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 porcine factor VIII (B-domain-deleted) EU/3/10/784 |
Treatment of haemophilia A | Withdrawn | 20 Sep 2010 |
| recombinant fusion protein consisting of human coagulation factor VIII attached to the Fc domain of human IgG1 (efmoroctocog alfa) EU/3/10/783 |
Treatment of haemophilia A | Withdrawn | 20 Sep 2010 |
| nafamostat mesilate EU/3/10/782 2 clinical trials → |
Treatment of cystic fibrosis | Active (positive) | 20 Sep 2010 |
| glutathione-pegylated liposomal doxorubicin hydrochloride EU/3/10/781 |
Treatment of glioma | Withdrawn | 20 Sep 2010 |
| Forodesine EU/3/10/780 |
Treatment of chronic lymphocytic leukaemia | Active (positive) | 20 Sep 2010 |
| eflornithine EU/3/10/779 6 clinical trials → |
Treatment of familial adenomatous polyposis (FAP) | Active (positive) | 20 Sep 2010 |
| cysteamine bitartrate (gastroresistant) (mercaptamine) EU/3/10/778 |
Treatment of cystinosis | Active (positive) | 20 Sep 2010 |
| cyclic pyranopterin monophosphate (fosdenopterin) EU/3/10/777 · now authorised as Nulibry |
Treatment of molybdenum-cofactor deficiency type A | Active (positive) | 20 Sep 2010 |
| autologous dendritic cells pulsed with recombinant human-fusion protein (mucin 1 - glutathione S transferase) coupled to oxidised polymannose EU/3/10/776 |
Treatment of ovarian cancer | Active (positive) | 20 Sep 2010 |
| autologous bone-marrow-derived mononuclear-cell fraction EU/3/10/775 |
Treatment of thromboangiitis obliterans (Buerger's disease) | Active (positive) | 20 Sep 2010 |
| allogeneic human dermal fibroblasts EU/3/10/774 |
Treatment of epidermolysis bullosa | Active (positive) | 20 Sep 2010 |
| allogeneic T cells encoding an exogenous TK gene EU/3/10/773 |
Treatment of acute myeloid leukaemia | Active (positive) | 20 Sep 2010 |
| adenovirus-associated viral vector serotype 10 carrying the human N-sulfoglucosamine sulfohydrolase and sulfatase modifying factor 1 cDNAs EU/3/10/772 |
Treatment of mucopolysaccharidosis, type IIIA (Sanfilippo A syndrome) | Active (positive) | 20 Sep 2010 |
| abarelix EU/3/10/771 |
Treatment of low-flow priapism | Withdrawn | 20 Sep 2010 |
| 1-[2-(Benzo[1,2,5]thiadiazol-5-ylamino)-6-(2,6-dichloro-phenyl)-pyrido[2,3-d]pyrimidin-7-yl]-3-tert-butyl-urea EU/3/10/770 |
Treatment of acute myeloid leukaemia | Withdrawn | 20 Sep 2010 |
| Lentiviral vector expressing the truncated form of human tyrosine hydroxylase gene, human aromatic L amino-acid decarboxylase gene, human GTP-cyclohydrolase 1 … - |
Treatment of off-periods in adult patients with advanced Parkinson's disease | Negative | 9 Sep 2010 |
| 11-(2-pyrrolidin-1-yl-ethoxy)-14,19-dioxa-5,7,26-triaza-tetracyclo[19.3.1.1(2,6).1(8,12)] heptacosa-1(25),2(26),3,5,8,10,12(27),16,21,23-decaene EU/3/10/769 · now authorised as Enpaxiq; Epjevy |
Treatment of post-polycythaemia vera myelofibrosis | Active (positive) | 25 Aug 2010 |
| 11-(2-pyrrolidin-1-yl-ethoxy)-14,19-dioxa-5,7,26-triaza-tetracyclo[19.3.1.1(2,6).1(8,12)] heptacosa-1(25),2(26),3,5,8,10,12(27),16,21,23-decaene EU/3/10/768 · now authorised as Enpaxiq; Epjevy |
Treatment of primary myelofibrosis | Active (positive) | 25 Aug 2010 |
| 11-(2-pyrrolidin-1-yl-ethoxy)-14,19-dioxa-5,7,26-triaza-tetracyclo[19.3.1.1(2,6).1(8,12)] heptacosa-1(25),2(26),3,5,8,10,12(27),16,21,23-decaene EU/3/10/767 · now authorised as Enpaxiq; Epjevy |
Treatment of post-essential thrombocythaemia myelofibrosis | Active (positive) | 25 Aug 2010 |
| Pyr-His-Trp-Ser-Tyr-D-Lys(doxorubicinylglutarate)-Leu-Arg-Pro-Gly-NH2, acetate salt EU/3/10/766 |
Treatment of ovarian cancer | Withdrawn | 4 Aug 2010 |
| Midostaurin EU/3/10/765 · now authorised as Rydapt 5 clinical trials → |
Treatment of mastocytosis | Active (positive) | 4 Aug 2010 |
| everolimus EU/3/10/764 53 clinical trials → |
Treatment of tuberous sclerosis | Expired | 4 Aug 2010 |
| dexamethasone EU/3/10/763 325 clinical trials → |
Treatment of non-infectious uveitis affecting the posterior segment of the eye | Withdrawn | 4 Aug 2010 |
| Bosutinib EU/3/10/762 6 clinical trials → |
Treatment of chronic myeloid leukaemia | Withdrawn | 4 Aug 2010 |
| 3-(6-(1-(2,2-Difluorobenzo [d] [1,3] dioxol-5-yl)cyclopropanecarboxamido)-3-methylpyridin-2-yl)benzoic acid (lumacaftor) EU/3/10/761 |
Treatment of cystic fibrosis | Withdrawn | 4 Aug 2010 |
| (3S)-3-{4-[7-(aminocarbonyl)-2H-indazol-2-yl] phenyl} piperidine tosilate monohydrate salt (niraparib) EU/3/10/760 · now authorised as Zejula |
Treatment of ovarian cancer | Active (positive) | 4 Aug 2010 |
| Pomalidomide EU/3/10/759 64 clinical trials → |
Treatment of post-essential thrombocythaemia myelofibrosis | Withdrawn | 27 Jul 2010 |
| Pomalidomide EU/3/10/758 64 clinical trials → |
Treatment of post-polycythaemia vera myelofibrosis | Withdrawn | 27 Jul 2010 |
| Pomalidomide EU/3/10/757 64 clinical trials → |
Treatment of primary myelofibrosis | Withdrawn | 27 Jul 2010 |
| tranilast EU/3/10/756 2 clinical trials → |
Prevention of scarring post glaucoma filtration surgery | Active (positive) | 27 Jul 2010 |
| octenidine dihydrochloride EU/3/10/755 |
Prevention of late-onset sepsis in premature infants of ≤ to 32 weeks of gestational age | Active (positive) | 27 Jul 2010 |
| heparin-activated recombinant human fibroblast growth factor 1 (on a biodegradable device made from alpha-calcium sulfate hemihydrate) EU/3/10/754 |
Treatment of traumatic spinal cord injury | Withdrawn | 27 Jul 2010 |
| 6alpha-ethyl-chenodeoxycholic acid EU/3/10/753 |
Treatment of primary biliary cirrhosis | Withdrawn | 27 Jul 2010 |
| molgramostim - 3 clinical trials → |
Treatment of cystic fibrosis | Negative | 16 Jul 2010 |
| Gastrin 17C diphtheria toxoid conjugate - |
Treatment of pancreatic cancer | Negative | 16 Jul 2010 |
| 2-Methoxymethyl-2-hydroxymethyl-1-azabicyclo[2,2,2]octan-3-one EU/3/10/742 |
Treatment of acute myeloid leukaemia | Active (positive) | 10 Jun 2010 |
| pralatrexate EU/3/10/741 3 clinical trials → |
Treatment of cutaneous T-cell lymphoma | Active (positive) | 10 Jun 2010 |
| perifosine EU/3/10/740 |
Treatment of multiple myeloma | Withdrawn | 10 Jun 2010 |
| Glyceryl tri-(4-phenylbutyrate) EU/3/10/739 |
Treatment of citrullinaemia type 2 | Active (positive) | 10 Jun 2010 |
| Glyceryl tri-(4-phenylbutyrate) EU/3/10/738 · now authorised as Ravicti |
Ornithine translocase deficiency (hyperornithinaemia-hyperammonaemia homocitrullinuria (HHH) syndrom | Active (positive) | 10 Jun 2010 |
| Glyceryl tri-(4-phenylbutyrate) EU/3/10/737 · now authorised as Ravicti |
Treatment of hyperargininaemia | Active (positive) | 10 Jun 2010 |
| Glyceryl tri-(4-phenylbutyrate) EU/3/10/736 · now authorised as Ravicti |
Treatment of argininosuccinic aciduria | Active (positive) | 10 Jun 2010 |
| Glyceryl tri-(4-phenylbutyrate) EU/3/10/735 · now authorised as Ravicti |
Treatment of citrullinaemia type 1 | Active (positive) | 10 Jun 2010 |
| Glyceryl tri-(4-phenylbutyrate) EU/3/10/734 · now authorised as Ravicti |
Treatment of ornithine carbamoyltransferase deficiency | Active (positive) | 10 Jun 2010 |
| Entinostat EU/3/10/732 6 clinical trials → |
Treatment of Hodgkin's lymphoma | Active (positive) | 10 Jun 2010 |
| bafetinib EU/3/10/731 |
Treatment of chronic myeloid leukaemia | Withdrawn | 10 Jun 2010 |
| raloxifene hydrochloride EU/3/10/730 |
Treatment of hereditary haemorrhagic telangiectasia | Active (positive) | 10 Jun 2010 |
| rifapentine EU/3/10/750 3 clinical trials → |
Treatment of tuberculosis | Active (positive) | 9 Jun 2010 |
| recombinant human anti-interferon gamma monoclonal antibody (emapalumab) EU/3/10/749 · now authorised as Gamifant |
Treatment of haemophagocytic lymphohistiocytosis | Active (positive) | 9 Jun 2010 |
| pravastatin;zoledronic acid EU/3/10/748 4 clinical trials → |
Treatment of Hutchinson-Gilford progeria | Active (positive) | 9 Jun 2010 |
| N-{2-Chloro-4-[(6,7-dimethoxy-4-quinolyl)oxy]phenyl}-N'-(5-methyl-3-isoxazolyl) urea hydrochloride monohydrate EU/3/10/747 |
Treatment of renal-cell carcinoma | Withdrawn | 9 Jun 2010 |
| Maytansinoid-conjugated humanised monoclonal antibody against CD56 EU/3/10/746 |
Treatment of Merkel cell carcinoma | Withdrawn | 9 Jun 2010 |
| dexamethasone EU/3/10/745 325 clinical trials → |
Treatment of multiple myeloma | Withdrawn | 9 Jun 2010 |
| adrenomedullin EU/3/10/744 2 clinical trials → |
Treatment of acute lung Injury | Active (positive) | 9 Jun 2010 |
| Pagibaximab EU/3/10/743 |
Prevention of sepsis caused by Gram-positive pathogens in premature infants less than or equal to 34 | Withdrawn | 9 Jun 2010 |
| velaglucerase alfa EU/3/10/752 |
Treatment of Gaucher disease | Expired | 6 Jun 2010 |
| synthetic double-stranded siRNA oligonucleotide directed against p53 mRNA (teprasiran) EU/3/10/751 |
Prevention of delayed graft function after renal transplantation | Withdrawn | 6 Jun 2010 |
EU/3/10/729 |
Treatment of acute myeloid leukaemia | Withdrawn | 23 Mar 2010 |
| Davunetide EU/3/10/728 |
Treatment of progressive supranuclear palsy | Withdrawn | 23 Mar 2010 |
| Lentiviral vector containing the human MYO7A gene EU/3/10/727 |
Treatment of retinitis pigmentosa in Usher syndrome 1B | Active (positive) | 23 Mar 2010 |
| taliglucerase alfa EU/3/10/726 · now authorised as Elelyso |
Treatment of Gaucher disease | Active (positive) | 23 Mar 2010 |
| recombinant fusion protein linking human coagulation factor IX with human albumin EU/3/09/723 · now authorised as Idelvion |
Treatment of haemophilia B | Active (positive) | 4 Feb 2010 |
| Glyceryl tri-(4-phenylbutyrate) EU/3/10/733 · now authorised as Ravicti |
Treatment of carbamoyl-phosphate synthase-1 deficiency | Active (positive) | 3 Feb 2010 |
| givinostat EU/3/09/719 |
Treatment of polycythaemia vera | Active (positive) | 3 Feb 2010 |
| Ecopipam EU/3/09/717 |
Treatment of Lesch-Nyhan disease | Active (positive) | 3 Feb 2010 |
| RNA, [P-deoxy-P-(dimethylamino)] (2',3'-dideoxy-2',3'-imino-2',3'-seco) (2'a→5') (C-m5U-m5U-A-C-A-G-G-C-m5U-C-C-A-A-m5U-A-G-m5U-G-G-m5U-C-A-G-m5U), 5' [P-[4-[[… EU/3/09/725 |
Treatment of Duchenne muscular dystrophy | Withdrawn | 2 Feb 2010 |
| recombinant human monoclonal antibody to human interleukin (IL)-17A of the IgG1/k class EU/3/09/724 |
Treatment of chronic non-infectious uveitis | Withdrawn | 2 Feb 2010 |
| pixantrone dimaleate EU/3/09/722 |
Treatment of diffuse large B-cell lymphoma | Withdrawn | 2 Feb 2010 |
| panobinostat EU/3/09/721 |
Treatment of Hodgkin's lymphoma | Withdrawn | 2 Feb 2010 |
| Lentiviral vector containing the human ABCA4 gene EU/3/09/720 |
Treatment of Stargardt's disease | Active (positive) | 2 Feb 2010 |
| Fingolimod EU/3/09/718 8 clinical trials → |
Treatment of chronic inflammatory demyelinating polyneuropathy | Withdrawn | 2 Feb 2010 |
| Benzamide, 3-(2-imidazo[1,2-b]pyridazin-3-ylethynyl)-4-methyl-N-[4-[(4-methyl-1-piperazinyl)methyl]-3-(trifluoromethyl)phenyl] (ponatinib) EU/3/09/716 |
Treatment of chronic myeloid leukaemia | Expired | 2 Feb 2010 |
| Benzamide, 3-(2-imidazo[1,2-b]pyridazin-3-ylethynyl)-4-methyl-N-[4-[(4-methyl-1-piperazinyl)methyl]-3-(trifluoromethyl)phenyl] (ponatinib) EU/3/09/715 |
Treatment of acute lymphoblastic leukaemia | Expired | 2 Feb 2010 |
| streptococcus pyogenes Su strain cells treated with benzylpenicillin EU/3/09/714 |
Treatment of congenital lymphatic malformations | Withdrawn | 29 Jan 2010 |
| veltuzumab EU/3/09/713 |
Treatment of chronic lymphocytic leukaemia | Withdrawn | 29 Jan 2010 |
| recombinant kallikrein inhibitor EU/3/09/712 |
Treatment of Netherton syndrome | Active (positive) | 29 Jan 2010 |
| recombinant human vascular endothelial growth factor EU/3/09/711 |
Treatment of amyotrophic lateral sclerosis | Active (positive) | 29 Jan 2010 |
| recombinant human elafin EU/3/09/710 |
Treatment of oesophagus carcinoma | Active (positive) | 28 Jan 2010 |
| recombinant fusion protein consisting of the extracellular portion of CD95 fused to the Fc part of a human IgG1 molecule EU/3/09/709 |
Treatment of glioma | Withdrawn | 28 Jan 2010 |
| pegylated recombinant phenylalanine ammonia lyase (pegvaliase) EU/3/09/708 · now authorised as Palynziq |
Treatment of hyperphenylalaninaemia | Active (positive) | 28 Jan 2010 |
| macitentan EU/3/09/707 4 clinical trials → |
Treatment of idiopathic pulmonary fibrosis | Withdrawn | 28 Jan 2010 |
| Lithium citrate tetrahydrate EU/3/09/706 |
Treatment of Huntington’s disease | Active (positive) | 28 Jan 2010 |
| human monoclonal antibody against Pseudomonas aeruginosa IATS-O1 EU/3/09/705 |
Treatment of pneumonia caused by serotype O1 Pseudomonas aeruginosa | Withdrawn | 28 Jan 2010 |
| givinostat EU/3/09/704 |
Treatment of systemic-onset juvenile idiopathic arthritis | Withdrawn | 28 Jan 2010 |
| brivudine EU/3/09/703 |
Treatment of pancreatic cancer | Active (positive) | 28 Jan 2010 |
| beta-artemether;lumefantrine EU/3/09/702 |
Treatment of malaria | Active (positive) | 28 Jan 2010 |
| 2-iminobiotin EU/3/09/701 |
Treatment of perinatal asphyxia | Active (positive) | 28 Jan 2010 |
| human MHC non-restricted cytotoxic T-cell line EU/3/09/696 |
Treatment of ovarian cancer | Withdrawn | 30 Nov 2009 |
| 8-[4-(1-aminocyclobutyl)phenyl]-9-phenyl-1,2,4-triazolo[3,4-f][1,6]naphthyridin-3(2H)-one mono-hydrochloride EU/3/09/695 |
Treatment of ovarian cancer | Withdrawn | 30 Nov 2009 |
| Vaccinia GM-CSF/TK-deactivated virus EU/3/09/700 |
Treatment of hepatocellular carcinoma (pexastimogene devacirepvec) | Active (positive) | 26 Nov 2009 |
| recombinant chimeric monoclonal antibody against CD20 (ublituximab) EU/3/09/699 |
Treatment of chronic lymphocytic leukaemia | Active (positive) | 26 Nov 2009 |
| pegylated carboxyhaemoglobin EU/3/09/698 |
Treatment of sickle cell disease | Active (positive) | 26 Nov 2009 |
| 6-Thioguanine EU/3/09/694 |
Treatment of acute lymphoblastic leukaemia | Active (positive) | 26 Nov 2009 |
| 1-Cyclopropyl-3-[3-(5-morpholin-4-ylmethyl-1H-benzoimidazol-2-yl)-1H-pyrazol-4-yl]-urea EU/3/09/693 |
Treatment of acute myeloid leukaemia | Withdrawn | 26 Nov 2009 |
| N-[6-(cis-2,6-dimethylmorpholin-4-yl)pyridine-3-yl]-2-methyl-4'-(trifluoromethoxy)[1,1'-biphenyl]-3-carboxamide EU/3/09/697 |
Treatment of naevoid basal-cell carcinoma syndrome (Gorlin syndrome) | Withdrawn | 25 Nov 2009 |
| 16-base single-stranded PNA oligonucleotide linked to a 7-aminoacid peptide EU/3/09/692 |
Treatment of neuroblastoma | Active (positive) | 25 Nov 2009 |
| human anthrax immunoglobulin EU/3/09/691 |
Treatment of inhalation anthrax disease | Withdrawn | 18 Nov 2009 |
| human anthrax immunoglobulin EU/3/09/690 |
Post-exposure prophylaxis of inhalation anthrax disease | Withdrawn | 9 Nov 2009 |
| Peptides mimicking antigen receptors on autoimmune B cells and autoimmune T cells associated with myasthenia gravis EU/3/09/689 |
Treatment of myasthenia gravis | Active (positive) | 9 Nov 2009 |
| NGR-human tumour necrosis factor EU/3/09/686 |
Treatment of hepatocellular carcinoma | Active (positive) | 9 Nov 2009 |
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.