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 |
|---|---|---|---|
| beloranib EU/3/14/1287 |
Treatment of Prader-Willi syndrome | Withdrawn | 4 Jul 2014 |
| Humanised Fc engineered monoclonal antibody against CD19 (tafasitamab) EU/3/14/1286 |
Treatment of chronic lymphocytic leukaemia / small lymphocytic lymphoma | Active (positive) | 4 Jul 2014 |
| afamelanotide EU/3/14/1285 |
Treatment of familial benign chronic pemphigus (Hailey-Hailey disease) | Active (positive) | 4 Jul 2014 |
| Isavuconazonium sulfate EU/3/14/1284 · now authorised as Cresemba |
Treatment of invasive aspergillosis | Active (positive) | 4 Jul 2014 |
| mixture of two adeno-associated viral vectors of serotye 8 containing the 5'-half sequence of human ABCA4 gene and the 3'-half sequence of human ABCA4 gene EU/3/14/1283 |
Treatment of Stargardt's disease | Active (positive) | 4 Jul 2014 |
| mixture of two adeno-associated viral vectors serotype 8 containing the 5'-half sequence of human MYO7A gene and the 3'-half sequence of human MYO7A gene EU/3/14/1282 |
Treatment of Usher syndrome | Active (positive) | 4 Jul 2014 |
| 1-(2,2-difluoro-1,3-benzodioxol-5-yl)-N-{1-[(2R)-2,3-dihydroxypropyl]-6-fluoro-2-(1-hydroxy-2-methylpropan-2-yl)-1H-indol-5-yl}cyclopropanecarboxamide (tezacafโฆ EU/3/14/1281 |
Treatment of cystic fibrosis | Active (positive) | 4 Jul 2014 |
| autologous CD34+ cells transduced with a lentiviral vector containing the human SGSH gene EU/3/14/1280 |
Treatment of mucopolysaccharidosis, type IIIA (Sanfilippo A syndrome) | Active (positive) | 10 Jun 2014 |
| aganirsen EU/3/14/1275 |
Treatment of central retinal vein occlusion | Active (positive) | 10 Jun 2014 |
| 4-(4-Methoxy-phenylamino)-6-methylcarbamyl-quinoline-3-carboxylic acid EU/3/14/1279 |
Prevention of scarring post glaucoma filtration surgery | Active (positive) | 4 Jun 2014 |
| adeno-associated viral vector serotype 2 containing the human CHM gene encoding human Rab escort protein 1 EU/3/14/1278 |
Treatment of choroideraemia | Active (positive) | 4 Jun 2014 |
| Plasmid DNA encoding the human cystic fibrosis transmembrane conductance regulator gene complexed with a non-viral, cationic lipid based gene transfer agent EU/3/14/1277 |
Treatment of cystic fibrosis | Active (positive) | 4 Jun 2014 |
| Isavuconazonium sulfate EU/3/14/1276 · now authorised as Cresemba |
Treatment of mucormycosis | Active (positive) | 4 Jun 2014 |
| Paclitaxel-succinate-Arg-Arg-Leu-Ser-Tyr-Ser-Arg-Arg-Arg-Phe EU/3/14/1274 |
Treatment of glioma | Active (positive) | 4 Jun 2014 |
| autologous dendritic cells pulsed with RNA from glioma stem cells EU/3/14/1273 |
Treatment of glioma | Active (positive) | 4 Jun 2014 |
| recombinant human alpha 1 chain homotrimer of type VII collagen EU/3/14/1272 |
Treatment of epidermolysis bullosa | Active (positive) | 4 Jun 2014 |
| 177Lu-tetraxetan-tetulomab EU/3/14/1271 |
Treatment of follicular lymphoma | Active (positive) | 4 Jun 2014 |
| (5R,5aR,8aR,9S)-9-[[4,6-O-[(R)-Ethylidene]-ฮฒ-D-glucopyranosyl]-oxy]-5-(4-({[(2,2-dimethyl-1,3-dioxolan-4-yl)methoxy]carbonyl}oxy)-3,5-dimethoxyphenyl)-5,8,8a,9โฆ EU/3/14/1270 |
Treatment of biliary tract cancer | Active (positive) | 4 Jun 2014 |
| Lutetium (177Lu) edotreotide EU/3/14/1269 |
Treatment of gastro-entero-pancreatic neuroendocrine tumours | Active (positive) | 4 Jun 2014 |
| Humanised monoclonal antibody against CD38 for the treatment of plasma cell myeloma (isatuximab) EU/3/14/1268 |
Treatment of plasma cell myeloma | Withdrawn | 29 Apr 2014 |
| synthetic double-stranded siRNA oligonucleotide directed against transthyretin mRNA and covalently linked to a ligand containing three N-acetylgalactosamine reโฆ EU/3/14/1267 |
Treatment of ATTR amyloidosis | Withdrawn | 29 Apr 2014 |
| autologous T cells transduced with lentiviral vector containing a chimeric antigen receptor directed against CD19 (tisagenlecleucel) EU/3/14/1266 · now authorised as Kymriah |
Treatment of B-lymphoblastic leukaemia/lymphoma | Active (positive) | 29 Apr 2014 |
| genetically modified serotype 5/3 adenovirus coding for granulocyte macrophage colony-stimulating factor for the treatment of ovarian cancer EU/3/14/1265 |
Treatment of ovarian cancer | Withdrawn | 29 Apr 2014 |
| Ibrutinib EU/3/14/1264 44 clinical trials → |
Treatment of lymphoplasmacytic lymphoma | Withdrawn | 29 Apr 2014 |
| autologous CD34+ haematopoietic stem cells transduced with lentiviral vector encoding the human beta A-T87Q-globin gene EU/3/14/1263 |
The treatment of sickle cell disease | Withdrawn | 29 Apr 2014 |
| recombinant human surfactant protein D EU/3/14/1262 |
Prevention of bronchopulmonary dysplasia | Active (positive) | 11 Apr 2014 |
| Caffeine citrate EU/3/14/1261 · now authorised as Blectifor 11 clinical trials → |
Prevention of bronchopulmonary dysplasia | Active (positive) | 11 Apr 2014 |
| amikacin sulfate EU/3/14/1259 · now authorised as Arikayce liposomal 5 clinical trials → |
Treatment of nontuberculous mycobacterial lung disease | Active (positive) | 8 Apr 2014 |
| N-({Carbamoylmethyl-[3-(2-oxo-pyrrolidin-1-yl)-propyl]-carbamoyl}-methyl)-2-[2-(2-fluoro-phenyl)-ethylamino]-N-isobutyl-acetamide EU/3/14/1248 |
Treatment of optic neuritis | Active (positive) | 31 Mar 2014 |
| 5-chloro-N2-[2-isopropoxy-5-methyl-4-(4-piperidinyl)phenyl]-N4-[2-(isopropylsulfonyl)phenyl]-2,4-pyrimidinediamine - |
Treatment of non-small cell lung cancer (NSCLC) that is anaplastic lymphoma kinase (ALK)-positive | Negative | 28 Mar 2014 |
| (RS)-Bacoflen;naltrexone hydrochloride;D-sorbitol EU/3/14/1260 10 clinical trials → |
Treatment of Charcot-Marie-Tooth disease type 1A | Active (positive) | 26 Mar 2014 |
| Doxorubicin(6-maleimidocaproyl)hydrazone (aldoxorubicin) EU/3/14/1258 |
Treatment of soft tissue sarcoma | Withdrawn | 26 Mar 2014 |
| autologous CD34+ cells transduced with a lentiviral vector containing the human RAG1 gene EU/3/14/1257 |
Treatment of recombination-activating gene 1 deficient severe combined immunodeficiency | Active (positive) | 26 Mar 2014 |
| adeno-associated viral vector serotype 8 containing the human GUCY2D gene EU/3/14/1256 |
Treatment of Leber's congenital amaurosis | Active (positive) | 26 Mar 2014 |
| volasertib EU/3/14/1255 |
Treatment of acute myeloid leukaemia | Active (positive) | 26 Mar 2014 |
| Eculizumab EU/3/14/1254 12 clinical trials → |
Prevention of graft rejection following solid organ transplantation | Withdrawn | 26 Mar 2014 |
| ex-vivo cultured human mesenchymal stromal cells EU/3/14/1253 |
Prevention of graft rejection following solid organ transplantation | Active (positive) | 26 Mar 2014 |
| cysteamine bitartrate (gastroresistant) (mercaptamine) EU/3/14/1252 |
Treatment of pancreatic cancer | Active (positive) | 26 Mar 2014 |
| recombinant human alpha-glucosidase conjugated with multiple copies of synthetic bismannose-6-phosphate-tetra-mannose glycan (avalglucosidase alfa) EU/3/14/1251 |
Treatment of glycogen storage disease type II (Pompe's disease) | Withdrawn | 26 Mar 2014 |
| phosphorothioate oligonucleotide targeted to transthyretin (inotersen) EU/3/14/1250 · now authorised as Tegsedi |
Treatment of ATTR amyloidosis | Active (positive) | 26 Mar 2014 |
| N-(3-(5-fluoro-2-(4-(2-methoxyethoxy)phenylamino)pyrimidin-4-ylamino)phenyl)acrylamide benzenesulfonic acid salt EU/3/13/1234 |
Treatment of chronic lymphocytic leukaemia / small lymphocytic lymphoma | Withdrawn | 16 Mar 2014 |
| obeticholic acid EU/3/13/1228 |
Treatment of primary sclerosing cholangitis | Withdrawn | 16 Mar 2014 |
| Diacerein EU/3/14/1236 |
Treatment of epidermolysis bullosa | Active (positive) | 20 Feb 2014 |
| mixture of recombinant human IgG1 monoclonal antibodies against human cytomegalovirus envelope glycoproteins EU/3/14/1235 |
Prevention of congenital cytomegalovirus infection following primary cytomegalovirus infection | Withdrawn | 20 Feb 2014 |
| phosphorothioate oligonucleotide targeted to apolipoprotein C-III (volanesorsen) EU/3/14/1249 · now authorised as Waylivra |
Treatment of familial chylomicronaemia syndrome | Active (positive) | 19 Feb 2014 |
| autologous dendritic cells pulsed with tumour antigen-derived synthetic peptides (MAGE-1, HER-2, AIM-2, TRP-2, gp-100, and interleukin-13 receptor alpha) EU/3/14/1247 |
Treatment of glioma | Active (positive) | 19 Feb 2014 |
| satoreotide trizoxetan EU/3/14/1246 |
Diagnosis of gastro-entero-pancreatic neuroendocrine tumours | Active (positive) | 19 Feb 2014 |
| pioglitazone EU/3/14/1245 13 clinical trials → |
Treatment of adrenoleukodystrophy | Active (positive) | 19 Feb 2014 |
| ruxolitinib EU/3/14/1244 70 clinical trials → |
Treatment of polycythaemia vera | Withdrawn | 19 Feb 2014 |
| recombinant human acid ceramidase EU/3/14/1243 |
Treatment of Farber disease | Active (positive) | 19 Feb 2014 |
| 3-Chloro-4-fluorophenyl-[4-fluoro-4-{[(5-methylpyrimidin-2-ylmethyl) amino]methyl}piperidin-1-yl]methanone EU/3/14/1242 |
Treatment of Rett syndrome | Active (positive) | 19 Feb 2014 |
| Asp-Arg-Val-Tyr-Ile-His-Pro (Angiotensin 1-7);talfirastide EU/3/14/1241 |
Treatment of Duchenne muscular dystrophy | Active (positive) | 19 Feb 2014 |
| cysteamine EU/3/14/1240 |
Treatment of cystic fibrosis | Active (positive) | 19 Feb 2014 |
| 11-(4-Dimethylamino-3-hydroxy-6-methyl-tetrahydro-pyran-2-yloxy)-2-ethyl-3,4,10-trihydroxy-3,5,6,8,10,12,14-heptamethyl-1-oxa-6-aza-cyclopentadecane-13,15-dione EU/3/14/1239 |
Treatment of cystic fibrosis | Active (positive) | 19 Feb 2014 |
| Eculizumab EU/3/14/1238 12 clinical trials → |
Prevention of delayed graft function after solid organ transplantation | Withdrawn | 19 Feb 2014 |
| Gallium [Ga-68]-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-Lโฆ EU/3/14/1237 |
Diagnosis of gastro-entero-pancreatic neuroendocrine tumours | Active (positive) | 19 Feb 2014 |
| allogeneic bone-marrow-derived ex-vivo-expanded multipotent adult progenitor cells EU/3/13/1233 |
Prevention of graft-versus-host disease | Active (positive) | 16 Jan 2014 |
| allantoin EU/3/13/1232 |
Treatment of epidermolysis bullosa | Withdrawn | 16 Jan 2014 |
EU/3/13/1231 |
Treatment of acute lymphoblastic leukaemia | Active (positive) | 16 Jan 2014 |
EU/3/13/1230 |
Treatment of acute myeloid leukaemia | Withdrawn | 16 Jan 2014 |
| autologous dendritic cells pulsed with allogeneic tumour cell lysate EU/3/13/1229 |
Treatment of malignant mesothelioma | Active (positive) | 16 Jan 2014 |
| adenovirus-specific T-cells derived from allogeneic donor leukocytes, expanded ex vivo EU/3/13/1227 |
Treatment of adenovirus infection in allogeneic haematopoietic stem cell transplant recipients | Withdrawn | 16 Jan 2014 |
| (6aS)-1,10-dimethoxy-6-methyl-5,6,6a,7-tetrahydro-4H-dibenzo[de,g]quinoline-2,9-diol EU/3/13/1226 |
Treatment of dystrophic myotonia | Active (positive) | 16 Jan 2014 |
| Lonafarnib EU/3/13/1225 |
Treatment of hepatitis delta virus infection | Active (positive) | 16 Jan 2014 |
| sodium nitrite EU/3/13/1224 |
Treatment of aneurysmal subarachnoid haemorrhage | Active (positive) | 16 Jan 2014 |
| Inecalcitol EU/3/13/1223 |
Treatment of chronic lymphocytic leukaemia / small lymphocytic lymphoma | Active (positive) | 16 Jan 2014 |
| amatuximab EU/3/13/1222 |
Treatment of malignant mesothelioma | Withdrawn | 16 Jan 2014 |
| Humanised monoclonal modified IgG4 antibody with bispecific structure targeting factors IX, IXa, X and Xa (emicizumab) EU/3/13/1221 |
Treatment of haemophilia A | Withdrawn | 16 Jan 2014 |
| Poly[2-[(4-{[1-carboxy-2-(hexadecylcarbamoyl)ethyl]sulfanyl}-2,3-bis({2-[((2S)-2-(2-{[(2R)-2-carbamoyl-(2-{[(2S)-1-ethoxy-3-(3-hydroxy-4oxo-1,4-dihydropyridin-โฆ EU/3/13/1220 |
Treatment of dengue | Active (positive) | 16 Jan 2014 |
| recombinant human type I pancreatic elastase for the prevention of arteriovenous access dysfunction in haemodialysis patients (vonapanitase) EU/3/13/1218 |
Prevention of arteriovenous access dysfunction in haemodialysis patients | Withdrawn | 16 Jan 2014 |
| (4R,5R)-1-[[4-[[4-[3,3-dibutyl-7-(dimethylamino)-2,3,4,5- tetrahydro-4-hydroxy-1,1-dioxido-1-benzothiepin-5-yl]phenoxy]methyl]phenyl]methyl]-4-aza-1-azoniabicyโฆ EU/3/13/1217 |
Treatment of primary sclerosing cholangitis | Withdrawn | 16 Jan 2014 |
| (4R,5R)-1-[[4-[[4-[3,3-dibutyl-7-(dimethylamino)-2,3,4,5- tetrahydro-4-hydroxy-1,1-dioxido-1-benzothiepin-5-yl]phenoxy]methyl]phenyl]methyl]-4-aza-1-azoniabicyโฆ EU/3/13/1216 · now authorised as Livmarli |
Treatment of progressive familial intrahepatic cholestasis | Active (positive) | 16 Jan 2014 |
| (4R,5R)-1-[[4-[[4-[3,3-dibutyl-7-(dimethylamino)-2,3,4,5- tetrahydro-4-hydroxy-1,1-dioxido-1-benzothiepin-5-yl]phenoxy]methyl]phenyl]methyl]-4-aza-1-azoniabicyโฆ EU/3/13/1215 |
Treatment of primary biliary cirrhosis | Withdrawn | 16 Jan 2014 |
| Lactobacillus acidophilus and Bifidobacterium bifidum EU/3/13/1213 2 clinical trials → |
Prevention of necrotising enterocolitis | Active (positive) | 16 Jan 2014 |
| Ibrutinib EU/3/13/1212 44 clinical trials → |
Treatment of follicular lymphoma | Withdrawn | 16 Jan 2014 |
| allogeneic and autologous haptenised and irradiated cells and cell lysates derived from glioma EU/3/13/1211 |
Treatment of glioma | Active (positive) | 16 Jan 2014 |
| recombinant human parathyroid hormone EU/3/13/1210 · now authorised as Natpar |
Treatment of hypoparathyroidism | Active (positive) | 16 Jan 2014 |
| nitric oxide EU/3/13/1209 4 clinical trials → |
Treatment of cystic fibrosis | Active (positive) | 16 Jan 2014 |
| fenfluramine hydrochloride EU/3/13/1219 · now authorised as Fintepla |
Treatment of Dravet syndrome | Active (positive) | 18 Dec 2013 |
| (4R,5R)-1-[[4-[[4-[3,3-dibutyl-7-(dimethylamino)-2,3,4,5- tetrahydro-4-hydroxy-1,1-dioxido-1-benzothiepin-5-yl]phenoxy]methyl]phenyl]methyl]-4-aza-1-azoniabicyโฆ EU/3/13/1214 · now authorised as Livmarli |
Treatment of Alagille syndrome | Active (positive) | 18 Dec 2013 |
| soraprazan EU/3/13/1208 |
Treatment of Stargardt's disease | Active (positive) | 13 Nov 2013 |
| trebananib EU/3/13/1207 |
Treatment of ovarian cancer | Active (positive) | 13 Nov 2013 |
| synthetic 12 amino acid peptide designed after subcommissural organ-spondin EU/3/13/1206 |
Treatment of spinal cord injury | Active (positive) | 13 Nov 2013 |
| human monoclonal antibody against human interleukin 13 EU/3/13/1205 |
Treatment of eosinophilic oesophagitis | Withdrawn | 13 Nov 2013 |
| sirolimus EU/3/13/1204 32 clinical trials → |
Prevention of arteriovenous access dysfunction in haemodialysis patients | Active (positive) | 13 Nov 2013 |
| Ibrutinib EU/3/13/1203 44 clinical trials → |
Treatment of diffuse large B-cell lymphoma | Withdrawn | 13 Nov 2013 |
| tivantinib EU/3/13/1202 |
Treatment of hepatocellular carcinoma | Withdrawn | 13 Nov 2013 |
| defibrotide EU/3/13/1201 |
Prevention of graft-versus-host disease | Withdrawn | 13 Nov 2013 |
| sorafenib tosilate EU/3/13/1200 |
Treatment of papillary thyroid cancer | Expired | 13 Nov 2013 |
| sorafenib tosilate EU/3/13/1199 |
Treatment of follicular thyroid cancer | Expired | 13 Nov 2013 |
| recombinant human insulin receptor monoclonal antibody-fused iduronate 2-sulfatase EU/3/13/1198 |
Treatment of mucopolysaccharidosis type II (Hunter's syndrome) | Withdrawn | 13 Nov 2013 |
| autologous ex-vivo-expanded leucocytes treated with 5-aza-2'-deoxycytidine EU/3/13/1197 |
Treatment of glioma | Active (positive) | 13 Nov 2013 |
| autologous regulatory T cells with an immunophenotype of CD4+CD25hiFoxP3+ EU/3/13/1171 |
Prevention of rejection following solid organ transplantation | Active (positive) | 8 Oct 2013 |
| autologous CD34+ cells transfected with lentiviral vector containing the Wiskott-Aldrich syndrome protein gene (etuvetidigene autotemcel) EU/3/13/1196 |
Treatment of Wiskott-Aldrich syndrome | Withdrawn | 7 Oct 2013 |
| antisense oligonucleotide targeting the F508delta mutation of CFTR (eluforsen) EU/3/13/1195 |
Treatment of cystic fibrosis | Withdrawn | 7 Oct 2013 |
| naproxcinod EU/3/13/1194 |
Treatment of Duchenne muscular dystrophy | Withdrawn | 7 Oct 2013 |
| 3,5-diiodothyropropionic acid EU/3/13/1193 |
Treatment of Allan-Herndon-Dudley syndrome | Active (positive) | 7 Oct 2013 |
| zoledronic acid EU/3/13/1192 11 clinical trials → |
Treatment of complex regional pain syndrome | Active (positive) | 7 Oct 2013 |
| L-Pyr-L-Glu-L-Gln-L-Leu-L-Glu-L-Arg-L-Ala-L-Leu-L-Asn-L-Ser-L-Ser (cibinetide) EU/3/13/1191 |
Treatment of sarcoidosis | Active (positive) | 7 Oct 2013 |
| recombinant human monoclonal IgM antibody targeting glucose regulated protein 78 EU/3/13/1190 |
Treatment of plasma cell myeloma | Active (positive) | 7 Oct 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.