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 |
|---|---|---|---|
| humanised IgG1 kappa monoclonal antibody against pregnancy-associated plasma protein A EU/3/25/3161 |
Treatment of autosomal dominant polycystic kidney disease | Active (positive) | 9 Dec 2025 |
| orvepitant maleate EU/3/25/3159 |
Treatment of idiopathic pulmonary fibrosis | Withdrawn | 21 Nov 2025 |
| resecabtagene autoleucel EU/3/25/3158 |
Treatment of systemic sclerosis | Active (positive) | 21 Nov 2025 |
| Infigratinib EU/3/25/3157 3 clinical trials → |
Treatment of hypochondroplasia | Active (positive) | 21 Nov 2025 |
| Felzartamab EU/3/25/3156 6 clinical trials → |
Treatment of primary membranous nephropathy | Active (positive) | 21 Nov 2025 |
| zanidatamab EU/3/25/3155 14 clinical trials → |
Treatment of oesophageal cancer | Active (positive) | 21 Nov 2025 |
| antisense oligonucleotide against ABCA4 pre-mRNA EU/3/25/3154 |
Treatment of non-syndromic inherited retinal dystrophies due to defects in the ABCA4 gene | Active (positive) | 21 Nov 2025 |
| vopimetostat EU/3/25/3153 |
Treatment of pancreatic cancer | Active (positive) | 21 Nov 2025 |
| 2'-O-(2-Methoxyethyl) modified antisense oligonucleotide against MECP2 pre-mRNA EU/3/25/3152 |
Treatment of MECP2 duplication syndrome | Active (positive) | 21 Nov 2025 |
| leuconostoc citreum, strain G511, live EU/3/25/3151 |
Treatment of primary sclerosing cholangitis | Active (positive) | 21 Nov 2025 |
| surovatamig EU/3/25/3150 9 clinical trials → |
Treatment of B-lymphoblastic leukaemia/lymphoma | Active (positive) | 21 Nov 2025 |
| (S)-1-(2,2-Difluoroethyl)-3-(difluoromethyl)-N-(1-(3-(2-methoxypyridin-4-yl)-1,2,4-oxadiazol-5-yl)ethyl)-1H-pyrazole-5-carboxamide EU/3/25/3149 |
Treatment of KCNT1-related epilepsy and neurodevelopmental disorders | Active (positive) | 21 Nov 2025 |
| 2-(3-Chlorophenyl)-N-[6-(trifluoromethyl)-1,3-benzothiazol-2-yl]acetamide EU/3/25/3148 |
Treatment of amyotrophic lateral sclerosis | Active (positive) | 21 Nov 2025 |
| nangibotide EU/3/25/3147 |
Treatment of sickle cell disease | Active (positive) | 21 Nov 2025 |
| autologous peripheral blood-derived CD34+ haematopoietic stem and progenitor cells transduced with a lentiviral vector containing the murine Glb1 gene EU/3/25/3146 |
Treatment of Glb1-related disorders | Active (positive) | 22 Oct 2025 |
| soquelitinib EU/3/25/3145 3 clinical trials → |
Treatment of peripheral T-cell lymphoma | Active (positive) | 20 Oct 2025 |
| fenfluramine hydrochloride EU/3/25/3144 |
Treatment of Rett syndrome | Active (positive) | 20 Oct 2025 |
| ceruloplasmin EU/3/25/3143 |
Treatment of congenital aceruloplasminemia | Active (positive) | 20 Oct 2025 |
| mezagitamab EU/3/25/3142 4 clinical trials → |
Treatment of primary IgA nephropathy | Active (positive) | 20 Oct 2025 |
| glial cell line-derived neurotrophic factor;sodium butyrate EU/3/25/3141 |
Treatment of Hirschsprung's disease | Active (positive) | 20 Oct 2025 |
| risvutatug rezetecan EU/3/25/3140 2 clinical trials → |
Treatment of pulmonary neuroendocrine carcinoma | Active (positive) | 20 Oct 2025 |
| bevantolol hydrochloride EU/3/25/3139 |
Treatment of Huntington's disease | Active (positive) | 20 Oct 2025 |
| sutacimig EU/3/25/3138 |
Treatment of Glanzmann thrombasthenia | Active (positive) | 20 Oct 2025 |
| Unasnemab EU/3/25/3137 |
Treatment of amyotrophic lateral sclerosis | Active (positive) | 20 Oct 2025 |
| ozisiran EU/3/25/3136 |
Treatment of Glanzmann thrombasthenia | Active (positive) | 20 Oct 2025 |
| iniparib EU/3/25/3175 |
Treatment of glioma | Active (positive) | 12 Sep 2025 |
| cyclo(arginyl-glycyl-aspartyl-D-tyrosyl-lysyl)-nizaracianine-1 EU/3/25/3173 |
Diagnosis of pancreatic cancer | Active (positive) | 12 Sep 2025 |
| biotin EU/3/25/3172 |
Treatment of biotin-thiamine-responsive basal ganglia disease (BTBGD) | Active (positive) | 12 Sep 2025 |
| puliretgene parvec EU/3/25/3171 |
Treatment of inherited retinal dystrophy due to dysfunction in the CYP4V2-gene | Active (positive) | 12 Sep 2025 |
| domvanalimab EU/3/25/3168 11 clinical trials → |
Treatment of oesophageal cancer | Active (positive) | 12 Sep 2025 |
| zimberelimab EU/3/25/3167 28 clinical trials → |
Treatment of gastric cancer | Active (positive) | 12 Sep 2025 |
| autologous peripheral blood-derived CD34+ haematopoietic stem and progenitor cells transduced with a lentiviral vector containing the human MAN2B1 gene EU/3/25/3165 |
Treatment of alpha-mannosidosis | Active (positive) | 12 Sep 2025 |
| human IgG1 monoclonal antibody against tumor necrosis factor receptor superfamily member 1B EU/3/25/3164 |
Treatment of cutaneous T-cell lymphoma | Active (positive) | 12 Sep 2025 |
| zimberelimab EU/3/25/3163 28 clinical trials → |
Treatment of oesophageal cancer | Active (positive) | 12 Sep 2025 |
| adeno-associated virus vector serotype 6.2 containing human TERT gene EU/3/25/3160 |
Treatment of idiopathic pulmonary fibrosis | Active (positive) | 12 Sep 2025 |
| methotrexate EU/3/25/3135 97 clinical trials → |
Treatment of primary large B-cell lymphoma of immune-privileged sites | Active (positive) | 22 Aug 2025 |
| sonpiretigene isteparvovec EU/3/25/3134 |
Treatment of non-syndromic macular dystrophy | Active (positive) | 22 Aug 2025 |
| sonpiretigene isteparvovec EU/3/25/3132 |
Treatment of non-syndromic inherited retinal dystrophies of the cone-dominant phenotype | Active (positive) | 22 Aug 2025 |
| sonpiretigene isteparvovec EU/3/25/3131 |
Treatment of non-syndromic inherited retinal dystrophies of the rod-dominant phenotype | Active (positive) | 22 Aug 2025 |
| sonpiretigene isteparvovec EU/3/25/3130 |
Treatment of syndromic inherited retinal dystrophies of the cone-dominant phenotype | Active (positive) | 22 Aug 2025 |
| sonpiretigene isteparvovec EU/3/25/3129 |
Treatment of syndromic inherited retinal dystrophies of the rod-dominant phenotype | Active (positive) | 22 Aug 2025 |
| 7-ethyl-10-hydroxycamptothecin EU/3/25/3128 |
Treatment of pancreatic cancer | Active (positive) | 22 Aug 2025 |
| varegacestat EU/3/25/3127 |
Treatment of soft tissue sarcoma | Active (positive) | 22 Aug 2025 |
| temozolomide EU/3/25/3125 97 clinical trials → |
Treatment of glioma | Active (positive) | 22 Aug 2025 |
| mezagitamab EU/3/25/3124 4 clinical trials → |
Treatment of immune thrombocytopenia | Active (positive) | 22 Aug 2025 |
| leniolisib EU/3/25/3123 4 clinical trials → |
Treatment of common variable immunodeficiency (CVID) | Active (positive) | 22 Aug 2025 |
| darovasertib EU/3/25/3122 5 clinical trials → |
Treatment of uveal melanoma | Active (positive) | 22 Aug 2025 |
| brepocitinib EU/3/25/3121 2 clinical trials → |
Treatment of idiopathic inflammatory myopathy | Active (positive) | 22 Aug 2025 |
| rilzabrutinib EU/3/25/3120 9 clinical trials → |
Treatment of immunoglobulin G4-related disease | Active (positive) | 22 Aug 2025 |
| zilurgisertib EU/3/25/3119 |
Treatment of fibrodysplasia ossificans progressiva | Active (positive) | 22 Aug 2025 |
| mRNA encoding Cas12HF endonuclease, Single guide RNA against the human HAO1 gene EU/3/25/3118 |
Treatment of primary hyperoxaluria | Active (positive) | 22 Aug 2025 |
| 3-tert-butyl-N-{(1R)-1-[4-(6-{6-[4-({1-[4-(2,4-dioxo-1,3-diazinan-1-yl)phenyl]piperidin-4-yl}methyl)piperazin-1-yl]pyridin-3-yl}-7H-pyrrolo[2,3-d]pyrimidin-4-y… EU/3/25/3117 |
Treatment of lymphoplasmacytic lymphoma | Active (positive) | 22 Aug 2025 |
| gildeuretinol, gildeuretinol acetate EU/3/25/3115 |
Treatment of non-syndromic inherited retinal dystrophies due to defects in the ABCA4 gene | Active (positive) | 22 Aug 2025 |
| RNA editing antisense oligonucleotide against the Z mutation of the human SERPINA1 mRNA transcript, sodium salt EU/3/25/3114 |
Treatment of alpha-1 antitrypsin deficiency | Withdrawn | 22 Aug 2025 |
| acetylcysteine amide EU/3/25/3113 |
Treatment of hereditary cerebral amyloid angiopathies | Active (positive) | 22 Aug 2025 |
| lentiviral vector containing the human MMUT transgene EU/3/25/3112 |
Treatment of methylmalonic acidaemia | Active (positive) | 22 Aug 2025 |
| pegtarazimod EU/3/25/3111 |
Treatment of graft-versus-host disease | Active (positive) | 22 Aug 2025 |
| emugrobart EU/3/25/3110 |
Treatment of spinal muscular atrophy | Active (positive) | 22 Aug 2025 |
| adeno-associated virus serotype 9 vector containing the human NDP gene EU/3/25/3109 |
Treatment of NDP gene-related disorders | Active (positive) | 22 Aug 2025 |
| cladribine EU/3/25/3108 7 clinical trials → |
Treatment of acute myeloid leukaemia | Active (positive) | 22 Aug 2025 |
| allogeneic cultured postnatal thymus-derived tissue EU/3/25/3107 |
Treatment of congenital athymia | Active (positive) | 22 Aug 2025 |
| RNA, [P-deoxy-P-(dimethylamino)](2',3'-dideoxy-2',3'-imino-2',3'-seco)(2'a->5') (C-A-G-C-A-G-C-A-G-C-A-G-C-A-G-C-A-G-C-A-[2'a-[39-[[1-acetyl-L-prolyl-L-lysyl-L… EU/3/25/3105 |
Treatment of dystrophic myotonia | Active (positive) | 22 Aug 2025 |
| 4-(2-{[2-Amino-6-(3-chloro-2-methylphenyl)pyrimidin-4-yl]amino}ethyl)benzene-1-sulfonamide EU/3/25/3104 |
Treatment of acute myeloid leukaemia | Active (positive) | 22 Aug 2025 |
| admilparant EU/3/25/3103 3 clinical trials → |
Treatment of idiopathic pulmonary fibrosis | Active (positive) | 22 Aug 2025 |
| mecasermin rinfabate EU/3/25/3102 |
Prevention of bronchopulmonary dysplasia | Active (positive) | 22 Aug 2025 |
| botaretigene sparoparvovec EU/3/16/1715 |
Treatment of inherited retinal dystrophies due to defects in the RPGR gene | Active (positive) | 23 Jul 2025 |
| adeno-associated viral vector serotype 9 containing the human CTNNB1 gene EU/3/25/3101 |
Treatment of autosomal dominant polycystic kidney disease | Active (positive) | 18 Jul 2025 |
| Elafibranor EU/3/25/3100 5 clinical trials → |
Treatment of primary sclerosing cholangitis | Active (positive) | 18 Jul 2025 |
| telitacicept EU/3/25/3099 22 clinical trials → |
Treatment of myasthenia gravis | Active (positive) | 18 Jul 2025 |
| human bispecific monoclonal antibody targeting ALK1 and BMPRII EU/3/25/3098 |
Treatment of hereditary haemorrhagic telangiectasia | Active (positive) | 18 Jul 2025 |
| humanised IgG1 kappa monoclonal antibody against tyrosine-protein phosphatase non-receptor type substrate 1, signal-regulatory protein beta-1 and signal-regula… EU/3/25/3097 |
Treatment of haemophagocytic lymphohistiocytosis | Active (positive) | 18 Jul 2025 |
| aglatimagene besadenovec EU/3/25/3096 5 clinical trials → |
Treatment of pancreatic cancer | Active (positive) | 18 Jul 2025 |
| luminol monosodium EU/3/25/3095 |
Treatment of Duchenne muscular dystrophy | Active (positive) | 18 Jul 2025 |
| resecabtagene autoleucel EU/3/25/3094 |
Treatment of idiopathic inflammatory myopathy | Active (positive) | 18 Jul 2025 |
| octreotide hydrochloride EU/3/25/3093 3 clinical trials → |
Treatment of autosomal dominant polycystic kidney disease | Active (positive) | 18 Jul 2025 |
| curcumin EU/3/25/3092 |
Treatment of Charcot-Marie-Tooth disease | Active (positive) | 18 Jul 2025 |
| allogeneic peripheral blood-derived T-cells, fratricide-resistant, transduced with a lentivirus vector expressing a chimeric antigen receptor against CD7 EU/3/25/3091 |
Treatment of acute lymphoblastic leukaemia | Active (positive) | 18 Jul 2025 |
| avutometinib;defactinib EU/3/25/3090 5 clinical trials → |
Treatment of ovarian cancer | Active (positive) | 18 Jul 2025 |
| methotrexate EU/3/25/3089 97 clinical trials → |
Treatment of non-syndromic inherited retinal dystrophies of the rod-dominant phenotype | Active (positive) | 18 Jul 2025 |
| nuvisertib EU/3/25/3088 |
Treatment of myelofibrosis | Active (positive) | 18 Jul 2025 |
| phosphorodiamidate morpholino oligonucleotide against the CUG repeat expansion of the DMPK gene mRNA transcript, conjugated to a cell penetrating peptide EU/3/25/3087 |
Treatment of dystrophic myotonia | Active (positive) | 18 Jul 2025 |
| ribavirin EU/3/25/3086 |
Treatment of hepatitis E | Active (positive) | 18 Jul 2025 |
| Ivosidenib EU/3/25/3085 26 clinical trials → |
Treatment of myelodysplastic syndromes | Active (positive) | 18 Jul 2025 |
| palmitoyl-conjugated tricyclo-DNA antisense oligonucleotide 5'-Palm-C6-*GGA GAT GgC AGT TTC-3 EU/3/25/3084 |
Treatment of Duchenne muscular dystrophy | Active (positive) | 18 Jul 2025 |
| sonlicromanol hydrochloride EU/3/25/3083 2 clinical trials → |
Treatment of inherited mitochondrial oxidative phosphorylation defects | Active (positive) | 18 Jul 2025 |
| zanidatamab EU/3/21/2458 14 clinical trials → |
Treatment of biliary tract cancer | Active (positive) | 27 Jun 2025 |
| Elesclomol-copper EU/3/23/2858 |
Treatment of Menkes disease | Active (positive) | 22 Jun 2025 |
| 2-[[Hydroxy[(R)-2-[((5Z,8Z,11Z,14Z)-eicosa-5,8,11,14-tetraenoyl)oxy]-3-(octadecyloxy)propoxy]phosphoryl]oxy]ethan-1-amine EU/3/25/3081 |
Treatment of Rett syndrome | Active (positive) | 20 Jun 2025 |
| 2-(2-(2-[2-(4-Benzothiazol-2-yl-phenoxy)-ethoxy]-ethoxy)-ethoxy)-ethanol EU/3/25/3080 |
Treatment of amyotrophic lateral sclerosis | Active (positive) | 20 Jun 2025 |
| 3-(Carbamoylamino)-5-[2-(3-fluorophenyl)ethynyl]-N-[(3S)-piperidin-3-yl]thiophene-2-carboxamide hydrochloric acid EU/3/25/3079 |
Treatment of acute myeloid leukaemia | Active (positive) | 20 Jun 2025 |
| 3-((4-(1-((1-(6-(((S)-2,6-Dioxopiperidin-3-yl)carbamoyl)pyridin-3-yl)piperidin-4-yl)methyl)piperidin-4-yl)phenyl)amino)-5-((R)-3-(3-methyl-2-oxoimidazolidin-1-… EU/3/25/3078 |
Treatment of lymphoplasmacytic lymphoma | Active (positive) | 20 Jun 2025 |
| Humanised IgG1 monoclonal antibody against adrenocorticotropic hormone EU/3/25/3077 |
Treatment of congenital adrenal hyperplasia | Active (positive) | 20 Jun 2025 |
| 4-Amino-1-[(2R,4S,5R)-4-hydroxy-5-(hydroxymethyl)oxolan-2-yl]-5-(hydroxymethyl)pyrimidin-2-one EU/3/25/3076 |
Treatment of glioma | Active (positive) | 20 Jun 2025 |
| Exidavnemab EU/3/25/3075 |
Treatment of multiple system atrophy | Active (positive) | 20 Jun 2025 |
| Alpha-N-acetyl glucosaminidase fused to a humanised monoclonal antibody against transferrin receptor EU/3/25/3073 |
Treatment of mucopolysaccharidosis type IIIB (Sanfilippo B syndrome) | Active (positive) | 20 Jun 2025 |
| Dipalmitoyl hydroxyproline EU/3/25/3072 |
Treatment of Netherton syndrome | Active (positive) | 20 Jun 2025 |
| beta-lapachone EU/3/25/3071 |
Treatment of primary sclerosing cholangitis | Active (positive) | 20 Jun 2025 |
| empasiprubart EU/3/25/3070 10 clinical trials → |
Treatment of multifocal motor neuropathy | Active (positive) | 20 Jun 2025 |
| Humanised IgG4 bispecific monoclonal antibody against sclerostin and dickkopf-related protein 1 EU/3/25/3069 |
Treatment of osteogenesis imperfecta | Active (positive) | 20 Jun 2025 |
| 1-(3-Bromobenzyl)-N3-(3,4-dichlorophenyl)-1H-1,2,4-triazole-3,5-diamine EU/3/25/3068 |
Treatment of radiation induced maculopathy | Active (positive) | 20 Jun 2025 |
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.