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 |
|---|---|---|---|
| 2-hydroxymethyl-2-methoxymethyl-1-azabicyclo[2,2,2]octan-3-one EU/3/14/1386 |
Treatment of ovarian cancer | Active (positive) | 16 Dec 2014 |
| Heat-killed Mycobacterium obuense (whole cell) EU/3/14/1385 |
Treatment of pancreatic cancer | Active (positive) | 16 Dec 2014 |
EU/3/14/1384 |
Treatment of WHIM syndrome | Active (positive) | 16 Dec 2014 |
| single-chain urokinase plasminogen activator EU/3/14/1383 |
Treatment of pleural empyema | Active (positive) | 16 Dec 2014 |
| allogeneic CD34+ cells expanded ex vivo with an aryl hydrocarbon receptor antagonistย (spanlecortemlocel) EU/3/14/1382 |
Treatment of acute lymphoblastic leukaemia | Withdrawn | 16 Dec 2014 |
| sodium ascorbate;menadione sodium bisulfite - |
Treatment of autosomal dominant polycystic kidney disease | Negative | 11 Dec 2014 |
| adeno-associated viral vector serotype 8 containing the human MD1 gene EU/3/14/1381 |
Treatment of Duchenne muscular dystrophy | Active (positive) | 19 Nov 2014 |
| ataluren EU/3/14/1380 |
Treatment of mucopolysaccharidosis type I | Withdrawn | 19 Nov 2014 |
| Dantrolene sodium EU/3/14/1379 |
Treatment of malignant hyperthermia | Active (positive) | 19 Nov 2014 |
| Diaspirin cross-linked haemoglobin EU/3/14/1378 |
Treatment of hepatocellular carcinoma | Active (positive) | 19 Nov 2014 |
| chloroquine EU/3/14/1377 2 clinical trials → |
Treatment of glioma | Active (positive) | 19 Nov 2014 |
| arimoclomol citrate EU/3/14/1376 · now authorised as Miplyffa; Meplyffa |
Treatment of Niemann-Pick's disease, type C | Active (positive) | 19 Nov 2014 |
| Pro-Pro-Thr-Val-Pro-Thr-Arg EU/3/14/1375 |
Treatment of xeroderma pigmentosum | Withdrawn | 19 Nov 2014 |
| (3S)-1-azabicyclo[2.2.2]oct-3-yl{2-[2-(4-fluorophenyl)-1,3-thiazol-4-yl]propan-2-yl}carbaNmate (venglustat) EU/3/14/1374 |
Treatment of Gaucher disease | Active (positive) | 19 Nov 2014 |
EU/3/14/1373 · now authorised as Vynpenta (previously AvacopanChemoCentryx); Tavneos |
Treatment of granulomatosis with polyangiitis | Active (positive) | 19 Nov 2014 |
| (2R,3S)-2-(4-cyclopentylaminophenyl)-1-(2-fluoro-6-methylbenzoyl)piperidine-3-carboxylic acid(4-methyl-3-trifluoromethylphenyl)amNide EU/3/14/1372 · now authorised as Vynpenta (previously AvacopanChemoCentryx); Tavneos |
Treatment of microscopic polyangiitis (avacopan) | Active (positive) | 19 Nov 2014 |
| Humanised IgG1 monoclonal antibody against human eotaxin-2 EU/3/14/1371 |
Treatment of systemic sclerosis | Active (positive) | 19 Nov 2014 |
| siponimod EU/3/14/1370 3 clinical trials → |
Treatment of polymyositis | Withdrawn | 19 Nov 2014 |
| siponimod EU/3/14/1369 3 clinical trials → |
Treatment of dermatomyositis | Withdrawn | 19 Nov 2014 |
| palovarotene EU/3/14/1368 · now authorised as Sohonos |
Treatment of fibrodysplasia ossificans progressiva | Withdrawn | 19 Nov 2014 |
| bazedoxifene acetate EU/3/14/1367 |
Treatment of hereditary haemorrhagic telangiectasia | Active (positive) | 19 Nov 2014 |
| recombinant human pentraxin-2 EU/3/14/1366 |
Treatment of primary myelofibrosis | Active (positive) | 19 Nov 2014 |
| recombinant human pentraxin-2 EU/3/14/1365 |
Treatment of post-polycythaemia vera myelofibrosis | Active (positive) | 19 Nov 2014 |
| olaptesed pegol EU/3/14/1364 |
Treatment of glioma | Active (positive) | 19 Nov 2014 |
| 4-[[(1S,4S)-5-[[4-[4-(oxazol-2-yl)phenoxy]phenyl]methyl]-2,5-diazabicyclo[2.2.1]hept-2-yl]methyl]benzoic acid (acebilustat) EU/3/14/1363 |
Treatment of cystic fibrosis | Active (positive) | 19 Nov 2014 |
| 1-(6-benzothiazolylsulfonyl)-5-chloro-1H-indole-2-butanoic acid (lanifibranor) EU/3/14/1362 |
Treatment of idiopathic pulmonary fibrosis | Active (positive) | 19 Nov 2014 |
| 1-(6-benzothiazolylsulfonyl)-5-chloro-1H-indole-2-butanoic acid (lanifibranor) EU/3/14/1361 |
Treatment of systemic sclerosis | Withdrawn | 19 Nov 2014 |
| A combination of H-Lys-Lys-Gly-Pro-Arg-Cys(SH)-Leu-Thr-Arg-Tyr-Tyr-Ser-Ser-Phe-Val-Asn-Met-Glu-Gly-Lys-Lys-OH and H-Lys-Lys-Gly-Asp-Asn-Ile-Met-Val-Thr-Phe-Argโฆ EU/3/14/1360 |
Treatment of haemophilia A | Withdrawn | 19 Nov 2014 |
| pentosan polysulfate sodium EU/3/14/1359 3 clinical trials → |
Treatment of mucopolysaccharidosis type I | Active (positive) | 19 Nov 2014 |
| recombinant human pentraxin-2 EU/3/14/1358 |
Treatment of post-essential thrombocythaemia myelofibrosis | Active (positive) | 19 Nov 2014 |
| imatinib EU/3/14/1357 32 clinical trials → |
Treatment of acute respiratory distress syndrome (ARDS) | Active (positive) | 19 Nov 2014 |
| Donor T lymphocytes depleted ex-vivo of host alloreactive T cells using photodynamic treatment EU/3/14/1356 · now authorised as Luxceptar |
Treatment of acute myeloid leukaemia | Withdrawn | 19 Nov 2014 |
| selinexor EU/3/14/1355 47 clinical trials → |
Treatment of plasma cell myeloma | Active (positive) | 19 Nov 2014 |
| selinexor EU/3/14/1354 47 clinical trials → |
Treatment of chronic lymphocytic leukaemia / small lymphocytic lymphoma | Active (positive) | 19 Nov 2014 |
| Mexiletine hydrochloride EU/3/14/1353 · now authorised as Namuscla 5 clinical trials → |
Treatment of myotonic disorders | Active (positive) | 19 Nov 2014 |
| S3,S13-cyclo(D-tyrolsyl-L-isoleucyl-L-cysteinyl-L-valyl-1-methyl-L-tryptophyl-L-glutaminyl-L-aspartyl-L-tryptophyl-N-methyl-L-glycyl-L-alanyl-L-histidyl-L-argiโฆ EU/3/14/1327 |
Treatment of paroxysmal nocturnal haemoglobinuria | Active (positive) | 22 Oct 2014 |
| raxibacumab EU/3/14/1352 |
Treatment of inhalation anthrax disease | Active (positive) | 15 Oct 2014 |
| recombinant human monoclonal IgG1 antibody for fibroblast growth factor 23 (burosumab) EU/3/14/1351 · now authorised as Crysvita |
Treatment of X-linked hypophosphataemia | Active (positive) | 15 Oct 2014 |
| recombinant human monoclonal antibody of the IgG1 kappa class against human macrophage colony-stimulating factor (lacnotuzumab) EU/3/14/1350 |
Treatment of tenosynovial giant cell tumour, localised and diffuse type | Withdrawn | 15 Oct 2014 |
| recombinant human insulin receptor monoclonal antibody-fused-ฮฑ-L-iduronidase EU/3/14/1349 |
Treatment of mucopolysaccharidosis type I | Withdrawn | 15 Oct 2014 |
| recombinant human bone morphogenetic protein 4 EU/3/14/1348 |
Treatment of glioma | Active (positive) | 15 Oct 2014 |
| pyridoxal 5'-phosphate EU/3/14/1347 |
Treatment of pyridoxamine 5'-phosphate oxidase deficiency | Active (positive) | 15 Oct 2014 |
| oxalobacter formigenes strain HC-1 EU/3/14/1346 |
Treatment of short bowel syndrome | Active (positive) | 15 Oct 2014 |
| osilodrostat EU/3/14/1345 · now authorised as Isturisa 2 clinical trials → |
Treatment of Cushing's syndrome | Active (positive) | 15 Oct 2014 |
| nitric oxide EU/3/14/1344 4 clinical trials → |
Treatment of cystic fibrosis | Active (positive) | 15 Oct 2014 |
| Immunoglobulin G1, anti-(human tumour-associated calcium signal transducer 2)(human-Mus musculus monoclonal hRS7 heavy chain), disulfide with human-Mus musculuโฆ EU/3/14/1343 |
Treatment of pancreatic cancer | Withdrawn | 15 Oct 2014 |
| cysteamine hydrochloride EU/3/14/1341 · now authorised as Dropcys |
Treatment of cystinosis | Active (positive) | 15 Oct 2014 |
| cultured allogeneic corneal limbal stem cells EU/3/14/1340 |
Treatment of limbal stem cell deficiency | Active (positive) | 15 Oct 2014 |
| cannabidiol EU/3/14/1339 · now authorised as Epidyolex 16 clinical trials → |
Treatment of Dravet syndrome | Active (positive) | 15 Oct 2014 |
| adeno-associated viral vector serotype 8 containing the human UGT1A1 gene (volrubigene ralaparvovec) EU/3/14/1338 |
Treatment of Crigler-Najjar syndrome | Active (positive) | 15 Oct 2014 |
| acamprosate calcium EU/3/14/1337 |
Treatment of fragile X syndrome | Active (positive) | 15 Oct 2014 |
| (S)-6-hydroxy-2,5,7,8-tetramethyl-N-((R)-piperidin-3-yl)chroman-2-carboxamide hydrochloride EU/3/14/1336 |
Treatment of Leigh syndrome | Withdrawn | 15 Oct 2014 |
| (S)-2-(1-((6-amino-5-cyanopyrimidin-4-yl)amino)ethyl)-4-oxo-3-phenyl-3,4-dihydropyrrolo[2,1-f][1,2,4]triazine-5-carbonitrile (tanziertib) EU/3/14/1335 |
Treatment of pemphigus | Withdrawn | 15 Oct 2014 |
EU/3/14/1334 |
Treatment of fragile X syndrome | Active (positive) | 15 Oct 2014 |
| Lumacaftor;ivacaftor EU/3/14/1333 3 clinical trials → |
Treatment of cystic fibrosis | Withdrawn | 22 Aug 2014 |
| Macromolecular conjugate of heparin sodium on a polymer backbone EU/3/14/1332 |
Prevention of ischaemia/reperfusion injury associated with solid organ transplantation | Active (positive) | 22 Aug 2014 |
| recombinant fusion protein consisting of a modified form of the extracellular domain of human activin receptor IIB linked to the human IgG1 Fc domainย (luspaterโฆ EU/3/14/1331 · now authorised as Reblozyl |
Treatment of myelodysplastic syndromes | Active (positive) | 22 Aug 2014 |
| Lentiviral vector containing the human liver and erythroid pyruvate kinase (PKLR) gene EU/3/14/1330 |
Treatment of pyruvate kinase deficiency | Active (positive) | 22 Aug 2014 |
| variant of recombinant human fibroblast growth factor 19 EU/3/14/1329 |
Treatment of primary biliary cirrhosis | Withdrawn | 22 Aug 2014 |
| 4-{[(2R,3S,4R,5S)-4-(4-chloro-2-fluoro-phenyl)-3-(3-chloro-2-fluoro-phenyl)-4-cyano-5-(2,2-dimethyl-propyl)-pyrrolidine-2-carbonyl]-amino}-3-methoxy-benzoic acโฆ EU/3/14/1328 |
Treatment of acute myeloid leukaemia | Withdrawn | 22 Aug 2014 |
| vector based on an adeno-associated virus serotype 2 backbone, pseudo-serotyped with a type 8 capsid, which carries the coding sequence of the human TYMP gene โฆ EU/3/14/1326 |
Treatment of mitochondrial neurogastrointestinal encephalomyopathy (MNGIE) | Active (positive) | 22 Aug 2014 |
| obinutuzumab EU/3/14/1325 88 clinical trials → |
Treatment of diffuse large B-cell lymphoma | Withdrawn | 22 Aug 2014 |
| obinutuzumab EU/3/14/1325 88 clinical trials → |
Treatment of diffuse large B-cell lymphoma | Active (positive) | 22 Aug 2014 |
| 2-(2-Methyl-5-nitro-1H-imidazol-1-yl)ethylsulfamide EU/3/14/1324 |
Treatment of small cell lung cancer | Active (positive) | 22 Aug 2014 |
| [5-Amino-1-(4-fluoro-phenyl)-1H-pyrazol-4-yl]-[3-(2,3-dihydroxy-propoxy)-phenyl]-methanone EU/3/14/1323 |
Treatment of pancreatic cancer | Active (positive) | 22 Aug 2014 |
| Humanised IgG1 monoclonal antibody against human KIR3DL2 EU/3/14/1322 |
Treatment of cutaneous T-cell lymphoma | Active (positive) | 22 Aug 2014 |
| adeno-associated viral vector serotype 8 containing the human UGT1A1 gene (volrubigene ralaparvovec) EU/3/14/1321 |
Treatment of Crigler-Najjar syndrome | Withdrawn | 22 Aug 2014 |
| recombinant human diamine oxidase EU/3/14/1320 |
Treatment of mastocytosis | Active (positive) | 22 Aug 2014 |
| recombinant factor VIIa modified with three terminal repeats derived from the ฮฒ chain of human chorionic gonadotropin EU/3/14/1319 |
Treatment of haemophilia B | Active (positive) | 22 Aug 2014 |
| ulinastatin EU/3/14/1318 3 clinical trials → |
Treatment of acute pancreatitis | Active (positive) | 22 Aug 2014 |
| (Z)-3-(3-(3,5-bis(trifluoromethyl)phenyl)-1H-1,2,4-triazol-1-yl)-N'-(pyrazin-2-yl)acrylohydrazide EU/3/14/1317 |
Treatment of diffuse large B-cell lymphoma | Active (positive) | 22 Aug 2014 |
| recombinant factor VIIa modified with three terminal repeats derived from the ฮฒ chain of human chorionic gonadotropin EU/3/14/1316 |
Treatment of haemophilia A | Active (positive) | 22 Aug 2014 |
| recombinant human apolipoprotein A-I in a complex with phospholipids EU/3/14/1315 |
Treatment of apolipoprotein A-I deficiency | Active (positive) | 22 Aug 2014 |
| recombinant human apolipoprotein A-I in a complex with phospholipids EU/3/14/1314 |
Treatment of ATP-binding cassette transporter A1 deficiency | Active (positive) | 22 Aug 2014 |
| (Z)-3-(3-(3,5-bis(trifluoromethyl)phenyl)-1H-1,2,4-triazol-1-yl)-N'-(pyrazin-2-yl)acrylohydrazide EU/3/14/1313 |
Treatment of acute myeloid leukaemia | Active (positive) | 22 Aug 2014 |
| recombinant factor VIIa modified with three terminal repeats derived from the ฮฒ chain of human chorionic gonadotropin EU/3/14/1312 |
Treatment of congenital factor VII deficiency | Active (positive) | 22 Aug 2014 |
| Gevokizumab EU/3/14/1311 |
Treatment of Schnitzler syndrome | Withdrawn | 22 Aug 2014 |
EU/3/14/1310 |
Treatment of Fabry disease | Active (positive) | 22 Aug 2014 |
| 17ฮฑ,21-Dihydroxy-16ฮฑ-methyl-pregna-1,4,9(11)-triene-3,20-dione (vamorolone) EU/3/14/1309 · now authorised as Agamree |
Treatment of Duchenne muscular dystrophy | Active (positive) | 22 Aug 2014 |
| sodium ascorbate;menadione sodium bisulfite EU/3/14/1308 |
Treatment of autosomal dominant polycystic liver disease | Active (positive) | 22 Aug 2014 |
| retinol EU/3/14/1307 |
Prevention of bronchopulmonary dysplasia | Active (positive) | 22 Aug 2014 |
| cysteamine bitartrate (gastroresistant) (mercaptamine) EU/3/14/1306 |
Treatment of Huntingtonโs disease | Active (positive) | 29 Jul 2014 |
| Humanised recombinant monoclonal antibody against epidermal growth factor receptor conjugated to maleimidocaproyl monomethylauristatin F(depatuxizumab mafodotin EU/3/14/1305 |
Treatment of glioma | Withdrawn | 29 Jul 2014 |
| Eculizumab EU/3/14/1304 · now authorised as Soliris 12 clinical trials → |
Treatment myasthenia gravis | Active (positive) | 29 Jul 2014 |
| cediranib EU/3/14/1303 10 clinical trials → |
Treatment of ovarian cancer | Active (positive) | 29 Jul 2014 |
| Oxytocin EU/3/14/1302 22 clinical trials → |
Treatment of Prader-Willi syndrome | Active (positive) | 29 Jul 2014 |
| Humanised anti-alpha ฮฝ beta 6 monoclonal antibody EU/3/14/1301 |
Treatment of idiopathic pulmonary fibrosis | Active (positive) | 29 Jul 2014 |
| recombinant fusion protein consisting of a modified form of the extracellular domain of human activin receptor IIB linked to the human IgG1 Fc domainย (luspaterโฆ EU/3/14/1300 · now authorised as Reblozyl |
Treatment of beta thalassaemia intermedia and major | Active (positive) | 29 Jul 2014 |
| riociguat EU/3/14/1299 2 clinical trials → |
Treatment of systemic sclerosis | Withdrawn | 29 Jul 2014 |
| synthetic double-stranded siRNA oligonucleotide directed against antithrombin mRNA and covalently linked to a ligand containing three N-acetylgalactosamine resโฆ EU/3/14/1298 |
Treatment of haemophilia B | Active (positive) | 29 Jul 2014 |
| synthetic double-stranded siRNA oligonucleotide directed against antithrombin mRNA and covalently linked to a ligand containing three N-acetylgalactosamine resโฆ EU/3/14/1297 |
Treatment of haemophilia A | Active (positive) | 29 Jul 2014 |
| adeno-associated viral vector serotype 9 containing the human cardiac calsequestrin gene EU/3/14/1296 |
Treatment of catecholaminergic polymorphic ventricular tachycardia | Active (positive) | 29 Jul 2014 |
| Marizomib EU/3/14/1295 |
Treatment of plasma cell myeloma | Active (positive) | 29 Jul 2014 |
| sodium acetate salt of the synthetic peptide H-D-Ala-Ser-Pro-Met-Leu-Val-Ala-Tyr-Asp-D-Ala-OH EU/3/14/1294 |
Treatment of necrotising soft tissue infections | Active (positive) | 29 Jul 2014 |
| recombinant monoclonal antibody to human serum amyloid Pย component (dezamizumab) EU/3/14/1293 |
Treatment of AL amyloidosis | Withdrawn | 29 Jul 2014 |
| carboxy pyrrolidine hexanoyl pyrrolidine carboxylateย (miridesap) EU/3/14/1292 |
Treatment of AL amyloidosis | Withdrawn | 29 Jul 2014 |
| rilotumumab EU/3/14/1291 |
Treatment of gastric cancer | Withdrawn | 29 Jul 2014 |
| adeno-associated viral vector serotype 2 containing the human REP1 gene EU/3/14/1290 |
Treatment of choroideraemia | Active (positive) | 4 Jul 2014 |
| recombinant human alpha-1-microglobulin EU/3/14/1289 |
Treatment of pre-eclampsia | Withdrawn | 4 Jul 2014 |
| Norursodeoxycholic acid EU/3/14/1288 |
Treatment of primary sclerosing cholangitis | Active (positive) | 4 Jul 2014 |
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.