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 |
|---|---|---|---|
| allogeneic motor neuron progenitor cells derived from human embryonic stem cells EU/3/12/1088 |
Treatment of 5q spinal muscular atrophy | Active (positive) | 24 Jan 2013 |
| recombinant modified human growth hormone EU/3/12/1087 · now authorised as Ngenla |
Treatment of growth hormone deficiency | Active (positive) | 24 Jan 2013 |
| eflornithine EU/3/12/1086 · now authorised as Flynpovi 6 clinical trials → |
Treatment of familial adenomatous polyposis (FAP) | Active (positive) | 24 Jan 2013 |
| voclosporin EU/3/12/1085 2 clinical trials → |
Treatment of non-infectious uveitis | Active (positive) | 6 Dec 2012 |
| Erdosteine EU/3/12/1084 2 clinical trials → |
Treatment of lead toxicity | Active (positive) | 6 Dec 2012 |
| Humanised single-chain monoclonal antibody against CD37 (otlertuzumab) EU/3/12/1083 |
Treatment chronic lymphocytic leukaemia | Withdrawn | 6 Dec 2012 |
| triheptanoin EU/3/12/1082 |
Treatment of long-chain 3-hydroxyacyl-coenzyme A dehydrogenase deficiency | Withdrawn | 6 Dec 2012 |
| triheptanoin EU/3/12/1081 |
Treatment of very-long-chain 3-hydroxyacyl-CoA-dehydrogenase deficiency | Withdrawn | 6 Dec 2012 |
| 4-(4-{[2-(4-Chlorophenyl)-4,4-dimethylcyclohex-1-en-1-yl]methyl}piperazin-1-yl)-N-({3-nitro-4-[(tetrahydro-2H-pyran-4-ylmethyl)amino]phenyl}sulfonyl)-2-(1H-pyr… EU/3/12/1080 |
Treatment of chronic lymphocytic leukaemia | Withdrawn | 6 Dec 2012 |
| artesunate EU/3/12/1079 5 clinical trials → |
Treatment of malaria | Active (positive) | 6 Dec 2012 |
| exon 55 specific phosphorothioate oligonucleotide EU/3/12/1078 |
Treatment of Duchenne muscular dystrophy | Active (positive) | 6 Dec 2012 |
| exon 52 specific phosphorothioate oligonucleotide EU/3/12/1077 |
Treatment of Duchenne muscular dystrophy | Active (positive) | 6 Dec 2012 |
| allopurinol sodium EU/3/12/1076 7 clinical trials → |
Treatment of perinatal asphyxia | Active (positive) | 6 Dec 2012 |
| Cyclo(-gamma-aminobutyryl-L-phenylalanyl-L-tryptophanyl-D-tryptophanyl-L-lysyl-L-threonyl-L phenylalanyl-N-3-carboxypropyl)-glycine amide, acetate salt EU/3/12/1075 |
Treatment of acromegaly | Active (positive) | 6 Dec 2012 |
| alisertib EU/3/12/1074 3 clinical trials → |
Treatment of peripheral T-cell lymphoma (nodal, other extranodal and leukaemic/disseminated) | Withdrawn | 6 Dec 2012 |
| Maytansinoid-conjugated human monoclonal antibody against mesothelin (anetumab ravtansine) EU/3/12/1073 |
Treatment of malignant mesothelioma | Withdrawn | 6 Dec 2012 |
| 17-(Dimethylaminoethylamino)-17-demethoxygeldanamycin EU/3/12/1007 |
Treatment of retinitis pigmentosa | Withdrawn | 28 Nov 2012 |
| Doxorubicin EU/3/12/1006 126 clinical trials → |
Treatment of glioma | Withdrawn | 28 Nov 2012 |
| Encapsulated human retinal pigment epithelial cell line transfected with plasmid vector expressing human ciliary neurotropic factor EU/3/12/1072 |
Treatment of macular telangiectasia type 2 | Active (positive) | 8 Nov 2012 |
| canakinumab EU/3/12/1071 |
Treatment of tumour-necrosis-factor-receptor-associated periodic syndrome | Withdrawn | 8 Nov 2012 |
| recombinant human dyskerin EU/3/12/1070 |
Treatment of dyskeratosis congenita | Active (positive) | 8 Nov 2012 |
| adeno-associated viral vector encoding an inducible short hairpin RNA targeting claudin-5 EU/3/12/1069 |
Treatment of retinitis pigmentosa | Withdrawn | 8 Nov 2012 |
| Melarsoprol EU/3/12/1068 |
Treatment of African trypanosomiasis | Active (positive) | 8 Nov 2012 |
| Erdosteine EU/3/12/1067 2 clinical trials → |
Treatment of mercury toxicity | Active (positive) | 8 Nov 2012 |
| tafamidis EU/3/12/1066 · now authorised as Vyndaqel |
Treatment of senile systemic amyloidosis | Active (positive) | 8 Nov 2012 |
| synthetic double-stranded siRNA oligonucleotide directed against claudin-5 complexed with polyethyleneimine EU/3/12/1065 |
Treatment of glioma | Withdrawn | 8 Nov 2012 |
| alisertib EU/3/12/1064 3 clinical trials → |
Treatment of ovarian cancer | Withdrawn | 8 Nov 2012 |
| panobinostat EU/3/12/1063 |
Treatment of multiple myeloma | Expired | 8 Nov 2012 |
| chimeric monoclonal antibody against GD2 EU/3/12/1062 · now authorised as Qarziba (previously Dinutuximab beta EUSA and Dinutuximab beta Apeiron) |
Treatment of neuroblastoma | Active (positive) | 8 Nov 2012 |
| tralokinumab EU/3/12/1061 2 clinical trials → |
Treatment of idiopathic pulmonary fibrosis | Withdrawn | 8 Nov 2012 |
| Ixazomib EU/3/12/1060 6 clinical trials → |
Treatment of systemic light chain amyloidosis | Withdrawn | 8 Nov 2012 |
| milciclib maleate EU/3/12/1059 |
Treatment of malignant thymoma | Withdrawn | 8 Nov 2012 |
| IL-12-secreting dendritic cells, loaded with autologous tumour lysate EU/3/12/1058 |
Treatment of glioma | Active (positive) | 8 Nov 2012 |
| naloxone hydrochloride dihydrate EU/3/12/1057 2 clinical trials → |
Treatment of cutaneous T-cell lymphoma | Active (positive) | 8 Nov 2012 |
| liposomal daunorubicin EU/3/12/1056 |
Treatment of acute myeloid leukaemia | Withdrawn | 10 Oct 2012 |
| belinostat EU/3/12/1055 2 clinical trials → |
Treatment of peripheral T-cell lymphoma (nodal, other extranodal and leukaemic/disseminated) | Active (positive) | 10 Oct 2012 |
| obinutuzumab EU/3/12/1054 88 clinical trials → |
Treatment of chronic lymphocytic leukaemia | Active (positive) | 10 Oct 2012 |
| lurbinectedin EU/3/12/1053 17 clinical trials → |
Treatment of ovarian cancer | Active (positive) | 10 Oct 2012 |
| Humanised monoclonal IgG4 antibody against tissue-factor-pathway inhibitor (concizumab) EU/3/12/1052 |
Treatment of haemophilia A | Withdrawn | 10 Oct 2012 |
| recombinant human lecithin cholesterol acyltransferase EU/3/12/1051 |
Treatment of lecithin-cholesterol-acyltransferase deficiency | Withdrawn | 10 Oct 2012 |
| [2-Cyano-3-cyclopropyl-3-hydroxy-N-(3-methyl-4-trifluoromethylphenyl)prop-2-enamide] EU/3/12/1050 |
Treatment of traumatic spinal cord injury | Active (positive) | 10 Oct 2012 |
| rucaparib EU/3/12/1049 4 clinical trials → |
Treatment of ovarian cancer | Withdrawn | 10 Oct 2012 |
| mixture of two allogeneic human pancreatic cancer cell lines stably transduced with a retroviral vector encoding the murine alpha-(1,3)-galactosyltransferase g… EU/3/12/1048 |
Treatment of pancreatic cancer | Withdrawn | 10 Oct 2012 |
| Asp-Arg-Val-Tyr-Ile-His-Pro EU/3/12/1047 |
Treatment of acute lung Injury | Withdrawn | 10 Oct 2012 |
| Mavoglurant EU/3/12/1046 |
Treatment of fragile X syndrome | Withdrawn | 10 Oct 2012 |
| alpha-1 proteinase inhibitor EU/3/12/1045 |
Treatment of cystic fibrosis | Active (positive) | 10 Oct 2012 |
| trans-4-[4-[5-[[6-(trifluoromethyl)-3-pyridinyl]amino]-2-pyridinyl]phenyl] cyclohexane acetic acid sodium salt EU/3/12/1036 |
Treatment of familial chylomicronaemia syndrome (type-I hyperlipoproteinaemia) | Withdrawn | 14 Sep 2012 |
| folic acid EU/3/12/1044 4 clinical trials → |
Diagnosis of positive folate-receptor status in ovarian cancer | Withdrawn | 10 Sep 2012 |
| N-[4-[[(2-amino-3,4-dihydro-4-oxo-6-pteridinyl)methyl]amino]benzoyl]-D-gamma-glutamyl-(2S)-2-amino-beta-alanyl-L-alpha-aspartyl-L-cysteine (etarfolatide) EU/3/12/1043 · now authorised as Folcepri |
Diagnosis of positive folate-receptor status in ovarian cancer | Withdrawn | 10 Sep 2012 |
| Covalently closed DNA plasmids coding for cytomegalovirus phosphoprotein 65 and glycoprotein B genes EU/3/12/1042 |
Prevention of cytomegalovirus disease in patients with impaired cell-mediated immunity | Withdrawn | 9 Aug 2012 |
EU/3/12/1041 |
Treatment of primary biliary cirrhosis | Active (positive) | 9 Aug 2012 |
EU/3/12/1040 · now authorised as Bylvay |
Treatment of Alagille syndrome | Withdrawn | 9 Aug 2012 |
| recombinant anti-CD3-bi-single-chain-Fv-diphtheria toxin fusion protein EU/3/12/1039 |
Treatment of cutaneous T-cell lymphoma | Active (positive) | 9 Aug 2012 |
| recombinant anti-CD3-bi-single-chain-Fv-diphtheria toxin fusion protein EU/3/12/1038 |
Treatment of peripheral T-cell lymphoma (nodal, other extranodal and leukaemic/disseminated) | Active (positive) | 9 Aug 2012 |
| Elotuzumab EU/3/12/1037 11 clinical trials → |
Treatment of multiple myeloma | Withdrawn | 9 Aug 2012 |
| recombinant human monoclonal antibody against activin receptor type IIB EU/3/12/1035 |
Treatment of inclusion body myositis | Withdrawn | 9 Aug 2012 |
| Humanised monoclonal antibody against P-selectin (crizanlizumab) EU/3/12/1034 |
Treatment of sickle cell disease | Withdrawn | 9 Aug 2012 |
| N-butyldeoxygalactonojirimycin EU/3/12/1033 |
Treatment of Fabry disease | Active (positive) | 9 Aug 2012 |
| vatreptacog alfa (activated) EU/3/12/1032 |
Treatment of haemophilia B | Withdrawn | 9 Aug 2012 |
| ketoconazole EU/3/12/1031 |
Treatment of Cushing's syndrome | Active (positive) | 9 Aug 2012 |
| vatreptacog alfa (activated) EU/3/12/1030 |
Treatment of haemophilia A | Withdrawn | 9 Aug 2012 |
| Humanised monoclonal antibody against epidermal-growth-factor receptor EU/3/12/1029 |
Treatment of glioma | Withdrawn | 9 Aug 2012 |
EU/3/12/1028 · now authorised as Bylvay |
Treatment of progressive familial intrahepatic cholestasis | Active (positive) | 17 Jul 2012 |
| human apotransferrin EU/3/12/1027 |
Treatment of congenital hypotransferrinaemia | Active (positive) | 17 Jul 2012 |
| Hexasodium phytate EU/3/12/1026 |
Treatment of calciphylaxis | Active (positive) | 17 Jul 2012 |
| Metreleptin EU/3/12/1025 · now authorised as Myalepta 5 clinical trials → |
Treatment of Berardinelli-Seip syndrome | Active (positive) | 17 Jul 2012 |
| Metreleptin EU/3/12/1024 · now authorised as Myalepta 5 clinical trials → |
Treatment of Lawrence syndrome | Active (positive) | 17 Jul 2012 |
| Metreleptin EU/3/12/1023 · now authorised as Myalepta 5 clinical trials → |
Treatment of Barraquer-Simons syndrome | Active (positive) | 17 Jul 2012 |
| Metreleptin EU/3/12/1022 · now authorised as Myalepta 5 clinical trials → |
Treatment of familial partial lipodystrophy | Active (positive) | 17 Jul 2012 |
| 1-[(2-Chloro-4-methoxyphenoxy)methyl]-4-[(2,6-dichlorophenoxy)methyl]benzene EU/3/12/1021 |
Prevention of poliomyelitis in patients with immunodeficiencies deemed at risk | Active (positive) | 17 Jul 2012 |
| recombinant human pentraxin-2 EU/3/12/1020 |
Treatment of idiopathic pulmonary fibrosis | Active (positive) | 17 Jul 2012 |
| ramucirumab EU/3/12/1019 30 clinical trials → |
Treatment of hepatocellular carcinoma | Withdrawn | 4 Jul 2012 |
| recombinant adeno-associated viral vector containing human acid alfa-glucosidase-gene EU/3/12/1018 |
Treatment of glycogen storage disease type II (Pompe's disease) | Active (positive) | 4 Jul 2012 |
| talarozole EU/3/12/1017 |
Treatment of recessive X-linked ichthyosis | Withdrawn | 4 Jul 2012 |
| 16-base single-stranded peptide nucleic acid oligonucleotide linked to 7-amino acid peptide EU/3/12/1016 |
Treatment of neuroblastoma | Active (positive) | 4 Jul 2012 |
| Eculizumab EU/3/12/1015 12 clinical trials → |
Treatment of infection-associated haemolytic uraemic syndrome | Withdrawn | 4 Jul 2012 |
| talarozole EU/3/12/1014 |
Treatment of keratinopathic ichthyosis | Withdrawn | 4 Jul 2012 |
| recombinant human interleukin-7 EU/3/12/1013 |
Treatment of progressive multifocal leukoencephalopathy | Active (positive) | 4 Jul 2012 |
| 2S, 4R ketoconazole EU/3/12/1012 |
Treatment of Cushing's syndrome | Active (positive) | 4 Jul 2012 |
| Levoglutamide EU/3/12/1011 · now authorised as Xyndari |
Treatment of sickle cell disease | Active (positive) | 4 Jul 2012 |
| ataluren EU/3/12/1010 |
Treatment of Becker muscular dystrophy | Withdrawn | 4 Jul 2012 |
| givinostat EU/3/12/1009 · now authorised as Duvyzat |
Treatment of Duchenne muscular dystrophy | Active (positive) | 4 Jul 2012 |
| human erythrocytes encapsulating inositol hexaphosphate EU/3/12/1008 |
Treatment of sickle cell disease | Active (positive) | 4 Jul 2012 |
| talarozole EU/3/12/1005 |
Treatment of autosomal recessive congenital ichthyosis | Withdrawn | 4 Jul 2012 |
| ramucirumab EU/3/12/1004 30 clinical trials → |
Treatment of gastric cancer | Withdrawn | 4 Jul 2012 |
| Letermovir EU/3/12/999 9 clinical trials → |
Treatment of cytomegalovirus disease in patients with impaired cell-mediated immunity | Withdrawn | 6 Jun 2012 |
| autologous CD34+ cells transfected with lentiviral vector containing the Wiskott-Aldrich syndrome protein gene (etuvetidigene autotemcel) EU/3/12/998 · now authorised as Waskyra |
Treatment of Wiskott-Aldrich syndrome | Active (positive) | 6 Jun 2012 |
| N-Hydroxy-4-(3-methyl-2-(S)-phenyl-butyrylamino) benzamide EU/3/12/997 |
Treatment of schwannoma | Withdrawn | 6 Jun 2012 |
| N-Hydroxy-4-(3-methyl-2-(S)-phenyl-butyrylamino) benzamide EU/3/12/996 |
Treatment of meningioma | Withdrawn | 6 Jun 2012 |
| autologous haematopoietic stem cells transduced with lentiviral vector Lenti-D encoding the human ABCD1 cDNA (elivaldogene autotemcel) EU/3/12/1003 |
Treatment of adrenoleukodystrophy | Withdrawn | 6 Jun 2012 |
| adenovirus-associated vector containing human Fas-c gene (ofranergene obadenovec) EU/3/12/1002 |
Treatment of glioma | Withdrawn | 6 Jun 2012 |
| 1-(4-{4-Amino-7-[1-(2-hydroxyethyl)-1H-pyrazol-4-yl]thieno[3,2-c]pyridin-3-yl}phenyl)-3-(3-fluorophenyl)urea EU/3/12/1001 |
Treatment of ovarian cancer | Withdrawn | 6 Jun 2012 |
| polyinosine-polycytidylic acid coupled with the polycationic polyethyleneimine EU/3/12/1000 |
Treatment of pancreatic cancer | Withdrawn | 6 Jun 2012 |
| yttrium (90Y)-DTPA-radiolabelled chimeric monoclonal antibody against frizzled homologue 10 EU/3/12/994 |
Treatment of soft tissue sarcoma | Active (positive) | 25 May 2012 |
| allogeneic human dendritic cells derived from a CD34+ progenitor cell line EU/3/12/969 |
Treatment of acute myeloid leukaemia | Active (positive) | 22 May 2012 |
| chlormethine EU/3/12/963 · now authorised as Ledaga |
Treatment of cutaneous T-cell lymphoma | Active (positive) | 22 May 2012 |
| chimeric monoclonal antibody against kappa myeloma antigen EU/3/12/962 |
Treatment of multiple myeloma | Withdrawn | 22 May 2012 |
| pegylated recombinant factor VIII (turoctocog alfa pegol) EU/3/12/995 |
Treatment of haemophilia A | Withdrawn | 26 Apr 2012 |
| N-Hydroxy-4-(3-methyl-2-(S)-phenyl-butyrylamino) benzamide EU/3/12/993 |
Treatment of neurofibromatosis type 2 | Active (positive) | 26 Apr 2012 |
| exon 53 specific phosphorothioate oligonucleotide EU/3/12/992 |
Treatment of Duchenne muscular dystrophy | Active (positive) | 26 Apr 2012 |
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.