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 benzoate EU/3/16/1787 · now authorised as Prohippur |
Treatment of argininosuccinic aciduria | Active (positive) | 18 Nov 2016 |
| recombinant adeno-associated viral vector serotype 2 carrying the gene for the human aromatic L-amino acid decarboxylase protein;Eladocagene exuparvovec EU/3/16/1786 · now authorised as Upstaza |
Treatment of aromatic L-amino acid decarboxylase deficiency | Active (positive) | 18 Nov 2016 |
| R-azasetron besilate EU/3/16/1785 |
Treatment of sudden sensorineural hearing loss | Active (positive) | 18 Nov 2016 |
| particles comprised of methacrylic acid based co-polymer, cross-linked with a bi-functional cross-linker, purified to bind L-phenylalanine and L-phenylalanine โฆ EU/3/16/1784 |
Treatment of hyperphenylalaninaemia | Active (positive) | 18 Nov 2016 |
| N-(5-(6-chloro-2,2-difluorobenzo[d][1,3]dioxol-5-yl)pyrazin-2-yl)-2-fluoro-6-methylbenzamide EU/3/16/1783 |
Treatment of acute pancreatitis | Active (positive) | 18 Nov 2016 |
| L-selenomethionine EU/3/16/1782 |
treatment of facioscapulohumeral muscular dystrophy | Active (positive) | 18 Nov 2016 |
| live-attenuated non-replicative Pseudomonas aeruginosa strain expressing large T antigen of Merkel cell polyomavirus EU/3/16/1781 |
Treatment of Merkel cell carcinoma | Active (positive) | 18 Nov 2016 |
| Ibrutinib EU/3/16/1780 44 clinical trials → |
Treatment of graft-versus-host disease | Withdrawn | 18 Nov 2016 |
| human monoclonal antibody against activin A EU/3/16/1779 |
Treatment of fibrodysplasia ossificans progressiva | Active (positive) | 18 Nov 2016 |
| Budesonide EU/3/16/1778 · now authorised as Kinpeygo 11 clinical trials → |
Treatment of primary IgA nephropathy | Active (positive) | 18 Nov 2016 |
| brincidofovir EU/3/16/1777 |
Treatment of smallpox | Active (positive) | 18 Nov 2016 |
| ascorbic acid EU/3/16/1776 2 clinical trials → |
treatment of facioscapulohumeral muscular dystrophy | Active (positive) | 18 Nov 2016 |
| alpha-tocopherol EU/3/16/1775 |
treatment of facioscapulohumeral muscular dystrophy | Active (positive) | 18 Nov 2016 |
| allogeneic peripheral blood mononuclear cells incubated ex-vivo with 16, 16-dimethyl prostaglandin E2;dexamethasone EU/3/16/1774 325 clinical trials → |
Treatment in haematopoietic stem cell transplantation | Withdrawn | 18 Nov 2016 |
| allogenic cytomegalovirus-specific cytotoxic T lymphocytes (adimlecleucel) EU/3/16/1773 |
Treatment of cytomegalovirus infection in patients with impaired cell-mediated immunity | Withdrawn | 18 Nov 2016 |
| adeno-associated viral vector serotype 8 containing the human UGT1A1 gene (volrubigene ralaparvovec) EU/3/16/1772 |
Treatment of Crigler-Najjar syndrome | Withdrawn | 18 Nov 2016 |
| adeno-associated viral vector serotype 8 containing the human glucose-6-phosphatase gene (pariglasgene brecaparvovec) EU/3/16/1771 |
treatment of glycogen storage disease type Ia | Active (positive) | 18 Nov 2016 |
| 5-[4-[2-(5-(1-hydroxyethyl)-2-pyridinyl)ethoxy]benzyl]-2,4-thiazolidinedione hydrochloride EU/3/16/1770 · now authorised as Nezglyal; Nezglyal |
Treatment of adrenoleukodystrophy | Active (positive) | 18 Nov 2016 |
| 2-hydroxy-6-((2-(1-isopropyl-1H-pyrazol-5-yl)pyridin-3-yl)methoxy)benzaldehyde (voxelotor) EU/3/16/1769 · now authorised as Oxbryta |
Treatment of sickle cell disease | Active (positive) | 18 Nov 2016 |
| xenon EU/3/16/1768 2 clinical trials → |
Treatment of ischaemia/reperfusion injury associated with cardiac arrest | Active (positive) | 14 Oct 2016 |
| venetoclax EU/3/16/1767 204 clinical trials → |
Treatment of multiple myeloma | Withdrawn | 14 Oct 2016 |
| venetoclax EU/3/16/1766 204 clinical trials → |
Treatment of diffuse large B-cell lymphoma | Withdrawn | 14 Oct 2016 |
| tadekinig alfa EU/3/16/1763 |
Treatment of haemophagocytic lymphohistiocytosis | Active (positive) | 14 Oct 2016 |
| synthetic 15-amino-acid macrocyclic peptide acylated with a polyethyleneglycol palmitoylated linker (zilucoplan) EU/3/16/1762 |
Treatment of paroxysmal nocturnal haemoglobinuria | Withdrawn | 14 Oct 2016 |
| radio-iodinated (131I) anti-CD45 murine monoclonal antibody EU/3/16/1760 |
Treatment in haematopoietic stem cell transplantation | Active (positive) | 14 Oct 2016 |
| recombinant adeno-associated viral vector encoding a human micro-dystrophin gene under the control of a muscle specific promoter EU/3/16/1759 |
Treatment of Duchenne muscular dystrophy | Active (positive) | 14 Oct 2016 |
| P-ethoxy growth factor receptor-bound protein 2 antisense oligonucleotide EU/3/16/1758 |
Treatment of acute myeloid leukaemia | Active (positive) | 14 Oct 2016 |
| N-[(2S)-5-{[(1R, 2S)-2-(4-fluorophenyl)cyclopropyl]amino}-1-(4-methylpiperazin-1-yl)-1-oxopentan-2-yl]-4-(1H-1,2,3-triazol-1-yl)benzamide, bis-tosilate salt (Bโฆ EU/3/16/1757 |
Treatment of myelofibrosis | Active (positive) | 14 Oct 2016 |
| mogamulizumab EU/3/16/1756 · now authorised as Poteligeo 3 clinical trials → |
Treatment of cutaneous T-cell lymphoma | Active (positive) | 14 Oct 2016 |
| melatonin EU/3/16/1755 23 clinical trials → |
Treatment of Smith-Magenis syndrome | Active (positive) | 14 Oct 2016 |
| human monoclonal IgG1 antibody against tissue factor pathway inhibitor EU/3/16/1752 |
Treatment of haemophilia A | Withdrawn | 14 Oct 2016 |
| Fenretinide EU/3/16/1751 2 clinical trials → |
Treatment of peripheral T-cell lymphoma | Withdrawn | 14 Oct 2016 |
| Exendin (9-39) EU/3/16/1750 |
Treatment of noninsulinoma pancreatogenous hypoglycaemia syndrome | Active (positive) | 14 Oct 2016 |
| crenolanib besilate EU/3/16/1748 |
Treatment of acute myeloid leukaemia | Active (positive) | 14 Oct 2016 |
| chemically modified human recombinant sulfamidase EU/3/16/1747 |
Treatment of mucopolysaccharidosis, type IIIA (Sanfilippo A syndrome) | Active (positive) | 14 Oct 2016 |
| carbamazepine EU/3/16/1746 6 clinical trials → |
Treatment of metaphyseal chondrodysplasia, Schmid type | Active (positive) | 14 Oct 2016 |
| autologous T cells transduced with lentiviral vector containing a chimeric antigen receptor directed against CD19 (tisagenlecleucel) EU/3/16/1745 |
Treatment of diffuse large B-cell lymphoma | Active (positive) | 14 Oct 2016 |
| autologous mononuclear cells derived from human cord blood EU/3/16/1744 |
Treatment of periventricular leukomalacia | Active (positive) | 14 Oct 2016 |
| autologous mononuclear cells derived from human cord blood EU/3/16/1743 |
Treatment of neonatal encephalopathy | Active (positive) | 14 Oct 2016 |
| acebutolol hydrochloride EU/3/16/1742 |
Treatment of Smith-Magenis syndrome | Active (positive) | 14 Oct 2016 |
| adeno-associated viral vector serotype 5 containing the human RLBP1 gene EU/3/16/1741 |
Treatment of retinitis pigmentosa | Active (positive) | 14 Oct 2016 |
| adeno-associated viral vector serotype 2/2 containing a gene encoding the channelrhodopsin-2 protein EU/3/16/1740 |
Treatment of retinitis pigmentosa | Withdrawn | 14 Oct 2016 |
| 2-(1,5-dimethyl-3-phenyl-1H-pyrrol-2-yl)-N-{4-[4-(5-fluoro-pyrimidin-2-yl)piperazin-1-yl]-phenyl}-2-oxo-acetamide (also known as F901318)(olorofim) EU/3/16/1738 |
Treatment of invasive aspergillosis | Active (positive) | 14 Oct 2016 |
| (E)-(6-((N-methyl-((3-methylbenzofuran-2-yl)methyl)amino)-3-oxoprop-1-en-1-yl)-2-oxo-3,4-dihydro-1,8-naphthyridin-1(2H)-yl)methyl phosphate, bis ethanolamine sโฆ EU/3/16/1737 |
Treatment of osteomyelitis | Active (positive) | 14 Oct 2016 |
| (6aR,10aR)-3-(1,1-dimethylheptyl)-delta8-tetrahydro-cannabinol-9-carboxylic acid EU/3/16/1736 |
Treatment of cystic fibrosis | Withdrawn | 14 Oct 2016 |
| naltrexone - 10 clinical trials → |
Treatment of fibromyalgia | Negative | 6 Oct 2016 |
| 6'-(R)-methyl-5-O-(5-amino-5,6-dideoxy-ฮฑ-L-talofuranosyl)-paromamine sulfate EU/3/16/1714 |
Treatment of mucopolysaccharidosis type I | Active (positive) | 22 Sep 2016 |
| ubiquinol EU/3/16/1765 |
Treatment of primary coenzyme Q10 deficiency syndrome | Active (positive) | 20 Sep 2016 |
| tetrofosmin EU/3/16/1764 |
Diagnosis of glioma | Active (positive) | 20 Sep 2016 |
| self-complementary adeno-associated viral vector serotype 9 containing the SGSH gene EU/3/16/1761 |
Treatment of mucopolysaccharidosis, type IIIA (Sanfilippo A syndrome) | Active (positive) | 20 Sep 2016 |
| Lutetium-177(3+), S2,S7-cyclo[N-{4,7,10-tricarboxymethyl-1,4,7,10-tetraaza-cyclododecan-1-yl-acetyl}-4-chloro-L-phenylalanyl-D-cysteinyl-4-[(4S)-2,6-dioxo-1,3-โฆ EU/3/16/1754 |
Treatment of gastro-entero-pancreatic neuroendocrine tumours | Active (positive) | 20 Sep 2016 |
| Haematopoietic stem cells modified with a lentiviral vector containing the CD18 gene EU/3/16/1753 |
Treatment of leukocyte adhesion deficiency type I | Active (positive) | 20 Sep 2016 |
| crenolanib besilate EU/3/16/1749 |
Treatment of soft tissue sarcoma | Active (positive) | 20 Sep 2016 |
| A non-covalent trimer of tumour necrosis factor fused to an antibody specific to the extra-domain B of fibronectin in single-chain variable fragment format EU/3/16/1739 |
Treatment of soft tissue sarcoma | Active (positive) | 20 Sep 2016 |
| zoledronic acid EU/3/16/1735 11 clinical trials → |
Treatment of glioma | Active (positive) | 29 Aug 2016 |
| valproic acid EU/3/16/1734 4 clinical trials → |
Treatment of McArdle's disease | Active (positive) | 29 Aug 2016 |
| temozolomide EU/3/16/1733 97 clinical trials → |
Treatment of glioma | Active (positive) | 29 Aug 2016 |
| synthetic ribonucleic acid oligonucleotide directed against superoxide dismutase 1 messenger ribonucleic acid (tofersen) EU/3/16/1732 · now authorised as Qalsody |
Treatment of amyotrophic lateral sclerosis | Active (positive) | 29 Aug 2016 |
| synthetic double-stranded siRNA oligonucleotide directed against delta-aminolevulinic acid synthase 1 mRNA, covalently linked to a ligand containing three N-acโฆ EU/3/16/1731 · now authorised as Givlaari |
Treatment of acute hepatic porphyria | Active (positive) | 29 Aug 2016 |
| sodium benzoate EU/3/16/1730 · now authorised as Prohippur |
Treatment of ornithine translocase deficiency | Active (positive) | 29 Aug 2016 |
| sodium benzoate EU/3/16/1729 · now authorised as Prohippur |
Treatment of lysinuric protein intolerance | Active (positive) | 29 Aug 2016 |
| recombinant humanised monoclonal antibody against human complement component C5a EU/3/16/1728 |
Treatment of graft-versus-host disease | Withdrawn | 29 Aug 2016 |
| recombinant human interleukin-12 EU/3/16/1727 |
Treatment of acute radiation syndrome | Active (positive) | 29 Aug 2016 |
| recombinant human acid alpha-glucosidase conjugated with mannose-6-phosphate analogues EU/3/16/1726 |
Treatment of glycogen storage disease type II (Pompe's disease) | Active (positive) | 29 Aug 2016 |
| recombinant protein derived from the saliva of the Ornithodoros moubata tick (nomacopan) EU/3/16/1725 |
Treatment of paroxysmal nocturnal haemoglobinuria | Active (positive) | 29 Aug 2016 |
| nintedanib EU/3/16/1724 7 clinical trials → |
Treatment of systemic sclerosis | Withdrawn | 29 Aug 2016 |
| methotrexate EU/3/16/1723 97 clinical trials → |
Treatment of alkaptonuria | Active (positive) | 29 Aug 2016 |
| Masitinib mesilate EU/3/16/1722 · now authorised as Alsitek; Masitinib AB Science 2 clinical trials → |
Treatment of amyotrophic lateral sclerosis | Active (positive) | 29 Aug 2016 |
| L-Pyr-L-Glu-L-Gln-L-Leu-L-Glu-L-Arg-L-Ala-L-Leu-L-Asn-L-Ser-L-Ser (cibinetide) EU/3/16/1721 |
Prevention of graft loss in pancreatic islet transplantation | Active (positive) | 29 Aug 2016 |
| Fimaporfin EU/3/16/1720 |
Treatment of cholangiocarcinoma | Active (positive) | 29 Aug 2016 |
| cisplatin EU/3/16/1719 473 clinical trials → |
Treatment of malignant mesothelioma | Active (positive) | 29 Aug 2016 |
| cannabidiol EU/3/16/1718 16 clinical trials → |
Treatment of graft-versus-host disease | Active (positive) | 29 Aug 2016 |
| autologous mesenchymal stromal cells on a decellularised tracheal scaffold from a cadaveric donor EU/3/16/1717 |
Treatment of tracheal stenosis | Active (positive) | 29 Aug 2016 |
| adeno-associated viral vector serotype 9 containing the human mini-dystrophin gene EU/3/16/1716 |
Treatment of Duchenne muscular dystrophy | Withdrawn | 29 Aug 2016 |
| 2-(1,5-dimethyl-3-phenyl-1H-pyrrol-2-yl)-N-{4-[4-(5-fluoro-pyrimidin-2-yl)piperazin-1-yl]-phenyl}-2-oxo-acetamide (also known as F901318) (olorofim) EU/3/16/1713 |
Treatment of scedosporiosis | Active (positive) | 29 Aug 2016 |
| 2-((2-ethyl-6-(4-(2-(3-hydroxyazetidin-1-yl)-2-oxoethyl)-piperazin-1-yl)-8-methylimidazo[1,2-alpha]pyridin-3-yl)-(methyl)amino)-4-(4-fluorophenyl)-thiazole-5-cโฆ EU/3/16/1712 |
Treatment of idiopathic pulmonary fibrosis | Withdrawn | 29 Aug 2016 |
| melatonin EU/3/16/1682 23 clinical trials → |
Treatment of necrotising enterocolitis | Active (positive) | 1 Aug 2016 |
| volanesorsen sodium EU/3/16/1711 |
Treatment of familial partial lipodystrophy | Active (positive) | 14 Jul 2016 |
| triheptanoin EU/3/16/1710 |
Treatment of McArdle's disease | Active (positive) | 14 Jul 2016 |
| sodium hypochlorite EU/3/16/1709 6 clinical trials → |
Treatment of partial deep dermal and full-thickness burns | Active (positive) | 14 Jul 2016 |
| sodium benzoate EU/3/16/1708 · now authorised as Prohippur |
Treatment of hyperargininaemia | Active (positive) | 14 Jul 2016 |
| sodium benzoate EU/3/16/1707 · now authorised as Prohippur |
Treatment of citrullinaemia type 1 | Active (positive) | 14 Jul 2016 |
| sodium benzoate EU/3/16/1706 · now authorised as Prohippur |
Treatment of carbamoyl-phosphate synthase-1 deficiency | Active (positive) | 14 Jul 2016 |
| sodium benzoate EU/3/16/1705 · now authorised as Prohippur |
Treatment of ornithine transcarbamylase deficiency | Active (positive) | 14 Jul 2016 |
| sirolimus EU/3/16/1704 32 clinical trials → |
Treatment of sporadic lymphangioleiomyomatosis | Active (positive) | 14 Jul 2016 |
| setmelanotide EU/3/16/1703 · now authorised as Imcivree 4 clinical trials → |
Treatment of pro-opiomelanocortin deficiency | Active (positive) | 14 Jul 2016 |
| recombinant human monoclonal antibody to insulin receptor EU/3/16/1702 |
Treatment of congenital hyperinsulinism | Active (positive) | 14 Jul 2016 |
| poly(oxy-1,2-ethanediyl), alpha-(carboxymethyl)-omega-methoxy-, amide with arginase 1 [cobalt cofactor] (synthetic human) (1:10), trimer (pegzilarginase) EU/3/16/1701 · now authorised as Loargys |
Treatment of hyperargininaemia | Active (positive) | 14 Jul 2016 |
| mifamurtide EU/3/16/1700 2 clinical trials → |
Treatment of hepatocellular carcinoma | Active (positive) | 14 Jul 2016 |
| mifamurtide EU/3/16/1699 2 clinical trials → |
Treatment of echinococcosis | Active (positive) | 14 Jul 2016 |
| dimethyl fumarate EU/3/16/1698 7 clinical trials → |
Treatment of bullous pemphigoid | Active (positive) | 14 Jul 2016 |
| brincidofovir EU/3/16/1697 |
Treatment of adenovirus infection in immunocompromised patients | Active (positive) | 14 Jul 2016 |
| autologous Epstein-Barr virus specific T cells derived from peripheral blood mononuclear cells, expanded ex vivo EU/3/16/1696 |
Treatment of post-transplantation lymphoproliferative disorders | Withdrawn | 14 Jul 2016 |
| autologous Epstein-Barr virus specific T cells derived from peripheral blood mononuclear cells, expanded ex vivo EU/3/16/1695 |
Treatment of extranodal NK/T-cell lymphoma, nasal type | Withdrawn | 14 Jul 2016 |
| autologous CD4+ and CD8+ T cells transduced with lentiviral vector containing an affinity-enhanced T-cell receptor targeting the New York esophageal antigen-1 EU/3/16/1694 |
Treatment of soft tissue sarcoma | Active (positive) | 14 Jul 2016 |
| adeno-associated viral vector serotype 2.7m8 containing the ChrimsonR-tdTomato gene EU/3/16/1693 |
Treatment of retinitis pigmentosa | Active (positive) | 14 Jul 2016 |
| 3-[4-(1H-imidazol-1-ylmethyl)phenyl]-5-(2-methylpropyl)thiophene-2-[(N-butyloxylcarbamate)-sulphonamide] sodium salt EU/3/16/1692 |
Treatment of idiopathic pulmonary fibrosis | Active (positive) | 14 Jul 2016 |
| 2-[4-(1-methyl-4-pyridin-4-yl-1H-pyrazol-3-yl)-phenoxymethyl]-quinoline succinic acid EU/3/16/1691 |
Treatment of Huntingtonโs disease | Withdrawn | 14 Jul 2016 |
| 16-base single-stranded peptide nucleic acid oligonucleotide linked to 7-amino acid peptide EU/3/16/1690 |
Treatment of soft tissue sarcoma | Active (positive) | 14 Jul 2016 |
| 3-(3-Methanesulfonyl-phenyl)-1-propyl-piperidine hydrochloride - |
Treatment of narcolepsy | Negative | 13 Jul 2016 |
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.