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 |
|---|---|---|---|
| 5-(Ethylsulfonyl)-2-(naphthalen-2-yl)benzo[d]oxazole (ezutromid) EU/3/08/591 |
Treatment of Duchenne muscular dystrophy | Withdrawn | 4 Dec 2008 |
| N2'-Deacetyl-N2'-[4-methyl-4-(oxobuthyldithio)-1-oxopentyl]-maytansine-chimerized anti-CD138 IgG4 monoclonal antibody EU/3/08/593 |
Treatment of multiple myeloma | Withdrawn | 3 Dec 2008 |
| recombinant human ADAMTS-13 EU/3/08/588 · now authorised as Adzynma |
Treatment of thrombotic thrombocytopenic purpura | Active (positive) | 3 Dec 2008 |
| cenersen EU/3/08/587 |
Treatment of chronic lymphocytic leukaemia | Active (positive) | 3 Dec 2008 |
| RNA, [P-deoxy-P-(dimethylamino)] (2',3'-dideoxy-2',3'-imino-2',3'-seco) (2'a→5') (C-m5U-C-C-A-A-C-A-m5U-C-A-A-G-G-A-A-G-A-m5U-G-G-C-A-m5U-m5U-m5U-C-m5U-A-G), P… EU/3/08/586 · now authorised as Exondys |
Treatment of Duchenne muscular dystrophy | Active (positive) | 3 Dec 2008 |
| daunorubicin EU/3/08/585 43 clinical trials → |
Treatment of acute myeloid leukaemia | Active (positive) | 3 Dec 2008 |
| palifosfamide EU/3/08/584 |
Treatment of soft tissue sarcoma | Withdrawn | 3 Dec 2008 |
| gadodiamide EU/3/08/583 |
Treatment of glioma | Active (positive) | 3 Dec 2008 |
| autologous urothelial and smooth muscle cells EU/3/08/574 |
Treatment of spinal cord injury | Withdrawn | 8 Nov 2008 |
| recombinant human heparan-N-sulfatase EU/3/08/582 |
Treatment of mucopolysaccharidosis, type IIIA (Sanfilippo A syndrome) | Withdrawn | 7 Nov 2008 |
| ofatumumab EU/3/08/581 11 clinical trials → |
Treatment of chronic lymphocytic leukaemia | Withdrawn | 7 Nov 2008 |
| filgrastim EU/3/08/580 14 clinical trials → |
Treatment of spinal cord injury | Active (positive) | 7 Nov 2008 |
| ex-vivo-expanded autologous human corneal epithelium-containing stem cells EU/3/08/579 · now authorised as Holoclar |
Corneal lesions, with associated corneal (limbal) stem cell deficiency, due to ocular burns | Active (positive) | 7 Nov 2008 |
| cysteamine hydrochloride EU/3/08/578 · now authorised as Cystadrops |
Treatment of cystinosis | Active (positive) | 7 Nov 2008 |
| carglumic acid EU/3/08/577 2 clinical trials → |
Treatment of propionic acidaemia | Expired | 7 Nov 2008 |
| carglumic acid EU/3/08/576 2 clinical trials → |
Treatment of methylmalonic acidaemia | Expired | 7 Nov 2008 |
| carglumic acid EU/3/08/575 2 clinical trials → |
Treatment of isovaleric acidaemia | Expired | 7 Nov 2008 |
| adeno-associated viral vector containing the human alpha-sarcoglycan gene EU/3/08/573 |
Treatment of alpha-sarcoglycanopathy | Withdrawn | 7 Nov 2008 |
| (R)-3-(4-(7H-pyrrolo[2,3-d]pyrimidin-4-yl)-1H-pyrazol-1-yl)-3-cyclopentylpropanenitrile phosphate EU/3/08/572 |
Treatment of chronic idiopathic myelofibrosis | Withdrawn | 7 Nov 2008 |
| Levofloxacin hemihydrate EU/3/08/566 10 clinical trials → |
Treatment of cystic fibrosis | Withdrawn | 23 Sep 2008 |
| avian polyclonal IgY antibody EU/3/08/564 |
Treatment of cystic fibrosis | Active (positive) | 23 Sep 2008 |
| recombinant human minibody against complement component C5 EU/3/08/571 |
Treatment of atypical haemolytic uraemic syndrome (aHUS) | Withdrawn | 22 Sep 2008 |
| recombinant human CXCL8 mutant EU/3/08/570 |
Prevention of delayed graft function after solid organ transplantation | Active (positive) | 22 Sep 2008 |
| pegylated L-asparaginase EU/3/08/569 |
Treatment of acute lymphoblastic leukaemia | Active (positive) | 22 Sep 2008 |
| N'-(5-chloro-2-hydroxy-3-methylbenzylidene)-2,4-dihydroxybenzhydrazide EU/3/08/568 |
Treatment of partial deep dermal and full-thickness burns | Active (positive) | 22 Sep 2008 |
| Miltefosine EU/3/08/567 |
Treatment of cutaneous T-cell lymphoma | Active (positive) | 22 Sep 2008 |
| drotrecogin alfa (activated) EU/3/08/565 |
Treatment of acute respiratory distress syndrome (ARDS) | Withdrawn | 22 Sep 2008 |
| 4-[[[4-(4-Chlorophenoxy)phenyl]sulfonyl]-methyl]tetrahydro-N-hydroxy-2H-pyran-4-carboxamide - |
Prevention of graft rejection after liver transplantation | Negative | 10 Sep 2008 |
| recombinant derivative of C3 transferase EU/3/08/563 |
Treatment of traumatic spinal cord injury | Active (positive) | 5 Sep 2008 |
| topotecan hydrochloride EU/3/08/562 46 clinical trials → |
Treatment of glioma | Active (positive) | 5 Sep 2008 |
| Donor lymphocyte preparation depleted of functional alloreactive T-cells EU/3/08/561 · now authorised as Luxceptar |
Prevention of graft-versus-host disease | Withdrawn | 5 Sep 2008 |
EU/3/08/560 |
Treatment of moderate and severe closed traumatic brain injury | Active (positive) | 5 Sep 2008 |
| Bosentan EU/3/08/559 3 clinical trials → |
Treatment of idiopathic pulmonary fibrosis | Withdrawn | 5 Sep 2008 |
| yttrium (90Y) edotreotide EU/3/08/589 |
Treatment of gastro-entero-pancreatic neuroendocrine tumours | Withdrawn | 4 Aug 2008 |
| sapacitabine EU/3/08/558 |
Treatment of acute myeloid leukaemia | Active (positive) | 10 Jul 2008 |
| beraprost sodium EU/3/08/554 |
Treatment of pulmonary arterial hypertension | Withdrawn | 10 Jul 2008 |
| sapacitabine EU/3/08/557 |
Treatment of myelodysplastic syndromes | Active (positive) | 8 Jul 2008 |
| sapacitabine EU/3/08/557 |
Treatment of myelodysplastic syndromes | Active (positive) | 8 Jul 2008 |
| N-(2,4-di-tert-butyl-5-hydroxyphenyl)-1,4-dihydro-4-oxoquinoline-3-carboxamide (ivacaftor) EU/3/08/556 |
Treatment of cystic fibrosis | Expired | 8 Jul 2008 |
| vincristine sulfate liposomes EU/3/08/555 |
Treatment of acute lymphoblastic leukaemia | Withdrawn | 8 Jul 2008 |
| pegylated recombinant factor VIIa EU/3/08/551 |
Treatment of haemophilia A | Withdrawn | 4 Jun 2008 |
| recombinant fusion protein of circulary-permuted IL-4 and pseudomonas exotoxin A, [IL-4(38-37)-PE38KDEL] EU/3/08/553 |
Treatment of glioma | Active (positive) | 3 Jun 2008 |
| pegylated recombinant factor VIIa EU/3/08/552 |
Treatment of haemophilia B | Withdrawn | 3 Jun 2008 |
| nimotuzumab EU/3/08/550 30 clinical trials → |
Treatment of pancreatic cancer | Active (positive) | 3 Jun 2008 |
| NGR-human tumour necrosis factor EU/3/08/549 · now authorised as Zafiride |
Treatment of malignant mesothelioma | Active (positive) | 3 Jun 2008 |
| carfilzomib EU/3/08/548 46 clinical trials → |
Treatment of multiple myeloma | Expired | 3 Jun 2008 |
| anti-von Willebrand aptamer EU/3/08/547 |
Treatment of thrombotic thrombocytopenic purpura | Withdrawn | 3 Jun 2008 |
EU/3/08/546 |
Treatment of congenital alpha-1 antitrypsin deficiency | Active (positive) | 3 Jun 2008 |
EU/3/08/545 |
Treatment of congenital erythropoietic porphyria | Active (positive) | 8 May 2008 |
| omigapil maleate EU/3/08/544 |
Treatment of congenital muscular dystrophy with merosin (laminin alpha 2) deficiency | Withdrawn | 8 May 2008 |
| sarsasapogenin EU/3/08/543 |
Treatment of amyotrophic lateral sclerosis | Withdrawn | 8 May 2008 |
| ribonucleotide reductase R2 specific phosphorothioate oligonucleotide EU/3/08/542 |
Treatment of acute myeloid leukaemia | Active (positive) | 8 May 2008 |
| [Nle4, D-Phe7]-alfa-melanocyte stimulating hormone (afamelanotide) EU/3/08/541 |
Treatment of erythropoietic protoporphyria | Expired | 8 May 2008 |
| omigapil maleate EU/3/08/540 |
Treatment of congenital muscular dystrophy with collagen VI deficiency | Withdrawn | 8 May 2008 |
| amrubicin hydrochloride EU/3/08/538 4 clinical trials → |
Treatment of small cell lung cancer | Withdrawn | 2 Apr 2008 |
| ammonium tetrathiomolybdate EU/3/08/539 |
Treatment of Wilson's disease | Active (positive) | 1 Apr 2008 |
| Humanised monoclonal antibody to the folate receptor alpha EU/3/08/535 |
Treatment of ovarian cancer | Withdrawn | 1 Apr 2008 |
| allogeneic human umbilical cord tissue-derived cells EU/3/08/534 |
Treatment of retinitis pigmentosa | Withdrawn | 1 Apr 2008 |
| recombinant human monoclonal antibody against transforming growth factor beta-1, 2 and 3 EU/3/08/533 |
Treatment of idiopathic pulmonary fibrosis | Withdrawn | 1 Apr 2008 |
| filgrastim EU/3/08/532 14 clinical trials → |
Treatment of amyotrophic lateral sclerosis | Active (positive) | 1 Apr 2008 |
| ascorbic acid EU/3/08/531 2 clinical trials → |
Treatment of Charcot-Marie-Tooth disease type 1A | Active (positive) | 1 Apr 2008 |
| autologous urothelial and smooth muscle cells EU/3/08/537 |
Treatment of spina bifida | Withdrawn | 18 Mar 2008 |
| chimeric antibody to mesothelin EU/3/08/536 |
Treatment of pancreatic cancer | Withdrawn | 17 Mar 2008 |
| recombinant human monoclonal antibody to human IL-1beta of the IgG1/K class EU/3/08/527 |
Treatment of systemic-onset juvenile idiopathic arthritis | Withdrawn | 5 Feb 2008 |
| heterologous human adult liver derived stem cells EU/3/08/530 |
Treatment of ornithinine transcarbamylase deficiency | Active (positive) | 4 Feb 2008 |
| tretazicar EU/3/08/529 |
Treatment of visceral leishmaniasis | Active (positive) | 4 Feb 2008 |
| Lumiliximab EU/3/08/528 |
Treatment chronic lymphocytic leukaemia | Withdrawn | 3 Feb 2008 |
| (R)-2-Methyl-6-nitro-2-{4-[4-(4-trifluoromethoxyphenoxy)piperidin-1-yl]phenoxymethyl}-2,3-dihydroimidazo[2,1-b]oxazole (delamanid) EU/3/07/524 |
Treatment of tuberculosis | Expired | 2 Feb 2008 |
| N- (2-Amino-phenyl)-4-[(4-pyridin-3-yl-pyrimidin-2-ylamino)-methyl] benzamide (mocetinostat) EU/3/07/526 |
Treatment of acute myeloid leukaemia | Withdrawn | 31 Jan 2008 |
| Iodine (131I) iobenguane EU/3/07/525 |
Treatment of neuroblastoma | Active (positive) | 31 Jan 2008 |
| Lutetium (177Lu)-N-[(4,7,10-Tricarboxymethyl-1,4,7,10-tetraazacyclododec-1-yl)acetyl]-D-phenylalanyl-L-cysteinyl-L-tyrosyl-D-tryptophanyl-L-lysyl-L-threoninyl-… EU/3/07/523 · now authorised as Lutathera |
Treatment of gastro-entero-pancreatic neuroendocrine tumours | Active (positive) | 31 Jan 2008 |
| (Manganese, dichloro [(4aR, 13aR, 17aR, 21aR)-1, 2, 3, 4, 4a, 5, 6, 12, 13, 13a, 14, 15, 16, 17, 17a, 18, 19, 20, 21, 21a-eicosahydro-11, 7-nitrilo-7H-dibenzo[… EU/3/07/522 |
Prevention of oral mucositis in head and neck cancer patients undergoing radiation therapy | Active (positive) | 31 Jan 2008 |
| Human-papilloma-virus-type-16 E6;E7 synthetic long peptides EU/3/07/520 |
Treatment of epithelial neoplasia of the vulva positive for human papilloma virus | Active (positive) | 20 Dec 2007 |
| Methyl 4,6-diamino-2-[1-(2-fluorobenzyl)-1H-pyrazolo[3,4-b]pyridine-3-yl]-5-pyrimidinyl(methyl)carbamate (riociguat) EU/3/07/518 |
Treatment of pulmonary arterial hypertension | Withdrawn | 20 Dec 2007 |
| N-[4-(3-amino-1H-indazol-4 yl)phenyl]-N'-(2-fluoro-5-methylphenyl) urea EU/3/07/517 |
Treatment of hepatocellular carcinoma | Withdrawn | 20 Dec 2007 |
| recombinant human histone H1.3 and recombinant human N-bis-met-histone H1.3 EU/3/07/516 |
Treatment of acute myeloid leukaemia | Active (positive) | 20 Dec 2007 |
| tegafur;gimeracil;oteracil potassium EU/3/07/515 2 clinical trials → |
Treatment of gastric cancer | Withdrawn | 20 Dec 2007 |
| H-Arg-Leu-Phe-Phe-Tyr-Arg-Lys-Ser-Val-OH, acetate salt& H-Tyr-Leu-Phe-Phe-Tyr-Arg-Lys-Ser-Val-OH, acetate salt EU/3/07/521 |
Treatment of TERT positive non-small cell lung cancer in HLA-A2 positive patients | Active (positive) | 18 Dec 2007 |
| Maribavir EU/3/07/519 · now authorised as Livtencity 7 clinical trials → |
Prevention of cytomegalovirus disease in patients with impaired cell-mediated immunity | Active (positive) | 18 Dec 2007 |
| artesunate EU/3/07/510 5 clinical trials → |
Treatment of malaria | Active (positive) | 7 Dec 2007 |
| recombinant human hepatitis C monoclonal antibody against C4 region of E1 EU/3/07/503 |
Prevention of recurrent hepatitis C virus induced liver disease in liver transplant recipients | Active (positive) | 6 Dec 2007 |
| Picoplatin EU/3/07/502 |
Treatment of small cell lung cancer | Withdrawn | 6 Dec 2007 |
| olaparib EU/3/07/501 113 clinical trials → |
Treatment of ovarian cancer | Withdrawn | 6 Dec 2007 |
EU/3/07/514 · now authorised as Cerdelga |
Treatment of Gaucher disease | Active (positive) | 4 Dec 2007 |
| 3-methoxy-pregnenolone EU/3/07/511 |
Treatment of spinal cord injury | Active (positive) | 4 Dec 2007 |
| chelidonii radix - |
Treatment of pancreatic cancer | Negative | 4 Dec 2007 |
| chimeric anti-interleukin-6 monoclonal antibody (siltuximab) EU/3/07/508 |
Treatment of Castleman's disease | Expired | 30 Nov 2007 |
| doxorubicin hydrochloride EU/3/07/507 126 clinical trials → |
Treatment of glioma | Withdrawn | 30 Nov 2007 |
| irinotecan hydrochloride EU/3/07/504 140 clinical trials → |
Treatment of glioma | Withdrawn | 30 Nov 2007 |
| (S)-2-nitro-6-(4-trifluoromethoxy)benzyloxy)-6,7-dihydro-5H-imidazo[2,1-b] [1,3] oxazine (pretomanid) EU/3/07/513 · now authorised as Dovprela (previously Pretomanid FGK) |
Treatment of tuberculosis | Active (positive) | 29 Nov 2007 |
| 17-(Allylamino)-17-demethoxygeldanamycin, hydroquinone hydrochloride EU/3/07/512 |
Treatment of malignant gastrointestinal stromal tumours | Withdrawn | 29 Nov 2007 |
| azacitidine EU/3/07/509 127 clinical trials → |
Treatment of acute myeloid leukaemia | Expired | 29 Nov 2007 |
| heterologous human adult liver-derived stem cells EU/3/07/506 |
Treatment of Crigler-Najjar syndrome | Active (positive) | 29 Nov 2007 |
| Interferon beta EU/3/07/505 |
Treatment of acute respiratory distress syndrome (ARDS) | Withdrawn | 29 Nov 2007 |
| recombinant human rod-derived cone viability factor EU/3/07/500 |
Treatment of retinitis pigmentosa | Withdrawn | 29 Nov 2007 |
| terguride EU/3/07/499 |
Treatment of pulmonary arterial hypertension and chronic thromboembolic pulmonary hypertension | Active (positive) | 29 Nov 2007 |
| lenalidomide EU/3/07/494 167 clinical trials → |
Treatment of chronic lymphocytic leukaemia | Withdrawn | 19 Nov 2007 |
| Polihexanide EU/3/07/498 · now authorised as Akantior |
Treatment of acanthamoeba keratitis | Active (positive) | 14 Nov 2007 |
| everolimus EU/3/07/488 53 clinical trials → |
Treatment of gastro-entero-pancreatic neuroendocrine tumours | Withdrawn | 14 Nov 2007 |
| 4-Ethoxy-2-(piperazin-1-yl)-7-(pyridin-4-yl)-5H-pyrimido[5,4-b]indol EU/3/07/487 |
Treatment of chronic lymphocytic leukaemia | Active (positive) | 14 Nov 2007 |
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.