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 |
|---|---|---|---|
| sildenafil EU/3/17/1885 13 clinical trials → |
Treatment of congenital diaphragmatic hernia | Active (positive) | 20 Jun 2017 |
| recombinant human factor IX protein modified with three point mutations (dalcinonacog alfa) EU/3/17/1884 |
Treatment of haemophilia B | Withdrawn | 20 Jun 2017 |
| pentamer formyl thiophene acetic acid EU/3/17/1883 |
Treatment of Creutzfeldt-Jakob disease | Active (positive) | 20 Jun 2017 |
| Ibutamoren mesilate EU/3/17/1882 |
Treatment of growth hormone deficiency | Active (positive) | 20 Jun 2017 |
| decitabine;tetrahydrouridine EU/3/17/1881 51 clinical trials → |
Treatment of sickle cell disease | Active (positive) | 20 Jun 2017 |
| avacopan EU/3/17/1880 4 clinical trials → |
Treatment of C3 glomerulopathy | Active (positive) | 20 Jun 2017 |
| Asp-Arg-Val-Tyr-Ile-His-Pro (Angiotensin 1-7);talfirastide EU/3/17/1879 |
Treatment of epidermolysis bullosa | Active (positive) | 20 Jun 2017 |
| ciclosporin EU/3/04/210 3 clinical trials → |
Treatment of bronchiolitis obliterans syndrome | Active (positive) | 24 May 2017 |
| ursodeoxycholic acid EU/3/17/1878 9 clinical trials → |
Treatment of Niemann-Pick disease | Active (positive) | 22 May 2017 |
| tamoxifen citrate EU/3/17/1877 34 clinical trials → |
Treatment of cystic fibrosis | Active (positive) | 22 May 2017 |
| sodium (1R,3R,4R,5S)-3-({2-N-acetylamino-2-deoxy-3-O-[(1S)-1-carboxylato-2-cyclohexylethyl]-beta-D-galactopyranosyl}oxy)-4-({6-deoxy-alpha-L-galactopyranosyl}o… EU/3/17/1876 |
Treatment of acute myeloid leukaemia | Active (positive) | 22 May 2017 |
| recombinant human interleukin-7 fused to a hybrid crystallisable fragment region of a human antibody- EU/3/17/1875 |
Treatment of idiopathic CD4 lymphocytopenia | Active (positive) | 22 May 2017 |
| recombinant adeno-associated viral vector serotype 6 encoding the B-domain-deleted human factor VIII EU/3/17/1874 |
Treatment of haemophilia A | Active (positive) | 22 May 2017 |
| Poly(oxy-1,2-ethanediyl),.alpha.-hydro-.omega.-hydroxy-,15,15'-diester with N-acetyl-L-isoleucyl-L-cysteinyl-L-valyl-1-methyl-L-tryptophyl-L-glutaminyl-L-.alph… EU/3/17/1873 · now authorised as Aspaveli |
Treatment of paroxysmal nocturnal haemoglobinuria | Active (positive) | 22 May 2017 |
| chimeric locked nucleic acid deoxynucleoside phosphorothioate-linked oligonucleotide inhibitor directed against microRNA-155-5p EU/3/17/1872 |
Treatment of cutaneous T-cell lymphoma | Withdrawn | 22 May 2017 |
| 225Ac-lintuzumab EU/3/17/1871 |
Treatment of acute myeloid leukaemia | Active (positive) | 22 May 2017 |
| thymidine;deoxycytidine EU/3/17/1870 · now authorised as Kygevvi |
Treatment of mitochondrial DNA depletion syndrome, myopathic form | Active (positive) | 20 Apr 2017 |
| rituximab EU/3/17/1869 239 clinical trials → |
Treatment in solid organ transplantation | Active (positive) | 20 Apr 2017 |
| N-[(1R)-1-phenylethyl]-6-{1H-pyrazolo[3,4-d]pyrimidin-4-yl}quinazolin-2-amine EU/3/17/1868 |
Treatment of fragile X syndrome | Withdrawn | 20 Apr 2017 |
| modified messenger ribonucleic acid encoding human ornithine transcarbamylase enzyme encapsulated into lipid nanoparticles EU/3/17/1867 |
Treatment of ornithine transcarbamylase deficiency | Active (positive) | 20 Apr 2017 |
| human normal immunoglobulin EU/3/17/1866 |
Treatment in solid organ transplantation | Active (positive) | 20 Apr 2017 |
| Estetrol EU/3/17/1865 |
Treatment of neonatal encephalopathy | Active (positive) | 20 Apr 2017 |
| emeramide EU/3/17/1864 |
Prevention of mercury toxicity | Active (positive) | 20 Apr 2017 |
| autologous T lymphocyte-enriched population of cells transduced with a lentiviral vector encoding a chimeric antigen receptor targeting human B cell maturation… EU/3/17/1863 · now authorised as Abecma |
Treatment of multiple myeloma | Active (positive) | 20 Apr 2017 |
| autologous adult bone marrow-derived non-expanded CD133+ haematopoietic stem cells EU/3/17/1862 |
Treatment of Asherman's syndrome | Active (positive) | 20 Apr 2017 |
| (S)-8-{2-amino-6-[1-(5-chloro-biphenyl-2-yl)-(R)-2,2,2-trifluoro-ethoxy]-pyrimidin-4-yl}-2,8-diaza-spiro[4.5]decane-3-carboxylic acid ethyl ester EU/3/17/1861 |
Treatment of pulmonary arterial hypertension | Active (positive) | 20 Apr 2017 |
| synthetic double-stranded siRNA oligonucleotide directed against transthyretin mRNA (also known as patisiran) EU/3/11/857 · now authorised as Onpattro |
Treatment of transthyretin-mediated amyloidosis | Active (positive) | 6 Apr 2017 |
| 20% Intravenous fat emulsion consisting of 20% soybean oil, 1.2% egg yolk phospholipids, 2.25% glycerin, and water for injection - |
Treatment of poisoning by local anaesthetics | Negative | 3 Apr 2017 |
| Poly-cyclodextrin-bis-cysteine-PEG3400-camptothecin-conjugate EU/3/17/1860 |
Treatment of ovarian cancer | Active (positive) | 20 Mar 2017 |
| Phosphoinositide 3-kinase gamma peptide EU/3/17/1859 |
Treatment of cystic fibrosis | Active (positive) | 20 Mar 2017 |
| Megestrol acetate EU/3/17/1858 12 clinical trials → |
Treatment of granulosa cell tumours | Active (positive) | 20 Mar 2017 |
| ketoconazole EU/3/17/1857 |
Treatment of granulosa cell tumours | Active (positive) | 20 Mar 2017 |
| inebilizumab EU/3/17/1856 12 clinical trials → |
Treatment of neuromyelitis optica spectrum disorders | Withdrawn | 20 Mar 2017 |
| cannabidiol EU/3/17/1855 · now authorised as Epidyolex 16 clinical trials → |
Treatment of Lennox-Gastaut syndrome | Active (positive) | 20 Mar 2017 |
| autologous adipose tissue-derived mesenchymal stem cells EU/3/17/1854 |
Treatment of thromboangiitis obliterans (Buerger's disease) | Withdrawn | 20 Mar 2017 |
| antisense oligonucleotide targeting the USH2A gene EU/3/17/1853 |
Treatment of retinitis pigmentosa | Active (positive) | 20 Mar 2017 |
| allogeneic ex-vivo-expanded umbilical cord blood-derived haematopoietic CD34+ progenitor cells and allogeneic non-expanded umbilical cord blood-derived haemato… EU/3/17/1852 |
Treatment in haematopoietic stem cell transplantation | Active (positive) | 20 Mar 2017 |
| Adeno-associated viral vector serotype rh.10 expressing beta-galactosidase EU/3/17/1851 |
Treatment of GM1 gangliosidosis | Active (positive) | 20 Mar 2017 |
| Adeno-associated viral vector serotype LK03 encoding human ornithine transcarbamylase EU/3/17/1850 |
Treatment of ornithine transcarbamylase deficiency | Active (positive) | 20 Mar 2017 |
| adeno-associated viral vector serotype 8 containing the human alpha-galactosidase A gene EU/3/17/1849 |
Treatment of Fabry disease | Active (positive) | 20 Mar 2017 |
| acetylleucine EU/3/17/1848 · now authorised as Aqneursa |
Treatment of Niemann-Pick disease | Active (positive) | 20 Mar 2017 |
| milademetan tosilate monohydrate EU/3/17/1847 |
Treatment of soft tissue sarcoma | Withdrawn | 20 Mar 2017 |
| vemurafenib EU/3/17/1846 11 clinical trials → |
Treatment of Erdheim-Chester disease | Active (positive) | 27 Feb 2017 |
| thalidomide EU/3/17/1845 12 clinical trials → |
Treatment of hereditary haemorrhagic telangiectasia | Active (positive) | 27 Feb 2017 |
| tauroursodeoxycholic acid EU/3/17/1844 |
Treatment of amyotrophic lateral sclerosis | Active (positive) | 27 Feb 2017 |
| soluble recombinant human fibroblast growth factor receptor 3 EU/3/17/1843 |
Treatment of achondroplasia | Withdrawn | 27 Feb 2017 |
| recombinant human club cell 10 KDa protein EU/3/17/1842 |
Treatment of bronchiolitis obliterans syndrome | Active (positive) | 27 Feb 2017 |
| propranolol hydrochloride EU/3/17/1841 21 clinical trials → |
Treatment of von Hippel-Lindau disease | Active (positive) | 27 Feb 2017 |
| N-(4-(1-cyanocyclopentyl)phenyl)-2-(4-pyridinylmethyl)amino-3-pyridinecarboxamide methanesulfonate EU/3/17/1840 |
Treatment of gastric cancer | Active (positive) | 27 Feb 2017 |
| Iodine (131I) murine IgG1 monoclonal antibody against CD276 EU/3/17/1839 · now authorised as Omblastys |
Treatment of neuroblastoma | Active (positive) | 27 Feb 2017 |
| Humanised IgG4 monoclonal antibody to the human toll-like receptor type 2 EU/3/17/1838 |
Treatment of pancreatic cancer | Active (positive) | 27 Feb 2017 |
| Humanised IgG4 monoclonal antibody to the human toll-like receptor type 2 EU/3/17/1837 |
Treatment of myelodysplastic syndromes | Active (positive) | 27 Feb 2017 |
| fenfluramine hydrochloride EU/3/17/1836 · now authorised as Fintepla |
Treatment of Lennox-Gastaut syndrome | Active (positive) | 27 Feb 2017 |
| ex-vivo-expanded autologous keratinocytes transduced with retroviral vector containing the COL7A1 gene (prademagene zamikeracel) EU/3/17/1835 |
Treatment of epidermolysis bullosa | Active (positive) | 27 Feb 2017 |
| Cyclo[L-alanyl-L-seryl-L-isoleucyl-L-prolyl-L-prolyl-L-glutaminyl-L-lysyl-L-tyrosyl-D-prolyl-L-prolyl-(2S)-2-aminodecanoyl-L-alpha-glutamyl-L-threonyl]acetate … EU/3/17/1834 |
Treatment of primary ciliary dyskinesia | Active (positive) | 27 Feb 2017 |
| autologous T-cells transduced with lentiviral vector encoding an anti-SLAMF7 CD28/CD3-zeta chimeric antigen receptor EU/3/17/1833 |
Treatment of plasma cell myeloma | Active (positive) | 27 Feb 2017 |
| alpha-tocopherol;ascorbic acid EU/3/17/1832 2 clinical trials → |
Treatment of fragile X syndrome | Active (positive) | 27 Feb 2017 |
| 505 amino acid protein, corresponding to amino acids 2-506 of the wild type human histidyl-tRNA synthetase EU/3/17/1831 |
Treatment of limb-girdle muscular dystrophy | Withdrawn | 27 Feb 2017 |
| 5-(4,6-dimorpholino-1,3,5-triazin-2-yl)-4-(trifluoromethyl)pyridin-2-amine (bimiralisib) EU/3/17/1830 |
Treatment of diffuse large B-cell lymphoma | Active (positive) | 27 Feb 2017 |
| 26 base synthetic single-stranded fully phosphorothioated 2'-O-methyl-RNA and DNA mixmer oligonucleotide-based compound EU/3/17/1829 |
Treatment of Dravet syndrome | Active (positive) | 27 Feb 2017 |
| ivacaftor;tezacaftor EU/3/17/1828 · now authorised as Symkevi 3 clinical trials → |
Treatment of cystic fibrosis | Active (positive) | 27 Feb 2017 |
| trans-resveratrol EU/3/16/1827 |
Treatment of spinocerebellar ataxia | Active (positive) | 12 Jan 2017 |
| recombinant IgG degrading enzyme of Streptococcus pyogenes (imlifidase) EU/3/16/1826 · now authorised as Idefirix |
Prevention of graft rejection following solid organ transplantation | Active (positive) | 12 Jan 2017 |
| recombinant adeno-associated viral vector serotype 9 containing the human N-alpha-acetylglucosaminidase gene EU/3/16/1825 |
Treatment of mucopolysaccharidosis type IIIB (Sanfilippo B syndrome) | Active (positive) | 12 Jan 2017 |
| Pr-D-Cys-Met-Pip-Arg-Leu-Arg-Sar-Cys-Lys-Arg-Pro-Tyr-Tle-Leu-OH EU/3/16/1824 |
Treatment of perinatal asphyxia | Active (positive) | 12 Jan 2017 |
| pioglitazone hydrochloride EU/3/16/1823 13 clinical trials → |
Treatment of sudden sensorineural hearing loss | Active (positive) | 12 Jan 2017 |
| pentosan polysulfate sodium EU/3/16/1822 3 clinical trials → |
Treatment of interstitial cystitis | Active (positive) | 12 Jan 2017 |
| leuprorelin acetate EU/3/16/1821 9 clinical trials → |
Treatment of congenital hypogonadotropic hypogonadism | Active (positive) | 12 Jan 2017 |
| hydroxychloroquine EU/3/16/1820 28 clinical trials → |
Treatment of antiphospholipid syndrome | Active (positive) | 12 Jan 2017 |
| Humanised IgG1 monoclonal antibody against the receptor-binding site of human placental growth factor EU/3/16/1819 |
Treatment of medulloblastoma | Active (positive) | 12 Jan 2017 |
| human hepatoma cell line HepaRG in bioartificial liver EU/3/16/1818 |
Treatment of acute liver failure | Active (positive) | 12 Jan 2017 |
| human donor haematopoietic stem and progenitor cells that have been treated ex vivo with the protein transduction domain of the HIV-1 transactivation protein f… EU/3/16/1817 |
Treatment in haematopoietic stem cell transplantation | Active (positive) | 12 Jan 2017 |
| genetically modified adeno-associated viral vector serotype 9 expressing shRNA as well as a codon-optimised shRNA-insensitive wildtype PABPN1 EU/3/16/1816 |
Treatment of oculopharyngeal muscular dystrophy | Active (positive) | 12 Jan 2017 |
| fluticasone propionate EU/3/16/1815 2 clinical trials → |
Treatment of eosinophilic oesophagitis | Active (positive) | 12 Jan 2017 |
| doxorubicin hydrochloride in a lipid-based pegylated nanoparticle modified with a 31-aminoacid peptide targeting nucleolin EU/3/16/1814 |
Treatment of malignant mesothelioma | Active (positive) | 12 Jan 2017 |
| autologous dendritic cells incubated ex vivo with zebularine and factor VIII EU/3/16/1813 |
Treatment of haemophilia A | Active (positive) | 12 Jan 2017 |
| antroquinonol EU/3/16/1812 |
Treatment of pancreatic cancer | Active (positive) | 12 Jan 2017 |
| 5-aminolevulinic acid EU/3/16/1811 |
Treatment of glioma | Active (positive) | 12 Jan 2017 |
| 3-pentylbenzeneacetic acid sodium salt EU/3/16/1810 |
Treatment of Alström syndrome | Withdrawn | 12 Jan 2017 |
| [5,10,15,20-tetrakis(4-carboxyphenyl)-21H,23H-porphine]manganese(III) chloride EU/3/16/1809 |
Treatment of Cockayne syndrome | Active (positive) | 12 Jan 2017 |
| (6aR, 10aR)-3-(1',1'-dimethylheptyl)-delta-8-tetrahydro-cannabinol-9-carboxylic acid EU/3/16/1808 |
Treatment of systemic sclerosis | Withdrawn | 12 Jan 2017 |
| udenafil EU/3/16/1807 |
Treatment of functional single ventricle congenital heart disease | Active (positive) | 12 Dec 2016 |
| propranolol EU/3/16/1805 21 clinical trials → |
Treatment of soft tissue sarcoma | Active (positive) | 12 Dec 2016 |
| pegylated recombinant human interleukin-10 EU/3/16/1804 |
Treatment of pancreatic cancer | Active (positive) | 12 Dec 2016 |
| Metformin EU/3/16/1803 105 clinical trials → |
Treatment of progressive myoclonic epilepsy type 2 (Lafora disease) | Active (positive) | 12 Dec 2016 |
| Ivosidenib EU/3/16/1802 · now authorised as Tibsovo; Tidhesco 26 clinical trials → |
Treatment of acute myeloid leukaemia | Active (positive) | 12 Dec 2016 |
| Ibudilast EU/3/16/1801 |
Treatment of amyotrophic lateral sclerosis | Active (positive) | 12 Dec 2016 |
| Dantrolene sodium EU/3/16/1800 |
Treatment of Wolfram syndrome | Withdrawn | 12 Dec 2016 |
| cabiralizumab EU/3/16/1799 |
Treatment of tenosynovial giant cell tumour, localised and diffuse type | Withdrawn | 12 Dec 2016 |
| avelumab EU/3/16/1798 31 clinical trials → |
Treatment of gastric cancer | Withdrawn | 12 Dec 2016 |
| arsenic trioxide EU/3/16/1797 11 clinical trials → |
Treatment of graft-versus-host disease | Active (positive) | 12 Dec 2016 |
| adeno-associated viral vector serotype 8 encoding engineered rhodopsin DNA-binding repressor and human rhodopsin expression cassettes EU/3/16/1796 |
Treatment of retinitis pigmentosa | Active (positive) | 12 Dec 2016 |
| adeno-associated viral vector serotype 8 containing the human CNGA3 gene under the control of a cone arrestin promoter EU/3/16/1795 |
Treatment of achromatopsia caused by mutations in the CNGA3 gene | Active (positive) | 12 Dec 2016 |
| 68Ga-DOTA-pABzA-DIG-dPhe-Gln-Trp-Ala-Val-Gly-His-NHCH[(CH2-CH(CH3)2]2 EU/3/16/1794 |
Diagnosis of gastrointestinal stromal tumours | Active (positive) | 12 Dec 2016 |
| zinc gluconate EU/3/16/1793 |
treatment of facioscapulohumeral muscular dystrophy | Active (positive) | 18 Nov 2016 |
| valproic acid EU/3/16/1792 4 clinical trials → |
Treatment of diffuse large B-cell lymphoma | Active (positive) | 18 Nov 2016 |
| vaccine consisting of 5 survivin peptides with different human leukocyte antigen restrictions EU/3/16/1791 |
Treatment of ovarian cancer | Active (positive) | 18 Nov 2016 |
| Tyr-Gly-Arg-Lys-Lys-Arg-Arg-Gln-Arg-Arg-Gly-Gly-Asp-Leu-Leu-Pro-Arg-Gly-Ser EU/3/16/1790 |
Treatment of Huntington’s disease | Active (positive) | 18 Nov 2016 |
| synthetic human hepcidin EU/3/16/1789 |
Treatment of sickle cell disease | Withdrawn | 18 Nov 2016 |
| sodium benzoate EU/3/16/1788 · now authorised as Prohippur |
Treatment of N-acetylglutamate synthetase (NAGS) deficiency | Active (positive) | 18 Nov 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.