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 |
|---|---|---|---|
| Mexiletine hydrochloride EU/3/13/1189 5 clinical trials → |
Treatment of myotonic disorders | Active (positive) | 7 Oct 2013 |
| recombinant fusion protein linking coagulation factor VIIa with albumin EU/3/13/1188 |
Treatment of congenital factor VII deficiency | Active (positive) | 7 Oct 2013 |
| heterologous human adult liver-derived progenitor cells EU/3/13/1166 |
Treatment of citrullinaemia type 2 | Active (positive) | 17 Aug 2013 |
| recombinant human monoclonal antibody against hepatitis-B virus (active ingredient lenvervimab) EU/3/13/1156 |
Prevention of hepatitis-B re-infection following liver transplantation | Active (positive) | 15 Aug 2013 |
| Idelalisib EU/3/13/1187 6 clinical trials → |
Treatment of splenic marginal-zone lymphoma | Withdrawn | 5 Aug 2013 |
| Idelalisib EU/3/13/1186 6 clinical trials → |
Treatment of extranodal marginal-zone lymphoma of mucosa-associated lymphoid tissue (MALT lymphoma) | Withdrawn | 5 Aug 2013 |
| Eculizumab EU/3/13/1185 · now authorised as Soliris 12 clinical trials → |
Treatment of neuromyelitis optica spectrum disorders | Active (positive) | 5 Aug 2013 |
EU/3/13/1184 |
Treatment of sickle cell disease | Withdrawn | 5 Aug 2013 |
| sacrosidase EU/3/13/1183 |
Treatment of congenital sucrase-isomaltase deficiency | Active (positive) | 5 Aug 2013 |
| cladribine EU/3/13/1182 7 clinical trials → |
Treatment of mastocytosis | Active (positive) | 5 Aug 2013 |
| Budesonide EU/3/13/1181 · now authorised as Jorveza 11 clinical trials → |
Treatment of eosinophilic oesophagitis | Active (positive) | 5 Aug 2013 |
| apremilast EU/3/13/1180 10 clinical trials → |
Treatment of Behรงet's Disease | Withdrawn | 5 Aug 2013 |
| recombinant human growth hormone modified by fusion with two hydrophilic polypeptide chains (somavaratan) EU/3/13/1179 |
Treatment of growth hormone deficiency | Withdrawn | 5 Aug 2013 |
| pegylated recombinant anti-Pseudomonas aeruginosa antibody EU/3/13/1178 |
Treatment of Pseudomonas aeruginosa lung infection in cystic fibrosis | Withdrawn | 5 Aug 2013 |
| chimaeric monoclonal antibody against claudin-18 splice variant 2 EU/3/13/1177 |
Treatment of pancreatic cancer | Active (positive) | 5 Aug 2013 |
| human allogeneic bone-marrow-derived osteoblastic-like cells EU/3/13/1176 |
Treatment of non-traumatic osteonecrosis | Active (positive) | 5 Aug 2013 |
| tolvaptan EU/3/13/1175 2 clinical trials → |
Treatment of autosomal dominant polycystic kidney disease | Withdrawn | 5 Aug 2013 |
| trans-N1-((1R,2S)-2-phenylcyclopropyl)cyclohexane-1,4-diamine bis-hydrochloride EU/3/13/1174 |
Treatment of acute myeloid leukaemia | Active (positive) | 5 Aug 2013 |
| Idelalisib EU/3/13/1173 6 clinical trials → |
Treatment of chronic lymphocytic leukaemia / small lymphocytic lymphoma | Withdrawn | 5 Aug 2013 |
| Idelalisib EU/3/13/1172 6 clinical trials → |
Treatment of nodal marginal-zone lymphoma | Withdrawn | 5 Aug 2013 |
| octreotide acetate EU/3/13/1170 3 clinical trials → |
Treatment of acromegaly | Withdrawn | 5 Aug 2013 |
| cisplatin EU/3/13/1169 473 clinical trials → |
Treatment of osteosarcoma | Active (positive) | 5 Aug 2013 |
| ex-vivo-expanded autologous human corneal epithelium containing stem cells EU/3/13/1168 |
Treatment of limbal stem cell deficiency | Active (positive) | 17 Jul 2013 |
| heterologous human adult liver-derived progenitor cells EU/3/13/1167 |
Treatment of ornithine-translocase deficiency | Active (positive) | 17 Jul 2013 |
| heterologous human adult liver-derived progenitor cells EU/3/13/1165 |
Treatment of N-acetylglutamate synthetase (NAGS) deficiency | Active (positive) | 17 Jul 2013 |
| heterologous human adult liver-derived progenitor cells EU/3/13/1164 |
Treatment of hyperargininaemia | Active (positive) | 17 Jul 2013 |
| heterologous human adult liver-derived progenitor cells EU/3/13/1163 |
Treatment of argininosuccinic aciduria | Active (positive) | 17 Jul 2013 |
| heterologous human adult liver-derived progenitor cells EU/3/13/1162 |
Treatment of citrullinaemia type 1 | Active (positive) | 17 Jul 2013 |
| heterologous human adult liver-derived progenitor cells EU/3/13/1161 |
Treatment of carbamoyl-phosphate synthase-1 deficiency | Active (positive) | 17 Jul 2013 |
| Idelalisib EU/3/13/1160 6 clinical trials → |
Treatment of lymphoplasmacytic lymphoma | Withdrawn | 17 Jul 2013 |
| Idelalisib EU/3/13/1159 6 clinical trials → |
Treatment of follicular lymphoma | Withdrawn | 17 Jul 2013 |
| Dexamethasone sodium phosphate encapsulated in human autologous erythrocytes EU/3/13/1158 |
Treatment of ataxia telangiectasia | Withdrawn | 17 Jul 2013 |
| (S)-3-(1-(9H-purin-6-ylamino)ethyl)-8-chloro-2-phenylisoquinolin-1(2H)-one EU/3/13/1157 |
Treatment of follicular lymphoma | Active (positive) | 17 Jul 2013 |
| allogeneic motor neuron progenitor cells derived from human embryonic stem cells EU/3/13/1155 |
Treatment of amyotrophic lateral sclerosis | Active (positive) | 17 Jul 2013 |
| Fosbretabulin tromethamine EU/3/13/1154 |
Treatment of ovarian cancer | Withdrawn | 17 Jul 2013 |
| daratumumab EU/3/13/1153 98 clinical trials → |
Treatment of plasma-cell myeloma | Expired | 17 Jul 2013 |
| (1-methyl-2-nitro-1H-imidazole-5-yl)methyl N,N'-bis(2-bromoethyl)diamidophosphate EU/3/13/1152 |
Treatment of pancreatic cancer | Withdrawn | 17 Jul 2013 |
| belinostat EU/3/13/1151 2 clinical trials → |
Treatment of malignant thymoma | Active (positive) | 17 Jul 2013 |
| human haemin EU/3/13/1149 |
Prevention of ischaemia/reperfusion injury associated with solid organ transplantation | Active (positive) | 17 Jul 2013 |
| autologous bone-marrow-derived mesenchymal stromal cells secreting neurotrophic factors EU/3/13/1148 |
Treatment of amyotrophic lateral sclerosis | Active (positive) | 17 Jul 2013 |
| Granulocyte-macrophage colony-stimulating factor EU/3/13/1147 |
Treatment of pulmonary alveolar proteinosis | Active (positive) | 17 Jul 2013 |
| copper meso-5,15-bis[3-[(1,2-dicarba-closo-dodecaboranyl)methoxy]phenyl]-meso-10,20-dinitroporphyrin EU/3/13/1138 |
Treatment of squamous-cell carcinoma of the head and neck in patients undergoing radiotherapy | Withdrawn | 27 Jun 2013 |
| unoprostone isopropyl EU/3/13/1146 |
Treatment of retinitis pigmentosa | Withdrawn | 19 Jun 2013 |
| genetically modified serotype 5/3 adenovirus coding for granulocyte-macrophage colony-stimulating factor EU/3/13/1145 |
Treatment of soft tissue sarcoma | Withdrawn | 19 Jun 2013 |
| recombinant human alpha-N-acetylglucosaminidase EU/3/13/1144 |
Treatment of mucopolysaccharidosis type IIIB (Sanfilippo B syndrome) | Withdrawn | 19 Jun 2013 |
| immortalised human C3A hepatoblastoma cells EU/3/13/1143 |
Treatment of acute liver failure | Withdrawn | 19 Jun 2013 |
| adenovirus associated viral vector serotype 5 containing the human pde6ฮฒ gene EU/3/13/1142 |
Treatment of retinitis pigmentosa | Active (positive) | 19 Jun 2013 |
| synthetic double-stranded siRNA oligonucleotide directed against the keratin 6a N171K mutation EU/3/13/1141 |
Treatment of pachyonychia congenita | Active (positive) | 19 Jun 2013 |
| expanded human allogeneic neural retinal progenitor cells extracted from neural retina EU/3/13/1140 |
Treatment of retinitis pigmentosa | Active (positive) | 19 Jun 2013 |
| sodium chlorite EU/3/13/1139 |
Treatment of amyotrophic lateral sclerosis | Active (positive) | 19 Jun 2013 |
| 4,6,4'-trimethylangelicin EU/3/13/1137 |
Treatment of cystic fibrosis | Active (positive) | 19 Jun 2013 |
| 5-[1-(2,6-Dichlorobenzyl)piperidin-4-ylmethoxy]quinazoline-2,4-diamine dihydrochloride EU/3/13/1136 |
Treatment of 5q spinal muscular atrophy | Active (positive) | 7 Jun 2013 |
| recombinant human nerve growth factor EU/3/13/1135 |
Treatment of retinitis pigmentosa | Active (positive) | 7 Jun 2013 |
| autologous CD34+ cells transduced with a lentiviral vector containing the human ADA gene EU/3/13/1134 |
Treatment of adenosine-deaminase-deficient severe combined immunodeficiency | Active (positive) | 7 Jun 2013 |
| Maribavir EU/3/13/1133 · now authorised as Livtencity 7 clinical trials → |
Treatment of cytomegalovirus disease in patients with impaired cell-mediated immunity | Active (positive) | 7 Jun 2013 |
| N-methyl-4-({4-[({3-methyl(methylsulfonyl)aminopyrazin-2-yl}methyl)amino]-5-(trifluoromethyl)pyrimidin-2-yl}amino)benzamide hydrochloride (defactinib) EU/3/13/1132 |
Treatment of malignant mesothelioma | Withdrawn | 7 Jun 2013 |
| recombinant human CXCL8 mutant EU/3/13/1131 |
Treatment of cystic fibrosis | Active (positive) | 7 Jun 2013 |
| recombinant human transglutaminase 1 encapsulated into liposomes EU/3/13/1130 |
Treatment of transglutaminase-1-deficient autosomal recessive congenital ichthyosis | Active (positive) | 7 Jun 2013 |
| allogeneic bone-marrow-derived mesenchymal cells expanded ex vivo in synthetic media EU/3/13/1129 |
Treatment of graft-versus-host disease | Active (positive) | 7 Jun 2013 |
| N-[2,6-bis(1-methylethyl)phenyl]-N'-[[1-[4-(dimethylamino) phenyl]cyclopentyl]methyl]urea, hydrochloride salt EU/3/13/1128 |
Treatment of adrenocortical carcinoma | Active (positive) | 7 Jun 2013 |
| Inotuzumab ozogamicin EU/3/13/1127 · now authorised as Besponsa 13 clinical trials → |
Treatment of B-cell acute lymphoblastic leukaemia | Active (positive) | 7 Jun 2013 |
| Mexiletine hydrochloride EU/3/13/1126 5 clinical trials → |
Treatment of non-dystrophic myotonia | Active (positive) | 7 Jun 2013 |
| (S)-3-(1-(9H-purin-6-ylamino)ethyl)-8-chloro-2-phenylisoquinolin-1(2H)-one (duvelisib) EU/3/13/1125 |
Treatment of chronic lymphocytic leukaemia / small lymphocytic lymphoma | Active (positive) | 26 Apr 2013 |
| 2-Hydroxypropyl-ฮฒ-cyclodextrin EU/3/13/1124 |
Treatment of Niemann-Pick's disease, type C | Active (positive) | 26 Apr 2013 |
| nintedanib EU/3/13/1123 7 clinical trials → |
Treatment of idiopathic pulmonary fibrosis | Withdrawn | 26 Apr 2013 |
| R,S-O-(3-piperidino-2-hydroxy-1-propyl)-nicotinic acid amidoxime dihydrochloride EU/3/13/1122 |
Treatment of Duchenne muscular dystrophy | Active (positive) | 26 Apr 2013 |
| lenvatinib EU/3/13/1121 124 clinical trials → |
Treatment of papillary thyroid cancer | Withdrawn | 26 Apr 2013 |
| 4-[2-(6-Methylpyridin-2-yl)-5,6-dihydro-4H-pyrrolo[1,2-b]pyrazol-3-yl]-quinoline-6-carboxamide monohydrate EU/3/13/1120 |
Treatment of glioma | Withdrawn | 26 Apr 2013 |
| lenvatinib EU/3/13/1119 124 clinical trials → |
Treatment of follicular thyroid cancer | Withdrawn | 26 Apr 2013 |
| zoledronic acid - 11 clinical trials → |
Treatment of complex regional pain syndrome | Negative | 17 Apr 2013 |
| Cyclo[L-alanyl-L-seryl-L-isoleucyl-L-prolyl-L-prolyl-L-glutaminyl-L-lysyl-L-tyrosyl-D-prolyl-L-prolyl-(2S)-2-aminodecanoyl-L-alpha-glutamyl-L-threonyl]acetate โฆ EU/3/13/1114 |
Treatment of congenital alpha-1 antitrypsin deficiency | Active (positive) | 20 Mar 2013 |
| recombinant human tripeptidyl-peptidase 1 EU/3/13/1118 · now authorised as Brineura |
Treatment of neuronal ceroid lipofuscinosis type 2 | Active (positive) | 12 Mar 2013 |
| ramiprilat EU/3/13/1117 |
Treatment of Stargardt's disease | Withdrawn | 12 Mar 2013 |
| Mepolizumab EU/3/13/1116 16 clinical trials → |
Treatment of Churg-Strauss syndrome | Withdrawn | 12 Mar 2013 |
| 1-[(3R)-3-[4-amino-3-(4-phenoxyphenyl)-1H-pyrazolo[3,4 d]pyrimidin-1-yl]-1-piperidinyl]-2-propen-1-one (ibrutinib) EU/3/13/1115 |
Treatment of mantle cell lymphoma | Withdrawn | 12 Mar 2013 |
| murine IgM monoclonal antibody binding to alpha beta T-cell receptor EU/3/13/1113 |
Prevention of graft rejection following solid organ transplantation | Withdrawn | 12 Mar 2013 |
| Poloxamer 188 EU/3/13/1112 |
Treatment of sickle cell disease | Withdrawn | 12 Mar 2013 |
| Gevokizumab EU/3/13/1111 |
Treatment of chronic non-infectious uveitis | Withdrawn | 12 Mar 2013 |
| recombinant human heat-shock protein 70 EU/3/13/1110 |
Treatment of Niemann-Pick's disease, type C | Withdrawn | 12 Mar 2013 |
| 4-[2-(6-Methylpyridin-2-yl)-5,6-dihydro-4H-pyrrolo[1,2-b]pyrazol-3-yl]-quinoline-6-carboxamide monohydrate EU/3/13/1109 |
Treatment of hepatocellular carcinoma | Withdrawn | 12 Mar 2013 |
| 2-[4-Methoxy-3-(2-m-tolyl-ethoxy)-benzoylamino]-indan-2-carboxylic acid EU/3/13/1108 |
Treatment of systemic sclerosis | Withdrawn | 12 Mar 2013 |
| recombinant adeno-associated viral vector containing the human retinoschisin gene EU/3/13/1107 |
Treatment of X-linked juvenile retinoschisis | Active (positive) | 12 Mar 2013 |
| L-asparaginase encapsulated in erythrocytes EU/3/13/1106 |
Treatment of acute myeloid leukaemia | Active (positive) | 8 Feb 2013 |
| Humanised monoclonal antibody against myostatin (domagrozumab) EU/3/13/1105 |
Treatment of Duchenne muscular dystrophy | Withdrawn | 8 Feb 2013 |
| terguride EU/3/13/1104 |
Treatment of systemic sclerosis | Withdrawn | 8 Feb 2013 |
| treprostinil sodium EU/3/13/1103 · now authorised as Trepulmix 4 clinical trials → |
Treatment of chronic thromboembolic pulmonary hypertension | Active (positive) | 8 Feb 2013 |
| Cyclo-Cys-Gly-Gln-Arg-Glu-Thr-Pro-Glu-Gly-Ala-Glu-Ala-Lys-Pro-Trp-Tyr-Cys EU/3/13/1102 |
Treatment of high-altitude pulmonary oedema | Active (positive) | 8 Feb 2013 |
| progesterone EU/3/13/1101 9 clinical trials → |
Treatment of moderate and severe traumatic brain injury | Withdrawn | 8 Feb 2013 |
| Humanised IgG1 kappa antibody against serum amyloid A and AL amyloid EU/3/13/1100 |
Treatment of amyloid light-chain amyloidosis | Active (positive) | 8 Feb 2013 |
| recombinant adeno-associated viral vector containing the human CNGB3 gene EU/3/13/1099 |
Treatment of achromatopsia caused by mutations in the CNGB3 gene | Active (positive) | 8 Feb 2013 |
| Encapsulated human retinal pigment epithelial cell line transfected with plasmid vector expressing human ciliary neurotrophic factor EU/3/12/1098 |
Treatment of retinitis pigmentosa | Active (positive) | 24 Jan 2013 |
| lenalidomide EU/3/12/1097 167 clinical trials → |
Treatment of follicular lymphoma | Withdrawn | 24 Jan 2013 |
| terguride EU/3/12/1096 |
Treatment of systemic sclerosis | Withdrawn | 24 Jan 2013 |
| adeno-associated viral vector serotype 9 containing the human N-acetylglucosaminidase-alpha gene EU/3/12/1095 |
Treatment of mucopolysaccharidosis type IIIB (Sanfilippo B syndrome) | Withdrawn | 24 Jan 2013 |
| modified recombinant human C-type natriuretic peptide (Vosoritide) EU/3/12/1094 · now authorised as Voxzogo |
Treatment of achondroplasia | Active (positive) | 24 Jan 2013 |
| 1,2:5,6-Dianhydrogalactitol EU/3/12/1093 |
Treatment of glioma | Active (positive) | 24 Jan 2013 |
| chimeric monoclonal antibody against claudin 6 EU/3/12/1092 |
Treatment of ovarian cancer | Withdrawn | 24 Jan 2013 |
| autologous CD34+ haematopoietic stem cells transduced with lentiviral vector encoding the human betaA-T87Q-globin gene (betibeglogene autotemcel) EU/3/12/1091 |
Treatment of beta thalassaemia intermedia and major | Withdrawn | 24 Jan 2013 |
| recombinant human monoclonal antibody of the IgG1 kappa class against prostate stem-cell antigen EU/3/12/1090 |
Treatment of pancreatic cancer | Active (positive) | 24 Jan 2013 |
| choline tetrathiomolybdate EU/3/12/1089 |
Treatment of Wilson's disease | Active (positive) | 24 Jan 2013 |
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.