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 |
|---|---|---|---|
| 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 EU/3/18/2086 |
Treatment of cystic fibrosis | Active (positive) | 19 Nov 2018 |
| autologous human adipose perivascular stromal cells genetically modified to secrete soluble tumour necrosis factor-related apoptosis-inducing ligand EU/3/18/2085 |
Treatment of pancreatic cancer | Active (positive) | 19 Nov 2018 |
| anetumab ravtansine EU/3/18/2084 3 clinical trials → |
Treatment of ovarian cancer | Withdrawn | 19 Nov 2018 |
| allogeneic faecal microbiota, pooled EU/3/18/2083 |
Treatment of graft-versus-host disease | Active (positive) | 19 Nov 2018 |
| 5-{(1R,2R)-2-[(cyclopropylmethyl)amino]cyclopropyl}-N-(tetrahydro-2H-pyran-4-yl)thiophene-3-carboxamide monohydrochloride EU/3/18/2082 |
Treatment of Kabuki syndrome | Withdrawn | 19 Nov 2018 |
| 3-(3-(3,5-Dimethyl-1H-pyrazol-4-yl)propoxy)-4-fluorobenzoic acid EU/3/18/2081 |
Treatment of ATTR amyloidosis | Withdrawn | 19 Nov 2018 |
| Diacerein - |
Treatment of epidermolysis bullosa | Negative | 8 Nov 2018 |
| recombinant adeno-associated viral vector serotype S3 containing codon-optimised expression cassette encoding human coagulation factor IX variant EU/3/18/2080 |
Treatment of haemophilia B | Withdrawn | 26 Oct 2018 |
| recombinant adeno-associated viral vector containing a bioengineered capsid and a codon-optimised expression cassette to drive the expression of the SQ form ofโฆ EU/3/18/2079 |
Treatment of haemophilia A | Withdrawn | 26 Oct 2018 |
| Peptides YMFPNAPYL, SGQAYMFPNAPYLPSCLES, RSDELVRHHNMHQRNMTKL and PGCNKRYFKLSHLQMHSRKHTG EU/3/18/2078 |
Treatment of multiple myeloma | Active (positive) | 26 Oct 2018 |
| melatonin EU/3/18/2077 23 clinical trials → |
Treatment of acute radiation syndrome | Active (positive) | 26 Oct 2018 |
| glycine;L-alanine;L-arginine;L-aspartic acid;L-cysteine;L-glutamic acid;L-histidine;L-lysine monohydrate;L-methionine;L-phenylalanine;L-proline;L-serine;L-threโฆ EU/3/18/2076 · now authorised as Maapliv 2 clinical trials → |
Treatment of maple syrup urine disease | Active (positive) | 26 Oct 2018 |
| gefinitib EU/3/18/2075 |
Treatment of Fanconi anaemia type A | Active (positive) | 26 Oct 2018 |
| avapritinib EU/3/18/2074 · now authorised as Ayvakyt 8 clinical trials → |
Treatment of mastocytosis | Active (positive) | 26 Oct 2018 |
| autologous CD34+ haematopoietic stem and progenitor cells genetically modified with the lentiviral vector IDUA LV, encoding for the alpha-L-iduronidase cDNA EU/3/18/2073 |
Treatment of mucopolysaccharidosis type I | Active (positive) | 26 Oct 2018 |
| 6-(R)-methyl-5-O-(5-amino-5,6-dideoxy-alpha-L-talofuranosyl)-paromamine sulfate EU/3/18/2072 |
Treatment of cystic fibrosis | Active (positive) | 26 Oct 2018 |
| 6-(2-hydroxy-2-methylpropoxy)-4-(6-(6-((6-methoxypyridin-3-yl)methyl)-3,6-diazabicyclo[3.1.1]heptan-3-yl)pyridin-3-yl)pyrazolo[1,5-a]pyridine-3-carbonitrile EU/3/18/2071 |
Treatment of medullary thyroid carcinoma | Withdrawn | 26 Oct 2018 |
| tilorone EU/3/18/2069 |
Treatment of idiopathic pulmonary fibrosis | Active (positive) | 24 Aug 2018 |
| somapacitan EU/3/18/2068 · now authorised as Sogroya 7 clinical trials → |
Treatment of growth hormone deficiency | Active (positive) | 24 Aug 2018 |
| recombinant human monoclonal antibody against mannan-binding lectin-associated serine protease-2 EU/3/18/2067 · now authorised as Yartemlea |
Treatment in haematopoietic stem cell transplantation | Active (positive) | 24 Aug 2018 |
| pemigatinib EU/3/18/2066 · now authorised as Pemazyre 3 clinical trials → |
Treatment of biliary tract cancer | Active (positive) | 24 Aug 2018 |
| obiltoxaximab EU/3/18/2065 |
Treatment of anthrax | Withdrawn | 24 Aug 2018 |
| Copanlisib EU/3/18/2064 · now authorised as Aliqopa 4 clinical trials → |
Treatment of marginal zone lymphoma | Withdrawn | 24 Aug 2018 |
| CD34+ haematopoietic stem and progenitor cells with CD3+ T-cells EU/3/18/2063 |
Treatment in solid organ transplantation | Active (positive) | 24 Aug 2018 |
| autologous glioma tumour cells treated with antisense molecule directed against the insulin-like growth factor type 1 receptor EU/3/18/2061 |
Treatment of glioma | Active (positive) | 24 Aug 2018 |
| adeno-associated viral vector serotype 2 containing the human REP1 gene (timrepigene emparvovec) EU/3/18/2060 |
Treatment of spinal muscular atrophy | Withdrawn | 24 Aug 2018 |
| 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-phโฆ EU/3/18/2058 |
Treatment of beta thalassaemia intermedia and major | Withdrawn | 24 Aug 2018 |
| 1-(2-hydroxyethyl)-8-{[5-(4-methylpiperazin-1-yl)-2-(trifluoromethoxy) phenyl]amino}-4,5-dihydro-1H-pyrazolo[4,3-h]quinazoline-3-carboxamide fumarate salt (onvโฆ EU/3/18/2057 |
Treatment of acute myeloid leukaemia | Withdrawn | 24 Aug 2018 |
| (S)-(โ)-3-(4-aminophenyl)-2-methoxypropanoic acid EU/3/18/2056 |
Treatment of idiopathic pulmonary fibrosis | Withdrawn | 24 Aug 2018 |
EU/3/18/2055 |
Treatment of glioma | Active (positive) | 24 Aug 2018 |
| tetracosactide EU/3/18/2054 |
Treatment of Duchenne muscular dystrophy | Active (positive) | 31 Jul 2018 |
| tamibarotene EU/3/18/2053 |
Treatment of acute myeloid leukaemia | Active (positive) | 31 Jul 2018 |
| synthetic double-stranded siRNA oligonucleotide directed against lactate dehydrogenase A mRNA and containing four modified nucleosides which form a ligand clusโฆ EU/3/18/2052 |
Treatment of primary hyperoxaluria | Active (positive) | 31 Jul 2018 |
EU/3/18/2052 |
Treatment of primary hyperoxaluria | Active (positive) | 31 Jul 2018 |
| synthetic antisense oligonucleotide directed against human dystrophin pre-mRNA EU/3/18/2051 |
Treatment of Duchenne muscular dystrophy | Active (positive) | 31 Jul 2018 |
| selumetinib EU/3/18/2050 · now authorised as Koselugo 18 clinical trials → |
Treatment of neurofibromatosis type 1 | Active (positive) | 31 Jul 2018 |
| recombinant human ectonucleotide pyrophosphatase/phosphodiesterase 1 fused to the Fc fragment of IgG1 EU/3/18/2049 |
treatment of ectonucleotide pyrophosphatase/phosphodiesterase 1 deficiency | Active (positive) | 31 Jul 2018 |
| N-acetylgalactosamine-conjugated synthetic double-stranded oligomer specific to serpin family A member 1 gene EU/3/18/2048 |
Treatment of congenital alpha-1 antitrypsin deficiency | Active (positive) | 31 Jul 2018 |
| liposomal mannose-1-phosphate EU/3/18/2047 |
treatment of phosphomannomutase 2-congenital disorder of glycosylation | Active (positive) | 31 Jul 2018 |
| givinostat EU/3/18/2046 |
Treatment of Becker muscular dystrophy | Active (positive) | 31 Jul 2018 |
| ex-vivo fused autologous human bone marrow-derived mesenchymal stem cell with allogenic human myoblast EU/3/18/2045 |
Treatment of Duchenne muscular dystrophy | Active (positive) | 31 Jul 2018 |
| allogeneic bone marrow derived mesenchymal stromal cells, ex-vivo expanded EU/3/18/2044 |
Treatment of graft-versus-host disease | Active (positive) | 31 Jul 2018 |
| carboplatin;sodium valproate EU/3/18/2043 545 clinical trials → |
Treatment of glioma | Active (positive) | 31 Jul 2018 |
| adenovirus associated viral vector serotype 2/8 containing the human CNGA3 gene (entacingene turiparvovec) EU/3/18/2042 |
Treatment of achromatopsia | Active (positive) | 31 Jul 2018 |
| 2สน-O-(2-methoxyethyl) antisense oligonucleotide targeting microtubule-associated protein tau pre-mRNA EU/3/18/2041 |
treatment of behavioural variant frontotemporal dementia | Active (positive) | 31 Jul 2018 |
| recombinant human placental growth factor EU/3/18/2040 |
Treatment of pre-eclampsia | Active (positive) | 27 Jun 2018 |
| recombinant adeno-associated viral vector serotype 9 containing human iduronidase gene EU/3/18/2039 |
Treatment of mucopolysaccharidosis type I | Active (positive) | 27 Jun 2018 |
| palovarotene EU/3/18/2038 |
Treatment of multiple osteochondromas | Withdrawn | 27 Jun 2018 |
| omaveloxolone EU/3/18/2037 · now authorised as Skyclarys 5 clinical trials → |
Treatment of Friedreichโs ataxia | Active (positive) | 27 Jun 2018 |
| L-cystine bis(N-methylpiperazide) EU/3/18/2036 |
Treatment of cystinuria | Active (positive) | 27 Jun 2018 |
| Efpegsomatropin EU/3/18/2035 |
Treatment of growth hormone deficiency | Active (positive) | 27 Jun 2018 |
| Deferiprone EU/3/18/2034 · now authorised as Upkanz |
Treatment of neurodegeneration with brain iron accumulation | Active (positive) | 27 Jun 2018 |
| codon-optimised human ornithine transcarbamylase mRNA complexed with lipid-based nanoparticles EU/3/18/2033 |
Treatment of ornithine transcarbamylase deficiency | Withdrawn | 27 Jun 2018 |
| carmustine EU/3/18/2032 |
Treatment in haematopoietic stem cell transplantation | Active (positive) | 27 Jun 2018 |
| argon EU/3/18/2031 |
Treatment of perinatal asphyxia | Active (positive) | 27 Jun 2018 |
| 20-hydroxyecdysone EU/3/18/2030 |
Treatment of Duchenne muscular dystrophy | Active (positive) | 27 Jun 2018 |
| 2-[(2S)-2-methyl-1,4-dioxa-8-azaspiro[4.5]dec-8-yl]-8-nitro-6-trifluoromethyl-4H-1,3-benzothiazin-4-one EU/3/18/2029 |
Treatment of tuberculosis | Active (positive) | 27 Jun 2018 |
| R)-1-(3-(aminomethyl) phenyl)-N-(5-((3-cyanophenyl)(cyclopropylmethylamino)methyl)-2-fluorophenyl)-3-(trifluoromethyl)-1H-pyrazole-5-carboxamide dihydrochloridโฆ EU/3/18/2028 |
Treatment of hereditary angioedema | Active (positive) | 27 Jun 2018 |
| three human monoclonal antibodies against the EBOV glycoprotein EU/3/18/2027 |
Treatment of Ebola virus disease | Active (positive) | 25 May 2018 |
| synthetic double-stranded siRNA oligonucleotide targeted against transthyretin mRNA, with six phosphorothioate linkages in the backbone, and nine 2'-fluoro andโฆ EU/3/18/2026 · now authorised as Amvuttra |
Treatment of transthyretin-mediated amyloidosis (ATTR amyloidosis) | Active (positive) | 25 May 2018 |
| modified mRNA encoding human methylmalonyl-coenzyme A mutase encapsulated into lipid nanoparticles EU/3/18/2025 |
Treatment of methylmalonic acidaemia | Withdrawn | 25 May 2018 |
| Itraconazole EU/3/18/2024 35 clinical trials → |
Prevention of invasive aspergillosis | Active (positive) | 25 May 2018 |
| H-Arg-Pro-Lys-Pro-Gln-Gln-Phe-2Thi-Gly-Leu-Met(O2)-NH2-DOTA-225-Actinium EU/3/18/2023 |
Treatment of glioma | Active (positive) | 25 May 2018 |
| Glucagon analogue linked to a human immunoglobulin Fc fragment EU/3/18/2022 |
Treatment of congenital hyperinsulinism | Active (positive) | 25 May 2018 |
| Equine immunoglobulin F(ab')2 fragments targeting Shiga toxin EU/3/18/2021 |
Prevention of haemolytic uraemic syndrome | Active (positive) | 25 May 2018 |
| daratumumab EU/3/18/2020 98 clinical trials → |
Treatment of AL amyloidosis | Withdrawn | 25 May 2018 |
| Bardoxolone methyl EU/3/18/2019 · now authorised as Imbarkyd |
Treatment of Alport syndrome | Active (positive) | 25 May 2018 |
| autologous CD4+ and CD8+ T cells expressing a CD19-specific chimeric antigen receptor (lisocabtagene maraleucel) EU/3/18/2018 |
Treatment of follicular lymphoma | Withdrawn | 25 May 2018 |
| ambroxol hydrochloride EU/3/18/2017 2 clinical trials → |
Treatment of amyotrophic lateral sclerosis | Active (positive) | 25 May 2018 |
| adeno-associated viral vector serotype 9 containing the human CLN1 gene EU/3/18/2016 |
Treatment of neuronal ceroid lipofuscinosis | Active (positive) | 25 May 2018 |
| 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 EU/3/18/2015 |
Treatment of haemophilia A | Withdrawn | 25 May 2018 |
| 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) EU/3/18/2014 |
Treatment of Stargardt's disease | Active (positive) | 25 May 2018 |
| polatuzumab vedotin EU/3/18/2013 · now authorised as Polivy 23 clinical trials → |
Treatment of diffuse large B-cell lymphoma | Active (positive) | 16 Apr 2018 |
| genetically modified replication-incompetent herpes simplex virus-1 expressing collagen VII (beremagene geperpavec) EU/3/18/2012 · now authorised as Vyjuvek |
Treatment of epidermolysis bullosa | Active (positive) | 16 Apr 2018 |
| burosumab EU/3/18/2011 · now authorised as Crysvita |
Treatment of phosphaturic mesenchymal tumour | Active (positive) | 16 Apr 2018 |
| branaplam EU/3/18/2010 |
Treatment of spinal muscular atrophy | Withdrawn | 16 Apr 2018 |
| autologous dendritic cells pulsed with killed ovarian cancer cells and matured by TLR3 ligand ex vivo EU/3/18/2009 |
Treatment of ovarian cancer | Withdrawn | 16 Apr 2018 |
| adeno-associated viral vector serotype 9 encoding miRNA against human superoxide dismutase 1 EU/3/18/2008 |
Treatment of amyotrophic lateral sclerosis | Active (positive) | 16 Apr 2018 |
| adeno-associated viral vector serotype 8 containing the human acid alpha-glucosidase gene EU/3/18/2007 |
Treatment of glycogen storage disease type II (Pompe's disease) | Withdrawn | 16 Apr 2018 |
| autologous skeletal myoblasts expanded ex vivo - |
Treatment of oculopharyngeal muscular dystrophy | Negative | 13 Apr 2018 |
| tazemetostat EU/3/18/2006 7 clinical trials → |
Treatment of malignant mesothelioma | Withdrawn | 21 Mar 2018 |
| tazemetostat EU/3/18/2005 7 clinical trials → |
Treatment of follicular lymphoma | Active (positive) | 21 Mar 2018 |
| tazemetostat EU/3/18/2004 7 clinical trials → |
Treatment of diffuse large B-cell lymphoma | Withdrawn | 21 Mar 2018 |
| ribavirin EU/3/18/2002 |
Treatment of Crimean-Congo haemorrhagic fever | Active (positive) | 21 Mar 2018 |
| recombinant modified ricin toxin A-chain subunit EU/3/18/2001 |
Prevention of ricin poisoning | Active (positive) | 21 Mar 2018 |
| recombinant human acid alpha-glucosidase EU/3/18/2000 |
Treatment of glycogen storage disease type II (Pompe's disease) | Withdrawn | 21 Mar 2018 |
| recombinant adeno-associated viral vector containing a codon-optimized Padua derivative of human coagulation factor IX cDNA (etranacogene dezaparvovec) EU/3/18/1999 · now authorised as Hemgenix |
Treatment of haemophilia B | Active (positive) | 21 Mar 2018 |
| patidegib EU/3/18/1998 |
Treatment of naevoid basal-cell carcinoma syndrome (Gorlin syndrome) | Active (positive) | 21 Mar 2018 |
| Miransertib EU/3/18/1997 |
Treatment of Proteus syndrome | Active (positive) | 21 Mar 2018 |
| melatonin EU/3/18/1996 23 clinical trials → |
Treatment of neonatal encephalopathy | Active (positive) | 21 Mar 2018 |
| Larotrectinib EU/3/18/1995 9 clinical trials → |
Treatment of salivary gland cancer | Withdrawn | 21 Mar 2018 |
| Ivosidenib EU/3/18/1994 · now authorised as Tibsovo 26 clinical trials → |
Treatment of biliary tract cancer | Active (positive) | 21 Mar 2018 |
| Gemfibrozil EU/3/18/1993 |
Treatment of neuronal ceroid lipofuscinosis | Active (positive) | 21 Mar 2018 |
| Docosahexaenoic acid ethyl ester EU/3/18/1991 |
Treatment of sickle cell disease | Active (positive) | 21 Mar 2018 |
| dimethyl fumarate EU/3/18/1990 7 clinical trials → |
Treatment of Friedreichโs ataxia | Active (positive) | 21 Mar 2018 |
| (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 EU/3/18/1989 |
Treatment of C3 glomerulopathy | Withdrawn | 21 Mar 2018 |
| melatonin - 23 clinical trials → |
Treatment of non-traumatic subarachnoid haemorrhage | Negative | 15 Mar 2018 |
| melatonin - 23 clinical trials → |
Treatment of partial deep dermal and full-thickness burns | Negative | 13 Mar 2018 |
| (R)-2-(5-cyano-2-(6-(methoxycarbonyl)-7-methyl-3-oxo-8-(3-(trifluoromethyl)phenyl)-2,3,5,8-tetrahydro-[1,2,4]triazolo[4,3-a]pyrimidine-5-yl)phenyl)-N,N,N-trimeโฆ EU/3/18/1988 |
Treatment of cystic fibrosis | Active (positive) | 22 Feb 2018 |
| vocimagene amiretrorepvec EU/3/18/1987 |
Treatment of glioma | Active (positive) | 22 Feb 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.