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 |
|---|---|---|---|
| adeno-associated virus vector serotype SAN011 encoding a microRNA against DMPK mRNA EU/3/24/2980 |
Treatment of dystrophic myotonia type 1 | Active (positive) | 11 Oct 2024 |
| (E)-2-((4-((4-Benzyl(ethyl)amino)phenyl)diazinyl)phenyl)amino-N,N,N-triethyl-2-oxoethan-1-aminium chloride EU/3/24/2975 |
Treatment of syndromic inherited retinal dystrophies of the rod-dominant phenotype | Active (positive) | 21 Aug 2024 |
| 4-Benzoyl-D-phenylalanyl-D-seryl-D-tryptophyl-D-seryl-2,3,4,5,6-pentafluoro-D-phenylalanyl-3-cyclohexyl-D-alanyl-D-arginyl-D-arginyl-D-arginyl-D-glutaminyl-D-a… EU/3/24/2974 |
Treatment of pancreatic cancer | Active (positive) | 21 Aug 2024 |
| olezarsen sodium EU/3/24/2973 · now authorised as Tryngolza 4 clinical trials → |
Treatment of familial chylomicronaemia syndrome | Active (positive) | 21 Aug 2024 |
| sodium valproate EU/3/24/2972 3 clinical trials → |
Treatment of pulmonary arterial hypertension | Active (positive) | 21 Aug 2024 |
| Humanised IgG1 monoclonal antibody against misfolded immunoglobulin G, fused with pan-amyloid-reactive peptide p5R EU/3/24/2970 |
Treatment of ATTR amyloidosis | Active (positive) | 21 Aug 2024 |
| (R)-(3-(2'-cyclopropyl-3-(hydroxymethyl)-[1,1'-biphenyl]-4-yl) pyrrolidin-1-yl)(5-fluoropyridin-2-yl)methanone EU/3/24/2968 |
Treatment of punctate palmoplantar keratoderma | Active (positive) | 21 Aug 2024 |
| adeno-associated virus vector serotype 9 containing the human GCDH gene EU/3/24/2967 |
Treatment of glutaric aciduria | Active (positive) | 21 Aug 2024 |
| bemarituzumab EU/3/24/2966 |
Treatment of gastric cancer | Withdrawn | 21 Aug 2024 |
| 4-(1H-Pyrrolo[2,3-b]pyridin-2-yl)phenol hydrochloride EU/3/24/2965 |
Prevention of acute liver failure | Active (positive) | 21 Aug 2024 |
| autologous CD34+ cells edited with a CRISPR/Cas9 system and transduced with an adeno-associated vector containing a codon-optimized version of WAS gene EU/3/24/2964 |
Treatment of Wiskott-Aldrich syndrome | Active (positive) | 21 Aug 2024 |
| Polihexanide EU/3/24/2963 |
Treatment of fungal keratitis | Active (positive) | 21 Aug 2024 |
| obrixtamig EU/3/24/2962 11 clinical trials → |
Treatment of extrapulmonary neuroendocrine carcinoma | Active (positive) | 21 Aug 2024 |
| obrixtamig EU/3/24/2961 11 clinical trials → |
Treatment of pulmonary neuroendocrine carcinoma | Active (positive) | 21 Aug 2024 |
| gallium (68Ga) boclatixafortide EU/3/24/2959 |
Diagnosis of marginal zone lymphoma | Active (positive) | 21 Aug 2024 |
| N,N'-([Cyclohexylmethylene]di-4,1-phenylene)bis(2-[1-pyrrolidinyl]acetamide) EU/3/24/2958 |
Treatment of Creutzfeldt-Jakob disease | Active (positive) | 25 Jul 2024 |
| adeno-associated virus vector serotype rh.10 containing the human FXN gene EU/3/24/2957 |
Treatment of Friedreich's ataxia | Active (positive) | 25 Jul 2024 |
| adeno-associated virus serotype A101 containing the codon-optimized human CFTRdeltaR EU/3/24/2956 |
Treatment of cystic fibrosis | Active (positive) | 25 Jul 2024 |
| heterologous swine glyco-humanised polyclonal antibody against T lymphocytes EU/3/24/2955 |
Treatment of peripheral T-cell lymphoma | Active (positive) | 25 Jul 2024 |
| sodium phenylacetate;ursodoxicoltaurine EU/3/24/2954 |
Treatment of Wolfram syndrome | Active (positive) | 25 Jul 2024 |
| cannabidiol acid methyl ester EU/3/24/2953 |
Treatment of Prader-Willi syndrome | Active (positive) | 25 Jul 2024 |
| N-[(1R)-1-[(S)-(2-Chloro-3-fluorophenyl)hydroxymethyl]butyl]-7-fluoro-2,3-dihydro-2-oxo-1H-indole-4-carboxamide EU/3/24/2952 |
Treatment of congenital alpha-1 antitrypsin deficiency | Active (positive) | 25 Jul 2024 |
| befiradol fumarate EU/3/24/2951 |
Treatment of spinocerebellar ataxia | Active (positive) | 25 Jul 2024 |
| rivoceranib (mesilate) EU/3/24/2950 5 clinical trials → |
Treatment of hepatocellular carcinoma | Active (positive) | 25 Jul 2024 |
| 4-[[[4-[5-Chloro-2-[[trans-4-[[(1R)-2-methoxy-1-methyl-ethyl]amino]cyclohexyl]amino]-4-pyridinyl]-2-thiazolyl]amino]methyl]tetrahydro-2H-pyran-4-carbonitrile EU/3/24/2949 |
Treatment of peripheral T-cell lymphoma | Active (positive) | 25 Jul 2024 |
| adeno-associated virus vector serotype 8 containing the human TYMP gene (CpG-depleted) EU/3/24/2948 |
Treatment of mitochondrial neurogastrointestinal encephalomyopathy (MNGIE) | Active (positive) | 25 Jul 2024 |
| afamelanotide EU/3/24/2947 |
Treatment of variegate porphyria | Active (positive) | 25 Jul 2024 |
| urokinase, catalytic domain, fused with a single-chain antibody against von Willebrand factor EU/3/24/2946 3 clinical trials → |
Treatment of thrombotic thrombocytopenic purpura | Active (positive) | 25 Jul 2024 |
| emavusertib EU/3/24/2945 3 clinical trials → |
Treatment of primary large B-cell lymphoma of immune-privileged sites | Active (positive) | 25 Jul 2024 |
| (E)-2-((4-((4-Benzyl(ethyl)amino)phenyl)diazinyl)phenyl)amino-N,N,N-triethyl-2-oxoethan-1-aminium chloride EU/3/24/2944 |
Treatment of non-syndromic inherited retinal dystrophies of the rod-dominant phenotype | Active (positive) | 25 Jul 2024 |
| camrelizumab EU/3/24/2943 87 clinical trials → |
Treatment of hepatocellular carcinoma | Active (positive) | 25 Jul 2024 |
| omadacycline EU/3/23/2804 |
Treatment of nontuberculous mycobacterial lung disease | Active (positive) | 25 Jul 2024 |
| apilimod EU/3/24/2942 |
Treatment of amyotrophic lateral sclerosis | Active (positive) | 28 Jun 2024 |
| Ethyl(2E,4S)-4-({(2S)-2-[3-{[(5-methyl-1,2-oxazol-3-yl)carbonyl]amino}-2-oxopyridin-1(2H)-yl]pent-4-ynoyl}amino)-5-[(3S)-2-oxopyrrolidin-3-yl]pent-2-enoate, Po… EU/3/24/2941 |
Treatment of poliovirus infection | Active (positive) | 28 Jun 2024 |
| nizubaglustat EU/3/24/2940 5 clinical trials → |
Treatment of GM1 gangliosidosis | Active (positive) | 28 Jun 2024 |
| 2-(((2E)-3-(3-methoxy-4-(2-propyn-1-yloxy)phenyl)-1-oxo-2-propen-1-yl)amino)benzoic acid EU/3/24/2939 |
Treatment of systemic sclerosis | Active (positive) | 28 Jun 2024 |
| uridine triacetate EU/3/24/2938 |
Treatment of hereditary orotic aciduria | Active (positive) | 28 Jun 2024 |
| 4-[[[4-[5-Chloro-2-[[trans-4-[[(1R)-2-methoxy-1-methyl-ethyl]amino]cyclohexyl]amino]-4-pyridinyl]-2-thiazolyl]amino]methyl]tetrahydro-2H-pyran-4-carbonitrile EU/3/24/2937 |
Treatment of acute myeloid leukaemia | Active (positive) | 28 Jun 2024 |
| Complement factor H EU/3/24/2936 |
Treatment of C3 glomerulopathy | Active (positive) | 28 Jun 2024 |
| (R)-(3-(2'-cyclopropyl-3-(hydroxymethyl)-[1,1'-biphenyl]-4-yl) pyrrolidin-1-yl)(5-fluoropyridin-2-yl)methanone EU/3/24/2935 |
Treatment of pachyonychia congenita | Active (positive) | 28 Jun 2024 |
| L-methionine EU/3/24/2934 |
Treatment of pulmonary alveolar proteinosis | Active (positive) | 28 Jun 2024 |
| human IgG1 monoclonal antibody against hepatitis B virus, surface antigen EU/3/24/2933 |
Treatment of hepatitis D virus infection | Active (positive) | 28 Jun 2024 |
| topiramate EU/3/24/2932 3 clinical trials → |
Treatment of neonatal encephalopathy | Active (positive) | 28 Jun 2024 |
| nerandomilast EU/3/24/2931 6 clinical trials → |
Treatment of idiopathic pulmonary fibrosis | Withdrawn | 28 Jun 2024 |
| (R)-3-(1-Cyclopropyl-3-(2-fluoro-4-(trifluoromethoxy)benzyl)ureido)piperidine-1-carboxamide EU/3/24/2930 |
Treatment of hyperphenylalaninaemia | Active (positive) | 28 Jun 2024 |
| afatinib EU/3/24/2929 16 clinical trials → |
Treatment of complex regional pain syndrome | Active (positive) | 28 Jun 2024 |
| sargramostim EU/3/24/2928 6 clinical trials → |
Treatment of pulmonary alveolar proteinosis | Active (positive) | 28 Jun 2024 |
| enzastaurin hydrochloride EU/3/05/343 |
Treatment of glioma | Withdrawn | 27 May 2024 |
| adeno-associated virus serotype rh.74 vector containing the human PKP2 gene isoform EU/3/24/2927 |
Treatment of arrhythmogenic cardiomyopathy caused by pathogenic mutations in the PKP2 gene | Active (positive) | 24 May 2024 |
| afamelanotide EU/3/24/2926 |
Treatment of xeroderma pigmentosum | Active (positive) | 24 May 2024 |
| efineptakin alfa EU/3/24/2925 |
Treatment of acute radiation syndrome | Active (positive) | 24 May 2024 |
| autologous induced pluripotent stem cells-derived thymic epithelial cells transduced with a lentiviral vector encoding forkhead box protein N1 EU/3/24/2924 |
Treatment of DiGeorge syndrome | Active (positive) | 24 May 2024 |
| 7-ethyl-10-hydroxy-camptothecin;irinotecan hydrochloride trihydrate EU/3/24/2923 140 clinical trials → |
Treatment of small cell lung cancer | Active (positive) | 24 May 2024 |
| adeno-associated virus serotype 9 containing CRISPR/Cas13Y and guide RNA against the human MECP2 gene EU/3/24/2922 |
Treatment of MECP2 duplication syndrome | Active (positive) | 24 May 2024 |
| 2,4-Diamino-5-[[5-(1H-pyrazol-5-yl)-2-thienyl]methyl]-1H-pyrimidin-6-one EU/3/24/2921 |
Treatment of hyperphenylalaninaemia | Active (positive) | 24 May 2024 |
| allogeneic non-alloreactive T cells edited with mRNA to disrupt TRAC and CD52 genes and transduced with lentiviral vector expressing a chimeric antigen recepto… EU/3/24/2920 |
Treatment of acute lymphoblastic leukaemia | Active (positive) | 24 May 2024 |
| DNA plasmid containing the COL7A1 gene EU/3/24/2919 |
Treatment of epidermolysis bullosa | Active (positive) | 24 May 2024 |
| autologous CD34+ cells transduced with a lentiviral vector containing the human RAG1 gene EU/3/24/2918 |
Treatment of recombination-activating gene 1 (RAG1) deficiency | Active (positive) | 24 May 2024 |
| N-(1,3-Benzothiazol-2-yl)-4-[(2-hydroxy-3-methoxyphenyl)methylamino]benzenesulfonamide EU/3/24/2917 |
Treatment of Platelet-activating anti-Platelet factor 4 (PF4) disorders | Active (positive) | 24 May 2024 |
| quabodepistat EU/3/24/2916 |
Treatment of tuberculosis | Active (positive) | 24 May 2024 |
| serdexmethylphenidate EU/3/24/2915 |
Treatment of idiopathic hypersomnia | Withdrawn | 24 May 2024 |
| Humanised IgG1 (K322A) monoclonal antibody against disialoganglioside GD2 EU/3/24/2914 |
Treatment of neuroblastoma | Active (positive) | 16 Apr 2024 |
| acetylleucine EU/3/24/2913 |
Treatment of ataxia-oculomotor apraxia | Active (positive) | 16 Apr 2024 |
| autologous adipose-derived mesenchymal stem cells embedded in an extracellular matrix with hydroxyapatite/beta-tricalcium phosphate particles EU/3/24/2912 |
Treatment of congenital pseudarthrosis of long bones | Active (positive) | 16 Apr 2024 |
| sevasemten EU/3/24/2911 |
Treatment of Duchenne muscular dystrophy | Active (positive) | 16 Apr 2024 |
| annamycin EU/3/24/2910 |
Treatment of acute myeloid leukaemia | Active (positive) | 16 Apr 2024 |
| autologous adipose-derived stem cells EU/3/24/2909 |
Treatment of spinal cord injury | Active (positive) | 16 Apr 2024 |
| 4-(4-Methyl-piperazin-1-yl)-N-{6-[2-(4-trifluoromethyl-benzyloxy)-ethoxy]-1H-indazol-3-yl}-benzamide hemioxalate EU/3/24/2908 |
Treatment of acute myeloid leukaemia | Active (positive) | 21 Mar 2024 |
| sevasemten EU/3/24/2907 |
Treatment of Becker muscular dystrophy | Active (positive) | 21 Mar 2024 |
| zatolmilast EU/3/24/2906 |
Treatment of fragile X syndrome | Active (positive) | 21 Mar 2024 |
| autologous CD3-positive T-cells expressing a chimeric antigen receptor against B cell maturation agent EU/3/24/2905 |
Treatment of multiple myeloma | Active (positive) | 21 Mar 2024 |
| Mibavademab EU/3/24/2904 3 clinical trials → |
Treatment of Lawrence syndrome | Active (positive) | 21 Mar 2024 |
| Mibavademab EU/3/24/2903 3 clinical trials → |
Treatment of Berardinelli-Seip syndrome (congenital generalised lipodystrophy) | Active (positive) | 21 Mar 2024 |
| alrefimotide acetate;riletamotide acetate;tapderimotide acetate EU/3/24/2902 |
Treatment of mesothelioma | Withdrawn | 21 Mar 2024 |
| tislelizumab EU/3/20/2269 259 clinical trials → |
Treatment of hepatocellular carcinoma | Withdrawn | 6 Mar 2024 |
| andecaliximab EU/3/24/2901 2 clinical trials → |
Treatment of fibrodysplasia ossificans progressiva | Active (positive) | 19 Feb 2024 |
| autologous CD3-positive T-cells expressing a chimeric antigen receptor against B cell maturation agent EU/3/24/2900 |
Treatment of AL amyloidosis | Active (positive) | 19 Feb 2024 |
| Plerixafor EU/3/24/2899 |
Treatment of acute respiratory distress syndrome (ARDS) | Active (positive) | 19 Feb 2024 |
| donidalorsen EU/3/24/2898 2 clinical trials → |
Treatment of hereditary angioedema | Withdrawn | 19 Feb 2024 |
| repagermanium EU/3/24/2897 2 clinical trials → |
Treatment of focal segmental glomerulosclerosis | Active (positive) | 19 Feb 2024 |
| human IgG1 monoclonal antibody targeting amyloid transthyretin EU/3/24/2896 |
Treatment of ATTR amyloidosis | Active (positive) | 19 Feb 2024 |
| tinengotinib EU/3/24/2895 5 clinical trials → |
Treatment of biliary tract cancer | Active (positive) | 19 Feb 2024 |
| mRNA encoding the human CFTR gene EU/3/24/2894 |
Treatment of cystic fibrosis | Active (positive) | 19 Feb 2024 |
| paclitaxel EU/3/24/2893 400 clinical trials → |
Treatment of gastric cancer | Active (positive) | 19 Feb 2024 |
EU/3/24/2892 |
Treatment of small cell lung cancer | Active (positive) | 19 Feb 2024 |
| carboplatin EU/3/24/2891 545 clinical trials → |
Treatment of glioma | Active (positive) | 19 Feb 2024 |
| raludotatug deruxtecan EU/3/24/2890 |
Treatment of ovarian cancer | Active (positive) | 19 Feb 2024 |
| autologous CD34+ cells transduced with a lentiviral vector containing the human GAA gene EU/3/23/2751 |
Treatment of glycogen storage disease type II (Pompe's disease) | Active (positive) | 15 Feb 2024 |
| mitapivat sulfate EU/3/23/2889 · now authorised as Pyrukynd 12 clinical trials → |
Treatment of thalassaemia intermedia and major | Active (positive) | 12 Jan 2024 |
| recombinant human heparan-N-sulfatase EU/3/23/2888 |
Treatment of mucopolysaccharidosis type IIIA (Sanfilippo A syndrome) | Active (positive) | 12 Jan 2024 |
| zopapogene imadenovec EU/3/23/2887 |
Treatment of recurrent respiratory papillomatosis | Active (positive) | 12 Jan 2024 |
| human coagulation factor X EU/3/23/2886 |
Treatment of acquired factor X deficiency | Active (positive) | 12 Jan 2024 |
| motixafortide EU/3/23/2885 3 clinical trials → |
Treatment of patients undergoing haematopoietic stem cell transplantation | Active (positive) | 12 Jan 2024 |
| adeno-associated viral vector serotype 9 containing the human MECP2 gene, an intron encoding a miRNA generating sequence, and complementary miRNA binding sites EU/3/23/2884 |
Treatment of Rett syndrome | Active (positive) | 12 Jan 2024 |
| cutamesine EU/3/23/2883 |
alpha-thalassaemia X-linked intellectual disability syndrome (due to mutations in the ATRX gene) | Active (positive) | 12 Jan 2024 |
| Methyl-(1-{[6-{[(1S)-1-cyclopropylethyl]amino}-2-(pyrazolo[5,1-b][1,3]thiazol-7-yl)-pyrimidin-4-yl]carbonyl}piperidin-4-yl)carbamate mono(4-methylbenzenesulfon… EU/3/23/2882 |
Treatment of eosinophilic granulomatosis with polyangiitis | Active (positive) | 12 Jan 2024 |
| ziftomenib EU/3/23/2881 7 clinical trials → |
Treatment of acute myeloid leukaemia | Active (positive) | 12 Jan 2024 |
| adenine EU/3/23/2880 |
Treatment of epidermolysis bullosa | Active (positive) | 12 Jan 2024 |
| Ersodetug EU/3/23/2879 |
Treatment of insulinoma | Active (positive) | 12 Jan 2024 |
| golcadomide hydrochloride EU/3/23/2878 15 clinical trials → |
Treatment of diffuse large B-cell lymphoma | Active (positive) | 12 Jan 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.