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 |
|---|---|---|---|
| autologous adipose-derived mesenchymal stem cells EU/3/23/2772 |
Treatment of oesophageal atresia | Active (positive) | 21 Apr 2023 |
| adeno-associated viral vector serotype 1 containing the 3' portion of human OTOF gene, Adeno-associated viral vector serotype 1 containing the 5' portion of hu… EU/3/23/2771 |
Treatment of otoferlin gene-mediated hearing loss | Active (positive) | 20 Mar 2023 |
| 5,5-dimethyl-3-[2-(7-methylspiro[2H-benzofuran-3,1'-cyclopropane]-4-yl)oxypyrimidin-5-yl]imidazolidine-2,4-dione EU/3/23/2770 |
Treatment of fragile X syndrome | Active (positive) | 20 Mar 2023 |
| Indole-3-carboxaldehyde EU/3/23/2769 |
Treatment of primary CTLA-4 checkpoint related immunodeficiencies | Active (positive) | 20 Mar 2023 |
| fenfluramine hydrochloride EU/3/23/2768 · now authorised as Fintepla |
Treatment of CDKL5 deficiency disorder | Active (positive) | 20 Mar 2023 |
| bezuclastinib EU/3/23/2767 3 clinical trials → |
Treatment of gastrointestinal stromal tumours | Active (positive) | 20 Mar 2023 |
| 6-(4-(1-amino-3-hydroxycyclobutyl)phenyl)-1-ethyl-7-phenyl-1H-pyrido[2,3-b][1,4]oxazin-2(3H)-one, L-tartrate salt EU/3/23/2766 |
Treatment of hereditary haemorrhagic telangiectasia | Active (positive) | 20 Mar 2023 |
| anagrelide hydrochloride monohydrate EU/3/23/2765 2 clinical trials → |
Treatment of gastrointestinal stromal tumours | Active (positive) | 20 Mar 2023 |
| guanabenz acetate EU/3/23/2764 |
Treatment of vanishing white matter disease | Active (positive) | 20 Mar 2023 |
| Fosmanogepix EU/3/23/2763 2 clinical trials → |
Treatment of scedosporiosis | Active (positive) | 20 Mar 2023 |
| zedenoleucel EU/3/23/2806 |
Treatment of acute myeloid leukaemia | Active (positive) | 25 Feb 2023 |
EU/3/23/2762 |
Treatment of GM2 gangliosidosis | Active (positive) | 15 Feb 2023 |
| bitopertin EU/3/23/2761 2 clinical trials → |
Treatment of erythropoietic protoporphyria | Active (positive) | 15 Feb 2023 |
| isotretinoin EU/3/23/2760 8 clinical trials → |
Treatment of recessive X-linked ichthyosis | Withdrawn | 15 Feb 2023 |
| crisdesalazine EU/3/23/2759 |
Treatment of amyotrophic lateral sclerosis | Active (positive) | 15 Feb 2023 |
| H-L-tryphophanyl-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/3/23/2758 |
Treatment of spinal cord injury | Active (positive) | 15 Feb 2023 |
| melatonin EU/3/23/2757 23 clinical trials → |
Treatment of perinatal asphyxia | Active (positive) | 15 Feb 2023 |
| humanised IgG1 monoclonal antibody against TfR1 conjugated to double stranded siRNA oligonucleotide against DUX4 mRNA via a non-cleavable linker EU/3/23/2756 |
treatment of facioscapulohumeral muscular dystrophy | Active (positive) | 15 Feb 2023 |
| adeno-associated viral vector serotype 9 expressing fukutin-related protein EU/3/23/2755 |
Treatment of limb girdle muscular dystrophy | Active (positive) | 15 Feb 2023 |
EU/3/23/2754 |
Treatment of Huntington's disease | Withdrawn | 15 Feb 2023 |
| patidistrogene bexoparvovec EU/3/23/2753 |
Treatment of limb girdle muscular dystrophy | Active (positive) | 15 Feb 2023 |
| humanised IgG1 monoclonal antibody against annexin-A1 EU/3/23/2752 |
Treatment of pancreatic cancer | Active (positive) | 15 Feb 2023 |
| Fosmanogepix EU/3/23/2750 2 clinical trials → |
Treatment of lomentosporiosis | Active (positive) | 15 Feb 2023 |
| Fosmanogepix EU/3/23/2749 2 clinical trials → |
Treatment of mucormycosis | Active (positive) | 15 Feb 2023 |
| Fosmanogepix EU/3/23/2748 2 clinical trials → |
Treatment of fusariosis | Active (positive) | 15 Feb 2023 |
| autologous CD34+ cells transduced with a lentiviral vector encoding the human NCF1 gene EU/3/22/2747 |
Treatment of chronic granulomatous disease | Active (positive) | 13 Jan 2023 |
| adeno-associated viral vector serotype 2 containing the human SLC6A3 gene EU/3/22/2746 |
Treatment of dopamine transporter deficiency syndrome | Active (positive) | 13 Jan 2023 |
| opelconazole EU/3/22/2745 |
Treatment of invasive aspergillosis | Active (positive) | 13 Jan 2023 |
| Efzofitimod EU/3/22/2744 |
Treatment of sarcoidosis | Active (positive) | 13 Jan 2023 |
| retifanlimab EU/3/22/2743 · now authorised as Zynyz 8 clinical trials → |
Treatment of Merkel cell carcinoma | Active (positive) | 13 Jan 2023 |
| valganciclovir;nanatinostat EU/3/22/2742 5 clinical trials → |
Treatment of diffuse large B-cell lymphoma | Withdrawn | 13 Jan 2023 |
| autologous hematopoietic cells genetically modified with a lentiviral vector containing the human RAG2 gene EU/3/22/2741 |
Treatment of recombination-activating gene 2 deficient severe combined immunodeficiency | Active (positive) | 13 Jan 2023 |
| N1,N14-diethyl-3S,12S-dihydroxyhomospermine tetrahydrochloride EU/3/22/2740 |
Treatment of pancreatic cancer | Active (positive) | 13 Jan 2023 |
| 3-(1-(2',3'-dimethoxy-[1,1'-biphenyl]-4-yl)-1H-1,2,3-triazol-4-yl)benzoic acid EU/3/22/2739 |
Treatment of Duchenne muscular dystrophy | Active (positive) | 13 Jan 2023 |
| octreotide acetate EU/3/22/2738 3 clinical trials → |
Treatment of carcinoid syndrome | Withdrawn | 13 Jan 2023 |
| vorasidenib hemicitrate hemihydrate EU/3/22/2737 · now authorised as Voranigo |
Treatment of glioma | Active (positive) | 13 Jan 2023 |
| adeno-associated virus serotype rh79 containing the human OTC gene, adeno-associated virus serotype rh79 encoding a meganuclease for targeted editing of the hu… EU/3/22/2736 |
Treatment of ornithine transcarbamylase deficiency | Active (positive) | 9 Dec 2022 |
| (2S)-4-[2-methoxyethyl-[4-(5,6,7,8-tetrahydro-1,8-naphthyridin-2-yl)butyl]amino]-2-(quinazolin-4-ylamino)butanoic acid EU/3/22/2735 |
Treatment of idiopathic pulmonary fibrosis | Active (positive) | 9 Dec 2022 |
| potassium 2-chloro-3-(1-hydroxy-5,6,7,8-tetrahydronaphthalen-2-yl)-6-oxo-5-phenyl-6,7-dihydrothieno[2,3-b]pyridin-4-olate monohydrate EU/3/22/2734 |
Treatment of adrenoleukodystrophy | Active (positive) | 9 Dec 2022 |
| briquilimab EU/3/22/2733 |
Treatment in haematopoietic stem cell transplantation | Active (positive) | 9 Dec 2022 |
| autologous T-cells transduced with a lentiviral vector encoding a chimeric antigen receptor against CD7 EU/3/22/2732 |
Treatment of acute lymphoblastic leukaemia | Active (positive) | 9 Dec 2022 |
| Duvelisib EU/3/22/2730 4 clinical trials → |
Treatment of peripheral T-cell lymphoma | Active (positive) | 9 Dec 2022 |
| recombinant adeno-associated virus Olig001 containing human aspartoacylase cDNA EU/3/22/2729 |
Treatment of Canavan disease | Active (positive) | 9 Dec 2022 |
| utreloxastat EU/3/22/2728 |
Treatment of amyotrophic lateral sclerosis | Withdrawn | 9 Dec 2022 |
| Copper (64Cu) oxodotreotide EU/3/22/2727 · now authorised as Deqtynet |
Diagnosis of neuroendocrine neoplasms | Active (positive) | 9 Dec 2022 |
| Mebendazole EU/3/22/2726 2 clinical trials → |
Treatment of autosomal dominant polycystic kidney disease | Active (positive) | 9 Dec 2022 |
| Iodine (124I) evuzamitide EU/3/22/2725 |
Diagnosis of ATTR amyloidosis | Active (positive) | 9 Dec 2022 |
| 16-base single stranded RNA targeting miR-23b linked to oleic acid EU/3/22/2724 |
Treatment of myotonic disorders | Active (positive) | 9 Dec 2022 |
| upifitamab rilsodotin EU/3/22/2723 |
Treatment of ovarian cancer | Withdrawn | 9 Dec 2022 |
| melatonin - 23 clinical trials → |
Treatment of retinopathy of prematurity | Negative | 28 Nov 2022 |
| (R)-deuteropioglitazone hydrochloride EU/3/22/2720 |
Treatment of adrenoleukodystrophy | Active (positive) | 11 Nov 2022 |
| adeno-associated viral vector serotype 9 containing the human ABCD1 gene EU/3/22/2719 |
Treatment of adrenoleukodystrophy | Withdrawn | 11 Nov 2022 |
| cannabidiol EU/3/22/2718 16 clinical trials → |
Treatment of 22q11.2 deletion syndrome | Active (positive) | 11 Nov 2022 |
| tricaprilin EU/3/22/2717 |
Treatment of West syndrome | Active (positive) | 11 Nov 2022 |
| pegcetacoplan EU/3/22/2716 · now authorised as Aspaveli 7 clinical trials → |
Treatment of C3 glomerulopathy with or without immune complexes | Active (positive) | 11 Nov 2022 |
| rovatirelin EU/3/22/2715 |
Treatment of spinocerebellar ataxia | Active (positive) | 11 Nov 2022 |
| atorvastatin EU/3/22/2714 45 clinical trials → |
Treatment of familial cerebral cavernous malformations | Active (positive) | 11 Nov 2022 |
| propranolol EU/3/22/2713 21 clinical trials → |
Treatment of familial cerebral cavernous malformations | Active (positive) | 11 Nov 2022 |
| Iodine (124I) evuzamitide EU/3/22/2712 |
Diagnosis of AL amyloidosis | Active (positive) | 11 Nov 2022 |
| copper nanocluster conjugated to acetate, histidinate and ascorbate EU/3/22/2711 |
Treatment of Menkes disease | Active (positive) | 11 Nov 2022 |
| adeno-associated virus serotype 5 vector encoding C1-esterase inhibitor EU/3/22/2722 |
Treatment of hereditary angioedema | Withdrawn | 10 Nov 2022 |
| N-(4-(4-amino-5-(3-fluoro-4-((4-methylpyrimidin-2-yl)oxy)phenyl)-7-methyl-7H-pyrrolo[2,3-d] pyrimidin-6-yl)phenyl)methacrylamide hydrochloride EU/3/22/2721 |
Treatment of biliary tract cancer | Active (positive) | 10 Nov 2022 |
| Delpazolid EU/3/22/2710 |
Treatment of tuberculosis | Active (positive) | 10 Nov 2022 |
| melatonin N/A 23 clinical trials → |
Prevention of spaceflight-related radiation and microgravity | Negative | 14 Oct 2022 |
| ropeginterferon alfa-2b EU/3/22/2709 |
Treatment of essential thrombocythaemia | Active (positive) | 11 Oct 2022 |
| acetylleucine EU/3/22/2708 |
Treatment of GM1 gangliosidosis | Active (positive) | 11 Oct 2022 |
| Eculizumab EU/3/22/2707 12 clinical trials → |
Treatment of Guillain-Barré syndrome | Withdrawn | 11 Oct 2022 |
| Methyl(R)-4-((3S,5R,7R,8R,9S,10S,13R,14S,17R)-7-hydroxy-10,13-dimethyl-3-((4-((pyridin-3-ylmethyl)amino)butyl)amino)hexadecahydro-1H-cyclopenta[a]phenanthren-1… EU/3/22/2706 |
Treatment of Rett syndrome | Active (positive) | 11 Oct 2022 |
| selinexor EU/3/22/2705 47 clinical trials → |
Treatment of myelofibrosis | Active (positive) | 11 Oct 2022 |
| sodium ({(2S)-1,4-bis[2-(4-chloro-3-fluorophenoxy)acetamido]bicyclo[2.2.2]octan-2-yl}oxy)methyl hydrogen phosphate-2-amino-2-(hydroxymethyl)propane-1,3-diol (1… EU/3/22/2704 |
Treatment of vanishing white matter disease | Active (positive) | 11 Oct 2022 |
| Etidronate disodium EU/3/22/2703 2 clinical trials → |
Treatment of pseudoxanthoma elasticum | Active (positive) | 11 Oct 2022 |
| human IgG4k monoclonal antibody against CD89 EU/3/22/2702 |
Treatment of linear IgA bullous dermatosis | Active (positive) | 11 Oct 2022 |
| isotretinoin EU/3/22/2701 8 clinical trials → |
Treatment of autosomal recessive congenital ichthyosis | Withdrawn | 11 Oct 2022 |
| Adeno-associated viral vector serotype rh.10 encoding the CLN2 gene EU/3/22/2700 |
Treatment of neuronal ceroid lipofuscinosis | Active (positive) | 11 Oct 2022 |
| aglatimagene besadenovec EU/3/22/2699 5 clinical trials → |
Treatment of glioma | Active (positive) | 11 Oct 2022 |
| adeno-associated viral vector serotype 8 containing the 3' human otoferlin coding sequence, adeno?associated viral vector serotype 8 containing the 5' human ot… EU/3/22/2698 |
Treatment of otoferlin gene-mediated hearing loss | Active (positive) | 11 Oct 2022 |
| Inaxaplin EU/3/22/2697 |
Treatment of apolipoprotein L1-mediated kidney disease | Active (positive) | 11 Oct 2022 |
| velusetrag EU/3/22/2696 |
Treatment of intestinal pseudo-obstruction | Active (positive) | 11 Oct 2022 |
| Epeleuton EU/3/22/2695 |
Treatment of sickle cell disease | Active (positive) | 11 Oct 2022 |
| autologous T cells transduced with a lentiviral vector expressing a chimeric antigen receptor against CLL-1 EU/3/22/2694 |
Treatment of acute myeloid leukaemia | Active (positive) | 11 Oct 2022 |
| crofelemer EU/3/22/2693 2 clinical trials → |
Treatment of microvillus inclusion disease | Active (positive) | 11 Oct 2022 |
| Oxygen, sodium chloride solution 0.9% EU/3/22/2692 |
Treatment of amyotrophic lateral sclerosis | Active (positive) | 11 Oct 2022 |
| amphotericin B EU/3/22/2691 2 clinical trials → |
Treatment of cryptococcosis | Withdrawn | 10 Aug 2022 |
| Humanised IgG1 tetravalent monoclonal antibody against death receptor 5 EU/3/22/2690 |
Treatment of chondrosarcoma | Active (positive) | 10 Aug 2022 |
| (7S)-8,8-dimethyl-7-{[(2E)-3-phenyl-2-propen-1-yl]oxy}-7,8-dihydro-2H,6H-pyrano[3,2-g]chromen-2-one EU/3/22/2689 |
Treatment of Hutchinson-Gilford progeria syndrome | Active (positive) | 10 Aug 2022 |
| tamibarotene EU/3/22/2688 |
Treatment of myelodysplastic syndromes | Active (positive) | 10 Aug 2022 |
| clarithromycin;clofazimine;rifabutin EU/3/22/2687 11 clinical trials → |
Treatment of nontuberculous mycobacterial lung disease | Active (positive) | 10 Aug 2022 |
| Gold EU/3/22/2686 |
Treatment of amyotrophic lateral sclerosis | Active (positive) | 10 Aug 2022 |
| allogeneic adult liver-derived stem cells EU/3/22/2685 |
Treatment of urea cycle disorders | Active (positive) | 10 Aug 2022 |
| batoclimab EU/3/22/2684 |
Treatment of myasthenia gravis | Active (positive) | 10 Aug 2022 |
| sotuletinib EU/3/22/2683 |
Treatment of amyotrophic lateral sclerosis | Active (positive) | 10 Aug 2022 |
| vutrisiran EU/3/22/2682 3 clinical trials → |
Treatment of Stargardt's disease | Withdrawn | 10 Aug 2022 |
| autologous human bone marrow-derived haematopoietic and mesenchymal stem cells depleted of erythrocytes, monocytes and lymphocytes EU/3/22/2681 |
Treatment of frontotemporal dementia | Active (positive) | 10 Aug 2022 |
| Epetraborole EU/3/22/2680 |
Treatment of nontuberculous mycobacterial lung disease | Active (positive) | 10 Aug 2022 |
| allogeneic placenta-derived decidual stromal cells EU/3/22/2679 |
Treatment of graft-versus-host-disease | Active (positive) | 10 Aug 2022 |
| calmangafodipir EU/3/22/2678 |
Prevention of acute liver failure | Active (positive) | 10 Aug 2022 |
| H-Lys-Lys-Gly-Asp-Asn-Ile-Met-Val-Thr-Phe-Arg-Asn-Gln-Ala-Ser-Arg-Pro-Tyr-Gly-Lys-Lys-OH EU/3/22/2677 |
Treatment of haemophilia A | Withdrawn | 10 Aug 2022 |
| Mazindol EU/3/22/2676 |
Treatment of idiopathic hypersomnia | Active (positive) | 10 Aug 2022 |
| Bezafibrate EU/3/22/2675 |
Treatment of primary sclerosing cholangitis | Active (positive) | 10 Aug 2022 |
| Lithium carbonate EU/3/22/2674 4 clinical trials → |
Treatment of familial adenomatous polyposis (FAP) | Active (positive) | 10 Aug 2022 |
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.