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 |
|---|---|---|---|
| apilimod dimesilate EU/3/23/2877 |
Treatment of amyotrophic lateral sclerosis | Withdrawn | 12 Jan 2024 |
| tarlatamab EU/3/23/2876 · now authorised as Imdylltra 25 clinical trials → |
Treatment of small cell lung cancer | Active (positive) | 12 Jan 2024 |
| thiophene methylimidazole pentahydrogen EU/3/23/2875 |
Diagnosis of glioma | Active (positive) | 12 Jan 2024 |
| (4R)-3-(4-fluoro-2-hydroxyphenyl)-4-methyl-4,5-dihydro-1H-pyrazole-1-carboximidamide hydrochloride EU/3/23/2874 |
Treatment of systemic sclerosis | Withdrawn | 12 Jan 2024 |
| 3,3-Dimethyl-N-(6-methyl-5-{[2-(1-methyl-1H-pyrazol-4-yl)pyridine-4-yl]oxy}pyridine-2-yl)-2-oxopyrrolidine-1-carboxamide hydrochloride hydrate EU/3/23/2873 |
Treatment of tenosynovial giant cell tumour, localised and diffuse type | Active (positive) | 13 Dec 2023 |
| ovine polyclonal fragment antigen-binding against ricin EU/3/23/2872 |
Treatment of ricin poisoning | Active (positive) | 13 Dec 2023 |
| bersiporocin EU/3/23/2871 |
Treatment of idiopathic pulmonary fibrosis | Active (positive) | 13 Dec 2023 |
| selumetinib EU/3/23/2870 18 clinical trials → |
Treatment of amyotrophic lateral sclerosis | Active (positive) | 13 Dec 2023 |
| 2'-O, 4'-C-Methylene-P-thio-adenylyl-(3'->5')-2'-O, 4'-C-methylene-P-thioguanylyl-(3'->5')-2'-O, 4'-C-methylene-P-thio-adenylyl-(3'->5')-2'-deoxy-P-thioadenyly… EU/3/23/2869 |
Treatment of Angelman syndrome | Active (positive) | 13 Dec 2023 |
| autologous T lymphocytes containing a T-cell receptor against Epstein–Barr virus EU/3/23/2868 |
Treatment of peripheral T-cell lymphoma | Active (positive) | 13 Dec 2023 |
| Methyl 4-(2-acetamidoethylsulfanyl)-4-oxobutanoate EU/3/23/2867 |
Treatment of Leigh syndrome | Active (positive) | 13 Dec 2023 |
| marstacimab EU/3/23/2866 5 clinical trials → |
Treatment of haemophilia B | Withdrawn | 13 Dec 2023 |
| selumetinib EU/3/23/2865 18 clinical trials → |
Treatment of glioma | Active (positive) | 13 Dec 2023 |
| golcadomide hydrochloride EU/3/23/2864 15 clinical trials → |
Treatment of follicular lymphoma | Active (positive) | 13 Dec 2023 |
| brogidirsen EU/3/23/2863 2 clinical trials → |
Treatment of Duchenne muscular dystrophy | Active (positive) | 13 Dec 2023 |
| allogeneic umbilical cord-derived mesenchymal stromal cells, pooled EU/3/23/2862 |
Treatment of perinatal asphyxia | Active (positive) | 13 Dec 2023 |
| (S)-N-(1-(3-Fluoro-2'-methoxy-[1,1'-biphenyl]-4-yl)-2-oxopiperidin-3-yl)-5-(pyridin-2-yl)thiophene-2-sulfonamide EU/3/23/2861 |
Treatment of soft-tissue sarcoma | Active (positive) | 13 Dec 2023 |
| allogeneic amniotic fluid-derived mesenchymal stem cells with lung specificity EU/3/23/2860 |
Prevention of primary graft dysfunction following lung transplantation | Active (positive) | 13 Dec 2023 |
| cutamesine EMA/OD/0000149115 |
Treatment of amyotrophic lateral sclerosis | Active (positive) | 13 Dec 2023 |
| adeno-associated viral vector serotype 9 containing the human PLA2G6 gene EU/3/23/2845 |
Treatment of infantile neuroaxonal dystrophy | Active (positive) | 10 Dec 2023 |
| Adeno-associated viral vector serotype rh10 encoding miRNA against SOD1 mRNA EU/3/23/2859 |
Treatment of amyotrophic lateral sclerosis | Active (positive) | 8 Nov 2023 |
| Elesclomol-copper EU/3/23/2858 |
Treatment of Menkes disease | Active (positive) | 8 Nov 2023 |
| oregovomab EU/3/23/2857 4 clinical trials → |
Treatment of ovarian cancer | Active (positive) | 8 Nov 2023 |
| messenger RNA encoding Cas9, single guide RNA targeting the human KLKB1 gene EU/3/23/2856 |
Treatment of hereditary angioedema | Active (positive) | 8 Nov 2023 |
| 5-bromo-N-(prop-2-yn-1-yl)-2-(1H-1,2,4-triazol-1-yl)pyrimidine-4,6-diamine EU/3/23/2855 |
Treatment of Prader-Willi syndrome | Active (positive) | 8 Nov 2023 |
| Humanised IgG1 monoclonal antibody against CLDN6 conjugated to monomethyl auristatin E via a cathepsin hydrolysable dipeptide VC linker EU/3/23/2854 |
Treatment of ovarian cancer | Active (positive) | 8 Nov 2023 |
| riboflavin EU/3/23/2853 |
Treatment of medium-chain acyl-coenzyme A dehydrogenase deficiency | Active (positive) | 8 Nov 2023 |
| rucosopasem manganese EU/3/23/2852 |
Treatment of pancreatic cancer | Active (positive) | 8 Nov 2023 |
| N-{(2S,3R)-4,4-Difluoro-1-(2-hydroxy-2-methylpropanoyl)-2-[(2,3',5'-trifluoro[1,1'-biphenyl]-3-yl)methyl]pyrrolidin-3-yl}ethanesulfonamide EU/3/23/2851 |
Treatment of narcolepsy | Active (positive) | 8 Nov 2023 |
| setanaxib EU/3/23/2850 |
Treatment of Alport syndrome | Active (positive) | 8 Nov 2023 |
| ambroxol hydrochloride EU/3/23/2849 2 clinical trials → |
Treatment of Gaucher disease | Active (positive) | 8 Nov 2023 |
| bortezomib EU/3/23/2848 92 clinical trials → |
Treatment of patients with light chain (AL) amyloidosis | Active (positive) | 8 Nov 2023 |
| human IgG1 (296-cysteine,301-glycine,306-cysteine) monoclonal antibody against TREM2 EU/3/23/2847 |
Treatment of CSF1R-related leukoencephalopathy | Withdrawn | 13 Oct 2023 |
| sodium oxybate EU/3/23/2846 |
Treatment of narcolepsy | Active (positive) | 13 Oct 2023 |
| 2-(3-(3,5-Dimethyltriazol-4-yl)-5-((S)-oxan-4-yl(phenyl)methyl)pyrido(3,2-b)indol-7-yl)propan-2-ol EU/3/23/2844 |
Treatment of myelofibrosis | Active (positive) | 13 Oct 2023 |
| adeno-associated viral vector serotype 9 containing the human NPC1 gene EU/3/23/2843 |
Treatment of Niemann-Pick's disease, type C | Active (positive) | 13 Oct 2023 |
| Glycerol phenylbutyrate EU/3/23/2842 |
Treatment of STXBP1 developmental and epileptic encephalopathy | Active (positive) | 13 Oct 2023 |
| alpelisib EU/3/23/2841 22 clinical trials → |
Treatment of lymphatic malformations | Active (positive) | 13 Oct 2023 |
| (R)-(3-(2'-cyclopropyl-3-(hydroxymethyl)-[1,1'-biphenyl]-4-yl) pyrrolidin-1-yl)(5-fluoropyridin-2-yl)methanone EU/3/23/2840 |
Treatment of Olmsted syndrome | Active (positive) | 13 Oct 2023 |
| Humanised IgG1 monoclonal antibody against TfR1 conjugated to exon 44 specific phosphorodiamidate morpholino oligonucleotide via a non-cleavable linker EU/3/23/2838 |
Treatment of Duchenne muscular dystrophy | Active (positive) | 13 Oct 2023 |
| adeno-associated virus vector serotype 9/rh74 containing the human CAPN3 gene and a target sequence of cardiac-specific microRNA EU/3/23/2837 |
Treatment of limb-girdle muscular dystrophy | Active (positive) | 13 Oct 2023 |
| certepetide EU/3/23/2834 |
Treatment of pancreatic cancer | Active (positive) | 13 Oct 2023 |
| setmelanotide EU/3/23/2833 4 clinical trials → |
Treatment of acquired hypothalamic obesity | Active (positive) | 13 Oct 2023 |
| tasimelteon EU/3/23/2832 3 clinical trials → |
Treatment of Smith-Magenis syndrome | Active (positive) | 13 Oct 2023 |
| sildenafil citrate EU/3/23/2831 13 clinical trials → |
Treatment of Leigh syndrome | Active (positive) | 13 Oct 2023 |
| sodium selenate EU/3/23/2829 |
Treatment of progressive supranuclear palsy | Active (positive) | 13 Oct 2023 |
| eplontersen EU/3/23/2828 · now authorised as Wainzua 4 clinical trials → |
Treatment of transthyretin-mediated amyloidosis | Withdrawn | 13 Oct 2023 |
| mitapivat sulfate EU/3/23/2827 · now authorised as Pyrukynd 12 clinical trials → |
Treatment of thalassaemia intermedia and major | Active (positive) | 13 Oct 2023 |
| adeno-associated virus serotype 9 vector containing the human LAMP2 isoform B transgene EU/3/23/2826 |
Treatment of Danon disease | Active (positive) | 16 Aug 2023 |
| cedazuridine;decitabine EU/3/23/2825 51 clinical trials → |
Treatment of myelodysplastic syndromes | Withdrawn | 16 Aug 2023 |
| adeno-associated virus vector serotype 9 containing the PKP2 gene EU/3/23/2824 |
Treatment of arrhythmogenic right ventricular cardiomyopathy due to plakophilin-2 gene mutations | Active (positive) | 16 Aug 2023 |
| 2'-O-(2-methoxyethyl) modified antisense oligonucleotide targeting PLP1 pre-mRNA EU/3/23/2823 |
Treatment of Pelizaeus-Merzbacher disease | Active (positive) | 16 Aug 2023 |
| Lithium carbonate EU/3/23/2822 4 clinical trials → |
Treatment of Kleine Levin syndrome | Active (positive) | 16 Aug 2023 |
| Mitazalimab EU/3/23/2821 2 clinical trials → |
Treatment of pancreatic cancer | Active (positive) | 16 Aug 2023 |
| Herpes simplex virus-1, derived from strain F, with deletions on genes gamma (1) 34.5 and UL39 EU/3/23/2820 |
Treatment of glioma | Active (positive) | 16 Aug 2023 |
| virus-like particle containing Cas9/gRNA ribonucleoprotein targeting the human HTT gene EU/3/23/2819 |
Treatment of Huntington’s disease | Active (positive) | 16 Aug 2023 |
| allogeneic faecal microbiota, pooled EU/3/23/2818 |
Treatment in haematopoietic stem cell transplantation | Active (positive) | 16 Aug 2023 |
| alprostadil EU/3/23/2817 |
Treatment of in solid organ transplantation | Active (positive) | 16 Aug 2023 |
| rituximab EU/3/23/2816 239 clinical trials → |
Treatment of primary membranous nephropathy | Active (positive) | 25 Jul 2023 |
| allogeneic peripheral blood-derived haematopoietic stem and progenitor cells, regulatory T cells and conventional T cells EU/3/23/2815 |
Treatment in haematopoietic stem cell transplantation | Active (positive) | 25 Jul 2023 |
| tarperprumig EU/3/23/2814 |
Treatment of sickle cell disease | Active (positive) | 25 Jul 2023 |
| taldefgrobep alfa EU/3/23/2813 2 clinical trials → |
Treatment of spinal muscular atrophy | Active (positive) | 25 Jul 2023 |
| messenger ribonucleic acid coding for coiled-coil domain-containing protein 40 EU/3/23/2812 |
Treatment of primary ciliary dyskinesia | Active (positive) | 25 Jul 2023 |
| relacorilant EU/3/23/2811 · now authorised as Lifyorli 5 clinical trials → |
Treatment of ovarian cancer | Active (positive) | 25 Jul 2023 |
| nomacopan EU/3/23/2810 |
Treatment in haematopoietic stem cell transplantation | Active (positive) | 25 Jul 2023 |
| chimeric human-murine IgG1 kappa monoclonal antibody against TNF alfa EU/3/23/2809 |
Treatment of sarcoidosis | Active (positive) | 25 Jul 2023 |
| vonafexor EU/3/23/2808 |
Treatment of Alport syndrome | Active (positive) | 25 Jul 2023 |
| paclitaxel obaluronate EU/3/23/2807 |
Treatment of malignant mesothelioma | Active (positive) | 25 Jul 2023 |
| ulviprubart EU/3/23/2805 |
Treatment of inclusion body myositis | Active (positive) | 25 Jul 2023 |
| Ilginatinib maleate EU/3/23/2803 |
Treatment of myelofibrosis | Withdrawn | 25 Jul 2023 |
| lusvertikimab EU/3/23/2802 |
Treatment of acute lymphoblastic leukaemia | Active (positive) | 25 Jul 2023 |
| rhizobium rhizogenes;lipopolysaccharide EU/3/23/2801 3 clinical trials → |
Treatment of congenital diaphragmatic hernia | Active (positive) | 25 Jul 2023 |
| cannabidiol EU/3/23/2800 16 clinical trials → |
Treatment of Leigh syndrome | Active (positive) | 25 Jul 2023 |
| humanised IgG1 monoclonal antibody against muscle specific kinase EU/3/23/2799 |
Treatment of congenital myasthenic syndromes | Active (positive) | 25 Jul 2023 |
| pemafibrate EU/3/23/2798 3 clinical trials → |
Treatment of primary biliary cholangitis | Active (positive) | 25 Jul 2023 |
| vactosertib EU/3/23/2797 |
Treatment of osteosarcoma | Active (positive) | 25 Jul 2023 |
| [(2S,3S,4E,6S,7R,10R)-7,10-dihydroxy-3,7-dimethyl-12-oxo-2-[(2E,4E,6R)-6-pyridin-2-ylhepta-2,4-dien-2-yl]-1-oxacyclododec-4-en-6-yl]4-methylpiperazine-1-carbox… EU/3/23/2796 |
Treatment of myelodysplastic syndromes | Withdrawn | 20 Jun 2023 |
| Efzofitimod EU/3/23/2795 |
Treatment of systemic sclerosis | Active (positive) | 20 Jun 2023 |
| autologous CD34+ haematopoietic stem and progenitor cells transduced with a lentiviral vector encoding the interferon alpha-2 gene EU/3/23/2794 |
Treatment of glioma | Active (positive) | 20 Jun 2023 |
| exenatide EU/3/23/2793 2 clinical trials → |
Treatment of moderate and severe closed traumatic brain injury | Active (positive) | 20 Jun 2023 |
| (3S,3'S,3a'S,10a'S)-6-chloro-3'-(3-chloro-2-fluorophenyl)-1'-(cyclopropylmethyl)-6'-methyl-2-oxo-1,2,3',3a',10',10a'-hexahydro-1'H-spiro[indole-3,2'-pyrrolo[2'… EU/3/23/2792 |
Treatment of soft tissue sarcoma | Withdrawn | 20 Jun 2023 |
| 5-(3,4-Dichloro-phenyl)-N-((1R,2R)-2-hydroxy-cyclohexyl)-6-(2,2,2-trifluoro-ethoxy)-nicotinamide EU/3/23/2791 |
Treatment of Alport syndrome | Active (positive) | 20 Jun 2023 |
| autologous dendritic cells pulsed with allogeneic tumour cell lysate EU/3/23/2790 |
Treatment of pancreatic cancer | Active (positive) | 20 Jun 2023 |
| fasudil hydrochloride EU/3/23/2789 2 clinical trials → |
Treatment of amyotrophic lateral sclerosis | Active (positive) | 20 Jun 2023 |
| adeno-associated virus vector serotype 8 encoding the ABCA4 protein, C-region, adeno-associated virus vector serotype 8 encoding the ABCA4 protein, N-region EU/3/23/2788 |
Treatment of inherited retinal dystrophies | Active (positive) | 20 Jun 2023 |
| Evorpacept EU/3/23/2787 5 clinical trials → |
Treatment of gastric cancer | Active (positive) | 20 Jun 2023 |
| nicotinamide mononucleotide EU/3/23/2786 |
Treatment of sickle cell disease | Active (positive) | 20 Jun 2023 |
| doruxapapogene ralaplasmid EU/3/23/2785 |
Treatment of recurrent respiratory papillomatosis | Active (positive) | 22 May 2023 |
| Humanised IgG4 monoclonal antibody against active complement component 1, subcomponent s EU/3/23/2784 |
Treatment of chronic inflammatory demyelinating polyneuropathy | Active (positive) | 22 May 2023 |
| govorestat EU/3/23/2783 |
Treatment of Charcot-Marie-Tooth disease | Active (positive) | 22 May 2023 |
| aspacytarabine EU/3/23/2782 |
Treatment of myelodysplastic syndromes | Active (positive) | 22 May 2023 |
| Humanised IgG1 kappa fragment antibody targeting TfR1 conjugated to P125 oligonucleotide EU/3/23/2781 |
Treatment of myotonic disorders | Active (positive) | 22 May 2023 |
| adeno-associated virus serotype 9 expressing a transcription factor for the SCN1A gene EU/3/23/2780 |
Treatment of Dravet syndrome | Active (positive) | 22 May 2023 |
| 2-((2-fluoro-4-iodophenyl)amino)-N-(2-hydroxyethoxy)-1-methyl-1H-pyrrolo[2,3-b]pyridine-3-carboxamide EU/3/23/2779 |
Treatment of neurofibromatosis type 1 | Active (positive) | 22 May 2023 |
| Idronoxil EU/3/23/2778 |
Treatment of soft tissue sarcoma | Withdrawn | 22 May 2023 |
| autologous blood-derived tumour and hypoxia educated macrophages EU/3/23/2777 |
Treatment of spinal cord injury | Active (positive) | 22 May 2023 |
| Fingolimod EU/3/23/2776 8 clinical trials → |
Treatment of hypomyelinating leukodystrophy-18 | Active (positive) | 22 May 2023 |
| 2-{4-[4-(4-{5-[(1S)-1-amino-1-(4-fluorophenyl) ethyl]pyrimidin-2-yl}piperazin-1-yl)pyrrolo[2,1-f] [1,2,4]triazin-6-yl]-1H-pyrazol-1-yl}ethan-1-ol EU/3/23/2775 |
Treatment of mastocytosis | Active (positive) | 21 Apr 2023 |
| lurbinectedin EU/3/23/2774 17 clinical trials → |
Treatment of soft tissue sarcoma | Active (positive) | 21 Apr 2023 |
| Escherichia coli, strain Nissle 1917, expressing high affinity phenylalanine transporter, phenylalanine ammonia lyase and L-amino acid deaminase EU/3/23/2773 |
Treatment of hyperphenylalaninaemia | Active (positive) | 21 Apr 2023 |
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.