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 |
|---|---|---|---|
| sodium 2-(3'(-3-(1-(4-(tert-butyl)benzyl)-4-ethyl-5-oxo-4,5-dihydro-1H-1,2,4-triazol-3-yl)propyl)-4-ethoxy-[1,1'-biphenyl]-3-yl)acetate EU/3/25/3067 |
Treatment of hepatocellular carcinoma | Active (positive) | 20 Jun 2025 |
| iloprost EU/3/25/3066 4 clinical trials → |
Treatment of systemic sclerosis | Active (positive) | 20 Jun 2025 |
| bezuclastinib EU/3/25/3065 3 clinical trials → |
Treatment of mastocytosis | Active (positive) | 20 Jun 2025 |
| recombinant human ectonucleotide pyrophosphatase/phosphodiesterase 1 fused to the Fc fragment of IgG1 EU/3/25/3064 |
Treatment of calciphylaxis | Active (positive) | 22 May 2025 |
| Induced pluripotent stem cells-derived myogenic progenitor cells EU/3/25/3063 |
Treatment of Becker muscular dystrophy | Active (positive) | 22 May 2025 |
| adeno-associated viral vector serotype 2i8 containing the human LAMP2 isoform B transgene EU/3/25/3062 |
Treatment of Danon disease | Active (positive) | 22 May 2025 |
| radiprodil EU/3/25/3061 4 clinical trials → |
Treatment of GRIN-related neurodevelopmental disorder | Active (positive) | 22 May 2025 |
| Humanised IgG1 monoclonal antibody against human epidermal growth factor receptor 2 EU/3/25/3060 |
Treatment of gastric cancer | Active (positive) | 22 May 2025 |
| 1-{4-[2-(5-ethoxymethyl-2-methyl-phenylamino)-oxazol-5-yl]-phenyl}-imidazolidin-2-one EU/3/25/3059 |
Treatment of acute myeloid leukaemia | Active (positive) | 22 May 2025 |
| tranilast EU/3/25/3058 2 clinical trials → |
Treatment of idiopathic pulmonary fibrosis | Active (positive) | 22 May 2025 |
| Equine polyclonal immunoglobulin F(ab')2 fragments against ricin EU/3/25/3057 |
Treatment of ricin poisoning | Withdrawn | 22 May 2025 |
| Doxorubicin, liposomal, pegylated EU/3/25/3056 126 clinical trials → |
Treatment of soft-tissue sarcoma | Active (positive) | 22 May 2025 |
| florbetaben (18F) EU/3/25/3055 |
Diagnosis of ATTR amyloidosis | Active (positive) | 22 May 2025 |
| taladegib EU/3/25/3054 2 clinical trials → |
Treatment of idiopathic pulmonary fibrosis | Active (positive) | 22 May 2025 |
| tertomotide EU/3/25/3053 |
Treatment of progressive supranuclear palsy | Active (positive) | 22 May 2025 |
| Parahydroxybenzoic acid EU/3/25/3052 |
Treatment of primary ubiquinone deficiency | Active (positive) | 16 Apr 2025 |
| sirolimus EU/3/25/3051 32 clinical trials → |
Treatment of familial adenomatous polyposis (FAP) | Active (positive) | 16 Apr 2025 |
| small-interfering RNA against soluble fms-like tyrosine kinase-1 e15a mRNA, small-interfering RNA against soluble fms-like tyrosine kinase-1 i13 mRNA EU/3/25/3050 |
Treatment of pre-eclampsia | Active (positive) | 16 Apr 2025 |
| humanised IgG1 monoclonal antibody against muscle specific kinase EU/3/25/3049 |
Treatment of spinal muscular atrophy | Active (positive) | 16 Apr 2025 |
| Adeno-associated viral vector serotype LK03 containing the human CFI gene EU/3/25/3048 |
Treatment of primary IgA nephropathy | Active (positive) | 16 Apr 2025 |
| Fragment antibody targeting human TfR1 conjugated to phosphorodiamidate morpholino oligomer EU/3/25/3047 |
Treatment of Duchenne muscular dystrophy | Active (positive) | 16 Apr 2025 |
| vamikibart EU/3/25/3046 |
Treatment of non-infectious uveitis | Active (positive) | 16 Apr 2025 |
| bexmarilimab EU/3/25/3044 2 clinical trials → |
Treatment of myelodysplastic syndromes | Active (positive) | 25 Mar 2025 |
| povetacicept EU/3/25/3043 4 clinical trials → |
Treatment of primary IgA nephropathy | Active (positive) | 25 Mar 2025 |
| extract from Cannabis flower, containing high levels of cannabidiolic acid and EU/3/25/3042 |
Treatment of Rett syndrome | Active (positive) | 25 Mar 2025 |
| allopurinol EU/3/25/3041 7 clinical trials → |
Treatment of Marfan syndrome | Active (positive) | 25 Mar 2025 |
| deucrictibant monohydrate EU/3/25/3040 4 clinical trials → |
Treatment of bradykinin-mediated angioedema | Active (positive) | 25 Mar 2025 |
| single guide RNA containing a sequence complementary to human ALB locus gene, intron 1, target region, Ziclumeran EU/3/25/3039 |
Treatment of haemophilia B | Active (positive) | 25 Mar 2025 |
| adeno-associated virus vector serotype 8 containing the human F9 gene EU/3/25/3038 |
Treatment of haemophilia B | Active (positive) | 25 Mar 2025 |
| ixodes ricinus contact phase inhibitor EU/3/25/3037 |
Treatment of non-traumatic spontaneous intracerebral haemorrhage | Active (positive) | 25 Mar 2025 |
| autologous CD34+ cell enriched population containing haematopoietic stem and progenitor cells transduced ex vivo with a lentiviral vector encoding the human AD… EU/3/25/3034 |
Treatment of adenosine deaminase 2 deficiency (DADA2) | Active (positive) | 27 Feb 2025 |
| adeno-associated virus sector serotype rh74 containing the human SGCG gene EU/3/25/3031 |
Treatment of limb-girdle muscular dystrophy | Active (positive) | 27 Feb 2025 |
| paltusotine EU/3/25/3035 · now authorised as Palsonify |
Treatment of acromegaly | Active (positive) | 26 Feb 2025 |
| adeno-associated virus serotype rh.10 containing the human PKP2 gene EU/3/25/3033 |
Treatment of arrhythmogenic cardiomyopathy caused by pathogenic mutations in the PKP2 gene | Active (positive) | 26 Feb 2025 |
| (R)-(3-(2'-cyclopropyl-3-(hydroxymethyl)-[1,1'-biphenyl]-4-yl)pyrrolidin-1-yl)(5-hydroxy-6-methylpyridin-2-yl)methanone EU/3/25/3032 |
Treatment of Olmsted syndrome | Active (positive) | 26 Feb 2025 |
| hydroxocobalamin acetate EU/3/25/3030 |
Treatment of homocystinuria and/or methylmalonic acidaemia due to genetic defects of intracellular cobalamin processing | Active (positive) | 26 Feb 2025 |
| volixibat potassium EU/3/25/3029 2 clinical trials → |
Treatment of primary sclerosing cholangitis | Active (positive) | 26 Feb 2025 |
| epertinib EU/3/25/3028 |
Treatment of amyotrophic lateral sclerosis | Active (positive) | 26 Feb 2025 |
| tafasitamab EU/3/25/3027 13 clinical trials → |
Treatment of follicular lymphoma | Active (positive) | 26 Feb 2025 |
| adeno-associated virus vector serotype SNY001 containing the human PAH gene EU/3/25/3026 |
Treatment of hyperphenylalaninaemia | Active (positive) | 26 Feb 2025 |
| rimeporide EU/3/15/1478 |
Treatment of Duchenne muscular dystrophy | Active (positive) | 26 Feb 2025 |
EU/3/05/334 |
Treatment of X-linked hypohidrotic ectodermal dysplasia (Christ-Siemens-Touraine Syndrome) | Active (positive) | 26 Feb 2025 |
| autologous T cells transduced with lentiviral vector containing a chimeric antigen receptor directed against CD19 EU/3/24/3011 |
Treatment of myasthenia gravis | Active (positive) | 20 Feb 2025 |
| serplulimab EU/3/22/2731 84 clinical trials → |
Treatment of small cell lung cancer | Withdrawn | 20 Feb 2025 |
| seladelpar EU/3/17/1930 · now authorised as Lyvdelzi (previously Seladelpar Gilead) 2 clinical trials → |
Treatment of primary biliary cholangitis | Active (positive) | 20 Feb 2025 |
| ornithine phenylacetate EU/3/11/945 |
Treatment of acute liver failure | Active (positive) | 20 Feb 2025 |
| pelabresib monohydrate EU/3/20/2247 7 clinical trials → |
Treatment of myelofibrosis | Active (positive) | 17 Feb 2025 |
| sutimlimab EU/3/16/1609 · now authorised as Enjaymo |
Treatment of autoimmune haemolytic anaemia | Active (positive) | 11 Feb 2025 |
| Humanised IgG4 monoclonal antibody against C1q EU/3/23/2836 |
Treatment of Guillain-Barré syndrome | Active (positive) | 10 Feb 2025 |
| [4-(Methyl-1H-pyrazol-4-yl)-benzyl]-(6[7-(3-pyrrolidin-1-yl-propoxy)-imidazo[1,2-a]pyridin-3-yl]-pyrimidin-4-yl]-amine EU/3/24/3025 |
Treatment of gastrointestinal stromal tumours | Active (positive) | 16 Jan 2025 |
| clofutriben EU/3/24/3024 2 clinical trials → |
Treatment of Cushing's syndrome of endogenous origin | Active (positive) | 16 Jan 2025 |
| N-(2-Methoxyethyl)-6-methyl-N-[(3-methyl-2-thienyl)methyl]-2-oxo-1,2-dihydropyridine-4-carboxamide EU/3/24/3023 |
Treatment of glycogen storage disease type IV | Active (positive) | 16 Jan 2025 |
| allogenic umbilical cord-derived osteoblast cells EU/3/24/3022 |
Treatment of non-traumatic osteonecrosis | Active (positive) | 16 Jan 2025 |
| camostat mesilate EU/3/24/3021 |
Treatment of chronic pancreatitis | Active (positive) | 16 Jan 2025 |
| adeno-associated virus serotype 9 containing the human RPE65 gene EU/3/24/3020 |
inherited retinal dystrophy due to defects in the RPE65 gene | Active (positive) | 16 Jan 2025 |
| rilzabrutinib EU/3/24/3019 9 clinical trials → |
Treatment of autoimmune haemolytic anaemia | Active (positive) | 16 Jan 2025 |
| alvelestat EU/3/24/3018 |
Treatment of congenital alpha-1 antitrypsin deficiency | Active (positive) | 16 Jan 2025 |
| volixibat potassium EU/3/24/3017 2 clinical trials → |
Treatment of primary biliary cholangitis | Active (positive) | 16 Jan 2025 |
| Elraglusib EU/3/24/3016 |
Treatment of soft tissue sarcoma | Active (positive) | 16 Jan 2025 |
| Elraglusib EU/3/24/3015 |
Treatment of pancreatic cancer | Active (positive) | 16 Jan 2025 |
| arsenic trioxide EU/3/24/3014 11 clinical trials → |
Treatment of acute promyelocytic leukaemia | Active (positive) | 13 Dec 2024 |
| mRNA encoding Cas9-deaminase, single guide RNA against the human TGM1 gene EU/3/24/3013 |
Treatment of autosomal recessive congenital ichthyosis | Active (positive) | 13 Dec 2024 |
| tobevibart EU/3/24/3012 5 clinical trials → |
Treatment of hepatitis delta virus infection | Active (positive) | 13 Dec 2024 |
| elebsiran EU/3/24/3010 7 clinical trials → |
Treatment of hepatitis delta virus infection | Active (positive) | 13 Dec 2024 |
| 2'-O-4'-C-(S)-ethyl-P-thioadenylyl-(3'-O->5'-O)-2'-O-4'-C-(S)-ethyl-P-thioguanylyl-(3'-O->5'-O)-2'-O-methyl-P-thiocytidylyl-(3'-O->5'-O)-2'-fluoro-P-thioadenyl… EU/3/24/3009 |
Treatment of autosomal dominant polycystic kidney disease | Active (positive) | 13 Dec 2024 |
| diazoxide choline EU/3/24/3008 |
Treatment of glycogen storage disease type I | Active (positive) | 13 Dec 2024 |
| diazoxide choline EU/3/24/3008 |
Treatment of glycogen storage disease type I | Active (positive) | 13 Dec 2024 |
| Davunetide EU/3/24/3007 |
Treatment of activity-dependent neuroprotective protein (ADNP) syndrome | Active (positive) | 13 Dec 2024 |
| adeno-associated virus serotype 5 containing the human RORA gene EU/3/24/3006 |
Treatment of inherited retinal dystrophies due to dysfunction in the ABCA4 gene | Active (positive) | 13 Dec 2024 |
| 4-(4-(2-(Diethylamino)ethoxy)phenyl)-1-(4-methoxybenzyl)-1H-1,2,3-triazol-5-amine EU/3/24/3005 |
Treatment of neurofibromatosis type 2 | Active (positive) | 13 Dec 2024 |
| monepantel EU/3/24/3004 |
Treatment of amyotrophic lateral sclerosis | Active (positive) | 13 Dec 2024 |
| allogeneic cardiosphere-derived cells EU/3/24/3003 |
Treatment of Duchenne muscular dystrophy | Active (positive) | 13 Dec 2024 |
| Felzartamab EU/3/24/3002 6 clinical trials → |
Treatment of in solid organ transplantation | Active (positive) | 13 Dec 2024 |
| votoplam EU/3/24/3001 2 clinical trials → |
Treatment of Huntington's disease | Active (positive) | 13 Dec 2024 |
| roginolisib EU/3/24/3000 5 clinical trials → |
Treatment of uveal melanoma | Active (positive) | 13 Dec 2024 |
| mitapivat sulfate EU/3/24/2999 12 clinical trials → |
Treatment of sickle cell disease | Active (positive) | 13 Dec 2024 |
| vosoritide EU/3/24/2998 9 clinical trials → |
Treatment of hypochondroplasia | Active (positive) | 13 Dec 2024 |
| Colistimethate sodium EU/3/24/2997 3 clinical trials → |
Treatment of cystic fibrosis | Active (positive) | 13 Dec 2024 |
| divesiran EU/3/24/2996 |
Treatment of polycythaemia vera | Active (positive) | 13 Dec 2024 |
| Curcumin E100, Resveratrol EU/3/24/2995 2 clinical trials → |
Treatment of Dercum disease | Active (positive) | 11 Nov 2024 |
| Felzartamab EU/3/24/2994 6 clinical trials → |
Treatment of primary IgA nephropathy | Active (positive) | 11 Nov 2024 |
| 4-[[(4-Methoxyphenyl)thio]methyl]-N,N-dimethyl-1H-1,2,3-triazole-1-ethanamine EU/3/24/2993 |
Treatment of non-syndromic inherited retinal dystrophies with a rod-dominant phenotype | Active (positive) | 11 Nov 2024 |
| 3-chloro-4-fluorophenyl-(4-fluoro-4-(((5-methylpyrimidin-2-ylmethyl)amino)methyl)piperidin-1yl)methanone EU/3/24/2992 |
Treatment of fragile X syndrome | Active (positive) | 11 Nov 2024 |
| coramitug EU/3/24/2991 |
Treatment of ATTR amyloidosis | Active (positive) | 11 Nov 2024 |
| Mesenchymal stem cells-derived small extracellular vesicles loaded with siRNA against phosphatase and tensin homolog EU/3/24/2990 |
Treatment of spinal cord injury | Active (positive) | 11 Nov 2024 |
| avenciguat EU/3/24/2989 |
Treatment of systemic sclerosis | Withdrawn | 11 Nov 2024 |
| 7-ethyl-10-hydroxy-camptothecin EU/3/24/2988 |
Treatment of glioma | Active (positive) | 11 Nov 2024 |
| 3-(5-(2-Hydroxy-2-methylpropoxy)-6-methylpyrazin-2-yl)-1H-indole-7-carbonitrile EU/3/24/2987 |
Treatment of X-linked spinal and bulbar muscular atrophy (Kennedy's disease) | Active (positive) | 10 Nov 2024 |
| H-L-Tryptophanyl-L-seryl-glycyl-L-tryptophanyl-L-seryl-L-seryl-L-cysteinyl-L-seryl-L-arginyl-L-seryl-L-cysteinyl-glycyl-OH (disulfide bond), acetate salt EU/3/24/2986 |
Treatment of amyotrophic lateral sclerosis | Active (positive) | 10 Nov 2024 |
| atacicept EU/3/24/2985 4 clinical trials → |
Treatment of primary IgA nephropathy | Active (positive) | 10 Nov 2024 |
| octreotide hydrochloride EU/3/24/2984 3 clinical trials → |
Treatment of autosomal dominant polycystic liver disease | Active (positive) | 10 Nov 2024 |
| Interleukin-12, human, recombinant EU/3/24/2983 |
Treatment of cutaneous T-cell lymphoma | Active (positive) | 10 Nov 2024 |
| genetically modified human adenovirus encoding human PH20 hyaluronidase EU/3/24/2982 |
Treatment of retinoblastoma | Active (positive) | 10 Nov 2024 |
| sodium selenate EU/3/24/2981 |
Treatment of frontotemporal dementia | Active (positive) | 10 Nov 2024 |
| navenibart EU/3/24/2979 3 clinical trials → |
Treatment of hereditary angioedema | Active (positive) | 10 Nov 2024 |
| Ivosidenib EU/3/24/2978 26 clinical trials → |
Treatment of chondrosarcoma | Active (positive) | 10 Nov 2024 |
| glecirasib EU/3/24/2977 10 clinical trials → |
Treatment of pancreatic cancer | Active (positive) | 10 Nov 2024 |
| 4,9-Dimethyl-6-(4'-aminophenyl)-2H-furo[2,3-h]-1-benzopyran-2-one EU/3/24/2976 |
Treatment of cystic fibrosis | Active (positive) | 10 Nov 2024 |
| resminostat EU/3/23/2835 · now authorised as Kinselby |
Treatment of cutaneous T-cell lymphoma | Active (positive) | 13 Oct 2024 |
| 1-(4-((1R,2S,3R)-1,2,3,4-Tetrahydroxybutyl)-1H-imidazol-2-yl) ethanone oxime EU/3/23/2830 |
Treatment of pancreatic cancer | Active (positive) | 13 Oct 2024 |
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.