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 |
|---|---|---|---|
| Loncastuximab tesirine EU/3/21/2481 6 clinical trials → |
Treatment of diffuse large B-cell lymphoma | Withdrawn | 20 Aug 2021 |
| Bardoxolone methyl EU/3/21/2480 |
Treatment of autosomal dominant polycystic kidney disease | Active (positive) | 20 Aug 2021 |
| allogeneic anti-Epstein Barr virus cytotoxic T lymphocytes expanded ex vivo EU/3/19/2138 |
Treatment of post-transplant lymphoproliferative disorder | Withdrawn | 21 Jul 2021 |
| 2-Methoxy-5-[(1Z)-2-(3,4,5-trimethoxyphenyl)ethenyl]-phenol (fosbretabulin tromethamine) EU/3/04/195 |
Treatment of anaplastic thyroid cancer | Withdrawn | 21 Jul 2021 |
| recombinant human ectonucleotide pyrophosphatase/phosphodiesterase 1 fused to the Fc fragment of IgG1 EU/3/21/2479 |
Treatment of adenosine triphosphate binding cassette transporter protein subfamily C member 6 deficiency | Active (positive) | 19 Jul 2021 |
| adeno-associated virus vector serotype 1 containing the human GRN gene EU/3/21/2478 |
Treatment of frontotemporal dementia | Withdrawn | 19 Jul 2021 |
| Adeno-associated viral vector serotype Anc80 containing the 3' portion of human OTOF gene, adeno-associated viral vector serotype Anc80 containing the 5' porti… EU/3/21/2477 |
Treatment of otoferlin gene-mediated hearing loss | Active (positive) | 19 Jul 2021 |
| 3,5-diiodothyropropionic acid EU/3/21/2476 |
Treatment of Allan-Herndon-Dudley syndrome | Active (positive) | 19 Jul 2021 |
| Infigratinib EU/3/21/2475 3 clinical trials → |
Treatment of achondroplasia | Active (positive) | 19 Jul 2021 |
| autologous CD34+ hematopoietic stem and progenitor cells genetically modified with the lentiviral vector encoding for the human palmitoyl-protein thioesterase … EU/3/21/2474 |
Treatment of neuronal ceroid lipofuscinosis | Active (positive) | 19 Jul 2021 |
| adeno-associated viral vector serotype 9 containing the human SLC13A5 gene EU/3/21/2473 |
Treatment of SLC13A5-epileptic encephalopathy deficiencies | Active (positive) | 19 Jul 2021 |
| mRNA encoding the human glycogen debranching enzyme EU/3/21/2472 |
Treatment of glycogen storage disease type III | Active (positive) | 19 Jul 2021 |
| humanised IgG2k Fc-modified bispecific monoclonal antibody against CD3 and BCMA EU/3/21/2471 |
Treatment of multiple myeloma | Withdrawn | 19 Jul 2021 |
| pridopidine hydrochloride EU/3/21/2470 3 clinical trials → |
Treatment of amyotrophic lateral sclerosis | Active (positive) | 19 Jul 2021 |
EU/3/21/2469 |
Treatment of chronic myeloid leukaemia | Active (positive) | 19 Jul 2021 |
| melatonin EU/3/21/2468 23 clinical trials → |
Treatment of non-traumatic spontaneous intracerebral haemorrhage | Active (positive) | 19 Jul 2021 |
| sirolimus EU/3/21/2467 32 clinical trials → |
Treatment of perivascular epithelioid cell tumours | Active (positive) | 19 Jul 2021 |
| Lutetium (177Lu) omburtamab barzuxetan EU/3/21/2466 |
Treatment of medulloblastoma | Active (positive) | 19 Jul 2021 |
| Eftansomatropin alfa EU/3/21/2465 |
Treatment of growth hormone deficiency | Active (positive) | 19 Jul 2021 |
| tisagenlecleucel EU/3/21/2464 · now authorised as Kymriah 5 clinical trials → |
Treatment of follicular lymphoma | Active (positive) | 19 Jul 2021 |
| Itolizumab EU/3/21/2463 |
Treatment of graft-versus-host disease | Active (positive) | 19 Jul 2021 |
| 1-(4-(6-chloropyridazin-3-yl)piperazin-1-yl)-2-(4-cyclopropyl-3-fluorophenyl)ethan-1-one EU/3/21/2462 |
Treatment of propionic acidaemia | Withdrawn | 19 Jul 2021 |
| 1-(4-(6-chloropyridazin-3-yl)piperazin-1-yl)-2-(4-cyclopropyl-3-fluorophenyl)ethan-1-one EU/3/21/2461 |
Treatment of pantothenate kinase-associated neurodegeneration | Withdrawn | 19 Jul 2021 |
| saroglitazar magnesium EU/3/21/2460 5 clinical trials → |
Treatment of primary biliary cholangitis | Active (positive) | 19 Jul 2021 |
| synthetic double-stranded siRNA oligonucleotide directed against apolipoprotein C-III mRNA and covalently linked to a ligand containing three N-acetylgalactosa… EU/3/21/2459 · now authorised as Redemplo |
Treatment of familial chylomicronaemia syndrome | Active (positive) | 19 Jul 2021 |
| zanidatamab EU/3/21/2458 · now authorised as Ziihera 14 clinical trials → |
Treatment of biliary tract cancer | Active (positive) | 19 Jul 2021 |
| amivantamab - 30 clinical trials → |
Treatment of partial deep dermal and full-thickness burns | Negative | 28 Jun 2021 |
| adeno-associated viral vector LK03 encoding human methylmalonyl-CoA mutase EU/3/21/2457 |
Treatment of methylmalonic acidaemia | Active (positive) | 21 Jun 2021 |
| adeno-associated virus serotype 9 containing the human FXN gene isoform 1 EU/3/21/2456 |
Treatment of Friedreich's ataxia | Withdrawn | 21 Jun 2021 |
| H-D-valyl1-D-alanyl-D-glutamyl-D-alanyl-D-arginyl5-D-glutamyl-D-glutamyl-D-leucyl-D-glutamyl-D-arginyl10-D-leucyl-D-glutamyl-D-alanyl-D-arginyl-D-leucyl15-glyc… EU/3/21/2455 |
Treatment of glioma | Active (positive) | 21 Jun 2021 |
| Humanised monoclonal antibody derivative against fibroblast growth factor receptor 3 EU/3/21/2454 |
Treatment of achondroplasia | Active (positive) | 21 Jun 2021 |
| melatonin EU/3/21/2453 23 clinical trials → |
Prevention of retinopathy of prematurity | Active (positive) | 21 Jun 2021 |
| Hydrocortisone hydrogen succinate EU/3/21/2452 |
Prevention of bronchopulmonary dysplasia | Withdrawn | 21 Jun 2021 |
| Bomedemstat ditosilate EU/3/21/2451 |
Treatment of essential thrombocythaemia | Active (positive) | 21 Jun 2021 |
| (S)-5-amino-3-(4-((5-fluoro-2-methoxybenzamido)methyl)phenyl)-1-(1,1,1-trifluoropropane-2-yl)-1H-pyrazole-4-carboxamide EU/3/21/2450 |
Treatment of mantle cell lymphoma | Withdrawn | 21 Jun 2021 |
| imatinib EU/3/21/2449 32 clinical trials → |
Treatment of pulmonary arterial hypertension | Active (positive) | 21 Jun 2021 |
| allogenic placenta-derived mesenchymal stromal cells secretome EU/3/21/2448 |
Treatment of pre-eclampsia | Active (positive) | 21 Jun 2021 |
| adeno-associated virus serotype 9 expressing human CLN5 EU/3/21/2447 |
Treatment of neuronal ceroid lipofuscinosis | Active (positive) | 21 Jun 2021 |
| tislelizumab EU/3/21/2446 259 clinical trials → |
Treatment of nasopharyngeal cancer | Withdrawn | 21 Jun 2021 |
| L-ergothioneine EU/3/21/2445 |
Treatment of cystinuria | Active (positive) | 21 Jun 2021 |
| Humanised IgG2 monoclonal antibody against TNFSF13 EU/3/21/2444 |
Treatment of primary IgA nephropathy | Active (positive) | 21 Jun 2021 |
| dantrolene sodium, hemiheptahydrate EU/3/21/2443 |
Treatment of malignant hyperthermia | Withdrawn | 20 May 2021 |
| adeno-associated virus serotype 9 expressing the cDNA for human MECP2 EU/3/21/2442 |
Treatment of Rett syndrome | Withdrawn | 20 May 2021 |
| adeno-associated viral vector serotype 5 expressing the human Cone-Rod Homeobox gene EU/3/21/2441 |
Treatment of Leber's congenital amaurosis | Active (positive) | 20 May 2021 |
| autologous mobilised peripheral blood-derived CD34+ cells transduced ex vivo with a self-inactivating lentiviral vector containing a normal version of the codi… EU/3/21/2440 |
Treatment of X-linked severe combined immunodeficiency | Withdrawn | 20 May 2021 |
| melatonin EU/3/21/2439 23 clinical trials → |
Treatment of retinitis pigmentosa | Active (positive) | 20 May 2021 |
| zanubrutinib EU/3/21/2438 62 clinical trials → |
Treatment of marginal zone lymphoma | Withdrawn | 20 May 2021 |
| synthetic 2′-O-(2-methoxyethyl)-modified antisense oligonucleotide linked to a triantennary cluster of N-acetyl galactosamine sugars targeting transmembrane pr… EU/3/21/2437 |
Treatment of beta thalassaemia intermedia and major | Active (positive) | 20 May 2021 |
| trehalose EU/3/21/2436 |
Treatment of amyotrophic lateral sclerosis | Active (positive) | 20 May 2021 |
| (S)-2-amino-6-(2-hydroxypropanoyl)-7,8-dihydropteridin-4(3H)-one EU/3/21/2435 · now authorised as Sephience |
Treatment of hyperphenylalaninemia | Active (positive) | 20 May 2021 |
| 6-amino-5-chloro-N-((1R)-1-(5-(((5-chloro-4-(trifluoromethyl)-2-pyridinyl)amino)carbonyl)-2-thiazolyl)ethyl)-4-pyrimidinecarboxamide EU/3/21/2434 · now authorised as Ojemda |
Treatment of glioma | Active (positive) | 20 May 2021 |
| adeno-associated viral vector serotype 2.5T encoding the human cystic fibrosis transmembrane conductance regulator with a partial deletion in the R domain EU/3/21/2433 |
Treatment of cystic fibrosis | Active (positive) | 20 May 2021 |
| 2-[4-[3-(methylamino)-1-phenylpropoxy]phenyl]ethanol hydrochloride EU/3/21/2432 |
Treatment of fragile X syndrome | Active (positive) | 20 May 2021 |
| human IgG1 monoclonal antibody against alpha-synuclein EU/3/21/2431 |
Treatment of multiple system atrophy | Active (positive) | 20 May 2021 |
| elamipretide EU/3/21/2430 2 clinical trials → |
Treatment of Barth syndrome | Active (positive) | 20 May 2021 |
| eflornithine EU/3/21/2429 · now authorised as Ifinwil 6 clinical trials → |
Treatment of neuroblastoma | Active (positive) | 20 May 2021 |
| macitentan EU/3/21/2428 4 clinical trials → |
Treatment of functional single ventricle congenital heart disease | Withdrawn | 20 May 2021 |
| begelomab EU/3/21/2427 |
Treatment of dermatomyositis | Active (positive) | 20 May 2021 |
| ganglioside GM1 EU/3/21/2426 |
Treatment of amyotrophic lateral sclerosis | Active (positive) | 13 Apr 2021 |
| S-[5-(omega-methoxypoly(oxyethylene)-2-oxopentyl)]-L-cysteinylglycyl-L-serinylglycylgylcyl-L-isoleucyl-L-lysyl-L-glutamyl-L-phenylalanyl-L-leucyl-L-glutaminyl-… EU/3/21/2425 |
Treatment of cutaneous T-cell lymphoma | Withdrawn | 13 Apr 2021 |
| cevostamab EU/3/21/2424 11 clinical trials → |
Treatment of multiple myeloma | Active (positive) | 13 Apr 2021 |
| vatiquinone EU/3/21/2423 2 clinical trials → |
Treatment of Friedreich's ataxia | Active (positive) | 26 Mar 2021 |
| Lorcaserin hydrochloride EU/3/21/2422 |
Treatment of Dravet syndrome | Active (positive) | 26 Mar 2021 |
| Herpes simplex virus 1 expressing the human CFTR gene EU/3/21/2421 |
Treatment of cystic fibrosis | Active (positive) | 26 Mar 2021 |
| alpelisib EU/3/21/2420 · now authorised as Vijoice; Vijoice 22 clinical trials → |
Treatment of PIK3CA related overgrowth spectrum | Active (positive) | 26 Mar 2021 |
| single guide RNA targeting the human TTR gene, Ziclumeran EU/3/21/2419 |
Treatment of ATTR amyloidosis | Active (positive) | 26 Mar 2021 |
| Ilixadencel EU/3/21/2418 |
Treatment of gastrointestinal stromal tumours | Active (positive) | 26 Mar 2021 |
| adeno-associated virus serotype hu68 containing the human GALC gene EU/3/21/2417 |
Treatment of Krabbe disease | Active (positive) | 26 Mar 2021 |
| 5-fluoro-4-(7'-fluoro-2'-methylspiro[cyclopentane-1,3'-indol]-5'-yl)-N-(5-(1-methylpiperidin-4-yl)pyridin-2-yl)pyrimidin-2-amine;crozbaciclib EU/3/21/2416 |
Treatment of glioma | Withdrawn | 26 Mar 2021 |
| Lefitolimod EU/3/21/2415 |
Treatment of small cell lung cancer | Active (positive) | 26 Mar 2021 |
| lepunafusp alfa EU/3/21/2414 |
Treatment of mucopolysaccharidosis type I | Active (positive) | 26 Mar 2021 |
| adeno-associated viral vector serotype 9 expressing codon-optimized human GBA gene EU/3/21/2413 |
Treatment of Gaucher disease | Active (positive) | 19 Feb 2021 |
| Adeno-associated virus serotype 5 containing the human NR2E3 gene EU/3/21/2412 |
Treatment of Leber's congenital amaurosis | Active (positive) | 19 Feb 2021 |
| Gly-Arg-Lys-Lys-Arg-Arg-Gln-Arg-Arg-Arg-Cys-Asp-Met-Ala-Glu-His-Thr-Glu-Arg-Leu-Lys-Ala-Asn-Asp-Ser-Leu-Lys-Leu-Ser-Gln-Glu-Tyr-Glu-Ser-Ile-NH2 EU/3/21/2411 |
Treatment of spinal cord injury | Active (positive) | 19 Feb 2021 |
| 2-[6-(6,7-Dimethoxyquinolin-3-yl)pyridin-3-yl]-N-[3-(1,1,1-trifluoro-2-methylpropan-2-yl)-1,2-oxazol-5-yl]acetamide (zeteletinib) EU/3/21/2410 |
Treatment of medullary thyroid carcinoma | Withdrawn | 19 Feb 2021 |
| Dodecyl creatine ester, dodecyl creatine ester hydrochloride EU/3/21/2409 |
Treatment of creatine deficiency syndromes | Active (positive) | 19 Feb 2021 |
| Adeno-associated virus serotype 5 containing the human NR2E3 gene EU/3/21/2408 |
Treatment of retinitis pigmentosa | Active (positive) | 19 Feb 2021 |
| autologous CD34+ cells transduced with a lentiviral RNA vector that results in integrated cDNA encoding for functional cystinosin EU/3/21/2407 |
Treatment of cystinosis | Active (positive) | 19 Feb 2021 |
| autologous CD34+ hematopoietic stem and progenitor cells transfected with zinc finger nuclease mRNAs SB-mRENH1 and SB-mRENH2 EU/3/21/2406 |
Treatment of sickle cell disease | Active (positive) | 19 Feb 2021 |
| autologous T lymphocyte-enriched population of cells transduced with a lentiviral vector encoding a chimeric antigen receptor targeting human claudin 18.2 anti… EU/3/21/2405 |
Treatment of gastric cancer | Active (positive) | 19 Feb 2021 |
EU/3/21/2404 |
Treatment of leishmaniasis | Active (positive) | 19 Feb 2021 |
| 2-(2-{[2-(1h-benzimidazol-2-yl)ethyl]amino}ethyl)-n-[(3-fluoropyridin-2-yl)methyl]-1,3-oxazole-4-carboxamide trihydrochloride EU/3/21/2402 |
Treatment of sickle cell disease | Active (positive) | 19 Feb 2021 |
EU/3/21/2401 |
Treatment of paroxysmal nocturnal haemoglobinuria | Withdrawn | 19 Feb 2021 |
| anti-SIGLEC8 IgG1 humanised monoclonal antibody EU/3/21/2400 |
Treatment of eosinophilic gastroenteritis | Withdrawn | 19 Feb 2021 |
| 18-(p-[131I]-iodophenyl)octadecyl phosphocholine EU/3/21/2399 |
Treatment of lymphoplasmacytic lymphoma | Active (positive) | 19 Feb 2021 |
| 2'-O-methyl phosphorothioate RNA oligonucleotide, 5'-m5CUGm5CUGm5CUGm5CUGm5CUGm5CUGm5CUG-3' EU/3/21/2398 |
Treatment of spinocerebellar ataxia | Active (positive) | 19 Feb 2021 |
| tebentafusp EU/3/21/2397 · now authorised as Kimmtrak 4 clinical trials → |
Treatment of uveal melanoma | Active (positive) | 19 Feb 2021 |
| alpha galactosidase A EU/3/20/2396 |
Treatment of Fabry disease | Active (positive) | 6 Jan 2021 |
| celecoxib;ciprofloxacin EU/3/20/2395 23 clinical trials → |
Treatment of amyotrophic lateral sclerosis | Active (positive) | 6 Jan 2021 |
| Cyclo-L-glycyl-L-2-allylproline EU/3/20/2394 |
Treatment of Phelan-McDermid syndrome | Active (positive) | 6 Jan 2021 |
| Cyclo-L-glycyl-L-2-allylproline EU/3/20/2393 |
Treatment of Pitt-Hopkins syndrome | Active (positive) | 6 Jan 2021 |
| Humanised IgG1K monoclonal antibody against interferon beta EU/3/20/2392 |
Treatment of dermatomyositis | Active (positive) | 6 Jan 2021 |
| idursulfase EU/3/20/2391 2 clinical trials → |
Treatment of mucopolysaccharidosis type II (Hunter's syndrome) | Withdrawn | 6 Jan 2021 |
| rilonacept EU/3/20/2390 · now authorised as Rilonacept FGK Representative Service GmbH |
Treatment of idiopathic pericarditis | Active (positive) | 6 Jan 2021 |
| Cyclo-L-glycyl-L-2-allylproline EU/3/20/2389 |
Treatment of Angelman syndrome | Active (positive) | 6 Jan 2021 |
| human laminin-111, recombinant EU/3/20/2388 |
Treatment of congenital muscular dystrophy | Active (positive) | 6 Jan 2021 |
| setanaxib EU/3/20/2387 |
Treatment of primary biliary cholangitis | Active (positive) | 6 Jan 2021 |
| sodium oxybate;calcium oxybate;magnesium oxybate;potassium oxybate EU/3/20/2386 |
Treatment of idiopathic hypersomnia | Active (positive) | 6 Jan 2021 |
| rezafungin acetate EU/3/20/2385 · now authorised as Rezzayo |
Treatment of invasive candidiasis | Active (positive) | 6 Jan 2021 |
| sulindac EU/3/20/2384 |
Treatment of fragile X syndrome | Active (positive) | 9 Dec 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.