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 |
|---|---|---|---|
| 1-((2S,4S)-2-((((S)-(4-bromophenoxy)(((S)-1-oxo-1-(((S)-pentan-2-yl)oxy)propan-2-yl)amino)phosphoryl)oxy)methyl)-1,3-dioxolan-4-yl)-2-oxo-1,2-dihydropyrimidin-… EU/3/20/2285 |
Treatment of hepatocellular carcinoma | Active (positive) | 4 Jun 2020 |
| sodium phenylbutyrate;tauroursodeoxycholic acid EU/3/20/2284 · now authorised as Albrioza |
Treatment of amyotrophic lateral sclerosis | Active (positive) | 4 Jun 2020 |
| Ile-Ala-Leu-Ile-Leu-Glu-Pro-Ile-Cys-Cys-Gln-Glu-Arg-Ala-Ala-(discrete-polyethylene glycol)24 EU/3/20/2283 |
Treatment of neonatal encephalopathy | Active (positive) | 4 Jun 2020 |
| viltolarsen EU/3/20/2282 |
Treatment of Duchenne muscular dystrophy | Active (positive) | 4 Jun 2020 |
| (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/3/20/2281 · now authorised as Fabhalta |
Treatment of paroxysmal nocturnal haemoglobinuria | Active (positive) | 4 Jun 2020 |
| Lutetium (177Lu) lilotomab satetraxetan EU/3/20/2280 |
Treatment of marginal zone lymphoma | Active (positive) | 4 Jun 2020 |
| Lumacaftor EU/3/20/2279 |
Treatment of non-traumatic subarachnoid haemorrhage | Active (positive) | 4 Jun 2020 |
| rilzabrutinib EU/3/20/2278 9 clinical trials → |
Treatment of immune thrombocytopenia | Active (positive) | 4 Jun 2020 |
| autologous T lymphocyte-enriched population of cells transduced with a lentiviral vector encoding a chimeric antigen receptor targeting human B cell maturation… EU/3/20/2277 |
Treatment of multiple myeloma | Withdrawn | 4 Jun 2020 |
| methotrexate EU/3/20/2276 97 clinical trials → |
Treatment of retinal detachment | Active (positive) | 4 Jun 2020 |
| Glucagon analogue linked to a human immunoglobulin Fc fragment EU/3/20/2275 |
Treatment of insulin autoimmune syndrome | Active (positive) | 22 Apr 2020 |
| adeno-associated virus vector serotype hu37 encoding human factor VIII EU/3/20/2274 |
Treatment of haemophilia A | Withdrawn | 22 Apr 2020 |
| anti-(integrin beta-3) human monoclonal antibody EU/3/20/2273 |
Prevention of fetal and neonatal alloimmune thrombocytopenia due to human platelet antigen-1a incompatibility | Active (positive) | 22 Apr 2020 |
| rozanolixizumab EU/3/20/2272 · now authorised as Rystiggo 6 clinical trials → |
Treatment of myasthenia gravis | Active (positive) | 22 Apr 2020 |
| mitapivat sulfate EU/3/20/2270 · now authorised as Pyrukynd 12 clinical trials → |
Treatment of pyruvate kinase deficiency | Active (positive) | 22 Apr 2020 |
| florbetaben (18F) EU/3/20/2268 |
Diagnosis of AL amyloidosis | Active (positive) | 22 Apr 2020 |
| adeno-associated viral vector serotype 3B encoding human multidrug resistance protein 3A EU/3/20/2267 |
Treatment of progressive familial intrahepatic cholestasis | Withdrawn | 22 Apr 2020 |
| DNA plasmid encoding IL-12 p35 and p40 genes EU/3/20/2266 |
Treatment of ovarian cancer | Active (positive) | 22 Apr 2020 |
| cusatuzumab EU/3/20/2265 3 clinical trials → |
Treatment of acute myeloid leukaemia | Active (positive) | 22 Apr 2020 |
| 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]indo… EU/3/20/2271 · now authorised as Zemcelpro |
Treatment in haematopoietic stem cell transplantation | Active (positive) | 20 Apr 2020 |
| trifarotene EU/3/20/2264 |
Treatment of autosomal recessive congenital ichthyosis | Withdrawn | 24 Mar 2020 |
| Losmapimod EU/3/20/2263 |
treatment of facioscapulohumeral muscular dystrophy | Withdrawn | 24 Mar 2020 |
| fosgemcitabine palabenamide EU/3/20/2262 |
Treatment of biliary tract cancer | Withdrawn | 24 Mar 2020 |
| asciminib EU/3/20/2261 · now authorised as Scemblix 7 clinical trials → |
Treatment of chronic myeloid leukaemia | Active (positive) | 24 Mar 2020 |
| allogeneic multi-virus specific T lymphocytes targeting BK virus, cytomegalovirus, human herpesvirus-6, Epstein Barr virus and adenovirus EU/3/20/2260 |
Treatment in haematopoietic stem cell transplantation | Active (positive) | 24 Mar 2020 |
| 2-hydroxy-N,N,N-trimethylethan-1-aminium (Z)-4-(5-((3-benzyl-4-oxo-2-thioxothiazolidin-5-ylidene)methyl)furan-2-yl)benzoate EU/3/20/2259 |
Treatment of pancreatic cancer | Withdrawn | 24 Mar 2020 |
| sutimlimab EU/3/20/2258 |
Treatment of immune thrombocytopenia | Withdrawn | 28 Feb 2020 |
| sintilimab EU/3/20/2257 176 clinical trials → |
Treatment of peripheral T-cell lymphoma | Active (positive) | 28 Feb 2020 |
| reldesemtiv EU/3/20/2256 |
Treatment of amyotrophic lateral sclerosis | Active (positive) | 28 Feb 2020 |
| Luspatercept EU/3/20/2255 28 clinical trials → |
Treatment of myelofibrosis | Active (positive) | 28 Feb 2020 |
| Combination of three adeno-associated viral vectors of serotype 8 containing the 5'-, the body- and the 3'- coding sequences of human CEP290 fused to inteins EU/3/20/2254 |
Treatment of inherited retinal dystrophies | Active (positive) | 28 Feb 2020 |
| autologous skin equivalent graft composed of keratinocytes and fibroblasts genetically corrected by CRISPR/Cas9-mediated excision of mutation-carrying COL7A1 e… EU/3/20/2253 |
Treatment of epidermolysis bullosa | Active (positive) | 28 Feb 2020 |
| autologous human T cells genetically modified ex-vivo with a lentiviral vector encoding a chimeric antigen receptor for B-cell maturation antigen EU/3/20/2252 · now authorised as Carvykti |
Treatment of multiple myeloma | Active (positive) | 28 Feb 2020 |
| artesunate EU/3/20/2251 · now authorised as Artesunate Amivas 5 clinical trials → |
Treatment of malaria | Active (positive) | 28 Feb 2020 |
| adeno-associated virus serotype rh74 containing the human micro-dystrophin gene EU/3/20/2250 · now authorised as Elevidys |
Treatment of Duchenne muscular dystrophy | Active (positive) | 28 Feb 2020 |
| adeno-associated virus serotype 8 containing the human RdCVF sequence and the human RdCVFL sequence EU/3/20/2249 |
Treatment of inherited retinal dystrophies | Active (positive) | 28 Feb 2020 |
| Adeno-associated viral vector serotype S3 encoding human alpha-galactosidase A cDNA EU/3/20/2248 |
Treatment of Fabry disease | Active (positive) | 28 Feb 2020 |
| romilkimab EU/3/19/2246 |
Treatment of systemic sclerosis | Withdrawn | 9 Jan 2020 |
| setmelanotide EU/3/19/2245 4 clinical trials → |
Treatment of Alström syndrome | Active (positive) | 9 Jan 2020 |
| ziritaxestat EU/3/19/2244 |
Treatment of systemic sclerosis | Withdrawn | 9 Jan 2020 |
| motixafortide EU/3/19/2243 3 clinical trials → |
Treatment of pancreatic cancer | Active (positive) | 9 Jan 2020 |
| autologous CD34+ hematopoietic stem cells with a CRISPR-edited erythroid enhancer region of the BCL11A gene EU/3/19/2242 · now authorised as Casgevy |
Treatment of sickle cell disease | Active (positive) | 9 Jan 2020 |
| adeno-associated virus serotype 2/6 encoding human alpha-galactosidase A cDNA EU/3/19/2241 |
Treatment of Fabry disease | Active (positive) | 9 Jan 2020 |
| nicardipine EU/3/19/2240 2 clinical trials → |
Treatment of non-traumatic subarachnoid haemorrhage | Active (positive) | 9 Jan 2020 |
| tripotassium citrate monohydrate;potassium hydrogen carbonate EU/3/19/2239 |
Treatment of cystinuria | Active (positive) | 9 Jan 2020 |
| adeno-associated virus serotype 9 vector containing human n-acetylgalactosamine-6-sulfate sulfatase gene EU/3/19/2238 |
Treatment of mucopolysaccharidosis type IVA (Morquio A Syndrome) | Active (positive) | 9 Jan 2020 |
| 2-(3,7-Dimethyl-octa-2, 6-dienyl)-6-ethylamino-3-hydroxy-5-pentyl-[1,4]benzoquinone EU/3/19/2237 |
Treatment of Huntington's disease | Active (positive) | 9 Jan 2020 |
| dimethyl fumarate EU/3/19/2236 7 clinical trials → |
Treatment of adrenoleukodystrophy | Active (positive) | 9 Jan 2020 |
| synthetic double-stranded siRNA oligonucleotide directed against SERPINA1 mRNA and containing four modified nucleosides which form a ligand cluster of four N-a… EU/3/19/2235 |
Treatment of congenital alpha-1 antitrypsin deficiency | Withdrawn | 16 Dec 2019 |
| pamrevlumab EU/3/19/2234 |
Treatment of Duchenne muscular dystrophy | Withdrawn | 16 Dec 2019 |
| navitoclax EU/3/19/2233 6 clinical trials → |
Treatment of myelofibrosis | Withdrawn | 16 Dec 2019 |
| Lactobacillus plantarum EU/3/19/2232 |
Treatment of amyotrophic lateral sclerosis | Withdrawn | 16 Dec 2019 |
| H-Leu-Pro-Pro-Leu-Pro-Tyr-Pro-OH EU/3/19/2231 |
Treatment of amyotrophic lateral sclerosis | Active (positive) | 16 Dec 2019 |
| efgartigimod alfa EU/3/19/2230 |
Treatment of immune thrombocytopenia | Active (positive) | 16 Dec 2019 |
| adeno-associated virus vector encoding human phenylalanine hydroxylase EU/3/19/2229 |
Treatment of phenylalanine hydroxylase deficiency | Withdrawn | 16 Dec 2019 |
| 5,7-dichloro-2-((ethylamino)methyl)-8-hydroxy-3-methylquinazolin-4(3H)-one mesilate EU/3/19/2228 |
Treatment of multiple system atrophy | Active (positive) | 16 Dec 2019 |
| 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 EU/3/19/2227 · now authorised as Romvimza |
Treatment of tenosynovial giant cell tumour, localised and diffuse type | Active (positive) | 16 Dec 2019 |
| (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-… EU/3/19/2226 |
Treatment of osteosarcoma | Active (positive) | 16 Dec 2019 |
| Ex vivo fused normal allogeneic human myoblast with autologous human myoblast derived from Duchenne muscular dystrophy affected donor EU/3/18/2089 |
Treatment of Duchenne muscular dystrophy | Active (positive) | 19 Nov 2019 |
| replication-incompetent, non-integrating, herpes simplex virus 1 vector expressing the human transglutaminase-1 enzyme (Inetagugene geperpavec) EU/3/19/2225 |
Treatment of autosomal recessive congenital ichthyosis | Active (positive) | 13 Nov 2019 |
| ganaxolone EU/3/19/2224 · now authorised as Ztalmy |
Treatment of CDKL5 deficiency disorder | Active (positive) | 13 Nov 2019 |
| Exendin (9-39) EU/3/19/2223 |
Treatment of congenital hyperinsulinism | Active (positive) | 13 Nov 2019 |
| chimeric fibril-reactive IgG1k monoclonal antibody 11-1F4 EU/3/19/2222 |
Treatment of AL amyloidosis | Active (positive) | 13 Nov 2019 |
| camsirubicin EU/3/19/2221 |
Treatment of soft tissue sarcoma | Active (positive) | 13 Nov 2019 |
| autologous peripheral blood T cells CD4 and CD8 selected and CD3 and CD28 activated transduced with retroviral vector expressing anti CD19 CD28/CD3-zeta chimer… EU/3/19/2220 · now authorised as Tecartus |
Treatment of mantle cell lymphoma | Active (positive) | 13 Nov 2019 |
| 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 … EU/3/19/2219 |
Treatment of non-infectious uveitis | Active (positive) | 13 Nov 2019 |
EU/3/19/2218 |
Treatment of GM2 gangliosidosis | Active (positive) | 13 Nov 2019 |
| propranolol hydrochloride EU/3/19/2217 21 clinical trials → |
Treatment of retinopathy of prematurity | Active (positive) | 17 Oct 2019 |
| pemigatinib EU/3/19/2216 · now authorised as Pemazyre 3 clinical trials → |
Treatment of myeloid/lymphoid neoplasms with eosinophilia and rearrangement of PDGFRA, PDGFRB, or FGFR1, or with PCM1-JAK | Active (positive) | 17 Oct 2019 |
| paclitaxel EU/3/19/2215 400 clinical trials → |
Treatment of soft tissue sarcoma | Active (positive) | 17 Oct 2019 |
| nirogacestat EU/3/19/2214 · now authorised as Ogsiveo 9 clinical trials → |
Treatment of soft tissue sarcoma | Active (positive) | 17 Oct 2019 |
| lonapegsomatropin EU/3/19/2213 · now authorised as Skytrofa (previously Lonapegsomatropin Ascendis Pharma) |
Treatment of growth hormone deficiency | Active (positive) | 17 Oct 2019 |
| leriglitazone EU/3/19/2212 |
Treatment of Friedreich’s ataxia | Active (positive) | 17 Oct 2019 |
| besilesomab EU/3/19/2211 |
Treatment in haematopoietic stem cell transplantation | Active (positive) | 17 Oct 2019 |
| autologous CD34+ haematopoietic stem cells with a CRISPR-edited erythroid enhancer region of the BCL11A gene EU/3/19/2210 · now authorised as Casgevy |
Treatment of beta thalassaemia intermedia and major | Active (positive) | 17 Oct 2019 |
| anti-neonatal Fc receptor human monoclonal antibody EU/3/19/2209 |
Prevention of haemolytic disease of the foetus and newborn | Active (positive) | 17 Oct 2019 |
| 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 EU/3/19/2208 |
Treatment of Stargardt's disease | Active (positive) | 17 Oct 2019 |
| 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 (enc… EU/3/19/2207 |
Treatment of soft tissue sarcoma | Active (positive) | 17 Oct 2019 |
| 2'-O-(2-methoxyethyl)-D-ribose antisense oligonucleotide targeting glial fibrillary acidic protein messenger ribonucleic acid EU/3/19/2206 |
Treatment of Alexander disease | Active (positive) | 17 Oct 2019 |
| 2-(3-(4-(1H-Indazol-5-ylamino)quinazolin-2-yl)phenoxy)-N-isopropylacetamide-methane sulfonic acid salt EU/3/19/2205 · now authorised as Rezurock |
Treatment of graft-versus-host disease | Active (positive) | 17 Oct 2019 |
| 18-(p-(131I)-iodophenyl)octadecyl phosphocholine EU/3/19/2204 |
Treatment of multiple myeloma | Active (positive) | 17 Oct 2019 |
| (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-dim… EU/3/19/2203 |
Treatment of Netherton syndrome | Withdrawn | 17 Oct 2019 |
| (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 EU/3/19/2202 |
Treatment of glioma | Active (positive) | 17 Oct 2019 |
| melatonin;sorafenib - 23 clinical trials → |
Treatment of hepatocellular carcinoma | Negative | 23 Sep 2019 |
| 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-as… EU/3/19/2201 |
Treatment of C3 glomerulopathy | Active (positive) | 21 Aug 2019 |
| clofazimine EU/3/19/2200 5 clinical trials → |
Treatment of nontuberculous mycobacterial lung disease | Active (positive) | 21 Aug 2019 |
| veledimex EU/3/19/2199 |
Treatment of glioma | Withdrawn | 21 Aug 2019 |
| adenoviral vector serotype 5 encoding the human interleukin-12 p70 transgene under the control of activator ligand veledimex EU/3/19/2198 |
Treatment of glioma | Withdrawn | 21 Aug 2019 |
| recombinant self-complementary adeno-associated viral vector serotype 9 containing the human CLN6 gene EU/3/19/2197 |
Treatment of neuronal ceroid lipofuscinosis | Active (positive) | 21 Aug 2019 |
| recombinant self-complementary adeno-associated viral vector serotype 9 containing the human CLN3 gene EU/3/19/2196 |
Treatment of neuronal ceroid lipofuscinosis | Active (positive) | 21 Aug 2019 |
| 1-(2,2-diphenyltetrahydrofuran-3-yl)-N,N-dimethylmethanamine hydrochloride EU/3/19/2195 |
Treatment of Rett syndrome | Active (positive) | 21 Aug 2019 |
| 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 EU/3/19/2194 |
Treatment of congenital adrenal hyperplasia | Active (positive) | 21 Aug 2019 |
| acetazolamide EU/3/19/2193 11 clinical trials → |
Treatment of periodic paralysis | Active (positive) | 21 Aug 2019 |
| setmelanotide EU/3/19/2192 · now authorised as Imcivree 4 clinical trials → |
Treatment of Bardet-Biedl syndrome | Active (positive) | 21 Aug 2019 |
| relacorilant EU/3/19/2191 5 clinical trials → |
Treatment of pancreatic cancer | Active (positive) | 21 Aug 2019 |
| peginterferon lambda-1a EU/3/19/2190 |
Treatment of hepatitis D virus infection | Active (positive) | 21 Aug 2019 |
| gallium citrate EU/3/19/2189 |
Treatment of cystic fibrosis | Active (positive) | 21 Aug 2019 |
| temozolomide EU/3/19/2188 · now authorised as Kizfizo 97 clinical trials → |
Treatment of neuroblastoma | Active (positive) | 21 Aug 2019 |
| monoclonal antibody against human CD30 covalently linked to the cytotoxin monomethylauristatin E EU/3/08/595 |
Treatment of peripheral T-cell lymphoma | Withdrawn | 21 Aug 2019 |
| recombinant mutated extracellular domain of the human acetylcholine receptor subunit alpha1 EU/3/19/2187 |
Treatment of myasthenia gravis | Active (positive) | 25 Jul 2019 |
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.