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 |
|---|---|---|---|
| seletalisib EU/3/18/1986 |
Treatment of activated phosphoinositide 3-kinase delta syndrome | Withdrawn | 22 Feb 2018 |
| rusalatide acetate EU/3/18/1985 |
Treatment of acute radiation syndrome | Active (positive) | 22 Feb 2018 |
| recombinant human monoclonal antibody against mannan-binding lectin-associated serine protease-2 EU/3/18/1984 |
Treatment of primary IgA nephropathy | Active (positive) | 22 Feb 2018 |
| pyridoxal 5'-phosphate EU/3/18/1983 |
Treatment of pyridoxamine 5'-phosphate oxidase deficiency | Active (positive) | 22 Feb 2018 |
| N-(tert-butylcarbamoyl)-5-cyano-2-((4'-(difluoromethoxy)-[1,1'-biphenyl]-3-yl)oxy)benzenesulfonamide EU/3/18/1982 |
Treatment of pulmonary arterial hypertension | Active (positive) | 22 Feb 2018 |
| Mertansine functionalised gold nanoconjugate EU/3/18/1981 |
Treatment of hepatocellular carcinoma | Withdrawn | 22 Feb 2018 |
| Levosimendan EU/3/18/1980 3 clinical trials → |
Treatment of amyotrophic lateral sclerosis | Withdrawn | 22 Feb 2018 |
| human monoclonal IgG2 antibody against tissue factor pathway inhibitor EU/3/18/1979 |
Treatment of haemophilia A | Withdrawn | 22 Feb 2018 |
| flucytosine EU/3/18/1978 |
Treatment of glioma | Active (positive) | 22 Feb 2018 |
| cannabidivarin EU/3/18/1977 |
Treatment of fragile X syndrome | Active (positive) | 22 Feb 2018 |
| allogeneic CD4+ and CD25+ T lymphocytes ex vivo incubated with GP120 EU/3/18/1976 |
Treatment in haematopoietic stem cell transplantation | Active (positive) | 22 Feb 2018 |
| adenovirus-associated viral vector serotype 8 containing the human RPGR gene EU/3/18/1975 |
Treatment of retinitis pigmentosa | Active (positive) | 22 Feb 2018 |
| 6-{[(1R,2S)-2-aminocyclohexyl]amino}-7-fluoro-4-(1-methyl-1H-pyrazol-4-yl)-1,2-dihydro-3H-pyrrolo[3,4-c]pyridin-3-one monocitrate (also known as mivavotinib) EU/3/18/1974 |
Treatment of acute myeloid leukaemia | Active (positive) | 22 Feb 2018 |
| ultevursen EU/3/18/1973 |
Treatment of retinitis pigmentosa | Active (positive) | 22 Feb 2018 |
| 1-[[[4-(4-Fluoro-2-methyl-1H-indol-5-yloxy)-6-methoxyquinolin-7-yl]oxy]methyl]cyclopropanamine-dihydrochloride EU/3/18/1972 · now authorised as Qezzaqar |
Treatment of soft tissue sarcoma | Active (positive) | 22 Feb 2018 |
| ribavirin EU/3/18/2003 |
Treatment of Lassa fever | Active (positive) | 21 Feb 2018 |
| vatiquinone EU/3/17/1971 2 clinical trials → |
Treatment of RARS2 syndrome | Withdrawn | 17 Jan 2018 |
| sirolimus EU/3/17/1970 32 clinical trials → |
Treatment of sickle cell disease | Active (positive) | 17 Jan 2018 |
| recombinant adeno-associated viral vector serotype 2/1 encoding human beta-hexosaminidase alpha and beta subunits EU/3/17/1969 |
Treatment of GM2 gangliosidosis | Active (positive) | 17 Jan 2018 |
| pyrazolo[1,5-a]pyrimidine, 3-[4-chloro-2-(4-morpholinyl)-5-thiazolyl]-7-(1-ethylpropyl)-2,5-dimethyl-pyrazolo[1,3-a]pyrimidine EU/3/17/1968 |
Treatment of congenital adrenal hyperplasia | Active (positive) | 17 Jan 2018 |
| N-[2,6-bis(1-methylethyl)phenyl]-N'-[[1-[4-(dimethylamino)phenyl]cyclopentyl]methyl]urea, hydrochloride salt EU/3/17/1967 |
Treatment of congenital adrenal hyperplasia | Active (positive) | 17 Jan 2018 |
| N-(bromoacetyl)-3,3-dinitroazetidine EU/3/17/1966 |
Treatment of small cell lung cancer | Active (positive) | 17 Jan 2018 |
| metformin;l-citrulline EU/3/17/1965 105 clinical trials → |
Treatment of Duchenne muscular dystrophy | Active (positive) | 17 Jan 2018 |
| Itacitinib EU/3/17/1964 2 clinical trials → |
Treatment of graft-versus-host disease | Active (positive) | 17 Jan 2018 |
| Humanised Fc-engineered monoclonal antibody against CD19 EU/3/17/1962 |
Treatment of IgG4-related disease | Active (positive) | 17 Jan 2018 |
| Gilteritinib EU/3/17/1961 · now authorised as Xospata 16 clinical trials → |
Treatment of acute myeloid leukaemia | Active (positive) | 17 Jan 2018 |
| ciclopirox EU/3/17/1960 |
Treatment of congenital erythropoietic porphyria | Active (positive) | 17 Jan 2018 |
| cannabidiol EU/3/17/1959 · now authorised as Epidyolex 16 clinical trials → |
Treatment of tuberous sclerosis | Active (positive) | 17 Jan 2018 |
| allogeneic umbilical cord blood CD34+ cells cultured ex vivo with Notch ligand Delta1 (dilanubicel) EU/3/17/1958 |
Treatment in haematopoietic stem cell transplantation | Withdrawn | 17 Jan 2018 |
| adeno-associated viral vector serotype 5 encoding a microRNA targeted to human huntingtin gene EU/3/17/1957 |
Treatment of Huntington’s disease | Active (positive) | 17 Jan 2018 |
| adeno-associated viral vector serotype 2/6 encoding zinc-finger nucleases and the human iduronate 2-sulfatase gene EU/3/17/1956 |
Treatment of mucopolysaccharidosis type II (Hunter's syndrome) | Withdrawn | 17 Jan 2018 |
| adeno-associated viral vector serotype 2/6 encoding zinc-finger nucleases and the human alpha L-iduronidase gene (devafidugene civaparvovec) EU/3/17/1955 |
Treatment of mucopolysaccharidosis type I | Withdrawn | 17 Jan 2018 |
| venetoclax EU/3/17/1954 204 clinical trials → |
Treatment of mantle cell lymphoma | Withdrawn | 12 Dec 2017 |
| pegunigalsidase alfa EU/3/17/1953 3 clinical trials → |
Treatment of Fabry disease | Withdrawn | 12 Dec 2017 |
| modified messenger ribonucleic acid encoding human argininosuccinate lyase enzyme encapsulated into lipid nanoparticles EU/3/17/1952 |
Treatment of argininosuccinic aciduria | Active (positive) | 12 Dec 2017 |
| agammaglobulinaemia tyrosine kinase EU/3/17/1951 |
Treatment of pemphigus | Withdrawn | 12 Dec 2017 |
| adenovirus associated viral vector serotype 8 containing the human AIPL1 gene EU/3/17/1950 |
Treatment of Leber's congenital amaurosis | Active (positive) | 12 Dec 2017 |
| acetylleucine EU/3/17/1949 |
Treatment of GM2 gangliosidosis | Active (positive) | 12 Dec 2017 |
| 4-hydroxy-2,2,6,6-tetramethylpiperidine-N-oxyl EU/3/17/1948 |
Treatment of familial cerebral cavernous malformations | Active (positive) | 12 Dec 2017 |
| 2-isopropyl-3H-naphtho[1,2-d]imidazole-4,5-dione EU/3/17/1947 |
Treatment of mitochondrial encephalomyopathy, lactic acidosis and stroke-like episodes | Active (positive) | 12 Dec 2017 |
| (2S,4R)-1-(2-(3-acetyl-5-(2-methylpyrimidine-5-yl)-1H-indazol-1-yl)acetyl)-N-(6-bromopyridine-2-yl)-4-fluoropyrrolidine-2-carboxamide EU/3/17/1946 · now authorised as Voydeya |
Treatment of paroxysmal nocturnal haemoglobinuria | Active (positive) | 12 Dec 2017 |
| 1-[4-bromo-5-[1-ethyl-7-(methylamino)-2-oxo-1,2-dihydro-1,6-naphthyridin-3-yl]-2-fluorophenyl]-3-phenylurea EU/3/17/1936 · now authorised as Qinlock |
Treatment of gastrointestinal stromal tumours | Active (positive) | 8 Nov 2017 |
| synthetic cyclic 8 amino acid analogue of human unacylated ghrelin EU/3/17/1932 |
Treatment of Prader-Willi syndrome | Active (positive) | 16 Oct 2017 |
| siplizumab EU/3/17/1931 2 clinical trials → |
Treatment in solid organ transplantation | Active (positive) | 16 Oct 2017 |
| recombinant monoclonal antibody to sialic acid-binding Ig-like lectin 8 EU/3/17/1929 |
Treatment of mastocytosis | Withdrawn | 16 Oct 2017 |
| recombinant adeno-associated viral vector serotype 5 encoding Staphylococcus aureus Cas9 endonuclease and two guide RNAs complementary to two regions of intron… EU/3/17/1928 |
Treatment of Leber's congenital amaurosis | Withdrawn | 16 Oct 2017 |
| pracinostat EU/3/17/1927 |
Treatment of acute myeloid leukaemia | Withdrawn | 16 Oct 2017 |
| ofranergene obadenovec EU/3/17/1926 |
Treatment of ovarian cancer | Withdrawn | 16 Oct 2017 |
| Humanised monoclonal antibody targeting B-cell maturation antigen conjugated with maleimidocaproyl monomethyl auristatin F EU/3/17/1925 |
Treatment of multiple myeloma | Withdrawn | 16 Oct 2017 |
| Glucopyranosyl lipid A EU/3/17/1924 |
Treatment of follicular lymphoma | Withdrawn | 16 Oct 2017 |
| Glasdegib maleate EU/3/17/1923 · now authorised as Daurismo |
Treatment of acute myeloid leukaemia | Active (positive) | 16 Oct 2017 |
| Entospletinib EU/3/17/1922 |
Treatment of acute myeloid leukaemia | Withdrawn | 16 Oct 2017 |
| cannabidivarin EU/3/17/1921 |
Treatment of Rett syndrome | Active (positive) | 16 Oct 2017 |
| cannabidiol EU/3/17/1920 16 clinical trials → |
Treatment of West syndrome | Active (positive) | 16 Oct 2017 |
| bitopertin EU/3/17/1919 2 clinical trials → |
Treatment of beta thalassaemia intermedia and major | Withdrawn | 16 Oct 2017 |
| autologous ex-vivo-expanded peripheral polyclonal lymphocytes enriched in activated natural killer cells EU/3/17/1918 |
Treatment of multiple myeloma | Active (positive) | 16 Oct 2017 |
| adenoviral vector of serotype 5 modified to contain a chimeric sequence consisting of a minimal urokinase-type plasminogen activator receptor promoter preceded… EU/3/17/1917 |
Treatment of pancreatic cancer | Active (positive) | 16 Oct 2017 |
| 5-amino-1-(2-methyl-1H-benzo[d]imidazol-5-yl)-1H-pyrazol-4-yl 1H-indol-2-yl ketone mono[(S)-2-hydroxysuccinate] EU/3/17/1916 |
Treatment of biliary tract cancer | Withdrawn | 16 Oct 2017 |
| 2-[N-(2-hydroxyethyl)]-N-(4-methoxybenzenesulfonyl)]amino-N-(4-chlorocinnamyl)-N-methylbenzylamine EU/3/17/1915 |
Treatment of Charcot-Marie-Tooth disease | Active (positive) | 16 Oct 2017 |
| S)-3-((S)-2-(2-((2,6-difluorophenyl)amino)-2-oxoacetamido)propanamido)-4-oxo-5-(2,3,5,6-tetrafluorophenoxy)pentanoic acid EU/3/17/1914 |
Treatment of primary sclerosing cholangitis | Withdrawn | 16 Oct 2017 |
| tiratricol EU/3/17/1945 · now authorised as Emcitate |
Treatment of Allan-Herndon-Dudley syndrome | Active (positive) | 12 Oct 2017 |
| tamoxifen citrate EU/3/17/1944 34 clinical trials → |
Treatment of Duchenne muscular dystrophy | Active (positive) | 12 Oct 2017 |
| recombinant adeno-associated viral vector serotype 9 containing human iduronate-2-sulfatase gene EU/3/17/1943 |
Treatment of mucopolysaccharidosis type II (Hunter's syndrome) | Active (positive) | 12 Oct 2017 |
| N-(2-aminophenyl)-4-(1-[(1,3-dimethyl-1H-pyrazol-4-yl)methyl]piperidin)benzamide EU/3/17/1942 |
Treatment of peripheral T-cell lymphoma | Active (positive) | 12 Oct 2017 |
| diazoxide choline EU/3/17/1941 · now authorised as Viokat |
Treatment of Prader-Willi syndrome | Active (positive) | 12 Oct 2017 |
| concizumab EU/3/17/1940 5 clinical trials → |
Treatment of haemophilia B | Withdrawn | 12 Oct 2017 |
| C1-esterase-inhibitor human EU/3/17/1939 |
Treatment in solid organ transplantation | Active (positive) | 12 Oct 2017 |
| antisense oligonucleotide targeting exon 73 in the COL7A1 gene EU/3/17/1938 |
Treatment of epidermolysis bullosa | Withdrawn | 12 Oct 2017 |
| 4-amino-1-[(1S,4R,5S)-2-fluoro-4,5-dihydroxy-3-(hydroxymethyl)cyclopent-2-en-1-yl]pyrimidin-2-one EU/3/17/1937 |
Treatment of pancreatic cancer | Active (positive) | 12 Oct 2017 |
| 1,4-diamino-2,3-dicyano-1,4-bis[2-aminophenylthio] butadiene EU/3/17/1935 |
Treatment of non-traumatic subarachnoid haemorrhage | Active (positive) | 12 Oct 2017 |
| (R)-troloxamide quinone EU/3/17/1934 |
Treatment of amyotrophic lateral sclerosis | Active (positive) | 12 Oct 2017 |
EU/3/17/1933 |
Treatment of systemic sclerosis | Active (positive) | 12 Oct 2017 |
| teicoplanin EU/3/17/1913 2 clinical trials → |
Treatment of cystic fibrosis | Active (positive) | 23 Aug 2017 |
| tacrolimus EU/3/17/1912 40 clinical trials → |
Treatment of pulmonary arterial hypertension | Active (positive) | 23 Aug 2017 |
| sodium 2-hydroxylinoleate EU/3/17/1911 |
Treatment of pancreatic cancer | Active (positive) | 23 Aug 2017 |
| sirolimus EU/3/17/1910 · now authorised as Hyftor 32 clinical trials → |
Treatment of tuberous sclerosis | Active (positive) | 23 Aug 2017 |
| Salmonella typhi Ty21a strain transfected with a plasmid vector encoding the human vascular endothelial growth factor receptor 2 EU/3/17/1909 |
Treatment of glioma | Active (positive) | 23 Aug 2017 |
| recombinant truncated N-terminal fragment of human lens epithelium-derived growth factor EU/3/17/1908 |
Treatment of retinitis pigmentosa | Withdrawn | 23 Aug 2017 |
| recombinant fragment of human surfactant protein-D EU/3/17/1907 |
Prevention of bronchopulmonary dysplasia | Active (positive) | 23 Aug 2017 |
| recombinant adeno-associated viral vector serotype 5 carrying the gene for the human frataxin protein EU/3/17/1906 |
Treatment of Friedreich's ataxia | Active (positive) | 23 Aug 2017 |
| purified pasteurised and freeze-dried cell-wall fragments from Mycobacterium tuberculosis strain RUTI EU/3/17/1905 |
Treatment of tuberculosis | Active (positive) | 23 Aug 2017 |
| Picropodophyllin EU/3/17/1904 |
Treatment of glioma | Active (positive) | 23 Aug 2017 |
| odiparcil EU/3/17/1903 |
Treatment of mucopolysaccharidosis VI (Maroteaux-Lamy syndrome) | Active (positive) | 23 Aug 2017 |
| N-{2-[(6-{[(2,6-dichloro-3,5-dimethoxyphenyl)carbamoyl](methyl)amino}pyrimidin-4-yl)amino]-5-(4-ethylpiperazin-1-yl)phenyl}prop-2-enamide EU/3/17/1902 |
Treatment of hepatocellular carcinoma | Withdrawn | 23 Aug 2017 |
| Itraconazole EU/3/17/1901 35 clinical trials → |
Treatment of naevoid basal-cell carcinoma syndrome (Gorlin syndrome) | Withdrawn | 23 Aug 2017 |
| asunercept EU/3/17/1900 |
Treatment of myelodysplastic syndromes | Active (positive) | 23 Aug 2017 |
| antisense oligonucleotide targeting exon 13 in the USH2A gene EU/3/17/1899 |
Treatment of retinitis pigmentosa | Active (positive) | 23 Aug 2017 |
| Adeno-associated viral vector serotype Anc80 containing the truncated human ATP7B gene under the control of the human alpha-1 antitrypsin promoter EU/3/17/1898 |
Treatment of Wilson's disease | Withdrawn | 23 Aug 2017 |
| tirapazamine EU/3/17/1897 2 clinical trials → |
Treatment of hepatocellular carcinoma | Active (positive) | 17 Jul 2017 |
| sirolimus EU/3/17/1896 32 clinical trials → |
Treatment of pachyonychia congenita | Active (positive) | 17 Jul 2017 |
| retinol EU/3/17/1895 |
Prevention of retinopathy of prematurity | Active (positive) | 17 Jul 2017 |
| recombinant human antibody directed against misfolded human superoxide dismutase 1 EU/3/17/1894 |
Treatment of amyotrophic lateral sclerosis | Active (positive) | 17 Jul 2017 |
| Polyphenyl(disodium 3-O-sulfo-beta-D-glucopyranuronate)-(1->3)-beta-D-galactopyranoside EU/3/17/1893 |
Treatment of anti-MAG neuropathy | Withdrawn | 17 Jul 2017 |
| Oxymetazoline hydrochloride EU/3/17/1892 |
Treatment of spinal cord injury | Active (positive) | 17 Jul 2017 |
| Bacillus subtilis oxalate decarboxylase EU/3/17/1891 |
Treatment of primary hyperoxaluria | Active (positive) | 17 Jul 2017 |
| autologous CD4+ and CD8+ T cells expressing a CD19-specific chimeric antigen receptor (lisocabtagene maraleucel) EU/3/17/1890 |
Treatment of diffuse large B-cell lymphoma | Withdrawn | 17 Jul 2017 |
| (S)-1-(4-fluorophenyl)-1-(2-(4-(6-(1-methyl-1H-pyrazol-4-yl)pyrrolo[2,1-f][1,2,4]triazin-4-yl)piperazin-yl)pyrimidin-5-yl)ethan-1-amine EU/3/17/1889 · now authorised as Ayvakyt |
Treatment of gastrointestinal stromal tumours | Active (positive) | 17 Jul 2017 |
| tripotassium citrate monohydrate;potassium hydrogen carbonate EU/3/17/1888 |
Treatment of distal renal tubular acidosis | Withdrawn | 20 Jun 2017 |
| synthetic glucagon analogue modified to contain 7 amino acid substitutions EU/3/17/1887 |
Treatment of congenital hyperinsulinism | Active (positive) | 20 Jun 2017 |
| sirolimus EU/3/17/1886 32 clinical trials → |
Treatment of tuberous sclerosis | Active (positive) | 20 Jun 2017 |
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.