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 |
|---|---|---|---|
| recombinant human mesencephalic astrocyte-derived neurotrophic factor EU/3/15/1486 |
Treatment of retinitis pigmentosa | Active (positive) | 24 Apr 2015 |
| sodium 2-hydroxylinoleate EU/3/15/1485 |
Treatment of neuroblastoma | Active (positive) | 24 Apr 2015 |
| nitric oxide EU/3/15/1484 4 clinical trials → |
Treatment of cystic fibrosis | Withdrawn | 24 Apr 2015 |
| xenon EU/3/15/1483 2 clinical trials → |
Treatment of perinatal asphyxia | Active (positive) | 24 Apr 2015 |
| adeno-associated viral vector serotype 5 containing the human CHM gene EU/3/15/1482 |
Treatment of choroideraemia | Active (positive) | 24 Apr 2015 |
| phenol, 4-[2-(aminomethyl)-4-thiazolyl]-2,6-bis(1,1-dimethylethyl) monohydrochloride EU/3/15/1481 |
Treatment of Huntington’s disease | Withdrawn | 24 Apr 2015 |
| rintatolimod EU/3/15/1480 |
Treatment of Ebola virus disease | Active (positive) | 24 Apr 2015 |
| recombinant monoclonal IgG1 antibody against T-cell immune response cDNA 7 EU/3/15/1479 |
Prevention of graft rejection following solid organ transplantation | Active (positive) | 24 Apr 2015 |
| Human reovirus type 3 Dearing strain EU/3/15/1477 |
Treatment of pancreatic cancer | Active (positive) | 24 Apr 2015 |
| 1-(4-(N-glycylamido)phenyl)-3-trifluoromethyl-5-(phenanthren-2-yl)-pyrazole-hydrochloride EU/3/15/1476 |
Treatment of tularaemia | Active (positive) | 24 Apr 2015 |
| 1-(4-(N-glycylamido)phenyl)-3-trifluoromethyl-5-(phenanthren-2-yl)-pyrazole-hydrochloride EU/3/15/1475 |
Treatment of cryptococcosis | Active (positive) | 24 Apr 2015 |
| Ecothiopate iodide EU/3/15/1474 |
Treatment of Stargardt's disease | Withdrawn | 24 Apr 2015 |
| lenalidomide EU/3/15/1473 167 clinical trials → |
Treatment of marginal zone lymphoma | Withdrawn | 24 Apr 2015 |
| fluciclovine (18F) EU/3/15/1472 |
Diagnosis of glioma | Active (positive) | 24 Apr 2015 |
| trientine tetrahydrochloride EU/3/15/1471 |
Treatment of Wilson's disease | Withdrawn | 19 Mar 2015 |
| 5,10,15,20-tetrakis(2,6-difluoro-3-N-methylsulfamoylphenyl)bacteriochlorin (redaporfin) EU/3/15/1470 |
Treatment of biliary tract cancer | Active (positive) | 19 Mar 2015 |
| Human reovirus type 3 Dearing strain EU/3/15/1469 |
Treatment of ovarian cancer | Active (positive) | 19 Mar 2015 |
| adeno-associated viral vector serotype 9 containing the human glucocerebrosidase gene EU/3/15/1468 |
Treatment of Gaucher disease | Withdrawn | 19 Mar 2015 |
| ex-vivo-expanded autologous human keratinocytes containing epidermal stem cells transduced with a COL17A1-encoding retroviral vector EU/3/15/1467 |
Treatment of epidermolysis bullosa | Active (positive) | 19 Mar 2015 |
| ex-vivo-expanded autologous human keratinocytes containing epidermal stem cells transduced with a COL7A1-encoding retroviral vector EU/3/15/1466 |
Treatment of epidermolysis bullosa | Active (positive) | 19 Mar 2015 |
| ex-vivo-expanded autologous human keratinocytes containing epidermal stem cells transduced with a LAMB3-encoding retroviral vector EU/3/15/1465 |
Treatment of epidermolysis bullosa | Active (positive) | 19 Mar 2015 |
| autologous adipose tissue-derived stromal vascular fraction cells EU/3/15/1464 |
Treatment of systemic sclerosis | Active (positive) | 19 Mar 2015 |
| Melphalan flufenamide EU/3/15/1463 |
Treatment of plasma cell myeloma | Withdrawn | 19 Mar 2015 |
| sodium 3-[(4aR,6R,7R,7aS)-7-hydroxy-2-oxido-2-sulfanylidene-4a,6,7,7a-tetrahydro-4H-furo [3,2-d][1,3,2] dioxaphosphinin-6-yl]-2-bromo-6-phenyl-5H-imidazo[1,2-a… EU/3/15/1462 |
Treatment of retinitis pigmentosa | Active (positive) | 19 Mar 2015 |
| recombinant human monoclonal antibody binding to vascular adhesion protein-1 EU/3/15/1461 |
Treatment of primary sclerosing cholangitis | Active (positive) | 19 Mar 2015 |
| lenvatinib EU/3/15/1460 124 clinical trials → |
Treatment of hepatocellular carcinoma | Withdrawn | 19 Mar 2015 |
| Enoxacin EU/3/15/1459 |
Treatment of amyotrophic lateral sclerosis | Active (positive) | 19 Mar 2015 |
| Humanised anti-folate receptor 1 monoclonal antibody conjugated to maytansinoid DM4 EU/3/15/1458 · now authorised as Elahere |
Treatment of ovarian cancer | Active (positive) | 19 Mar 2015 |
| [5-(5-chloro-1H-pyrrolo[2,3-b]pyridin-3-ylmethyl)-pyridin-2-yl]-(6-trifluoromethyl-pyridin-3-ylmethyl)-amine hydrochloride EU/3/15/1457 · now authorised as Turalio |
Treatment of tenosynovial giant cell tumour, localised and diffuse type | Withdrawn | 19 Mar 2015 |
| recombinant human club cell 10 KDa protein EU/3/15/1456 |
Prevention of bronchopulmonary dysplasia | Active (positive) | 19 Mar 2015 |
| Human plasma-derived alpha-1 proteinase inhibitor EU/3/15/1455 |
Treatment of graft-versus-host disease | Active (positive) | 19 Mar 2015 |
| 6-ethoxy-7-methoxy-2-(2-methylsulfanylphenyl)-3,1-benzoxazin-4-one EU/3/15/1454 |
Treatment of Netherton syndrome | Active (positive) | 19 Mar 2015 |
| chimeric 2'-O-(2-methoxyethyl) modified oligonucleotide targeted to huntingtin RNA EU/3/15/1453 |
Treatment of Huntington’s disease | Active (positive) | 19 Mar 2015 |
| tideglusib EU/3/15/1452 3 clinical trials → |
Treatment of fragile X syndrome | Withdrawn | 19 Mar 2015 |
| 5'-ASCSASTSCSASGSTSCSTSGSASUSASASGSCSTSA-3' (lademirsen) EU/3/15/1451 |
Treatment of Alport syndrome | Withdrawn | 19 Mar 2015 |
| Gallium (68Ga)-edotreotide EU/3/15/1450 · now authorised as SomaKit TOC |
Diagnosis of gastro-entero-pancreatic neuroendocrine tumours | Active (positive) | 19 Mar 2015 |
| allogeneic CD4+ and CD8+ T lymphocytes ex vivo incubated with synthetic peptides of the viral antigens of cytomegalovirus, adenovirus and Epstein-Barr virus EU/3/15/1440 |
Treatment of Epstein-Barr virus infection following haematopoietic stem cell transplantation | Active (positive) | 19 Mar 2015 |
| allogeneic CD4+ and CD8+ T lymphocytes ex vivo incubated with synthetic peptides of the viral antigens of cytomegalovirus, adenovirus and Epstein-Barr virus EU/3/15/1438 |
Treatment of adenovirus infection following haematopoietic stem cell transplantation | Active (positive) | 19 Mar 2015 |
| nalbuphine hydrochloride - 5 clinical trials → |
Treatment of uraemic pruritus | Negative | 27 Feb 2015 |
| myriocin EU/3/15/1449 |
Treatment of retinitis pigmentosa | Active (positive) | 12 Feb 2015 |
| 505 amino acid protein, corresponding to amino acids 2-506 of the wild type human histidyl-tRNA synthetase EU/3/15/1448 |
treatment of facioscapulohumeral muscular dystrophy | Withdrawn | 12 Feb 2015 |
| olaratumab EU/3/15/1447 |
Treatment of soft tissue sarcoma | Withdrawn | 12 Feb 2015 |
| N-(3-(4-(3-(diisobutylamino)propyl)piperazin-1-yl)propyl)-1H-benzo[d]imidazol-2-amine disulfate salt EU/3/15/1446 |
Treatment of progressive supranuclear palsy | Active (positive) | 12 Feb 2015 |
| ulocuplumab EU/3/15/1445 |
Treatment of acute myeloid leukaemia | Withdrawn | 12 Feb 2015 |
| Mazindol EU/3/15/1444 |
Treatment of narcolepsy | Withdrawn | 12 Feb 2015 |
| recombinant human glutamate oxaloacetate transaminase 1 EU/3/15/1443 |
Treatment of glioma | Withdrawn | 12 Feb 2015 |
| fibrinogen-coated albumin spheres EU/3/15/1442 |
Treatment of Ebola virus disease | Active (positive) | 12 Feb 2015 |
| 5-hydroxymethyl-2-furfural EU/3/15/1441 |
Treatment of sickle cell disease | Withdrawn | 12 Feb 2015 |
| allogeneic CD4+ and CD8+ T lymphocytes ex vivo incubated with synthetic peptides of the viral antigens of cytomegalovirus, adenovirus and Epstein-Barr virus EU/3/15/1439 |
Treatment of cytomegalovirus infection following haematopoietic stem cell transplantation | Active (positive) | 12 Feb 2015 |
| alvocidib EU/3/15/1437 |
Treatment of acute myeloid leukaemia | Active (positive) | 12 Feb 2015 |
| Lactobacillus reuteri EU/3/15/1436 |
Prevention of necrotising enterocolitis | Active (positive) | 12 Feb 2015 |
| nitroglycerin EU/3/15/1435 6 clinical trials → |
Treatment of systemic sclerosis | Active (positive) | 12 Feb 2015 |
| chimeric group B adenovirus (11p/3) with deletions in the E3 and E4 regions (enadenotucirev) EU/3/15/1434 |
Treatment of ovarian cancer | Withdrawn | 12 Feb 2015 |
| sevuparin sodium EU/3/15/1433 |
Treatment of sickle cell disease | Active (positive) | 12 Feb 2015 |
| 2'-O-methyl phosphorothioate RNA oligonucleotide, 5'-m5CUGm5CUGm5CUGm5CUGm5CUGm5CUGm5CUG-3' EU/3/15/1432 |
Treatment of Huntington’s disease | Active (positive) | 12 Feb 2015 |
| 3-[2-(4-carbamimidoyl-phenylcarbamoyl)-5-methoxy-4-vinyl-phenyl]-6-(cyclopropylmethyl-carbamoyl)-pyridine-2-carboxylic acid (avoralstat) EU/3/15/1431 |
Treatment of hereditary angioedema | Withdrawn | 12 Feb 2015 |
| adeno-associated viral vector serotype 8 containing the human factor-VII gene EU/3/14/1430 |
Treatment of congenital factor VII deficiency | Active (positive) | 15 Jan 2015 |
| N-methyl-4-({4-[({3-methyl(methylsulfonyl)aminopyrazin-2-yl}methyl)amino]-5-(trifluoromethyl)pyrimidin-2-yl}amino)benzamide hydrochloride (defactinib) EU/3/14/1429 |
Treatment of ovarian cancer | Active (positive) | 15 Jan 2015 |
| sodium valproate fNor EU/3/14/1428 |
Treatment of Wolfram syndrome | Active (positive) | 15 Jan 2015 |
| recombinant human alkaline phosphatase EU/3/14/1427 |
Treatment of hypophosphatasia | Active (positive) | 15 Jan 2015 |
| allogeneic peripheral blood mononuclear cells induced to an early apoptotic state EU/3/14/1426 |
Prevention of graft-versus-host disease | Withdrawn | 15 Jan 2015 |
| ceftriaxone EU/3/14/1425 6 clinical trials → |
Treatment of spinocerebellar ataxia | Active (positive) | 15 Jan 2015 |
| Humanised Fc engineered monoclonal antibody against CD19 (tafasitamab) EU/3/14/1424 · now authorised as Minjuvi |
Treatment of diffuse large B-cell lymphoma | Active (positive) | 15 Jan 2015 |
| synthetic signal peptide of human mucin-1 (amino acids 1-21) EU/3/14/1423 |
Treatment of plasma cell myeloma | Active (positive) | 15 Jan 2015 |
| chimeric fusion protein of recombinant human alpha-N-acetylglucosaminidase and human insulin-like growth factor 2 (tralesinidase alfa) EU/3/14/1422 |
Treatment of mucopolysaccharidosis type IIIB | Active (positive) | 15 Jan 2015 |
| ponatinib hydrochloride EU/3/14/1421 11 clinical trials → |
Treatment of gastrointestinal stromal tumours | Withdrawn | 15 Jan 2015 |
| emtricitabine EU/3/14/1420 6 clinical trials → |
Treatment of Aicardi-Goutières syndrome | Active (positive) | 15 Jan 2015 |
| tenofovir disoproxil fumarate EU/3/14/1419 4 clinical trials → |
Treatment of Aicardi-Goutières syndrome | Active (positive) | 15 Jan 2015 |
| A lentiviral vector pseudotyped by the New-Jersey serotype of the vesicular stomatitis virus G protein encoding an antigen derived from the Tax, HBZ, p12I and … EU/3/14/1418 |
Treatment of adult T-cell leukaemia/lymphoma | Active (positive) | 15 Jan 2015 |
| A lentiviral vector pseudotyped by the Indiana serotype of the vesicular stomatitis virus G protein encoding an antigen derived from the Tax, HBZ, p12I and p30… EU/3/14/1417 |
Treatment of adult T-cell leukaemia/lymphoma | Active (positive) | 15 Jan 2015 |
| chimeric monoclonal antibody to O-acetyl-GD2 antigen EU/3/14/1416 |
Treatment of neuroblastoma | Active (positive) | 15 Jan 2015 |
| adenoviral vector serotype 5 containing the vascular endothelial growth factor D isoform (preprocessed short form) from a CMV promoter EU/3/14/1415 |
Treatment of placental insufficiency | Active (positive) | 15 Jan 2015 |
| sodium thiosulfate EU/3/14/1414 5 clinical trials → |
Treatment for calciphylaxis | Active (positive) | 15 Jan 2015 |
| allogeneic, umbilical cord blood-derived, ex vivo-expanded, haematopoietic CD133+ cells;allogeneic, umbilical cord blood-derived, non-expanded, haematopoietic … EU/3/14/1413 |
Treatment of acute myeloid leukaemia | Withdrawn | 15 Jan 2015 |
| Herpes simplex type 1 virus containing cellular B-myb gene as tumour-specific promoter EU/3/14/1412 |
Treatment of pancreatic cancer | Active (positive) | 15 Jan 2015 |
| pentosan polysulfate sodium EU/3/14/1411 3 clinical trials → |
Treatment of interstitial cystitis | Withdrawn | 15 Jan 2015 |
| recombinant human aspartylglucosaminidase EU/3/14/1410 |
Treatment of aspartylglucosaminuria | Withdrawn | 15 Jan 2015 |
| pegylated recombinant arginine deiminase (pegargiminase) EU/3/14/1409 |
Treatment of malignant mesothelioma | Active (positive) | 15 Jan 2015 |
| 5,5'-(4-(trifluromethyl)benzylazanediyl)bis(methylene)diquinolin-8-ol EU/3/14/1408 |
Treatment of glioma | Active (positive) | 16 Dec 2014 |
| allogeneic adipose-derived adult mesenchymal stem cells contained in a fibrin-based bioengineered dermis EU/3/14/1407 |
Treatment of epidermolysis bullosa | Active (positive) | 16 Dec 2014 |
| chenodeoxycholic acid EU/3/14/1406 · now authorised as Chenodeoxycholic acid Leadiant (previously Chenodeoxycholic acid sigma-tau) |
Treatment of inborn errors in primary bile acid synthesis | Active (positive) | 16 Dec 2014 |
| autologous collagen type II-specific regulatory T cells EU/3/14/1405 |
Treatment of non-infectious uveitis | Withdrawn | 16 Dec 2014 |
| 1-(2-isopropoxyethyl)-2-thioxo-1,2,3,5-tetrahydro-pyrrolo[3,2-d] pyrimidin-4-one (verdiperstat) EU/3/14/1404 |
Treatment of multiple system atrophy | Active (positive) | 16 Dec 2014 |
| Plerixafor EU/3/14/1403 |
Treatment of WHIM syndrome | Active (positive) | 16 Dec 2014 |
| benserazide hydrochloride EU/3/14/1402 |
Treatment of beta thalassaemia intermedia and major | Active (positive) | 16 Dec 2014 |
| riluzole EU/3/14/1401 7 clinical trials → |
Treatment of traumatic spinal cord injury | Active (positive) | 16 Dec 2014 |
| (1S,4R,5R,7S)-3,4-dibenzyl-2-oxo-6,8-dioxa-3-azabyciclo[3.2.1]octane-7-carboxylic acid-L-lysine EU/3/14/1400 |
Treatment of neurotrophic keratitis | Active (positive) | 16 Dec 2014 |
| edaravone EU/3/14/1399 2 clinical trials → |
Treatment of amyotrophic lateral sclerosis | Active (positive) | 16 Dec 2014 |
| genetically modified serotype 5/3 adenovirus coding for granulocyte macrophage colony-stimulating factor EU/3/14/1398 |
Treatment of malignant mesothelioma | Withdrawn | 16 Dec 2014 |
| amikacin sulfate EU/3/14/1397 5 clinical trials → |
Treatment of Pseudomonas aeruginosa lung infection in cystic fibrosis | Active (positive) | 16 Dec 2014 |
| adenovirus serotype 5 containing partial E1A deletion and an integrin-binding domain (tasadenoturev) EU/3/14/1396 |
Treatment of glioma | Active (positive) | 16 Dec 2014 |
| allogeneic ex vivo-generated natural killer cells from CD34+ umbilical cord blood progenitor cells EU/3/14/1395 |
Treatment of acute myeloid leukaemia | Active (positive) | 16 Dec 2014 |
| pegylated recombinant human hyaluronidase PH20(pegvorhyaluronidase alfa) EU/3/14/1394 |
Treatment of pancreatic cancer | Withdrawn | 16 Dec 2014 |
| autologous T cells transduced with retroviral vector encoding an anti-CD19 CD28/CD3 zeta chimeric antigen receptor (axicabtagene ciloleucel) EU/3/14/1393 · now authorised as Yescarta |
Treatment of diffuse large B-cell lymphoma | Active (positive) | 16 Dec 2014 |
| 5-bromo-N-(prop-2-yn-1-yl)-2-(1H-1,2,4-triazol-1-yl)pyrimidine-4,6-diamine EU/3/14/1392 |
Treatment of Huntington’s disease | Active (positive) | 16 Dec 2014 |
| 5-[8-methyl-9-(1-methylethyl)-2-(4-morpholinyl)-9H-purin-6-yl]-2-pyrimidinamine EU/3/14/1391 |
Treatment of malignant mesothelioma | Withdrawn | 16 Dec 2014 |
| bevacizumab EU/3/14/1390 · now authorised as Orblid 400 clinical trials → |
Treatment of hereditary haemorrhagic telangiectasia | Active (positive) | 16 Dec 2014 |
| Adeno-associated viral vector serotype rh.rh.10 carrying the human N-sulfoglucosamine sulfohydrolase cDNA EU/3/14/1389 |
Treatment of mucopolysaccharidosis, type IIIA (Sanfilippo A syndrome) | Active (positive) | 16 Dec 2014 |
| allogeneic bone marrow derived mesenchymal cells expanded ex vivo in synthetic media EU/3/14/1388 |
Prevention of graft-versus-host disease | Active (positive) | 16 Dec 2014 |
| Exisulind EU/3/14/1387 |
Treatment of familial cerebral cavernous malformations | Active (positive) | 16 Dec 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.