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 |
|---|---|---|---|
| Combretastatin A1 diphosphate EU/3/15/1587 |
Treatment of acute myeloid leukaemia | Active (positive) | 14 Dec 2015 |
| recombinant human nerve growth factor (cenegermin) EU/3/15/1586 · now authorised as Oxervate |
Treatment of neurotrophic keratitis | Active (positive) | 14 Dec 2015 |
| sirolimus EU/3/15/1585 32 clinical trials → |
Treatment of beta thalassaemia intermedia and major | Active (positive) | 14 Dec 2015 |
| variant of recombinant human fibroblast growth factor 19 EU/3/15/1584 |
Treatment of primary sclerosing cholangitis | Withdrawn | 14 Dec 2015 |
| 2-amino-2-[2-[2-chloro-4-[[3-(phenylmethoxy)phenyl]thio]phenyl]ethyl]-1,3-propanediol hydrochloride EU/3/15/1583 |
Prevention of graft-versus-host disease | Withdrawn | 14 Dec 2015 |
| Magrolimab EU/3/15/1582 2 clinical trials → |
Treatment of acute myeloid leukaemia | Withdrawn | 11 Nov 2015 |
| sodium phenylbutyrate EU/3/15/1581 |
Treatment of pyruvate dehydrogenase complex deficiency | Active (positive) | 11 Nov 2015 |
| N-[5-(3,5-difluorobenzyl)-1H-indazol-3-yl]-4-(4 methylpiperazin-1-yl)-2-(tetrahydro-2H-pyran-4-ylamino) benzamide (entrectinib) EU/3/15/1580 |
Treatment of neuroblastoma | Withdrawn | 11 Nov 2015 |
| autologous T cells transduced with retroviral vector encoding an anti-CD19 CD28/CD3-zeta chimeric antigen receptor (axicabtagene ciloleucel) EU/3/15/1579 · now authorised as Yescarta |
Treatment of follicular lymphoma | Active (positive) | 11 Nov 2015 |
| adenovirus associated viral vector serotype 8 containing the human CNGB3 gene EU/3/15/1578 |
Treatment of achromatopsia caused by mutations in the CNGB3 gene | Active (positive) | 11 Nov 2015 |
| adenovirus associated viral vector serotype 5 containing the human RPE65 gene EU/3/15/1577 |
Treatment of Leber's congenital amaurosis | Active (positive) | 11 Nov 2015 |
| xevinapant EU/3/15/1576 |
Treatment of ovarian cancer | Withdrawn | 11 Nov 2015 |
| humanised fusion protein consisting of extracellular domain of CD24 linked to IgG1 Fc domain EU/3/15/1575 |
Prevention of graft-versus-host disease | Withdrawn | 11 Nov 2015 |
| 4'-[(2-butyl-4-oxo-1,3-diazaspiro[4.4]non-1-en-3-yl)methyl]-N-(4,5-dimethyl-3-isoxazolyl)-2'-(ethoxymethyl)-[1,1'-biphenyl]-2-sulfonamide EU/3/15/1574 |
Treatment of focal segmental glomerulosclerosis | Active (positive) | 11 Nov 2015 |
| adeno-associated viral vector serotype 8 encoding the human ATP7B gene under the control of the human alpha-1 antitrypsin promoter EU/3/15/1573 |
Treatment of Wilson's disease | Active (positive) | 11 Nov 2015 |
| autologous T cells transduced with retroviral vector encoding an anti-CD19 CD28/CD3-zeta chimeric antigen receptor (axicabtagene ciloleucel) EU/3/15/1572 |
Treatment of chronic lymphocytic leukaemia / small lymphocytic lymphoma | Withdrawn | 11 Nov 2015 |
| autologous T cells transduced with retroviral vector encoding an anti-CD19 CD28/CD3-zeta chimeric antigen receptor (axicabtagene ciloleucel) EU/3/15/1571 |
Treatment of acute lymphoblastic leukaemia | Withdrawn | 11 Nov 2015 |
| azacitidine EU/3/15/1570 127 clinical trials → |
Treatment of nasopharyngeal carcinoma | Withdrawn | 11 Nov 2015 |
| pentetrazol EU/3/15/1569 |
Treatment of idiopathic hypersomnia | Withdrawn | 11 Nov 2015 |
| interferon alfa-n3 EU/3/15/1568 |
Treatment of Middle East respiratory syndrome | Active (positive) | 11 Nov 2015 |
| recombinant human interleukin-3 truncated diphtheria toxin fusion protein EU/3/15/1567 · now authorised as Elzonris |
Treatment of blastic plasmacytoid dendritic cell neoplasm | Active (positive) | 11 Nov 2015 |
| sialic acid (also known as aceneuramic acid) EU/3/12/972 |
Treatment of GNE myopathy | Withdrawn | 16 Oct 2015 |
| autologous human peripheral blood Vdelta1+ T lymphocytes activated in vitro by cytokine and monoclonal antibody treatment EU/3/15/1566 |
Treatment of chronic lymphocytic leukaemia / small lymphocytic lymphoma | Active (positive) | 9 Oct 2015 |
| 2-Chloro-N6-(3-iodobenzyl)adenosine-5'-N-methyluronamide (namodenoson) EU/3/15/1565 |
Treatment of hepatocellular carcinoma | Active (positive) | 9 Oct 2015 |
| Dronabinol;cannabidiol EU/3/15/1564 3 clinical trials → |
Treatment of glioma | Withdrawn | 9 Oct 2015 |
| recombinant human interleukin-3 truncated diphtheria toxin fusion protein EU/3/15/1563 |
Treatment of acute myeloid leukaemia | Active (positive) | 9 Oct 2015 |
| staphylococcus aureus protein A EU/3/15/1562 |
Treatment of immune thrombocytopenia | Active (positive) | 9 Oct 2015 |
| ataluren EU/3/15/1561 |
Treatment of aniridia | Withdrawn | 9 Oct 2015 |
| N-(2-((4Z,7Z,10Z,13Z,16Z,19Z)-docosa-4,7,10,13,16,19-hexaenamido)ethyl)-2-hydroxybenzamideย (edasalonexent) EU/3/15/1560 |
Treatment of Duchenne muscular dystrophy | Withdrawn | 9 Oct 2015 |
| 2-(2-chlorophenyl)-4-[3-(dimethylamino)phenyl]-5-methyl-1H-pyrazolo[4,3-C]pyridine-3,6(2H,5H)-dione EU/3/15/1559 |
Treatment of systemic sclerosis | Active (positive) | 9 Oct 2015 |
| three chimeric human/murine monoclonal antibodies against the Ebola (Zaire) surface glycoprotein (porgaviximab) EU/3/15/1558 |
Treatment for Ebola virus disease | Withdrawn | 9 Oct 2015 |
| sirolimus EU/3/15/1557 32 clinical trials → |
Treatment of tuberous sclerosis | Active (positive) | 9 Oct 2015 |
| recombinant adeno-associated viral vector expressing the human CNGA3 gene EU/3/15/1556 |
Treatment of achromatopsia caused by mutations in the CNGA3 gene | Active (positive) | 9 Oct 2015 |
| synthetic hepcidin EU/3/15/1555 |
Treatment of beta thalassaemia intermedia and major | Withdrawn | 9 Oct 2015 |
| nimodipine EU/3/15/1554 2 clinical trials → |
Treatment of aneurysmal subarachnoid haemorrhage | Active (positive) | 9 Oct 2015 |
| autologous T cells transduced with retroviral vector encoding an anti-CD19 CD28/CD3-zeta chimeric antigen receptor (axicabtagene ciloleucel) EU/3/15/1553 · now authorised as Yescarta |
Treatment of primary mediastinal large B-cell lymphoma | Active (positive) | 9 Oct 2015 |
| autologous T cells transduced with retroviral vector encoding an anti-CD19 CD28/CD3-zeta chimeric antigen receptor (axicabtagene ciloleucel) EU/3/15/1552 |
Treatment of mantle cell lymphoma | Withdrawn | 9 Oct 2015 |
| recombinant human IgG1 kappa light chain monoclonal antibody targeting plasma kallikreinย (lanadelumab) EU/3/15/1551 · now authorised as Takhzyro |
Treatment of hereditary angioedema | Active (positive) | 9 Oct 2015 |
| 3-pentylbenzeneacetic acid sodium salt EU/3/15/1550 |
Treatment of idiopathic pulmonary fibrosis | Withdrawn | 9 Oct 2015 |
| synthetic peptide L-cysteine, L-cysteinylglycyl-L-glutaminyl-L-arginyl-L-.alpha.-glutamyl-L-threonyl-L-prolyl-L-.alpha.-glutamylglycyl-L-alanyl-L-.alpha.-glutaโฆ EU/3/15/1549 |
Treatment of primary graft dysfunction following lung transplantation | Active (positive) | 9 Oct 2015 |
| ovine specific immunoglobulin (Fab) fragments raised against Vipera berus veno EU/3/15/1548 |
Treatment of snakebite envenomation | Active (positive) | 9 Oct 2015 |
| Mazindol EU/3/15/1547 |
Treatment of narcolepsy | Active (positive) | 9 Oct 2015 |
| allogeneic umbilical cord blood cells treated ex vivo with 16,16-dimethyl prostaglandin E2 EU/3/15/1546 |
Treatment of acute lymphoblastic leukaemia | Withdrawn | 10 Aug 2015 |
| CD33-directed antibody-drug conjugate consisting of an antibody conjugated to a DNA cross-linking pyrrolobenzodiazepine dimer drug (vadastuximab talirine) EU/3/15/1545 |
Treatment of acute myeloid leukaemia | Withdrawn | 10 Aug 2015 |
| 2-(2-phenylvinyl)-4-[4- methylpiperazin-1-yl)]-6-(5-methyl-2H-pyrazol-3-yl-amino)-pyrimidine L(+) tartrate salt EU/3/15/1544 |
Treatment of hepatocellular carcinoma | Active (positive) | 10 Aug 2015 |
| (S)-6-hydroxy-2,5,7,8-tetramethyl-N-((R)-piperidin-3-yl)chroman-2-carboxamide hydrochloride EU/3/15/1543 |
Treatment of mitochondrial encephalomyopathy, lactic acidosis and stroke-like episodes | Withdrawn | 10 Aug 2015 |
| 2'-deoxyguanosylyl-(3',5'-phosphoryl)-2'-deoxythymidylyl-(3',5'-phosphoryl)- 2'-deoxyguanosylyl-(3',5'-phosphoryl)-2'-deoxycytidylyl-(3',5'-phosphoryl)-2'-deoxโฆ EU/3/15/1542 |
Treatment of diffuse large B-cell lymphoma | Withdrawn | 10 Aug 2015 |
| Ibrutinib EU/3/15/1541 44 clinical trials → |
Treatment of marginal zone lymphoma | Withdrawn | 10 Aug 2015 |
| adeno-associated viral vector serotype 9 containing the human iduronate-2-sulfatase gene EU/3/15/1540 |
Treatment of mucopolysaccharidosis type II (Hunter's syndrome) | Withdrawn | 10 Aug 2015 |
| adeno-associated viral vector serotype 8 containing the human MTM1 gene (resamirigene bilparvovec) EU/3/15/1539 |
Treatment of X-linked myotubular myopathy | Active (positive) | 10 Aug 2015 |
| fosfomycin disodium;tobramycin EU/3/15/1538 3 clinical trials → |
Treatment of cystic fibrosis | Active (positive) | 10 Aug 2015 |
| verucerfont EU/3/15/1537 |
Treatment of congenital adrenal hyperplasia | Withdrawn | 10 Aug 2015 |
| recombinant human acid ceramidase EU/3/15/1536 |
Treatment of cystic fibrosis | Active (positive) | 10 Aug 2015 |
| fibrinogen-coated albumin spheres EU/3/15/1535 |
Treatment of acute radiation syndrome | Active (positive) | 10 Aug 2015 |
| Glycyl-L-2-methylprolyl-L-glutamic acid;trofinetide EU/3/15/1534 · now authorised as Daybu |
Treatment of Rett syndrome | Active (positive) | 10 Aug 2015 |
| human allogeneic bone-marrow-derived osteoblastic cells EU/3/15/1533 |
Treatment of osteogenesis imperfecta | Active (positive) | 10 Aug 2015 |
| insulin human EU/3/15/1532 |
Treatment of short bowel syndrome | Active (positive) | 10 Aug 2015 |
| Lanreotide acetate EU/3/15/1514 5 clinical trials → |
Treatment of autosomal dominant polycystic kidney disease | Active (positive) | 10 Aug 2015 |
| sarizotan hydrochloride EU/3/15/1531 |
Treatment of Rett syndrome | Active (positive) | 28 Jul 2015 |
| Glycyl-L-2-methylprolyl-L-glutamic acid EU/3/15/1529 |
Treatment of fragile X syndrome | Active (positive) | 28 Jul 2015 |
| synthetic double-stranded RNA oligonucleotide specific to hydroxyacid oxidase 1 gene EU/3/15/1528 |
Treatment of primary hyperoxaluria type 1 | Withdrawn | 28 Jul 2015 |
| Hydrocinnamate-[Orn-Pro-dCha-Trp-Arg]acetate EU/3/15/1527 |
Treatment of amyotrophic lateral sclerosis | Active (positive) | 28 Jul 2015 |
| triheptanoin EU/3/15/1526 |
Treatment of carnitine palmitoyltransferase II deficiency | Active (positive) | 28 Jul 2015 |
| triheptanoin EU/3/15/1525 |
Treatment of mitochondrial trifunctional protein deficiency | Active (positive) | 28 Jul 2015 |
| triheptanoin EU/3/15/1524 |
Treatment of long-chain 3-hydroxyacyl-coenzyme A dehydrogenase deficiency | Active (positive) | 28 Jul 2015 |
| Inecalcitol EU/3/15/1523 |
Treatment of acute myeloid leukaemia | Active (positive) | 28 Jul 2015 |
| Humanised IgG4 monoclonal antibody against extracellular tau EU/3/15/1522 |
Treatment of progressive supranuclear palsy | Active (positive) | 28 Jul 2015 |
| artesunate EU/3/15/1521 · now authorised as Garsun 5 clinical trials → |
Treatment of malaria | Active (positive) | 28 Jul 2015 |
| cannabidiol EU/3/15/1520 16 clinical trials → |
Treatment of perinatal asphyxia | Active (positive) | 28 Jul 2015 |
| allogeneic human adult stem cells, isolated from skeletal muscle and expanded ex vivo EU/3/15/1519 |
Treatment of Duchenne muscular dystrophy | Active (positive) | 28 Jul 2015 |
| adenovirus-associated viral vector serotype 2 containing the human RPE65 gene (voretigene neparvovec) EU/3/15/1518 · now authorised as Luxturna |
Treatment of retinitis pigmentosa | Active (positive) | 28 Jul 2015 |
| 2-((3-((4-((3-aminopropyl)amino)butyl)amino)propyl)amino)-N-((5S,5aS,8aR,9R)-9-(4-hydroxy-3,5-dimethoxyphenyl)-8-oxo-5,5a,6,8,8a,9-hexahydrofuro[3',4':6,7]naphโฆ EU/3/15/1517 |
Treatment of acute myeloid leukaemia | Active (positive) | 28 Jul 2015 |
| modified adenovirus serotype 5/35 containing a CMV promoter-driven transgene cassette with the human transgenes for a membrane-bound CD40 ligand and full lengtโฆ EU/3/15/1516 |
Treatment of pancreatic cancer | Active (positive) | 28 Jul 2015 |
| synthetic hypericin EU/3/15/1515 |
Treatment of cutaneous T-cell lymphoma | Active (positive) | 28 Jul 2015 |
| Doxorubicin EU/3/15/1513 126 clinical trials → |
Treatment of hepatoblastoma | Active (positive) | 28 Jul 2015 |
| anti-H5N1 equine immunoglobulin F(ab')2 fragments EU/3/15/1512 |
Treatment of avian influenza | Active (positive) | 28 Jul 2015 |
| Human plasminogen EU/3/15/1511 |
Treatment of plasminogen deficiency | Active (positive) | 28 Jul 2015 |
| edaravone EU/3/15/1510 · now authorised as Radicava 2 clinical trials → |
Treatment of amyotrophic lateral sclerosis | Active (positive) | 19 Jun 2015 |
| adeno-associated viral vector serotype 9 containing the human SMN gene (onasemnogene abeparvovec) EU/3/15/1509 · now authorised as Zolgensma; Itvisma |
Treatment of spinal muscular atrophy | Active (positive) | 19 Jun 2015 |
| triheptanoin EU/3/15/1508 |
Treatment of very long-chain acyl-CoA dehydrogenase deficiency | Active (positive) | 19 Jun 2015 |
| 3-{[2,3,5,6-tetrafluoro-3'-(trifluoromethoxy)biphenyl-4-yl]carbamoyl}thiophene-2-carboxylic acid EU/3/15/1507 |
Treatment of non-infectious uveitis | Active (positive) | 19 Jun 2015 |
| antisense oligonucleotide directed against TGF-ฮฒ2 mRNA EU/3/15/1506 |
Prevention of scarring post glaucoma filtration surgery | Active (positive) | 19 Jun 2015 |
| obinutuzumab EU/3/15/1505 88 clinical trials → |
Treatment of marginal zone lymphoma | Withdrawn | 19 Jun 2015 |
| obinutuzumab EU/3/15/1504 88 clinical trials → |
Treatment of follicular lymphoma | Withdrawn | 19 Jun 2015 |
| allogeneic ex-vivo-expanded human umbilical cord blood-derived mesenchymal stem cells EU/3/15/1503 |
Prevention of bronchopulmonary dysplasia | Active (positive) | 19 Jun 2015 |
| trehalose EU/3/15/1502 |
Treatment of spinocerebellar ataxia | Active (positive) | 19 Jun 2015 |
| adeno-associated viral vector containing the human factor IX gene EU/3/15/1501 |
Treatment of haemophilia B | Withdrawn | 19 Jun 2015 |
EU/3/15/1500 · now authorised as Hepcludex |
Treatment of hepatitis delta virus infection | Active (positive) | 19 Jun 2015 |
| adult human bone-marrow-derived, ex-vivo-expanded, pooled allogeneic mesenchymal stromal cells EU/3/15/1499 |
Treatment of thromboangiitis obliterans (Buerger's disease) | Active (positive) | 21 May 2015 |
| 2-(7-ethoxy-4-(3-fluorophenyl)-1-oxophthalazin-2(1H)-yl)-N-methyl-N-(2-methylbenzo[d]oxazol-6-yl) acetamide EU/3/15/1498 |
Treatment of cystic fibrosis | Withdrawn | 21 May 2015 |
| 5,7-dichloro-2-dimethylaminomethyl-8-hydroxyquinoline hydrochloride EU/3/15/1497 |
Treatment of Huntingtonโs disease | Withdrawn | 21 May 2015 |
| trehalose EU/3/15/1496 |
Treatment of oculopharyngeal muscular dystrophy | Active (positive) | 21 May 2015 |
| triheptanoin EU/3/15/1495 |
Treatment of glucose transporter type-1 deficiency syndrome | Withdrawn | 21 May 2015 |
| allopurinol sodium EU/3/15/1493 7 clinical trials → |
Treatment of perinatal asphyxia | Active (positive) | 21 May 2015 |
| adeno-associated viral vector serotype 9 containing the human HGSNAT gene EU/3/15/1491 |
Treatment of mucopolysaccharidosis IIIC (Sanfilippo C syndrome) | Active (positive) | 21 May 2015 |
| triamcinolone acetonide EU/3/15/1490 12 clinical trials → |
Treatment of non-infectious uveitis | Active (positive) | 21 May 2015 |
| Fusion proteins composed by a genetically modified cholera toxin subunit A1, peptides from the acetylcholine receptor alpha chain and a dimer of the D fragmentโฆ EU/3/15/1489 |
Treatment of myasthenia gravis | Withdrawn | 21 May 2015 |
| {2-amino-8-[4-(pyrrolidinylcarbonyl)phenyl]-(3H-benzo[f]azepin-4-yl)}-N,N-dipropylcarboxamide EU/3/15/1488 |
Treatment of ovarian cancer | Withdrawn | 21 May 2015 |
| reduced oxidised N-acetyl heparin (roneparstat) EU/3/15/1487 |
Treatment of plasma cell myeloma | Withdrawn | 21 May 2015 |
| AASSGVSTPGSAGHDIITEQPRS EU/3/15/1492 |
Treatment of Huntingtonโs disease | Active (positive) | 12 May 2015 |
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.