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 |
|---|---|---|---|
| patupilone EU/3/09/688 |
Treatment of fallopian tube cancer | Withdrawn | 5 Nov 2009 |
| patupilone EU/3/09/687 |
Treatment of primary peritoneal cancer | Withdrawn | 5 Nov 2009 |
| cholic acid EU/3/09/683 |
Treatment of inborn errors in primary bile acid synthesis responsive to treatment with cholic acid | Withdrawn | 29 Oct 2009 |
| Masitinib mesilate EU/3/09/684 · now authorised as Masican; Masiviera 2 clinical trials → |
Treatment of pancreatic cancer | Active (positive) | 28 Oct 2009 |
| anti-EphA2 monoclonal antibody conjugated to maleimidocaproyl monomethylauristatin phenylalanine EU/3/09/682 |
Treatment of ovarian cancer | Withdrawn | 28 Oct 2009 |
| 5'-O-(trans-9''-octadecenoyl)-1-beta-D-2'-deoxy-2',2'-difluorocytidine EU/3/09/680 |
Treatment of pancreatic cancer | Withdrawn | 28 Oct 2009 |
| 4-Benzyl-2-naphtalen-1-yl-1,2,4-thiadiazolidine-3,5-dione EU/3/09/679 |
Treatment of progressive supranuclear palsy | Withdrawn | 28 Oct 2009 |
| sequence-modified recombinant human factor VIIa EU/3/09/675 |
Treatment of haemophilia A | Withdrawn | 9 Oct 2009 |
| recombinant human serum amyloid P EU/3/09/674 |
Prevention of scarring post glaucoma filtration surgery | Withdrawn | 9 Oct 2009 |
| recombinant antibody construct against human CD30 and CD16A EU/3/09/673 |
Treatment of Hodgkin's lymphoma | Active (positive) | 9 Oct 2009 |
| dextran sulfate EU/3/09/669 |
Prevention of graft rejection during pancreatic islet transplantation | Active (positive) | 9 Oct 2009 |
| hydroxychloroquine sulfate EU/3/17/1963 28 clinical trials → |
Treatment of LIPIN1 disease | Active (positive) | 8 Oct 2009 |
| recombinant self-complementary adeno-associated viral vector serotype 9 containing the human CLN3 gene EU/3/16/1806 |
Treatment of neuronal ceroid lipofuscinosis | Withdrawn | 8 Oct 2009 |
| beloranib EU/3/15/1530 |
Treatment of craniopharyngioma | Withdrawn | 8 Oct 2009 |
| Glucagon EU/3/14/1342 2 clinical trials → |
Treatment of congenital hyperinsulinism | Active (positive) | 8 Oct 2009 |
| human heterologous liver cells EU/3/10/818 · now authorised as Heparesc |
Treatment of citrullinaemia type 1 | Active (positive) | 8 Oct 2009 |
| N-[(2S)-2,3-dihydroxypropyl]-3-[(2-fluoro-4-iodophenyl)amino]isonicotinamide hydrochloride EU/3/09/685 |
Treatment of pancreatic cancer | Withdrawn | 8 Oct 2009 |
| 6-chloro-2,3,4,9-tetrahydro-1H-carbazole-1-carboxamide (selisistat) EU/3/09/681 |
Treatment of Huntington’s disease | Active (positive) | 8 Oct 2009 |
EU/3/09/678 |
Treatment of soft tissue sarcoma | Withdrawn | 8 Oct 2009 |
| Human tumour necrosis factor alpha-derived peptide Cys-Gly-Gln-Arg-Glu-Thr-Pro-Glu-Gly-Ala-Glu-Ala-Lys-Pro-Trp-Tyr-Cys EU/3/09/677 |
Treatment of acute lung Injury | Active (positive) | 8 Oct 2009 |
| sequence-modified recombinant human factor VIIa EU/3/09/676 |
Treatment of haemophilia B | Withdrawn | 8 Oct 2009 |
| Pomalidomide EU/3/09/672 64 clinical trials → |
Treatment of multiple myeloma | Expired | 8 Oct 2009 |
| pasireotide EU/3/09/671 |
Treatment of Cushing's disease | Expired | 8 Oct 2009 |
| pasireotide EU/3/09/670 |
Treatment of acromegaly | Expired | 8 Oct 2009 |
| human C1 inhibitor EU/3/09/668 |
Treatment of angioedema caused by C1 inhibitor deficiency | Withdrawn | 8 Oct 2009 |
| expanded human allogeneic mesenchymal adult stem cells extracted from adipose tissue (darvadstrocel) EU/3/09/667 |
Treatment of anal fistula | Withdrawn | 8 Oct 2009 |
| Eicosapentaenoic acid EU/3/09/666 |
Treatment of familial adenomatous polyposis (FAP) | Active (positive) | 8 Oct 2009 |
| allogeneic ex-vivo-expanded umbilical-cord blood cells EU/3/09/665 |
Treatment of chronic myeloid leukaemia | Withdrawn | 8 Oct 2009 |
| allogeneic ex-vivo-expanded umbilical-cord blood cells EU/3/09/664 |
Treatment of myelodysplastic syndromes | Withdrawn | 8 Oct 2009 |
| adeno-associated viral vector containing modified U1 snRNA EU/3/09/663 |
Treatment of Duchenne muscular dystrophy | Withdrawn | 8 Oct 2009 |
| 26 base single stranded phosphodiester DNA oligonucleotide EU/3/09/662 |
Treatment of acute myeloid leukaemia | Withdrawn | 8 Oct 2009 |
| alpha-1-acid glycoprotein EU/3/03/164 |
Treatment of cocaine poisoning | Withdrawn | 8 Oct 2009 |
| trabedersen EU/3/09/660 |
Treatment of pancreatic cancer | Active (positive) | 24 Jul 2009 |
| tosedostat EU/3/09/659 |
Treatment of acute myeloid leukaemia | Withdrawn | 24 Jul 2009 |
| tamibarotene EU/3/09/658 |
Treatment of acute promyelocytic leukaemia | Withdrawn | 24 Jul 2009 |
| recombinant human N-acetylgalactosamine-6-sulfatase (elosulfase alfa) EU/3/09/657 |
Treatment of mucopolysaccharidosis, type IVA (Morquio A syndrome) | Withdrawn | 24 Jul 2009 |
| recombinant histidine-tagged idiotype immunoglobulin Fab fragment of clonal B-cell receptors EU/3/09/656 |
Treatment of diffuse large B-cell lymphoma | Active (positive) | 24 Jul 2009 |
| octocog alfa EU/3/09/655 |
Treatment of haemophilia A | Withdrawn | 24 Jul 2009 |
| Hypothiocyanite;lactoferrin EU/3/09/654 |
Treatment of cystic fibrosis | Active (positive) | 24 Jul 2009 |
| Eculizumab EU/3/09/653 12 clinical trials → |
Treatment of atypical haemolytic uraemic syndrome (aHUS) | Active (positive) | 24 Jul 2009 |
| ciprofloxacin EU/3/09/652 11 clinical trials → |
Treatment of cystic fibrosis | Active (positive) | 24 Jul 2009 |
| ciclosporin EU/3/09/651 3 clinical trials → |
Treatment of atopic keratoconjunctivitis | Active (positive) | 24 Jul 2009 |
| blinatumomab EU/3/09/650 · now authorised as Blincyto 43 clinical trials → |
Treatment of acute lymphoblastic leukaemia | Active (positive) | 24 Jul 2009 |
| allogeneic ex-vivo-expanded umbilical-cord blood cells EU/3/09/649 |
Treatment of Hodgkin's lymphoma | Withdrawn | 24 Jul 2009 |
| afamelanotide EU/3/09/648 |
Treatment of solar urticaria | Active (positive) | 24 Jul 2009 |
| (S)-3'-(OH)-desazadesferrithiocin-polyether, magnesium salt (deferitazole) EU/3/09/647 |
Treatment of chronic iron overload requiring chelation therapy | Withdrawn | 24 Jul 2009 |
| talampanel EU/3/09/646 |
Treatment of amyotrophic lateral sclerosis | Withdrawn | 12 Jun 2009 |
| octreotide hydrochloride EU/3/09/645 3 clinical trials → |
Treatment of acromegaly | Withdrawn | 12 Jun 2009 |
| Murine monoclonal antibody to GD2 EU/3/09/644 |
Treatment of neuroblastoma | Withdrawn | 12 Jun 2009 |
| Desipramine chlorhydrate EU/3/09/643 |
Treatment of Rett syndrome | Withdrawn | 12 Jun 2009 |
| chimeric anti-interleukin-6 monoclonal antibody EU/3/09/642 |
Treatment of multiple myeloma | Withdrawn | 12 Jun 2009 |
| alicaforsen EU/3/09/641 |
Treatment of pouchitis | Active (positive) | 15 May 2009 |
| pegylated recombinant human factor IX (nonacog beta pegol) EU/3/09/640 |
Treatment of haemophilia B | Expired | 15 May 2009 |
| 4,6,8-Trihydroxy-10-(3,7,11-trimethyldodeca-2,6,10-trienyl)-5,10-dihydrodibenzo[b,e][1,4] diazepin-11-one EU/3/09/639 |
Treatment of glioma | Withdrawn | 15 May 2009 |
| Humanised IgG4 monoclonal antibody to the human toll-like receptor type 2 EU/3/09/638 |
Prevention of ischaemia/reperfusion injury associated with solid organ transplantation | Active (positive) | 15 May 2009 |
| 2',3',5'-Tri-O-acetyluridine EU/3/09/637 |
Treatment of 5-fluorouracil overdose | Active (positive) | 15 May 2009 |
| Dexamethasone phosphate EU/3/09/636 325 clinical trials → |
Treatment of corneal graft rejection | Withdrawn | 15 May 2009 |
| treprostinil diethanolamine EU/3/09/635 |
Treatment of systemic sclerosis | Active (positive) | 15 May 2009 |
| S-[2,3-bispalmitoyloxy-(2R)-propyl]-cysteinyl-GNNDESNISFKEK EU/3/09/634 |
Treatment of pancreatic cancer | Active (positive) | 15 May 2009 |
| L-asparaginase encapsulated in erythrocytes EU/3/09/633 |
Treatment of pancreatic cancer | Active (positive) | 15 May 2009 |
| skin equivalent graft genetically corrected with a COL7A1-encoding SIN retroviral vector EU/3/09/630 |
Treatment of dystrophic epidermolysis bullosa | Active (positive) | 30 Apr 2009 |
| nanobody directed towards the human A1 domain of von Willebrand factor (caplacizumab) EU/3/09/629 · now authorised as Cablivi |
Treatment of thrombotic thrombocytopenic purpura | Active (positive) | 30 Apr 2009 |
| Mercaptopurine EU/3/09/628 15 clinical trials → |
Treatment of acute lymphoblastic leukaemia | Expired | 30 Apr 2009 |
| Lintuzumab EU/3/09/627 |
Treatment of acute myeloid leukaemia | Withdrawn | 30 Apr 2009 |
| adeno-associated viral vector containing porphobilinogen deaminase gene EU/3/09/632 |
Treatment of acute intermittent porphyria | Withdrawn | 29 Apr 2009 |
| talampanel EU/3/09/631 |
Treatment of glioma | Withdrawn | 29 Apr 2009 |
| Lintuzumab EU/3/09/626 |
Treatment of myelodysplastic syndromes | Withdrawn | 29 Apr 2009 |
| Guanabenz EU/3/09/625 |
Treatment of traumatic spinal cord injury | Active (positive) | 29 Apr 2009 |
| autologous Tumor-Derived gp96 Heat Shock Protein-Peptide Complex EU/3/09/624 |
Treatment of glioma | Active (positive) | 29 Apr 2009 |
| autologous haematopoietic stem cells transduced with lentiviral vector encoding the human beta-globin gene EU/3/09/623 |
Treatment of beta thalassaemia intermedia and major | Active (positive) | 29 Apr 2009 |
| (R)-3-(4-(7H-pyrrolo[2,3-d]pyrimidin-4-yl)-1H-pyrazol-1-yl)-3-cyclopentylpropanenitrile phosphate EU/3/09/620 |
Treatment of myelofibrosis secondary to polycythaemia vera or essential thrombocythaemia | Withdrawn | 3 Apr 2009 |
| N-(5-tert-Butylisoxazol-3-yl)-N'-{4-[7-(2-(morpholin-4-yl)ethoxy) imidazo[2,1-b][1,3]benzothiazol-2-yl]phenyl}urea di-hydrochloride salt EU/3/09/622 |
Treatment of acute myeloid leukaemia | Withdrawn | 23 Mar 2009 |
| 2,2-Dimethylbutyric acid, sodium salt EU/3/09/621 |
Treatment of sickle cell disease | Active (positive) | 18 Mar 2009 |
| tobramycin EU/3/09/613 2 clinical trials → |
Treatment of Pseudomonas aeruginosa lung infection in cystic fibrosis | Withdrawn | 28 Feb 2009 |
| allogeneic ex-vivo-expanded umbilical-cord blood cells EU/3/09/619 |
Treatment of acute myeloid leukaemia | Withdrawn | 27 Feb 2009 |
| allogeneic ex-vivo-expanded umbilical-cord blood cells EU/3/09/618 |
Treatment of acute lymphoblastic leukaemia | Withdrawn | 27 Feb 2009 |
| 2,2-Dimethylbutyric acid, sodium salt EU/3/09/617 |
Treatment of beta thalassaemia intermedia and major | Active (positive) | 27 Feb 2009 |
| (6R)-4,5,6,7-tetrahydro-N6-propyl-2,6-benzothiazolediamine dihydrochloride monohydrate EU/3/09/616 |
Treatment of amyotrophic lateral sclerosis | Active (positive) | 27 Feb 2009 |
| N-terminal hexaglutamine-tagged recombinant human N-acetylgalactosamine-6-sulfate sulfatase EU/3/09/615 |
Treatment of mucopolysaccharidosis, type IVA (Morquio A syndrome) | Active (positive) | 27 Feb 2009 |
| mifepristone EU/3/09/614 3 clinical trials → |
Treatment of hypercortisolism (Cushing's syndrome) of endogenous origin | Active (positive) | 27 Feb 2009 |
| exon 51 specific phosphorothioate oligonucleotide EU/3/08/599 |
Treatment of Duchenne muscular dystrophy | Active (positive) | 27 Feb 2009 |
| exon 44 specific phosphorothioate oligonucleotide EU/3/08/598 |
Treatment of Duchenne muscular dystrophy | Active (positive) | 27 Feb 2009 |
| recombinant human proinsulin EU/3/08/612 |
Treatment of retinitis pigmentosa | Active (positive) | 11 Feb 2009 |
| recombinant human hepatocarcinoma-intestine-pancreas;pancreatic associated protein EU/3/08/611 |
Treatment of acute liver failure | Active (positive) | 11 Feb 2009 |
| type I native bovine skin collagen EU/3/08/607 |
Treatment of systemic sclerosis | Active (positive) | 9 Feb 2009 |
| adeno-associated viral vector serotype 5 containing the human ABCA4 gene EU/3/08/609 |
Treatment of Stargardt's disease | Withdrawn | 7 Feb 2009 |
| Cyclopropane-1,1-dicarboxylic acid [4-(6,7-dimethoxy-quinolin-4-yloxy)-phenyl]-amide (4-fluoro-phenyl)-amide, (L)-malate salt (cabozantinib) EU/3/08/610 |
Treatment of medullary thyroid carcinoma | Active (positive) | 6 Feb 2009 |
| yttrium (90Y)-DOTA-radiolabelled humanized monoclonal antibody against mucin 1 EU/3/08/608 |
Treatment of pancreatic cancer | Withdrawn | 6 Feb 2009 |
| 2,3,4,5 Tetrahydro-2,8-dimethyl-5-[2-(6-methyl-3-pyridinyl)ethyl]-1H-pyrido[4,3-b]indole dihydrochloride EU/3/08/597 |
Treatment of Huntington’s disease | Withdrawn | 21 Jan 2009 |
| recombinant human minibody against complement component C5 fused with RGD-motif EU/3/08/604 |
Prevention of ischaemia/reperfusion injury associated with solid organ transplantation | Withdrawn | 20 Jan 2009 |
| human anti-intercellular adhesion molecule-1 monoclonal antibody EU/3/08/600 |
Treatment of multiple myeloma | Active (positive) | 20 Jan 2009 |
| recombinant human residue 41 glutamic acid to glutamine variant of interferon alfa-2b EU/3/08/606 |
Treatment of Behçet's Disease | Withdrawn | 19 Jan 2009 |
| recombinant human monoclonal antibody to human Nogo-A protein of the IgG4/kappa class (atinumab) EU/3/08/605 |
Treatment of spinal cord injury | Withdrawn | 19 Jan 2009 |
| pralatrexate EU/3/08/603 3 clinical trials → |
Treatment of non-papillary transitional cell carcinoma of the urinary bladder | Active (positive) | 19 Jan 2009 |
| Milatuzumab EU/3/08/602 |
Treatment of chronic lymphocytic leukaemia | Withdrawn | 19 Jan 2009 |
| Milatuzumab EU/3/08/601 |
Treatment of multiple myeloma | Withdrawn | 19 Jan 2009 |
| monoclonal antibody against human CD30 covalently linked to the cytotoxin monomethylauristatin E (brentuximab vedotin) EU/3/08/596 · now authorised as Adcetris |
Treatment of Hodgkin's lymphoma | Withdrawn | 15 Jan 2009 |
| murine anti-CD22 antibody variable region fused to truncated Pseudomonas exotoxin 38 ( Moxetumomab pasudotox) EU/3/08/592 |
Treatment of hairy cell leukaemia | Withdrawn | 5 Dec 2008 |
| 2-[[3-({4-[(5-{2-[(3-Fluorophenyl)amino]-2-oxoethyl}-1H-pyrazol-3-yl)amino]-quinazolin-7-yl}oxy)propyl](ethyl)amino]ethyl dihydrogen phosphate trihydrate EU/3/08/590 |
Treatment of acute myeloid leukaemia | Withdrawn | 5 Dec 2008 |
| recombinant human tissue non-specific alkaline phosphatase - Fc - deca-aspartate fusion protein EU/3/08/594 · now authorised as Strensiq |
Treatment of hypophosphatasia | Active (positive) | 4 Dec 2008 |
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.