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 |
|---|---|---|---|
| Ibuprofen L-Lysinate (salt) - |
Prevention of patent ductus arteriosus in premature neonates of less than 34 week of gestational age | Negative | 7 Jul 2005 |
| Ibuprofen L-Lysinate (salt) - |
Treatment of patent ductus arteriosus in premature neonates of less than 34 week gestational age | Negative | 24 Jun 2005 |
| Humanised antibody fragment (EP-CAM)-truncated Pseudomonas exotoxin A fusion protein EU/3/05/290 |
Treatment of Ep-CAM-YES squamous cell carcinoma of the head and neck | Active (positive) | 20 Jun 2005 |
| pegylated arginine deiminase EU/3/05/289 |
Treatment of hepatocellular carcinoma | Active (positive) | 20 Jun 2005 |
| 4-[3-(Methylsulfonyl)phenyl]-1-propylpiperidine x HC1 (pridopidine hydrochloride) EU/3/05/288 · now authorised as Nurzigma |
Treatment of Huntington’s disease | Active (positive) | 20 Jun 2005 |
| bovine bile extract EU/3/05/287 |
Treatment of pancreatic cancer | Active (positive) | 20 Jun 2005 |
| N-(methyl-diazacyclohexyl-methylbenzamide)-azaphenyl-aminothiopyrrole EU/3/05/286 |
Treatment of multiple myeloma | Active (positive) | 20 Jun 2005 |
| soluble yeast beta-1,3/1,6-glucan EU/3/05/294 |
Prevention of oral mucositis in head and neck cancer patients undergoing radiation therapy | Active (positive) | 16 Jun 2005 |
| nelarabine EU/3/05/293 |
Treatment of acute lymphoblastic leukaemia | Expired | 16 Jun 2005 |
| H-D-Asp-D-Gln-D-Ser-D-Arg-D-Pro-D-Val-D-Gln-D-Pro-D-Phe-D-Leu-D-Asn-D-Leu-D-Thr-D-Thr-D-Pro-D-Arg-D-Lys-D-Pro-D-Arg-D-Pro-D-Pro-D-Arg-D-Arg-D-Arg-D-Gln-D-Arg-D… EU/3/05/292 |
Hearing loss (acute acoustic trauma, sudden deafness and surgery induced acoustic trauma) | Active (positive) | 16 Jun 2005 |
| human monoclonal antibody against HLA-DR EU/3/05/291 |
Treatment of Hodgkin's lymphoma | Withdrawn | 16 Jun 2005 |
| sudismase - |
Treatment of active phase of Peyronie's disease | Negative | 16 Jun 2005 |
| bimosiamose disodium EU/3/05/285 |
Treatment of acute lung Injury | Active (positive) | 27 May 2005 |
| sodium butyrate EU/3/05/284 |
Prevention of radiation proctitis | Active (positive) | 27 May 2005 |
| recombinant megakaryopoiesis-stimulating protein EU/3/05/283 |
Treatment of idiopathic thrombocytopenic purpura | Expired | 27 May 2005 |
| Miltefosine EU/3/05/282 |
Treatment of acanthamoeba keratitis | Active (positive) | 27 May 2005 |
| Interferon gamma EU/3/05/281 |
Treatment of idiopathic pulmonary fibrosis | Withdrawn | 27 May 2005 |
| acadesine EU/3/05/280 |
Treatment of B-cell chronic lymphocytic leukaemia | Active (positive) | 27 May 2005 |
| (E)-(1S,4S,10S,21R)-7-[(Z)-ethylidene]-4,21-diisopropyl-2-oxa-12,13-dithia-5,8,20,23- tetraazabicyclo[8.7.6]tricos-16-ene-3,6,9,19,22-pentone EU/3/05/279 |
Treatment of cutaneous T-cell lymphoma | Active (positive) | 27 May 2005 |
| 3-[5-(2-Fluoro-phenyl)-[1,2,4]oxadiazole-3-yl]-benzoic acid (ataluren) EU/3/05/278 |
Treatment of Duchenne muscular dystrophy | Active (positive) | 27 May 2005 |
| 3-[5-(2-Fluoro-phenyl)-[1,2,4]oxadiazole-3-yl]-benzoic acid EU/3/05/277 |
Treatment of cystic fibrosis | Withdrawn | 27 May 2005 |
| Humanised agonistic anti-CD28 monoclonal antibody EU/3/05/276 |
Treatment of B-cell chronic lymphocytic leukaemia | Active (positive) | 11 Apr 2005 |
| estradiol hemihydrate;progesterone EU/3/05/275 11 clinical trials → |
Bronchopulmonary dysplasia in premature neonates of less than 30 weeks of gestational age | Active (positive) | 11 Apr 2005 |
| melatonin EU/3/05/274 23 clinical trials → |
Treatment of non-24-hour sleep-wake disorders in blind people with no light perception | Withdrawn | 11 Apr 2005 |
| ambrisentan EU/3/05/273 3 clinical trials → |
Treatment of pulmonary arterial hypertension and chronic thromboembolic pulmonary hypertension | Withdrawn | 11 Apr 2005 |
| histamine dihydrochloride EU/3/05/272 |
Treatment of acute myeloid leukaemia | Expired | 11 Apr 2005 |
| Paromomycin sulfate EU/3/05/271 |
Treatment of visceral leishmaniasis | Withdrawn | 11 Apr 2005 |
| autologous Tumor-Derived gp96 Heat Shock Protein-Peptide Complex EU/3/05/270 · now authorised as Oncophage |
Treatment of renal-cell carcinoma | Active (positive) | 11 Apr 2005 |
| tipifarnib EU/3/05/269 |
Treatment of acute myeloid leukaemia | Withdrawn | 10 Mar 2005 |
| (Z)-N-[2-(Diethylamino)ethyl]-5-[(5-fluoro-2-oxo-1,2-dihydro-3H-indol-3-ylidene)methyl]-2,4-dimethyl-1H-pyrrole-3-carboxamide (S)-2-hydroxysyccinate EU/3/05/268 |
Treatment of renal-cell carcinoma | Withdrawn | 10 Mar 2005 |
| (Z)-N-[2-(Diethylamino)ethyl]-5-[(5-fluoro-2-oxo-1,2-dihydro-3H-indol-3-ylidene)methyl]-2,4-dimethyl-1H-pyrrole-3-carboxamide (S)-2-hydroxysyccinate EU/3/05/267 |
Treatment of malignant gastrointestinal stromal tumours | Withdrawn | 10 Mar 2005 |
| ciclosporin EU/3/05/266 3 clinical trials → |
Prevention of graft rejection after lung transplantation | Withdrawn | 10 Mar 2005 |
| ciclosporin EU/3/05/265 3 clinical trials → |
Treatment of graft rejection after lung transplantation | Withdrawn | 10 Mar 2005 |
| cholest-4-en-3-one, oxime EU/3/05/264 |
Treatment of 5q spinal muscular atrophy | Withdrawn | 10 Mar 2005 |
| Fluocinolone acetonide EU/3/05/261 · now authorised as Retisert; Retisert |
Treatment of non-infectious uveitis affecting the posterior segment of the eye | Active (positive) | 7 Mar 2005 |
| Dimethyl sulfoxide EU/3/05/263 |
Treatment of severe closed traumatic brain injury | Active (positive) | 3 Mar 2005 |
| titanium dioxide;bisoctrizole EU/3/05/262 |
Treatment of ultraviolet-A- and visible-light-induced photosensitivity disorders | Withdrawn | 3 Mar 2005 |
| recombinant human alpha-Mannosidase (velmanase alfa) EU/3/04/260 · now authorised as Lamzede |
Treatment of alpha-mannosidosis | Active (positive) | 26 Jan 2005 |
| acetylcysteine EU/3/04/259 2 clinical trials → |
Treatment of idiopathic pulmonary fibrosis | Withdrawn | 26 Jan 2005 |
| L-Asparaginase EU/3/04/258 2 clinical trials → |
Treatment of acute lymphoblastic leukaemia | Withdrawn | 26 Jan 2005 |
| recombinant human bile-salt-stimulated lipase EU/3/04/257 |
Treatment of cystic fibrosis | Withdrawn | 26 Jan 2005 |
| 17-allylamino-17-demethoxygeldanamycin EU/3/04/256 |
Treatment of chronic myeloid leukaemia | Withdrawn | 26 Jan 2005 |
| recombinant histidine-tagged idiotype immunoglobulin Fab fragment of clonal B-cell receptors EU/3/04/249 |
Treatment of follicular lymphoma | Active (positive) | 23 Dec 2004 |
| sabarubicin EU/3/04/255 |
Treatment of small cell lung cancer | Withdrawn | 21 Dec 2004 |
| Val-Leu-Gln-Glu-Leu-Asn-Val-Thr-Val (Pr1 nanopeptide, sequence 169-177, of proteinase 3) EU/3/04/254 |
Treatment of myelodysplastic syndromes | Withdrawn | 21 Dec 2004 |
| Val-Leu-Gln-Glu-Leu-Asn-Val-Thr-Val (Pr1 nanopeptide, sequence 169-177, of proteinase 3) EU/3/04/253 |
Treatment of acute myeloid leukaemia | Withdrawn | 21 Dec 2004 |
| Val-Leu-Gln-Glu-Leu-Asn-Val-Thr-Val (Pr1 nanopeptide, sequence 169-177, of proteinase 3) EU/3/04/252 |
Treatment of chronic myeloid leukaemia | Withdrawn | 21 Dec 2004 |
| N-(methyl-diazacyclohexyl-methylbenzamide)-azaphenyl-aminothiopyrrole EU/3/04/251 |
Treatment of malignant gastrointestinal stromal tumours | Active (positive) | 21 Dec 2004 |
| recombinant histidine-tagged idiotype immunoglobulin Fab fragment of clonal B-cell receptors EU/3/04/250 |
Treatment of mantle cell lymphoma | Active (positive) | 21 Dec 2004 |
| recombinant histidine-tagged idiotype immunoglobulin Fab fragment of clonal B-cell receptors EU/3/04/248 |
Treatment of multiple myeloma | Active (positive) | 21 Dec 2004 |
| 17-allylamino-17-demethoxygeldanamycin EU/3/04/247 |
Treatment of multiple myeloma | Withdrawn | 21 Dec 2004 |
| valproic acid EU/3/04/246 4 clinical trials → |
Treatment of familial adenomatous polyposis (FAP) | Withdrawn | 30 Nov 2004 |
| aplidine EU/3/04/245 · now authorised as Aplidin |
Treatment of multiple myeloma | Active (positive) | 16 Nov 2004 |
| alpha-1 antitrypsin EU/3/04/244 · now authorised as Infinia |
Treatment of emphysema secondary to congenital alpha-1 antitrypsin deficiency | Active (positive) | 16 Nov 2004 |
| alpha-1 antitrypsin EU/3/04/243 |
Treatment of cystic fibrosis | Active (positive) | 16 Nov 2004 |
| N-(methyl-diazacyclohexyl-methylbenzamide)-azaphenyl-aminothiopyrrole EU/3/04/242 · now authorised as Masipro |
Treatment of mastocytosis | Active (positive) | 16 Nov 2004 |
| Pirfenidone EU/3/04/241 13 clinical trials → |
Treatment of idiopathic pulmonary fibrosis | Expired | 16 Nov 2004 |
| recombinant human insulin-like growth factor-I;recombinant human insulin-like growth factor binding protein-3 EU/3/04/238 |
Treatment of leprechaunism | Withdrawn | 21 Oct 2004 |
| recombinant human insulin-like growth factor-I;recombinant human insulin-like growth factor binding protein-3 EU/3/04/237 |
Treatment of Rabson-Mendenhall syndrome | Withdrawn | 21 Oct 2004 |
| recombinant human insulin-like growth factor-I;recombinant human insulin-like growth factor binding protein-3 EU/3/04/236 |
Treatment of type A extreme insulin resistance syndrome | Withdrawn | 21 Oct 2004 |
| sitaxentan sodium EU/3/04/234 |
Treatment of pulmonary arterial hypertension and chronic thromboembolic pulmonary hypertension | Withdrawn | 21 Oct 2004 |
| adeno-associated viral vector containing the human gamma-sarcoglycan gene EU/3/04/233 |
Treatment of gamma sarcoglycanopathy | Withdrawn | 21 Oct 2004 |
| Doxorubicine polyisohexylcyanoacrylate nanoparticles EU/3/04/229 |
Treatment of hepatocellular carcinoma | Withdrawn | 21 Oct 2004 |
| rufinamide EU/3/04/240 |
Treatment of Lennox-Gastaut syndrome | Expired | 20 Oct 2004 |
| Deuterium oxide EU/3/04/239 |
Treatment of pancreatic cancer | Withdrawn | 20 Oct 2004 |
| recombinant human insulin-like growth factor-I;recombinant human insulin-like growth factor binding protein-3 EU/3/04/235 |
Treatment of type B extreme insulin resistance syndrome | Withdrawn | 20 Oct 2004 |
| biotinylated anti-tenascin monoclonal antibody EU/3/04/232 |
Treatment of glioma | Active (positive) | 20 Oct 2004 |
| Deferoxamine mesilate EU/3/04/231 |
Treatment of traumatic spinal cord injury | Withdrawn | 20 Oct 2004 |
| Dexamethasone sodium phosphate encapsulated in human erythrocytes EU/3/04/230 |
Treatment of cystic fibrosis | Withdrawn | 20 Oct 2004 |
| Homoharringtonine EU/3/04/228 14 clinical trials → |
Treatment of acute myeloid leukaemia | Withdrawn | 20 Oct 2004 |
| 1, 1'-[1,4-Phenylenebis (methylene)]-bis-1,4,8,11-tetraazacyclotetradecane (plerixafor) EU/3/04/227 |
Treatment to mobilize progenitor cells prior to stem cell transplantation | Expired | 20 Oct 2004 |
| recombinant human interleukin-21 EU/3/04/226 |
Treatment of renal-cell carcinoma | Withdrawn | 2 Sep 2004 |
| sodium dichloroacetate EU/3/04/225 |
Treatment of systemic monochloroacetate poisoning | Withdrawn | 2 Sep 2004 |
| Homoharringtonine EU/3/04/224 14 clinical trials → |
Treatment of chronic myeloid leukaemia | Withdrawn | 2 Sep 2004 |
| heparin sodium EU/3/04/223 15 clinical trials → |
Treatment of idiopathic pulmonary fibrosis | Withdrawn | 2 Sep 2004 |
| Pancreatic enzymes (cross linked enzyme crystal lipase, protease, amylase) EU/3/04/222 |
Treatment of malabsorption due to exocrine pancreatic enzyme insufficiency | Active (positive) | 2 Sep 2004 |
| 5,10-Methylene-tetrahydrofolic acid EU/3/04/221 |
Treatment of pancreatic cancer in combination with 5-fluorouracil | Withdrawn | 2 Sep 2004 |
| anti epidermal growth factor receptor antibody h-R3 EU/3/04/220 · now authorised as Theraloc; Theraloc |
Treatment of glioma | Active (positive) | 2 Sep 2004 |
| HLA-B27-derived peptide (amino acid 125-138) EU/3/04/219 |
Treatment of autoimmune uveitis | Active (positive) | 2 Sep 2004 |
| histamine dihydrochloride - |
Treatment of malignant melanoma | Negative | 24 Aug 2004 |
| (R, S)-3-(bromomethyl)-3-butanol-1-yl-disphosphate EU/3/04/218 |
Treatment of renal-cell carcinoma | Withdrawn | 29 Jul 2004 |
| sinapultide;Dipalmitoylphosphatidylcholine;palmitoyloleoyl phosphatidylglycerol;palmitic acid EU/3/04/217 |
Respiratory distress syndrome in premature neonates of less then 37 weeks of gestational age | Active (positive) | 29 Jul 2004 |
| sinapultide;Dipalmitoylphosphatidylcholine;palmitoyloleoyl phosphatidylglycerol;palmitic acid EU/3/04/216 · now authorised as Surfaxin; Surfaxin |
Respiratory distress syndrome in premature neonates of less than 32 weeks of gestational age | Active (positive) | 29 Jul 2004 |
| porfimer sodium EU/3/04/215 |
Treatment of cholangiocarcinoma | Withdrawn | 29 Jul 2004 |
| Midostaurin EU/3/04/214 · now authorised as Rydapt 5 clinical trials → |
Treatment of acute myeloid leukaemia | Active (positive) | 29 Jul 2004 |
| Mepolizumab EU/3/04/213 · now authorised as Bosatria; Bosatria 16 clinical trials → |
Treatment of hypereosinophilic syndrome | Withdrawn | 29 Jul 2004 |
| defibrotide EU/3/04/212 |
Treatment of hepatic veno-occlusive disease | Active (positive) | 29 Jul 2004 |
| defibrotide EU/3/04/211 |
Prevention of hepatic veno-occlusive disease | Withdrawn | 29 Jul 2004 |
| ciclosporin EU/3/04/209 3 clinical trials → |
Prevention of graft rejection after lung transplantation | Active (positive) | 29 Jul 2004 |
| acetylsalicylic acid EU/3/04/208 15 clinical trials → |
Treatment of polycythaemia vera | Withdrawn | 29 Jul 2004 |
| sorafenib tosilate EU/3/04/207 |
Treatment of renal-cell carcinoma | Expired | 29 Jul 2004 |
| muramyl tripeptide phosphatidyl ethanolamine EU/3/04/206 |
Treatment of osteosarcoma | Expired | 21 Jun 2004 |
| suberolylanilide hydroxamic acid EU/3/04/205 |
Treatment of cutaneous T-cell lymphoma | Withdrawn | 21 Jun 2004 |
| aztreonam lysinate EU/3/04/204 |
Treatment of Gram-negative bacterial lung infection in cystic fibrosis | Expired | 21 Jun 2004 |
| 5'-CTG CCA CGT TCT CCT GC-(2' methoxy)A-(2' methoxy)C-(2' methoxy)C-3' EU/3/04/203 |
Treatment of myasthenia gravis | Withdrawn | 21 Jun 2004 |
| HLA-A2 restricted CD8 T-cell line expressing MART-1 T-cell receptor EU/3/04/202 |
Treatment of MART-1 YES malignant melanoma in HLA-A2 YES patients | Active (positive) | 21 Jun 2004 |
| vascular endothelial growth factor-D gene in an adenoviral vector for use with a collagen collar EU/3/04/201 |
Prevention of stenosis in synthetic grafts used in haemodialysis | Active (positive) | 8 Jun 2004 |
| (2-aminoethyl) carbamic acid (2R,5S,8S,11S,14R,17S,19aS)-11-(4-aminobutyl)-5-benzyl-8-(4-benzyloxy benzyl)-14-(1H-indol-3-ylmethyl)-4,7,10,13,16,19-hexaoxo-17-… EU/3/04/200 |
Treatment of functional gastro-entero-pancreatic endocrine tumours | Withdrawn | 8 Jun 2004 |
| tetrahydrobiopterin EU/3/04/199 |
Treatment of hyperphenylalaninaemia | Withdrawn | 8 Jun 2004 |
| human monoclonal antibody against CD4 EU/3/04/198 |
Treatment of cutaneous T-cell lymphoma | Active (positive) | 14 Apr 2004 |
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.