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 |
|---|---|---|---|
| exon 45 specific phosphorothioate oligonucleotide EU/3/12/991 |
Treatment of Duchenne muscular dystrophy | Active (positive) | 26 Apr 2012 |
| vosaroxin EU/3/12/990 |
Treatment of acute myeloid leukaemia | Active (positive) | 26 Apr 2012 |
| 2-Allyl-1-[6-(1-hydroxy-1-methylethyl)pyridin-2-yl]-6-{[4-(4-methylpiperazin-1-yl)phenyl]amino}-1,2-dihydro-3H-pyrazolo[3,4-d]pyrimidin-3-one EU/3/12/989 |
Treatment of ovarian cancer | Withdrawn | 26 Apr 2012 |
| Halofuginone hydrobromide EU/3/12/988 |
Treatment of Duchenne muscular dystrophy | Withdrawn | 26 Apr 2012 |
| (E)-2,4,6-trimethoxystyryl-3-carboxymethylamino-4-methoxybenzyl-sulfone sodium salt (rigosertib sodium) EU/3/12/987 |
Treatment of myelodysplastic syndromes | Withdrawn | 26 Apr 2012 |
| Pomalidomide EU/3/12/986 64 clinical trials → |
Treatment of systemic sclerosis | Withdrawn | 26 Apr 2012 |
| recombinant human methionine proinsulin EU/3/12/985 |
Treatment of retinitis pigmentosa | Active (positive) | 26 Apr 2012 |
| 1-[(3R)-3-[4-amino-3-(4-phenoxyphenyl)-1H-pyrazolo[3,4 d]pyrimidin-1-yl]-1-piperidinyl]-2-propen-1-one (ibrutinib) EU/3/12/984 |
Treatment of chronic lymphocytic leukaemia | Withdrawn | 26 Apr 2012 |
| heterologous human adult liver-derived stem cells EU/3/12/983 |
Treatment of acute liver failure | Active (positive) | 26 Apr 2012 |
| ketoconazole EU/3/12/965 |
Treatment of Cushing's syndrome | Expired | 23 Apr 2012 |
| oleylphosphocholine EU/3/12/964 |
Treatment of leishmaniasis | Active (positive) | 23 Apr 2012 |
| Dipalmitoylphosphatidylcholine, 1-palmitoyl-2-oleoyl-sn-glycero-3-phosphoglycerol, sodium salt, synthetic surfactant protein C analogue and synthetic surfactan… EU/3/12/982 |
Respiratory distress syndrome in premature neonates of less than 37 weeks of gestational age | Active (positive) | 2 Apr 2012 |
| adenovirus-associated viral vector serotype 2 containing the human RPE65 gene (voretigene neparvovec) EU/3/12/981 · now authorised as Luxturna |
Treatment of Leber's congenital amaurosis | Active (positive) | 2 Apr 2012 |
| Genistein sodium salt dihydrate EU/3/12/980 |
Treatment of mucopolysaccharidosis type III (Sanfilippo syndrome) | Active (positive) | 2 Apr 2012 |
| sodium thiosulfate EU/3/12/979 5 clinical trials → |
Treatment of calciphylaxis | Active (positive) | 2 Apr 2012 |
| melatonin EU/3/12/978 23 clinical trials → |
Treatment of perinatal asphyxia | Active (positive) | 2 Apr 2012 |
| Linsitinib EU/3/12/977 2 clinical trials → |
Treatment of adrenal cortical carcinoma | Withdrawn | 2 Apr 2012 |
| antisense oligonucleotide targeted to the SMN2 gene (nusinersen) EU/3/12/976 · now authorised as Spinraza |
Treatment of 5q spinal muscular atrophy | Active (positive) | 2 Apr 2012 |
| Doxycycline hyclate EU/3/12/955 10 clinical trials → |
Treatment of familial amyloid polyneuropathy | Active (positive) | 2 Apr 2012 |
| carbetocin EU/3/12/975 9 clinical trials → |
Treatment of Prader-Willi syndrome | Withdrawn | 21 Mar 2012 |
| adeno-associated viral vector of serotype 5 containing the human alanine-glyoxylate-aminotransferase gene EU/3/12/974 |
Treatment of primary hyperoxaluria type 1 | Withdrawn | 21 Mar 2012 |
| recombinant human beta-glucuronidase (vestronidase alfa) EU/3/12/973 · now authorised as Mepsevii |
Treatment of mucopolysaccharidosis type VII (Sly syndrome) | Active (positive) | 21 Mar 2012 |
| tariquidar - |
treatment of P-gp positive breast cancer | Negative | 21 Mar 2012 |
| heterologous human adult liver-derived stem cells EU/3/12/971 |
Treatment of carbamoyl-phosphate synthase-1 deficiency | Active (positive) | 5 Mar 2012 |
| 6-ethynyl-1-(pentan-3-yl)-1H-imidazo[4,5-b]pyrazin-2(3H)-one (tirasemtiv) EU/3/12/970 |
Treatment of amyotrophic lateral sclerosis | Active (positive) | 5 Mar 2012 |
| human monoclonal antibody targeting Staphylococcus aureus alpha-toxin (tosatoxumab) EU/3/12/968 |
Treatment of pneumonia caused by Staphylococcus aureus | Active (positive) | 5 Mar 2012 |
| sodium nitrite EU/3/12/967 |
Treatment of pulmonary arterial hypertension | Active (positive) | 5 Mar 2012 |
| (1-methyl-2-nitro-1H-imidazole-5-yl)methyl N,N'-bis(2-bromoethyl)diamidophosphate EU/3/12/966 |
Treatment of soft tissue sarcoma | Withdrawn | 5 Mar 2012 |
| Doxycycline hyclate EU/3/12/961 10 clinical trials → |
Treatment of systemic amyloidosis caused by beta-2 microglobulin | Active (positive) | 5 Mar 2012 |
| Glucagon EU/3/12/960 2 clinical trials → |
Treatment of congenital hyperinsulinism | Active (positive) | 5 Mar 2012 |
| Vincaleukoblastin-23-oic acid, O4-deacetyl-, 2-[(2-mercaptoethoxy)carbonyl]hydrazide, disulfide with N-[4-[[(2-amino-3,4-dihydro-4-oxo-6-pteridinyl)methyl]amin… EU/3/12/959 |
Treatment of ovarian cancer | Withdrawn | 9 Feb 2012 |
| N-[4-[[(2-amino-3,4-dihydro-4-oxo-6-pteridinyl)methyl]amino]benzoyl]-D-gamma-glutamyl-(2S)-2-amino-beta-alanyl-L-alpha-aspartyl-L-cysteine;folic acid EU/3/12/958 4 clinical trials → |
Diagnosis of positive folate-receptor status in ovarian cancer | Withdrawn | 9 Feb 2012 |
| autologous haematopoietic cells genetically modified with a lentiviral vector containing the human gp91(phox) gene EU/3/12/957 |
Treatment of X-linked chronic granulomatous disease | Active (positive) | 9 Feb 2012 |
| human monoclonal antibody against Fas ligand EU/3/12/956 |
Treatment of pemphigus | Active (positive) | 9 Feb 2012 |
| S[+] apomorphine EU/3/12/954 |
Treatment of amyotrophic lateral sclerosis | Active (positive) | 9 Feb 2012 |
EU/3/12/953 |
Treatment of West syndrome | Withdrawn | 9 Feb 2012 |
| nimorazole maleate EU/3/12/952 |
Treatment of squamous-cell carcinoma of the head and neck in patients undergoing radiotherapy | Withdrawn | 9 Feb 2012 |
| sodium phenylbutyrate EU/3/12/951 |
Treatment of carbamoyl-phosphate synthase-1 deficiency | Withdrawn | 9 Feb 2012 |
| sodium phenylbutyrate EU/3/12/950 |
Treatment of ornithine-transcarbamylase deficiency | Withdrawn | 9 Feb 2012 |
| sodium phenylbutyrate EU/3/12/949 |
Treatment of citrullinaemia type 1 | Withdrawn | 9 Feb 2012 |
| sodium phenylbutyrate EU/3/11/948 |
Treatment of 5q spinal muscular atrophy | Withdrawn | 11 Jan 2012 |
| recombinant protein consisting of modified human growth hormone releasing hormone and the translocation and endopeptidase domains of botulinum toxin serotype D EU/3/11/947 |
Treatment of acromegaly | Withdrawn | 11 Jan 2012 |
| recombinant homodimer of the human annexin V EU/3/11/946 |
Prevention of ischaemia/reperfusion injury associated with solid organ transplantation | Withdrawn | 11 Jan 2012 |
| N,N'-bis(2-mercaptoethyl)isophthalamide (emeramide) EU/3/11/944 |
Treatment of mercury toxicity | Active (positive) | 11 Jan 2012 |
| mogamulizumab EU/3/11/943 3 clinical trials → |
Treatment of peripheral T-cell lymphoma (nodal, other extranodal and leukaemic/disseminated) | Active (positive) | 11 Jan 2012 |
| cytarabine;daunorubicin EU/3/11/942 · now authorised as Vyxeos liposomal (previously Vyxeos) 103 clinical trials → |
Treatment of acute myeloid leukaemia | Active (positive) | 11 Jan 2012 |
| Lipopolysaccharide of Ochrobactrum intermedium EU/3/11/941 |
Prevention of sepsis in at-risk premature infants of less than or equal to 32 weeks | Active (positive) | 11 Jan 2012 |
| chimeric locked nucleic acid-deoxynucleoside phosphorothioate-linked oligonucleotide directed against microRNA-451 EU/3/11/940 |
Treatment of polycythaemia vera | Withdrawn | 11 Jan 2012 |
| Brentuximab vedotin EU/3/11/939 21 clinical trials → |
Treatment of cutaneous T-cell lymphoma | Withdrawn | 11 Jan 2012 |
| adeno-associated viral vector containing the human factor IX gene EU/3/11/938 |
Treatment of haemophilia B | Active (positive) | 11 Jan 2012 |
| alpha-tocotrienol quinone EU/3/11/937 |
Treatment of Leigh syndrome | Withdrawn | 9 Dec 2011 |
| human haptoglobin EU/3/11/936 |
Treatment of sickle cell disease | Active (positive) | 9 Dec 2011 |
| Interferon gamma EU/3/11/935 |
Treatment of Friedreich's ataxia | Withdrawn | 9 Dec 2011 |
| 4-[[9-[(3S)-tetrahydro-3-furanyl]-8-[(2,4,6-trifluorophenyl)amino]-9H-purin-2-yl]amino]-trans-cyclohexanol (tanzisertib) EU/3/11/934 |
Treatment of idiopathic pulmonary fibrosis | Withdrawn | 9 Dec 2011 |
| nanoliposomal irinotecan EU/3/11/933 · now authorised as Onivyde pegylated liposomal (previously Onivyde) 5 clinical trials → |
Treatment of pancreatic cancer | Active (positive) | 9 Dec 2011 |
| pegylated proline-interferon alpha-2b (ropeginterferon alfa-2b) EU/3/11/932 |
Treatment of polycythaemia vera | Withdrawn | 9 Dec 2011 |
| Plerixafor EU/3/11/931 |
Adjunctive treatment to cytotoxic therapy in acute myeloid leukaemia | Withdrawn | 9 Dec 2011 |
| resminostat EU/3/11/930 |
Treatment of Hodgkin's lymphoma | Withdrawn | 9 Dec 2011 |
| adeno-associated viral vector serotype 8 containing the human AIPL1 gene EU/3/11/929 |
Treatment of Leber's congenital amaurosis type 4 | Withdrawn | 9 Dec 2011 |
| cysteamine EU/3/11/928 |
Treatment of cystic fibrosis | Active (positive) | 9 Dec 2011 |
| sinapultide;Dipalmitoylphosphatidylcholine;palmitoyl-oleoyl phosphatidylglycerol sodium salt;palmitic acid EU/3/11/927 |
Treatment of cystic fibrosis | Active (positive) | 27 Oct 2011 |
| recombinant human minibody against complement component C5 EU/3/11/926 |
Treatment of primary membranoproliferative glomerulonephritis | Withdrawn | 27 Oct 2011 |
| mifepristone EU/3/11/925 · now authorised as Corluxin 3 clinical trials → |
Treatment of hypercortisolism (Cushing's syndrome) of endogenous origin | Active (positive) | 27 Oct 2011 |
| lenalidomide EU/3/11/924 167 clinical trials → |
Treatment of mantle cell lymphoma | Withdrawn | 27 Oct 2011 |
| L-cysteine, L-leucyl-L-alpha-glutamyl-L-alpha-glutamyl-L-lysyl-L-lysylglycyl-L-asparaginyl-L-tyrosyl-L-valyl-L-valyl-L-threonyl-L-alpha-aspartyl-L-histidyl-S-[… EU/3/11/923 |
Treatment of glioma | Withdrawn | 27 Oct 2011 |
| Human platelet antigen-1a immunoglobulin EU/3/11/922 |
Prevention of fetal and neonatal alloimmune thrombocytopenia | Withdrawn | 27 Oct 2011 |
| Glycosylation independent lysosomal targeting (GILT)-tagged recombinant human acid alpha glucosidase EU/3/11/921 |
Treatment of glycogen storage disease type II (Pompe's disease) | Active (positive) | 27 Oct 2011 |
| Gallium (68Ga)-pasireotide tetraxetan EU/3/11/920 |
Diagnosis of gastro-entero-pancreatic neuroendocrine tumours | Active (positive) | 27 Oct 2011 |
| clonidine hydrochloride EU/3/11/919 8 clinical trials → |
Prevention of oral mucositis in head and neck cancer patients undergoing radiation therapy | Active (positive) | 27 Oct 2011 |
| brivanib alaninate EU/3/11/918 |
Treatment of hepatocellular carcinoma | Withdrawn | 27 Oct 2011 |
| adeno-associated viral vector containing the human alpha-N-acetylglucosaminidase gene EU/3/11/917 |
Treatment of mucopolysaccharidosis type IIIB (Sanfilippo B syndrome) | Active (positive) | 27 Oct 2011 |
| 2-Hydroxyoleic acid EU/3/11/916 |
Treatment of glioma | Active (positive) | 27 Oct 2011 |
| 1-(4-{4-Amino-7-[1-(2-hydroxyethyl)-1H-pyrazol-4-yl]thieno[3,2-c]pyridin-3-yl}phenyl)-3-(3-fluorophenyl)urea EU/3/11/915 |
Treatment of acute myeloid leukaemia | Withdrawn | 27 Oct 2011 |
| smilagenin EU/3/11/914 |
Treatment of amyotrophic lateral sclerosis | Active (positive) | 27 Sep 2011 |
| resminostat EU/3/11/913 |
Treatment of hepatocellular carcinoma | Withdrawn | 27 Sep 2011 |
| reparixin EU/3/11/912 |
Prevention of graft loss in pancreatic islet transplantation | Active (positive) | 27 Sep 2011 |
| recombinant human galactocerebrosidase EU/3/11/911 |
Treatment of globoid cell leukodystrophy (Krabbe disease) | Withdrawn | 27 Sep 2011 |
| NH2-Cys-Ser-Ser-Val-Thr-Ala-Trp-Thr-Thr-Gly-Cys-Gly-CONH2 EU/3/11/910 |
Treatment of traumatic spinal cord injury | Active (positive) | 27 Sep 2011 |
| macitentan EU/3/11/909 4 clinical trials → |
Treatment of pulmonary arterial hypertension | Withdrawn | 27 Sep 2011 |
| Kifunensine EU/3/11/908 |
Treatment of gamma sarcoglycanopathy | Withdrawn | 27 Sep 2011 |
| Kifunensine EU/3/11/907 |
Treatment of delta sarcoglycanopathy | Withdrawn | 27 Sep 2011 |
| Kifunensine EU/3/11/906 |
Treatment of beta sarcoglycanopathy | Withdrawn | 27 Sep 2011 |
| Kifunensine EU/3/11/905 |
Treatment of alpha-sarcoglycanopathy | Withdrawn | 27 Sep 2011 |
| heterologous human adult liver-derived stem cells EU/3/11/904 |
Treatment of ornithine transcarbamylase deficiency | Active (positive) | 27 Sep 2011 |
| genetically modified Lactococcus lactis bacteria containing the human trefoil factor 1 gene EU/3/11/903 |
Prevention of oral mucositis in head and neck cancer patients undergoing radiation therapy | Active (positive) | 27 Sep 2011 |
| eflornithine EU/3/11/902 6 clinical trials → |
Treatment of neuroblastoma | Withdrawn | 27 Sep 2011 |
| Dinaciclib EU/3/11/901 |
Treatment of chronic lymphocytic leukaemia | Withdrawn | 27 Sep 2011 |
| 20-pentaerythritol poly (oxy-1,2-ethanediyl)-carboxymethyl-glycinate-7-ethyl-10-hydroxycamptothecine 10-[1,4'-bipiperidine]-1'-carboxylate EU/3/11/900 |
Treatment of ovarian cancer | Active (positive) | 27 Sep 2011 |
| 2,2'-{2-[(1R)-1-({[(2,5-dichlorobenzoyl)amino]acetyl}amino)-3-methylbutyl]-5-oxo-1,3,2-dioxaborolane-4,4-diyl}diacetic acid EU/3/11/899 · now authorised as Ninlaro |
Treatment of multiple myeloma | Active (positive) | 27 Sep 2011 |
| sirolimus EU/3/11/898 · now authorised as Xegafri 32 clinical trials → |
Treatment of chronic non-infectious uveitis | Withdrawn | 30 Aug 2011 |
| N-{[(5S)-3-(3-fluoro-4-thiomorpholin-4-ylphenyl)-2-oxo-1,3-oxazolidin-5-yl]methyl}acetamide EU/3/11/897 |
Treatment of tuberculosis | Active (positive) | 30 Aug 2011 |
| multilamellar microvesicle comprising phosphatidylcholine, sphingomyelin, phosphatidylethanolamine, phosphatidylserine, phosphatidylinositol and cholesterol EU/3/11/896 |
Treatment of cystic fibrosis | Withdrawn | 30 Aug 2011 |
| hydroxy-propyl-beta-cyclodextrin (hydroxypropylbetadex) EU/3/11/895 |
Treatment of Niemann-Pick's disease, type C | Active (positive) | 30 Aug 2011 |
| everolimus EU/3/11/894 53 clinical trials → |
Treatment of gastric cancer | Withdrawn | 30 Aug 2011 |
| cardiotrophin-1 EU/3/11/893 |
Treatment of acute liver failure | Active (positive) | 30 Aug 2011 |
| 5-[1-(2,6-Dichlorobenzyl)piperidin-4-ylmethoxy]quinazoline-2,4-diamine dihydrochloride EU/3/11/892 |
Treatment of 5q spinal muscular atrophy | Active (positive) | 30 Aug 2011 |
| human anthrax monoclonal antibody EU/3/11/891 |
For post-exposure prophylaxis of inhalation anthrax disease | Withdrawn | 5 Aug 2011 |
| peretinoin EU/3/11/890 |
Treatment of hepatocellular carcinoma | Active (positive) | 5 Aug 2011 |
| pegylated recombinant Erwinia chrysanthemi L-asparaginase EU/3/11/889 |
Treatment of acute lymphoblastic leukaemia | Active (positive) | 5 Aug 2011 |
| N-(cyanomethyl)-4-(2-{[4-(morpholin-4-yl)phenyl]amino}pyrimidin-4-yl)benzamide, dihydrochloride salt (momelotinib) EU/3/11/888 · now authorised as Omjjara |
Treatment of primary myelofibrosis | Active (positive) | 5 Aug 2011 |
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.