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 |
|---|---|---|---|
| perflubron EU/3/20/2383 |
Treatment of respiratory distress syndrome | Withdrawn | 9 Dec 2020 |
| erlotinib EU/3/20/2382 14 clinical trials → |
Treatment of patients with Olmsted syndrome | Active (positive) | 9 Dec 2020 |
| (S)-N-(5-(4-(1-(benzo[d][1,3]dioxol-5-yl)ethyl)piperazin-1-yl)-1,3,4-thiadiazol-2-yl)acetamide, hydrochloride salt EU/3/20/2381 |
Treatment of progressive supranuclear palsy | Active (positive) | 9 Dec 2020 |
| allogeneic retinal pigment epithelial cells genetically modified with a non-viral vector to express beta-domain deleted human factor VIII EU/3/20/2380 |
Treatment of haemophilia A | Withdrawn | 9 Dec 2020 |
| synthetic oligonucleotide selectively targeting UBE3A antisense RNA transcripts EU/3/20/2379 |
Treatment of Angelman syndrome | Active (positive) | 9 Dec 2020 |
| adeno-associated viral vector serotype 9 encoding human ATP7B EU/3/20/2378 |
Treatment of Wilson's disease | Active (positive) | 9 Dec 2020 |
| 2โ-O-(2-methoxyethyl) modified antisense oligonucleotide targeting glycogen synthase 1 preโmRNA EU/3/20/2377 |
Treatment of progressive myoclonic epilepsy type 2 (Lafora disease) | Active (positive) | 9 Dec 2020 |
| 4-[(3S)-3-aminopyrrolidin-1-yl]-6-cyano-5-(3,5-difluorophenyl)-N-[(2S)-1,1,1-trifluoropropan-2-yl]pyridine-3-carboxamide EU/3/20/2376 |
Treatment of congenital hyperinsulinism | Withdrawn | 9 Dec 2020 |
| 2'-O-(2-methoxyethyl) phosphorothioate antisense oligonucleotide targeting CD49d RNA EU/3/20/2375 |
Treatment of Duchenne muscular dystrophy | Active (positive) | 9 Dec 2020 |
| trametinib dimethyl sulfoxide EU/3/20/2374 · now authorised as Spexotras 3 clinical trials → |
Treatment of glioma | Active (positive) | 9 Dec 2020 |
| Humanised IgG1 monoclonal antibody against the extracellular domain of receptor tyrosine kinase-like orphan receptor 1 coupled via a proteolytically cleavable โฆ EU/3/20/2373 |
Treatment of mantle cell lymphoma | Active (positive) | 9 Dec 2020 |
| dabrafenib mesilate EU/3/20/2372 · now authorised as Finlee 24 clinical trials → |
Treatment of glioma | Active (positive) | 9 Dec 2020 |
| aspacytarabine EU/3/20/2371 |
Treatment of acute myeloid leukaemia | Active (positive) | 9 Dec 2020 |
| tremelimumab EU/3/20/2370 47 clinical trials → |
Treatment of hepatocellular carcinoma | Withdrawn | 9 Dec 2020 |
| sotatercept EU/3/20/2369 · now authorised as Winrevair 8 clinical trials → |
Treatment of pulmonary arterial hypertension | Active (positive) | 9 Dec 2020 |
| human interleukin 12 fused with immunoglobulin G4 C-terminal Fc fragment EU/3/20/2368 |
Treatment of glioma | Active (positive) | 9 Dec 2020 |
| (R)-3-(1-(2,3-dichloro-4-(pyrazin-2-yl)phenyl)-2,2,2-trifluoroethyl)-1-methyl-1-(1-methylpiperidin-4-yl)urea fumarate EU/3/20/2367 |
Treatment of Prader-Willi syndrome | Active (positive) | 9 Dec 2020 |
| autologous CD4+ and CD8+ T cells genetically modified with a lentiviral vector encoding a B-cell maturation antigen-specific chimeric antigen receptor EU/3/20/2366 |
Treatment of multiple myeloma | Withdrawn | 9 Dec 2020 |
| adeno-associated virus serotype rh74 containing the human sarcoglycan beta gene EU/3/20/2365 |
Treatment of limb-girdle muscular dystrophy | Active (positive) | 9 Dec 2020 |
| 3,5-diamino-6-chloro-N-(N-(4-(4-(2-(hexyl((2S,3R,4R,5R)-2,3,4,5,6-pentahydroxyhexyl)amino)ethoxy)phenyl)butyl)-carbamimidoyl)pyrazine-2-carboxamide, Sodium chlโฆ EU/3/20/2363 |
Treatment of primary ciliary dyskinesia | Active (positive) | 16 Nov 2020 |
| DNA plasmid encoding human transferrin gene EU/3/20/2364 |
Treatment of retinitis pigmentosa | Active (positive) | 13 Nov 2020 |
| autologous bone marrow derived CD34+ cells transduced ex vivo with a self-inactivating lentiviral vector containing a normal version of the coding region of thโฆ EU/3/20/2362 |
Treatment of X-linked severe combined immunodeficiency | Withdrawn | 13 Nov 2020 |
| perflubron EU/3/20/2361 |
Treatment of congenital pulmonary hypoplasia | Active (positive) | 13 Nov 2020 |
| triheptanoin EU/3/20/2360 |
Treatment of carnitine palmitoyltransferase I deficiency | Active (positive) | 13 Nov 2020 |
| adeno-associated viral vector serotype 9 expressing codon-optimized human GBA gene EU/3/20/2359 |
Treatment of frontotemporal dementia | Active (positive) | 13 Nov 2020 |
| L-pyroglutamyl-L-asparaginyl-L-prolyl-D-tyrosyl-D-tryptophan amide EU/3/20/2358 |
Treatment of amyotrophic lateral sclerosis | Active (positive) | 13 Nov 2020 |
| tislelizumab EU/3/20/2357 259 clinical trials → |
Treatment of oesophageal cancer | Withdrawn | 13 Nov 2020 |
| autologous CD34+ cells transduced ex vivo with a lentiviral vector containing a modified gamma-globin gene EU/3/20/2356 |
Treatment of sickle cell disease | Withdrawn | 13 Nov 2020 |
| anti-(pancreatic adenocarcinoma upregulated factor) IgG1 humanised monoclonal antibody EU/3/20/2355 |
Treatment of pancreatic cancer | Active (positive) | 13 Nov 2020 |
| alisitol;retinol palmitate;zinc gluconate EU/3/20/2354 |
Treatment of microvillus inclusion disease | Active (positive) | 13 Nov 2020 |
| zanidatamab EU/3/20/2353 14 clinical trials → |
Treatment of gastric cancer | Active (positive) | 13 Nov 2020 |
| (R)โtetrahydrofuranโ3โyl 4โ(6โ(5โ(4โethoxyโ1โisopropylpiperidinโ4โyl)pyridin-2-yl)pyrrolo[1,2-b]pyridazin-4-yl)piperazine-1-carboxylate sesquisuccinate EU/3/20/2352 |
Treatment of fibrodysplasia ossificans progressiva | Active (positive) | 13 Nov 2020 |
| naltrexone - 10 clinical trials → |
Treatment of fibromyalgia | Negative | 7 Nov 2020 |
| adeno-associated virus serotype 5 containing the human RDH12 gene EU/3/20/2351 |
Treatment of RDH12 mutation associated retinal dystrophy | Active (positive) | 19 Oct 2020 |
| Poly(oxy-1,2-ethanediyl), alpha-hydro-omega-methoxy, ether with N-[[[2-[[6-[[1-[3-[[3-(2,3-dihydroxypropoxy)propyl]amino]-3-oxopropyl]-2,5-dioxo-3-pyrrolidinylโฆ EU/3/20/2350 · now authorised as Yorvipath |
Treatment of hypoparathyroidism | Active (positive) | 19 Oct 2020 |
| miglustat EU/3/20/2349 3 clinical trials → |
Treatment of neuronal ceroid lipofuscinosis | Active (positive) | 19 Oct 2020 |
| poly(oxy-1,2-ethanediyl), alpha-(carboxymethyl)-omega-methoxy-, amide with cystathionine ฮณ-lyase [Pyridoxal 5โ-phosphate cofactor] (synthetic engineered human)โฆ EU/3/20/2348 |
Treatment of homocystinuria | Active (positive) | 19 Oct 2020 |
| trehalose EU/3/20/2347 |
Treatment of neuronal ceroid lipofuscinosis | Withdrawn | 19 Oct 2020 |
| adeno-associated virus serotype hu68 containing the human GLB1 gene EU/3/20/2346 |
Treatment of GM1 gangliosidosis | Active (positive) | 19 Oct 2020 |
| sparsentan EU/3/20/2345 · now authorised as Filspari 5 clinical trials → |
Treatment of primary IgA nephropathy | Active (positive) | 19 Oct 2020 |
| autologous peripheral blood T cells CD4 and CD8 selected and CD3 and CD28 activated transduced with retroviral vector expressing anti-CD19 CD28/CD3-zeta chimerโฆ EU/3/20/2344 · now authorised as Tecartus |
Treatment of acute lymphoblastic leukaemia | Active (positive) | 19 Oct 2020 |
| retifanlimab EU/3/20/2343 8 clinical trials → |
Treatment of anal cancer | Active (positive) | 19 Oct 2020 |
| Highly branched poly (beta-amino ester) complexed with a nanoplasmid containing the human COL7A1 gene EU/3/20/2342 |
Treatment of epidermolysis bullosa | Active (positive) | 19 Oct 2020 |
| autologous CD34+ cells transduced with a lentiviral vector encoding galactosidase alpha EU/3/20/2341 |
Treatment of Fabry disease | Withdrawn | 19 Oct 2020 |
| 2-(2-(18F)fluoropyridin-4-yl)-9H-pyrrolo[2,3-b:4,5-c']dipyridine EU/3/20/2340 |
Diagnosis of corticobasal degeneration | Active (positive) | 19 Oct 2020 |
| leniolisib EU/3/20/2339 · now authorised as Joenja 4 clinical trials → |
Treatment of activated phosphoinositide 3-kinase delta syndrome | Active (positive) | 19 Oct 2020 |
| decitabine;tetrahydrouridine EU/3/20/2338 51 clinical trials → |
Treatment of sickle cell disease | Active (positive) | 19 Oct 2020 |
| ribitol EU/3/20/2337 3 clinical trials → |
Treatment of limb-girdle muscular dystrophy | Active (positive) | 19 Oct 2020 |
| (4-{(2S,4S)-4-ethoxy-1-[(5-methoxy-7-methyl-1H-indol-4-yl)methyl]piperidin-2-yl}benzoic acid-hydrogen chloride(1/1)) EU/3/20/2336 |
Treatment of primary IgA nephropathy | Active (positive) | 19 Oct 2020 |
| (S)-1-(5-((2,3-dihydro-[1,4]dioxino[2,3-b]pyridin-7-yl)sulfonyl)-3,4,5,6-tetrahydropyrrolo[3,4-c]pyrrol-2(1H)-yl)-3-hydroxy-2-phenylpropan-1-one EU/3/20/2335 |
Treatment of sickle cell disease | Active (positive) | 19 Oct 2020 |
| adeno-associated viral vector serotype 9 encoding a codon-optimised human aspartylglucosaminidase transgene EU/3/20/2334 |
Treatment of aspartylglucosaminuria | Withdrawn | 19 Oct 2020 |
| tipifarnib EU/3/20/2333 |
Treatment of peripheral T-cell lymphoma | Withdrawn | 19 Oct 2020 |
| retigabine EU/3/20/2332 |
Treatment of KCNQ2 developmental and epileptic encephalopathy | Active (positive) | 19 Oct 2020 |
| 1-(3-methylbutanoyl)-L-aspartyl-L-threonyl-L-histidyl-L-phenylalanyl-L-prolyl-(L-cystinyl-L-isoleucyl-[(N6-(S)-4-carboxy-4-palmitamidobutanoyl)-L-lysinyl]-L-phโฆ EU/3/20/2330 |
Treatment of polycythaemia vera | Active (positive) | 19 Oct 2020 |
| Infigratinib EU/3/20/2329 · now authorised as Febseltiq 3 clinical trials → |
Treatment of cholangiocarcinoma | Withdrawn | 21 Aug 2020 |
| human frataxin fused to TAT cell-penetrating peptide EU/3/20/2328 |
Treatment of Friedreichโs ataxia | Active (positive) | 21 Aug 2020 |
| autologous T cells transduced with lentiviral vector containing a tandem chimeric antigen receptor directed against CD20 and CD19 EU/3/20/2327 2 clinical trials → |
Treatment of diffuse large B-cell lymphoma | Active (positive) | 21 Aug 2020 |
| autologous CD34+ cells transduced with a lentiviral vector encoding glucosylceramidase beta EU/3/20/2326 |
Treatment of Gaucher disease | Withdrawn | 21 Aug 2020 |
| venglustat EU/3/20/2325 4 clinical trials → |
Treatment of GM2 gangliosidosis | Active (positive) | 21 Aug 2020 |
| 3-(((1S,2S,3R)-2,3-difluoro-1-hydroxy-7-(methylsulfonyl)-2,3-dihydro-1H-inden-4-yl)oxy)-5-fluorobenzonitrile EU/3/20/2324 |
Treatment of von Hippel-Lindau disease | Withdrawn | 21 Aug 2020 |
| trehalose EU/3/20/2323 |
Treatment of mucopolysaccharidosis type III (Sanfilippo syndrome) | Active (positive) | 21 Aug 2020 |
| 6-((3S,4S)-4-methyl-1-(pyrimidin-2-yl-methyl)pyrrolidin-3-yl)-3-tetrahydropyran-4-yl-7H-imidazo(1,5-a)pyrazin-8-one (Tovinontrine) EU/3/20/2322 |
Treatment of sickle cell disease | Withdrawn | 21 Aug 2020 |
| adeno-associated viral vector serotype 3B encoding shortened human ATP7B EU/3/20/2321 |
Treatment of Wilson's disease | Withdrawn | 21 Aug 2020 |
| copper histidinate EU/3/20/2320 |
Treatment of Menkes disease | Active (positive) | 21 Aug 2020 |
| sodium (4-{(E)-3-(4-fluorophenyl)-3-[4-(3-morpholin-4-yl-prop1ynyl)phenyl]allyloxy}-2-methylphenoxy)acetate EU/3/20/2319 |
Treatment of long-chain 3-hydroxyacyl-coenzyme A dehydrogenase deficiency | Withdrawn | 21 Aug 2020 |
| dextran sulfate low molecular weight EU/3/20/2318 |
Treatment of amyotrophic lateral sclerosis | Active (positive) | 21 Aug 2020 |
| allogeneic T-cell precursors, mobilised peripheral blood-derived, ex vivo cultured EU/3/20/2317 |
Treatment in haematopoietic stem cell transplantation | Active (positive) | 21 Aug 2020 |
| protein-based delivery vector carrying a DNA payload encoding an RNA-guided nuclease that targets stx genes of Shiga toxin-producing Escherichia coli EU/3/20/2316 |
Prevention of haemolytic uraemic syndrome | Active (positive) | 21 Aug 2020 |
| pentosan polysulfate sodium EU/3/20/2315 3 clinical trials → |
Treatment of mucopolysaccharidosis VI (Maroteaux-Lamy syndrome) | Active (positive) | 21 Aug 2020 |
| Humanised IgG1 monoclonal antibody against human eotaxin-2;nebokitug EU/3/20/2314 |
Treatment of primary sclerosing cholangitis | Active (positive) | 21 Aug 2020 |
| 2-(2-(18F)fluoropyridin-4-yl)-9H-pyrrolo[2,3-b:4,5-c']dipyridine EU/3/20/2313 |
Diagnosis of progressive supranuclear palsy | Active (positive) | 21 Aug 2020 |
| allogeneic hepatoblastoma cells encapsulated in alginate, ex vivo expanded EU/3/20/2312 |
Treatment of acute liver failure | Active (positive) | 21 Aug 2020 |
| sodium (4-{(E)-3-(4-fluorophenyl)-3-[4-(3-morpholin-4-yl-prop1ynyl)phenyl]allyloxy}-2-methylphenoxy)acetate EU/3/20/2311 |
Treatment of mitochondrial encephalomyopathy, lactic acidosis and stroke-like episodes | Withdrawn | 21 Aug 2020 |
| Adeno-associated virus serotype 2/8 vector containing the human PDE6A gene EU/3/20/2310 |
Treatment of retinitis pigmentosa | Active (positive) | 21 Aug 2020 |
| Bis-(3-deoxy-3-(4-(3-fluorophenyl)-1H-1,2,3-triazol-1-yl)-beta-D-galactopyranosyl) sulfane EU/3/20/2309 |
Treatment of idiopathic pulmonary fibrosis | Withdrawn | 21 Aug 2020 |
| allogeneic umbilical cord tissue-derived mesenchymal stromal cells ex vivo expanded EU/3/20/2308 |
Prevention of bronchopulmonary dysplasia | Active (positive) | 21 Aug 2020 |
| tinostamustine EU/3/20/2307 |
Treatment of T-cell prolymphocytic leukaemia | Active (positive) | 27 Jul 2020 |
| retinol palmitate EU/3/20/2306 |
Prevention of bronchopulmonary dysplasia | Active (positive) | 27 Jul 2020 |
| imetelstat sodium EU/3/20/2305 4 clinical trials → |
Treatment of myelodysplastic syndromes | Active (positive) | 27 Jul 2020 |
| Maralixibat chloride EU/3/20/2304 4 clinical trials → |
Treatment of biliary atresia | Active (positive) | 27 Jul 2020 |
| adeno-associated viral vector expressing acid alpha-glucosidase gene EU/3/20/2303 |
Treatment of glycogen storage disease type II (Pompe's disease) | Active (positive) | 27 Jul 2020 |
| triheptanoin EU/3/20/2302 |
Treatment of carnitine-acylcarnitine translocase deficiency | Active (positive) | 27 Jul 2020 |
| pegylated adrenomedullin EU/3/20/2301 |
Treatment of acute respiratory distress syndrome (ARDS) | Withdrawn | 27 Jul 2020 |
| fasudil hydrochloride EU/3/20/2300 2 clinical trials → |
Treatment of non-traumatic subarachnoid haemorrhage | Active (positive) | 27 Jul 2020 |
| C-type natriuretic peptide conjugated to multi-arm polyethylene glycol carrier through a cleavable linker EU/3/20/2299 |
Treatment of achondroplasia | Active (positive) | 27 Jul 2020 |
| Hemopexin EU/3/20/2298 |
Treatment of sickle cell disease | Active (positive) | 27 Jul 2020 |
| melatonin - 23 clinical trials → |
Treatment of intracerebral haemorrhage | Negative | 6 Jul 2020 |
| adeno-associated virus serotype HSC15 expressing human arylsulfatase A gene EU/3/20/2297 |
treatment of metachromatic leukodystrophy | Withdrawn | 26 Jun 2020 |
| axicabtagene ciloleucel EU/3/20/2296 13 clinical trials → |
Treatment of marginal zone lymphoma | Active (positive) | 26 Jun 2020 |
| Lys40(NODAGA-68Ga)NH2-exendin-4 EU/3/20/2295 |
Diagnosis of insulinoma | Active (positive) | 26 Jun 2020 |
| sodium cromoglicate EU/3/20/2294 |
Treatment of idiopathic pulmonary fibrosis | Withdrawn | 26 Jun 2020 |
EU/3/20/2293 |
Treatment of Becker muscular dystrophy | Withdrawn | 26 Jun 2020 |
| anti-CD123 IgG1 humanised monoclonal antibody conjugated to N1-(2-(2,5-dioxo-2,5-dihydro-1H-pyrrol-1-yl)ethyl)-N6-((S)-1-(((S)-1-((3-((((S)-8-methoxy-6-oxo-11,โฆ EU/3/20/2292 2 clinical trials → |
Treatment of blastic plasmacytoid dendritic cell neoplasm | Active (positive) | 26 Jun 2020 |
| onfekafusp alfa EU/3/20/2291 6 clinical trials → |
Treatment of glioma | Active (positive) | 26 Jun 2020 |
| stiripentol EU/3/20/2290 |
Treatment of primary hyperoxaluria | Active (positive) | 26 Jun 2020 |
| nomacopan EU/3/20/2289 |
Treatment of bullous pemphigoid | Active (positive) | 26 Jun 2020 |
| Magrolimab EU/3/20/2288 2 clinical trials → |
Treatment of myelodysplastic syndromes | Withdrawn | 26 Jun 2020 |
| benzyl benzoate;beta-caryophyllene;cineole;cinnamaldehyde;cinnamyl acetate;linalool;trans-2-methoxycinnamaldehyde - |
Treatment of eumycetoma | Negative | 18 Jun 2020 |
| adeno-associated virus serotype 9 containing the human ASPA gene EU/3/20/2287 |
Treatment of Canavan disease | Active (positive) | 4 Jun 2020 |
| autologous CD4+ and CD8+ T cells transduced with a lentiviral vector encoding an affinity enhanced T cell receptor specific to MAGE-A4 EU/3/20/2286 |
Treatment of soft tissue sarcoma | Active (positive) | 4 Jun 2020 |
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.