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 |
|---|---|---|---|
| pevonedistat EU/3/19/2186 |
Treatment of acute myeloid leukaemia | Withdrawn | 25 Jul 2019 |
| Parsaclisib EU/3/19/2185 2 clinical trials → |
Treatment of marginal zone lymphoma | Withdrawn | 25 Jul 2019 |
| N-((R)-2,3-dihydroxypropoxyl)-3,4-difluro-2-(2-fluoro-4-iodo-phenylamino)-benzamide EU/3/19/2184 · now authorised as Ezmekly |
Treatment of neurofibromatosis type 1 | Active (positive) | 25 Jul 2019 |
| mavorixafor EU/3/19/2183 · now authorised as Xolremdi |
Treatment of WHIM syndrome | Active (positive) | 25 Jul 2019 |
| Elafibranor EU/3/19/2182 · now authorised as Iqirvo 5 clinical trials → |
Treatment of primary biliary cholangitis | Active (positive) | 25 Jul 2019 |
| 7-ethyl-10-hydroxy-camptothecin EU/3/19/2181 |
Treatment of soft tissue sarcoma | Active (positive) | 25 Jul 2019 |
| 2-(hydroxymethyl)-2-(methoxymethyl)-1-azabicyclo[2.2.2]octan-3-one EU/3/19/2180 |
Treatment of myelodysplastic syndromes | Active (positive) | 25 Jul 2019 |
| sodium benzoate;sodium phenylacetate EU/3/19/2179 |
Treatment of hyperargininaemia | Active (positive) | 28 Jun 2019 |
| sodium benzoate;sodium phenylacetate EU/3/19/2178 |
Treatment of argininosuccinic aciduria | Active (positive) | 28 Jun 2019 |
| regorafenib EU/3/19/2177 39 clinical trials → |
Treatment of glioma | Withdrawn | 28 Jun 2019 |
| recombinant human coagulation factor VIII Fc - von Willebrand factor - XTEN fusion protein EU/3/19/2176 · now authorised as Altuvoct |
Treatment of haemophilia A | Active (positive) | 28 Jun 2019 |
| recombinant adeno-associated viral vector containing a bioengineered capsid serotype AAV-rh74 and a codon-optimised expression cassette to drive the expression… EU/3/19/2175 |
Treatment of glycogen storage disease type II (Pompe's disease) | Withdrawn | 28 Jun 2019 |
| rasagiline EU/3/19/2174 |
Treatment of Duchenne muscular dystrophy | Withdrawn | 28 Jun 2019 |
| Imidazolyl ethanamide pentandioic acid EU/3/19/2173 |
Treatment of acute radiation syndrome | Active (positive) | 28 Jun 2019 |
| Gaboxadol monohydrate EU/3/19/2172 |
Treatment of Angelman syndrome | Active (positive) | 28 Jun 2019 |
| 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) EU/3/19/2171 |
Treatment of centronuclear myopathies | Active (positive) | 28 Jun 2019 |
| 2-(2-{[2-(1H-benzimidazol-2-yl)ethyl]amino}ethyl)-N-[(3-fluoropyridine-2-yl)methyl]-1,3-oxazole-4-carboxamide trihydrochloride EU/3/19/2170 |
Treatment of beta thalassaemia intermedia and major | Active (positive) | 28 Jun 2019 |
| reldesemtiv EU/3/19/2169 |
Treatment of spinal muscular atrophy | Active (positive) | 28 Jun 2019 |
| (S)-6-hydroxy-2,5,7,8-tetramethyl-N-((R)-piperidine-3-yl)chroman-2-carboxamide hydrochloride EU/3/19/2168 |
Treatment of maternally-inherited diabetes and deafness | Withdrawn | 28 Jun 2019 |
| zanubrutinib EU/3/19/2167 62 clinical trials → |
Treatment of lymphoplasmacytic lymphoma | Withdrawn | 29 May 2019 |
| sodium benzoate EU/3/19/2166 |
Treatment of carbamoyl-phosphate synthase-1 deficiency | Active (positive) | 29 May 2019 |
| sodium phenylacetate;sodium benzoate EU/3/19/2165 |
Treatment of citrullinaemia type 1 | Active (positive) | 29 May 2019 |
| relacorilant EU/3/19/2164 5 clinical trials → |
Treatment of Cushing's syndrome | Active (positive) | 29 May 2019 |
| N-(trans-3-(5-((R)-1-hydroxyethyl)-1,3,4-oxadiazol-2-yl)cyclobutyl)-3-phenylisoxazole-5-carboxamide EU/3/19/2163 |
Treatment of cystic fibrosis | Withdrawn | 29 May 2019 |
| emixustat hydrochloride EU/3/19/2162 |
Treatment of Stargardt's disease | Active (positive) | 29 May 2019 |
| Diacerein EU/3/19/2161 |
Treatment of epidermolysis bullosa | Active (positive) | 29 May 2019 |
| allogeneic skin-derived ABCB5-positive mesenchymal stem cells EU/3/19/2160 |
Treatment of epidermolysis bullosa | Active (positive) | 29 May 2019 |
| (S)-5-(1-(6-chloro-2-oxo-1,2-dihydroquinolin-3-yl)ethylamino-1-methyl-6-oxo-1,6-dihydropyridine-2-carbonitrile (olutasidenib) EU/3/19/2159 |
Treatment of acute myeloid leukaemia | Active (positive) | 29 May 2019 |
| (S)-3-((3-(1-((6-(3,4-dimethoxyphenyl)pryazin-2-yl)amino)ethyl)phenyl)carbamoyl)-5-methylpridin-1-ium (seralutinib) EU/3/19/2158 |
Treatment of pulmonary arterial hypertension | Active (positive) | 29 May 2019 |
| sodium benzoate;sodium phenylacetate EU/3/19/2157 |
Treatment of ornithine transcarbamylase deficiency | Active (positive) | 24 Apr 2019 |
| modified messenger ribonucleic acid encoding human propionyl-coenzyme A carboxylase alpha and beta subunits encapsulated into lipid nanoparticle EU/3/19/2156 |
Treatment of propionic acidaemia | Active (positive) | 24 Apr 2019 |
| human culture expanded autologous mesenchymal stromal cells EU/3/19/2155 |
Treatment of amyotrophic lateral sclerosis | Active (positive) | 24 Apr 2019 |
| autologous human bone marrow-derived haematopoietic and mesenchymal stem cells depleted of erythrocytes, monocytes and lymphocytes EU/3/19/2153 |
Treatment of spinal cord injury | Active (positive) | 24 Apr 2019 |
| 3-(3-(3,5-bis(trifluoromethyl)phenyl)-1h-pyrazol-1-yl)propanoic acid EU/3/19/2152 |
Treatment of Stargardt's disease | Withdrawn | 24 Apr 2019 |
| balipodect EU/3/19/2154 |
Treatment of fragile X syndrome | Active (positive) | 14 Apr 2019 |
| Marzeptacog alfa (activated) EU/3/19/2151 |
Treatment of haemophilia B | Withdrawn | 1 Apr 2019 |
| codon-optimised human cystic fibrosis transmembrane conductance regulator messenger ribonucleic acid complexed with lipid-based nanoparticles;pomulmeran EU/3/19/2150 |
Treatment of cystic fibrosis | Withdrawn | 1 Apr 2019 |
| Adeno-associated viral vector serotype rh10 containing the human cholesterol 24-hydroxylase gene EU/3/19/2149 |
Treatment of Huntington’s disease | Withdrawn | 1 Apr 2019 |
| 4-hydroxy-6-{2-[4-(trifluoromethyl)phenyl]ethyl}pyridazin-3(2H)-one EU/3/19/2148 |
Treatment of Friedreich’s ataxia | Withdrawn | 1 Apr 2019 |
| 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-(hydroxymet… EU/3/19/2147 |
Treatment of primary sclerosing cholangitis | Withdrawn | 1 Apr 2019 |
| 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) EU/3/19/2146 |
Treatment of biliary tract cancer | Withdrawn | 1 Apr 2019 |
| risdiplam EU/3/19/2145 6 clinical trials → |
Treatment of spinal muscular atrophy | Withdrawn | 26 Feb 2019 |
| Poly(N-acetyl, N-arginyl)glucosamine EU/3/19/2144 |
Treatment of cystic fibrosis | Active (positive) | 26 Feb 2019 |
| lurbinectedin EU/3/19/2143 · now authorised as Zepzelca 17 clinical trials → |
Treatment of small cell lung cancer | Active (positive) | 26 Feb 2019 |
| losartan EU/3/19/2142 10 clinical trials → |
Treatment of epidermolysis bullosa | Active (positive) | 26 Feb 2019 |
| Lentiviral vector encoding human coagulation factor IX EU/3/19/2141 |
Treatment of haemophilia B | Active (positive) | 26 Feb 2019 |
| Humanised IGg1 monoclonal antibody targeting human transferrin receptor conjugated to human iduronate-2-sulfatase EU/3/19/2140 |
Treatment of mucopolysaccharidosis type II (Hunter's syndrome) | Active (positive) | 26 Feb 2019 |
| autologous adult live cultured osteoblasts EU/3/19/2139 |
Treatment of non-traumatic osteonecrosis | Active (positive) | 26 Feb 2019 |
| allogeneic cultured postnatal thymus-derived tissue EU/3/19/2137 |
Treatment of severe combined immunodeficiency due to FOXN1 deficiency | Withdrawn | 26 Feb 2019 |
| allogeneic cultured postnatal thymus-derived tissue EU/3/19/2136 |
Treatment of CHARGE syndrome | Withdrawn | 26 Feb 2019 |
| allogeneic cultured postnatal thymus-derived tissue EU/3/19/2135 |
Treatment of DiGeorge syndrome | Withdrawn | 26 Feb 2019 |
| 9-cis, 12-cis-11,11-D2-linoleic acid ethyl ester EU/3/19/2134 |
Treatment of infantile neuroaxonal dystrophy | Active (positive) | 26 Feb 2019 |
| vinorelbine tartrate EU/3/18/2133 33 clinical trials → |
Treatment of soft tissue sarcoma | Active (positive) | 11 Jan 2019 |
| synthetic double-stranded siRNA oligonucleotide directed against TMPRSS6 mRNA and covalently linked to a ligand containing three N-acetylgalactosamine residues EU/3/18/2132 |
Treatment of beta thalassaemia intermedia and major | Withdrawn | 11 Jan 2019 |
| rozanolixizumab EU/3/18/2131 6 clinical trials → |
Treatment of immune thrombocytopenia | Withdrawn | 11 Jan 2019 |
| ralinepag EU/3/18/2130 |
Treatment of pulmonary arterial hypertension | Active (positive) | 11 Jan 2019 |
| miglustat EU/3/18/2129 3 clinical trials → |
Treatment of glycogen storage disease type II (Pompe's disease) | Withdrawn | 11 Jan 2019 |
| Mercaptamine-pantetheine disulfide EU/3/18/2128 |
Treatment of Rett syndrome | Active (positive) | 11 Jan 2019 |
| melatonin EU/3/18/2127 23 clinical trials → |
Treatment of perinatal asphyxia | Active (positive) | 11 Jan 2019 |
| human glucagon-like peptide-2 analogue linked to a human immunoglobulin Fc fragment EU/3/18/2126 |
Treatment of short bowel syndrome | Active (positive) | 11 Jan 2019 |
| benserazide hydrochloride EU/3/18/2125 |
The treatment of sickle cell disease | Active (positive) | 11 Jan 2019 |
| acetylleucine EU/3/18/2124 |
Treatment of ataxia telangiectasia | Active (positive) | 11 Jan 2019 |
| 6,8-bis(benzylthio)octanoic acid EU/3/18/2123 |
Treatment of acute myeloid leukaemia | Active (positive) | 14 Dec 2018 |
| venglustat EU/3/18/2122 4 clinical trials → |
Treatment of autosomal dominant polycystic kidney disease | Withdrawn | 14 Dec 2018 |
| sodium 2-hydroxylinoleate EU/3/18/2121 |
Treatment of biliary tract cancer | Active (positive) | 14 Dec 2018 |
| pevonedistat EU/3/18/2120 |
Treatment of myelodysplastic syndromes | Withdrawn | 14 Dec 2018 |
| Marizomib EU/3/18/2119 |
Treatment of glioma | Active (positive) | 14 Dec 2018 |
| Lonafarnib EU/3/18/2118 · now authorised as Zokinvy |
Treatment of Hutchinson-Gilford progeria | Active (positive) | 14 Dec 2018 |
| Ivacaftor, potassium(benzenesulfonyl)({[6-(3-{2-[1-(trifluoromethyl) cyclopropyl]ethoxy}-1H-pyrazol-1-yl)-2-[(4S)-2,2,4-trimethylpyrrolidin-1-yl]pyridin-3-yl]c… EU/3/18/2117 3 clinical trials → |
Treatment of cystic fibrosis | Active (positive) | 14 Dec 2018 |
| Ivacaftor, N-(1,3-dimethyl-1H-pyrazole-4-sulfonyl)-6-[3-(3,3,3-trifluoro-2,2-dimethylpropoxy)-1H-pyrazol-1-yl]-2-[(4S)-2,2,4-trimethylpyrrolidin-1-yl]pyridine-… EU/3/18/2116 · now authorised as Kaftrio 3 clinical trials → |
Treatment of cystic fibrosis | Active (positive) | 14 Dec 2018 |
| human anti-promyostatin monoclonal antibody (apitegromab) EU/3/18/2115 · now authorised as Isembyld |
Treatment of spinal muscular atrophy | Active (positive) | 14 Dec 2018 |
| C1 esterase inhibitor (human) EU/3/18/2114 |
Treatment in solid organ transplantation | Withdrawn | 14 Dec 2018 |
| bromelain EU/3/18/2113 |
Treatment of pseudomyxoma peritonei | Active (positive) | 14 Dec 2018 |
| Bifunctional fusion protein composed of two extracellular domains of transforming growth factor beta receptor II fused with a human immunoglobulin G1 monoclona… EU/3/18/2112 |
Treatment of biliary tract cancer | Withdrawn | 14 Dec 2018 |
| allogeneic ABCB5-positive limbal stem cells EU/3/18/2111 |
Treatment of limbal stem cell deficiency | Active (positive) | 14 Dec 2018 |
| afatinib EU/3/18/2110 16 clinical trials → |
Treatment of Fanconi anaemia | Active (positive) | 14 Dec 2018 |
| adeno-associated virus serotype HSC15 expressing human phenylalanine hydroxylase EU/3/18/2109 |
Treatment of phenylalanine hydroxylase deficiency | Withdrawn | 14 Dec 2018 |
| adeno-associated viral vector expressing human 21- hydroxylase EU/3/18/2108 |
Treatment of congenital adrenal hyperplasia | Active (positive) | 14 Dec 2018 |
| acetylcysteine EU/3/18/2107 2 clinical trials → |
Treatment of pseudomyxoma peritonei | Active (positive) | 14 Dec 2018 |
| 6-fluoro-9-methyl-9H-pyrido[3,4-b]-indole EU/3/18/2106 |
Treatment of sudden sensorineural hearing loss | Active (positive) | 14 Dec 2018 |
| 6,8-bis(benzylthio)octanoic acid EU/3/18/2105 |
Treatment of pancreatic cancer | Active (positive) | 14 Dec 2018 |
| (4-{(2S,4S)-4-ethoxy-1-[(5-methoxy-7-methyl-1H-indol-4-yl)methyl]piperidin-2-yl}benzoic acid-hydrogen chloride(1/1)) EU/3/18/2104 |
Treatment of C3 glomerulopathy | Active (positive) | 14 Dec 2018 |
EU/3/18/2103 |
Treatment of biliary atresia | Active (positive) | 14 Dec 2018 |
| bertilimumab EU/3/18/2062 |
Treatment of bullous pemphigoid | Active (positive) | 24 Nov 2018 |
| acetylleucine EU/3/18/2059 |
Treatment of spinocerebellar ataxia | Active (positive) | 22 Nov 2018 |
| apraglutide EU/3/18/2102 2 clinical trials → |
Treatment of short bowel syndrome | Active (positive) | 19 Nov 2018 |
| setmelanotide EU/3/18/2101 · now authorised as Imcivree 4 clinical trials → |
Treatment of leptin receptor deficiency | Active (positive) | 19 Nov 2018 |
| propagermanium EU/3/18/2100 |
Treatment of focal segmental glomerulosclerosis | Withdrawn | 19 Nov 2018 |
| lisocabtagene maraleucel EU/3/18/2099 9 clinical trials → |
Treatment of primary mediastinal large B-cell lymphoma | Withdrawn | 19 Nov 2018 |
| Larotrectinib EU/3/18/2098 9 clinical trials → |
Treatment of papillary thyroid cancer | Withdrawn | 19 Nov 2018 |
| Larotrectinib EU/3/18/2097 9 clinical trials → |
Treatment of glioma | Withdrawn | 19 Nov 2018 |
| imlifidase EU/3/18/2096 2 clinical trials → |
Treatment of anti-glomerular basement membrane disease | Active (positive) | 19 Nov 2018 |
| Ile-Ser-Ile-Thr-Glu-Ile-Lys-Gly-Val-Ile-Val-His-Arg-Ile-Glu-Thr-Ile-Leu-Phe-Lys-Lys-Lys-Lys-Glu-Met-Pro-Ser-Glu-Glu-Gly-Tyr-Gln-Asp EU/3/18/2095 |
Treatment of multiple system atrophy | Active (positive) | 19 Nov 2018 |
| anti-GD2 monoclonal antibody 3F8 humanised EU/3/18/2094 |
Treatment of neuroblastoma | Active (positive) | 19 Nov 2018 |
| human apotransferrin EU/3/18/2093 |
Treatment of beta thalassaemia intermedia and major | Active (positive) | 19 Nov 2018 |
| H-Arg-Pro-Lys-Pro-Gln-Gln-Phe-2Thi-Gly-Leu-Met(O2)-NH2-DOTA-213-bismuth EU/3/18/2092 |
Treatment of glioma | Active (positive) | 19 Nov 2018 |
| Glucagon EU/3/18/2091 2 clinical trials → |
Treatment of noninsulinoma pancreatogenous hypoglycaemia syndrome | Active (positive) | 19 Nov 2018 |
| fidanacogene elaparvovec EU/3/18/2090 |
Treatment of haemophilia B | Withdrawn | 19 Nov 2018 |
| Ex vivo fused normal allogeneic human myoblast with another normal allogeneic human myoblast EU/3/18/2088 |
Treatment of Duchenne muscular dystrophy | Active (positive) | 19 Nov 2018 |
| Etamsylate EU/3/18/2087 |
Treatment of hereditary haemorrhagic telangiectasia | Active (positive) | 19 Nov 2018 |
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.