic cancer | Active (positive) | 15 Apr 2011 |
| Darinaparsin EU/3/11/850 |
Treatment of peripheral T-cell lymphoma (nodal, other extranodal and leukaemic/disseminated) | Active (positive) | 15 Apr 2011 |
| (S)-{8-Fluoro-2-2[4-(3-methoxyphenyl)-1-piperazinyl]-3-[2-methoxy-5-(trifluoromethyl)-phenyl]-3, 4-dihydro-4-quinazolinyl} acetic acidย (letermovir) EU/3/11/849 · now authorised as Prevymis |
Prevention of cytomegalovirus disease in patients with impaired cell-mediated immunity | Active (positive) | 15 Apr 2011 |
| sodium thiosulfate EU/3/10/848 5 clinical trials → |
Treatment of calciphylaxis | Withdrawn | 23 Feb 2011 |
| pegylated B-domain-deleted sequence-modified recombinant human factor VIII (damoctocog alfa pegol) EU/3/10/847 |
Treatment of haemophilia A | Withdrawn | 23 Feb 2011 |
| paclitaxel EU/3/10/846 400 clinical trials → |
Treatment of oesophagus carcinoma | Active (positive) | 23 Feb 2011 |
| dry extract from birch bark (DER 5-10 : 1), extraction solvent n-heptane 95% (w/w) (birch bark extract) EU/3/10/845 · now authorised as Filsuvez |
Treatment of epidermolysis bullosa | Active (positive) | 23 Feb 2011 |
| axitinib EU/3/10/844 22 clinical trials → |
Treatment of renal-cell carcinoma | Withdrawn | 23 Feb 2011 |
| allogeneic aortic endothelial cells cultured in a porcine gelatin matrix EU/3/10/843 |
Prevention of arteriovenous access failure in haemodialysis patients | Withdrawn | 23 Feb 2011 |
| nimorazole EU/3/10/842 |
Treatment of squamous-cell carcinoma of the head and neck in patients undergoing radiotherapy | Withdrawn | 23 Feb 2011 |
| tasimelteon EU/3/10/841 3 clinical trials → |
Treatment of non-24-hour sleep-wake disorders in blind people with no light perception | Expired | 23 Feb 2011 |
| recombinant human histone H1.3 and recombinant human N-bis-met-histone H1.3 EU/3/10/840 |
Treatment of acute lymphoblastic leukaemia | Active (positive) | 23 Feb 2011 |
| plitidepsin EU/3/10/839 |
Treatment of post-polycythaemia vera myelofibrosis | Withdrawn | 23 Feb 2011 |
| plitidepsin EU/3/10/838 |
Treatment of post-essential thrombocythaemia myelofibrosis | Withdrawn | 23 Feb 2011 |
| plitidepsin EU/3/10/837 |
Treatment of primary myelofibrosis | Withdrawn | 23 Feb 2011 |
| paquinimod EU/3/10/836 |
Treatment of systemic sclerosis | Active (positive) | 23 Feb 2011 |
| Maytansinoid-conjugated humanised monoclonal antibody against CD56 EU/3/10/835 |
Treatment of multiple myeloma | Withdrawn | 23 Feb 2011 |
| Human plasmin EU/3/10/834 |
Treatment of acute peripheral arterial occlusion | Withdrawn | 23 Feb 2011 |
| doxorubicin hydrochloride EU/3/10/833 126 clinical trials → |
Treatment of hepatocellular carcinoma | Active (positive) | 23 Feb 2011 |
| Deferiprone EU/3/10/832 |
Treatment of sickle cell disease | Active (positive) | 23 Feb 2011 |
| autologous tumour-derived immunoglobulin idiotype coupled to keyhole limpet hemocyanin EU/3/10/831 |
Treatment of mantle cell lymphoma | Active (positive) | 23 Feb 2011 |
| nabilone - 2 clinical trials → |
Treatment of amyotrophic lateral sclerosis | Negative | 16 Jan 2011 |
| veliparib EU/3/10/830 3 clinical trials → |
Treatment of ovarian cancer | Withdrawn | 17 Dec 2010 |
| tesetaxel EU/3/10/829 |
Treatment of gastric cancer | Active (positive) | 17 Dec 2010 |
| silibinin-C-2',3-dihydrogensuccinate, disodium salt EU/3/10/828 |
Prevention of recurrent hepatitis C in liver transplant recipients | Active (positive) | 17 Dec 2010 |
| recombinant human lysosomal acid lipase EU/3/10/827 · now authorised as Kanuma |
Treatment of lysosomal acid lipase deficiency | Active (positive) | 17 Dec 2010 |
| para-aminosalicylic acid EU/3/10/826 |
Treatment of tuberculosis | Active (positive) | 17 Dec 2010 |
| ovine anti-colchicine polyclonal antibody fragments EU/3/10/825 |
Treatment of colchicine poisoning | Withdrawn | 17 Dec 2010 |
| N-[(2S)-2,3-dihydroxypropyl]-3-[(2-fluoro-4-iodophenyl)amino]isonicotinamide hydrochloride EU/3/10/824 |
Treatment of acute myeloid leukaemia | Withdrawn | 17 Dec 2010 |
| Lomitapide EU/3/10/823 |
Treatment of familial chylomicronaemia | Withdrawn | 17 Dec 2010 |
| Lentiviral vector carrying the Fanconi anaemia-A (FANCA) gene EU/3/10/822 · now authorised as Fanskya |
Treatment of Fanconi anaemia type A | Active (positive) | 17 Dec 2010 |
| human heterologous liver cells EU/3/10/821 · now authorised as Heparesc |
Treatment of carbamoyl-phosphate synthase-1 deficiency | Active (positive) | 17 Dec 2010 |
| human heterologous liver cells EU/3/10/820 · now authorised as Heparesc |
Treatment of argininosuccinic aciduria | Active (positive) | 17 Dec 2010 |
| human heterologous liver cells EU/3/10/819 · now authorised as Heparesc |
Treatment of hyperargininaemia | Active (positive) | 17 Dec 2010 |
| adeno-associated viral vector containing DNA encoding an RNAi targeting rhodopsin;adeno-associated viral vector containing a rhodopsin gene EU/3/10/817 |
Treatment of rhodopsin-linked retinitis pigmentosa | Withdrawn | 17 Dec 2010 |
| 7-beta-hydroxy cholesteryl-3-beta-oleate EU/3/10/816 |
Treatment of glioma | Active (positive) | 17 Dec 2010 |
| sildenafil citrate EU/3/10/815 13 clinical trials → |
treatment of postcardiotomy right ventricular failure | Active (positive) | 26 Nov 2010 |
| recombinant human von Willebrand factor EU/3/10/814 |
Treatment of von Willebrand disease | Withdrawn | 26 Nov 2010 |
| recombinant human arylsulfatase A EU/3/10/813 |
treatment of metachromatic leukodystrophy | Active (positive) | 26 Nov 2010 |
| recombinant fusion protein consisting of the extracellular portion of human activin receptor IIB linked to the human IgG1 Fc domain EU/3/10/812 |
Treatment of Duchenne muscular dystrophy | Withdrawn | 26 Nov 2010 |
| N-tert-butyl-3-[(5-methyl-2-{[4-(2-pyrrolidin-1-ylethoxy)phenyl]amino}pyrimidin-4-yl)amino] benzenesulfonamide dihydrochloride monohydrate (fedratinib) EU/3/10/811 · now authorised as Inrebic |
Treatment of post-polycythaemia vera myelofibrosis | Active (positive) | 26 Nov 2010 |
| N-tert-butyl-3-[(5-methyl-2-{[4-(2-pyrrolidin-1-ylethoxy)phenyl]amino}pyrimidin-4-yl)amino] benzenesulfonamide dihydrochloride monohydrate (fedratinib) EU/3/10/810 · now authorised as Inrebic |
Treatment of post-essential thrombocythaemia myelofibrosis | Active (positive) | 26 Nov 2010 |
| nanoparticle albumin-bound paclitaxel EU/3/10/809 2 clinical trials → |
Treatment of pancreatic cancer | Withdrawn | 26 Nov 2010 |
| murine monoclonal antibody against CD26 EU/3/10/808 · now authorised as Begedina |
Treatment of graft-versus-host disease | Active (positive) | 26 Nov 2010 |
| Methylthioninium EU/3/10/807 |
treatment of frontotemporal dementia with parkinsonism-17 | Active (positive) | 26 Nov 2010 |
| Methylthioninium EU/3/10/806 |
treatment of progressive non-fluent aphasia | Active (positive) | 26 Nov 2010 |
| Methylthioninium EU/3/10/805 |
treatment of behavioural variant frontotemporal dementia | Active (positive) | 26 Nov 2010 |
| Methylthioninium EU/3/10/804 |
Treatment of progressive supranuclear palsy | Active (positive) | 26 Nov 2010 |
| chimeric monoclonal antibody against claudin-18 splice variant 2 EU/3/10/803 · now authorised as Vyloy |
Treatment of gastric cancer | Active (positive) | 26 Nov 2010 |
| 2-(2-chlorophenyl)-4-[3-(dimethylamino)phenyl]-5-methyl-1H-pyrazolo[4,3-C]pyridine-3,6(2H,5H)-dione EU/3/10/802 |
Treatment of idiopathic pulmonary fibrosis | Active (positive) | 26 Nov 2010 |
| tecovirimat EU/3/10/799 2 clinical trials → |
Treatment of cowpox infection | Withdrawn | 1 Oct 2010 |
| synthetic double-stranded short interfering RNA oligonucleotide directed against proopiomelanocortin EU/3/10/798 |
Treatment of adrenocorticotropin-dependent Cushing's syndrome | Active (positive) | 1 Oct 2010 |
| recombinant humanised monoclonal antibody to human Nogo-A protein of the IgG1/kappa class EU/3/10/797 |
Treatment of amyotrophic lateral sclerosis | Withdrawn | 1 Oct 2010 |
| recombinant humanised anti-human interleukin-1 beta monoclonal antibody EU/3/10/796 |
Treatment of Behรงet's Disease | Withdrawn | 1 Oct 2010 |
| pralatrexate EU/3/10/795 3 clinical trials → |
Treatment of Hodgkin's lymphoma | Active (positive) | 1 Oct 2010 |
| N-tert-butyl-3-[(5-methyl-2-{[4-(2-pyrrolidin-1-ylethoxy)phenyl]amino}pyrimidin-4-yl)amino] benzenesulfonamide dihydrochloride monohydrate (fedratinib) EU/3/10/794 · now authorised as Inrebic |
Treatment of primary myelofibrosis | Active (positive) | 1 Oct 2010 |
| N-(6-(2-aminophenylamino)-6-oxohexyl)-4-methylbenzamide EU/3/10/793 |
Treatment of Friedreich's ataxia | Active (positive) | 1 Oct 2010 |
| Maytansinoid-conjugated humanised monoclonal antibody against CD56 EU/3/10/792 |
Treatment of small cell lung cancer | Withdrawn | 1 Oct 2010 |
| ciclosporin EU/3/10/791 3 clinical trials → |
Treatment of moderate and severe closed traumatic brain injury | Active (positive) | 1 Oct 2010 |
| ambrisentan EU/3/10/790 3 clinical trials → |
Treatment idiopathic pulmonary fibrosis | Withdrawn | 1 Oct 2010 |
| 16-base single-stranded peptide nucleic acid oligonucleotide linked to 7-amino acid peptide EU/3/10/789 |
Treatment of medulloblastoma | Active (positive) | 1 Oct 2010 |
| (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']indolizinoโฆ EU/3/10/788 |
Treatment of hepatocellular carcinoma | Active (positive) | 1 Oct 2010 |
| (3S)-3-{4-[7-(aminocarbonyl)-2H-indazol-2-yl] phenyl} piperidine tosilate monohydrate salt EU/3/10/787 |
Treatment of mantle cell lymphoma | Withdrawn | 1 Oct 2010 |
| Heat-killed Mycobacterium vaccae (whole cell) EU/3/10/786 |
Treatment of tuberculosis | Active (positive) | 20 Sep 2010 |
| vorinostat EU/3/10/785 5 clinical trials → |
Treatment of malignant mesothelioma | Withdrawn | 20 Sep 2010 |
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.