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 |
|---|---|---|---|
| 18‐mer antisense oligonucleotide complementary to SCN1A mRNA;sodium salt EU/3/22/2579 |
Treatment of Dravet syndrome | Active (positive) | 24 Feb 2022 |
| aldesleukin EU/3/22/2578 42 clinical trials → |
Treatment of amyotrophic lateral sclerosis | Active (positive) | 24 Feb 2022 |
| [Ala1,3,12,Gln10,Arg11,Trp14]PTH(1-14)/[Ala18,22, Lys26]PTHrP(15-36)COOH EU/3/22/2577 |
Treatment of hypoparathyroidism | Active (positive) | 24 Feb 2022 |
| Gadolinium-chelated polysiloxane nanoparticles EU/3/22/2576 |
Treatment of pancreatic cancer | Active (positive) | 24 Feb 2022 |
| bovactant EU/3/22/2575 |
Treatment of respiratory distress syndrome | Active (positive) | 24 Feb 2022 |
| pyridoxal 5'-phosphate EU/3/22/2574 |
Treatment of pyridoxamine 5'-phosphate oxidase deficiency | Active (positive) | 24 Feb 2022 |
| vatiquinone EU/3/22/2573 2 clinical trials → |
Treatment of mitochondrial encephalomyopathy, lactic acidosis, and stroke-like episodes | Withdrawn | 24 Feb 2022 |
| valemetostat tosilate EU/3/22/2572 |
Treatment of peripheral T-cell lymphoma | Withdrawn | 24 Feb 2022 |
| rintatolimod EU/3/21/2403 |
Treatment of pancreatic cancer | Active (positive) | 19 Feb 2022 |
EU/3/21/2571 |
Treatment of Leber’s hereditary optic neuropathy | Active (positive) | 14 Jan 2022 |
| sirolimus EU/3/21/2570 32 clinical trials → |
Treatment of bronchiolitis obliterans syndrome (BOS) | Active (positive) | 14 Jan 2022 |
| trans N-ethyl-2-((4-(7-((4-(ethylsulfonamido)cyclohexyl)methyl)-2,7-diazaspiro[3.5]nonan-2-yl)pyrimidin-5-yl)oxy)-5-fluoro-N-isopropylbenzamide sesquifumarate EU/3/21/2569 |
Treatment of acute myeloid leukaemia | Active (positive) | 14 Jan 2022 |
| azithromycin dihydrate EU/3/21/2568 14 clinical trials → |
Prevention of bronchopulmonary dysplasia | Active (positive) | 14 Jan 2022 |
| melatonin EU/3/21/2567 23 clinical trials → |
Treatment of pre-eclampsia | Active (positive) | 14 Jan 2022 |
| adeno-associated virus serotype 9 expressing the human fukutin related protein and target sequence of the miR-208a EU/3/21/2566 |
Treatment of limb girdle muscular dystrophy | Active (positive) | 14 Jan 2022 |
| 5' moemC-(sp)-moemC-(p)-moeA-(p)-moemC-(p)-moeG-(p)-moeA-(p)-dmC-(sp)-dA-(sp)-dT-(sp)-dA-(sp)-dT-(sp)-dT-(sp)-dT-(sp)-dT-(sp)-dT-(sp)-dmC-(sp)-moeT-(p)-moeA-(s… EU/3/21/2565 |
Treatment of SCN2A developmental and epileptic encephalopathy | Active (positive) | 14 Jan 2022 |
| 5-((4'-(3,3-difluorocyclobutyl)-[1,1'-biphenyl]-4-yl)oxy)-1H-1,2,3-triazole-4-carboxylic acid EU/3/21/2564 |
Treatment of primary hyperoxaluria | Withdrawn | 14 Jan 2022 |
| olorofim EU/3/21/2563 |
Treatment of invasive Scopulariopsis | Active (positive) | 14 Jan 2022 |
| Gadolinium-chelated polysiloxane nanoparticles EU/3/21/2562 |
Treatment of glioma | Active (positive) | 14 Jan 2022 |
| chimeric peptide of human glucagon-like peptide-1, glucagon and gastric inhibitory polypeptide analogues linked to a human immunoglobulin Fc fragment EU/3/21/2561 2 clinical trials → |
Treatment of primary biliary cholangitis | Active (positive) | 14 Jan 2022 |
| posnafusp alfa EU/3/21/2560 |
Treatment of mucopolysaccharidosis type IIIA (Sanfilippo A syndrome) | Active (positive) | 14 Jan 2022 |
| fasudil hydrochloride EU/3/21/2559 2 clinical trials → |
Treatment of amyotrophic lateral sclerosis | Active (positive) | 14 Jan 2022 |
| mRNA encoding human glucose-6-phosphatase variant S298C EU/3/21/2558 |
treatment of glycogen storage disease type Ia | Active (positive) | 14 Jan 2022 |
| insulin human EU/3/21/2557 |
Prevention of retinopathy of prematurity | Active (positive) | 14 Jan 2022 |
| navtemadlin EU/3/21/2556 5 clinical trials → |
Treatment of Merkel cell carcinoma | Active (positive) | 14 Jan 2022 |
| efgartigimod alfa EU/3/21/2555 |
Treatment of chronic inflammatory demyelinating polyneuropathy | Active (positive) | 14 Jan 2022 |
| Enterovirus B, echovirus 7, live - |
Treatment of uveal melanoma | Negative | 11 Jan 2022 |
| octreotide acetate EU/3/21/2554 3 clinical trials → |
Treatment of idiopathic intracranial hypertension | Active (positive) | 10 Dec 2021 |
| troriluzole hydrochloride EU/3/21/2553 3 clinical trials → |
Treatment of spinocerebellar ataxia | Active (positive) | 10 Dec 2021 |
| 2’-O-(2-methoxyethyl)-5-methyl-P-thiocytidylyl-(3’-O->5’-O)-2’-O-(2-methoxyethyl)-5-methyl-P-thiouridylyl-(3’-O->5’-O)-2’-O-(2-methoxyethyl)-P-thioguanylyl-(3’… EU/3/21/2552 |
Treatment of cystic fibrosis | Active (positive) | 10 Dec 2021 |
| adeno-associated virus vector serotype 9 encoding the human GRN gene EU/3/21/2551 |
Treatment of frontotemporal dementia | Active (positive) | 10 Dec 2021 |
| unesbulin EU/3/21/2550 |
Treatment of soft tissue sarcoma | Withdrawn | 10 Dec 2021 |
| Norrin (25-133), Lys86Pro EU/3/21/2549 |
Treatment of familial exudative vitreoretinopathy | Active (positive) | 10 Dec 2021 |
| cedazuridine;decitabine EU/3/21/2548 51 clinical trials → |
Treatment of acute myeloid leukaemia | Withdrawn | 10 Dec 2021 |
| chimeric peptide of human glucagon-like peptide-1, glucagon and gastric inhibitory polypeptide analogues linked to a human immunoglobulin Fc fragment EU/3/21/2547 2 clinical trials → |
Treatment of primary sclerosing cholangitis | Active (positive) | 10 Dec 2021 |
| 6-(4-(tert-butyl)phenoxy)pyridin-3-amine EU/3/21/2546 |
Treatment of acute lymphoblastic leukaemia | Withdrawn | 10 Dec 2021 |
| N-[1-((5-cyanopyridin-2-yl)methyl)-1H-pyrazol- 3-yl]-2-[4-(1-(trifluoromethyl)cyclopropyl) phenyl]acetamide EU/3/21/2545 |
Treatment of epileptic encephalopathy with continuous spike-and-wave during sleep | Active (positive) | 10 Dec 2021 |
| ribonucleoprotein complex composed of two sgRNA and a Cas9 nuclease targeting the human COL7A1 gene EU/3/21/2544 |
Treatment of epidermolysis bullosa | Active (positive) | 10 Dec 2021 |
| copper (64Cu) chloride EU/3/21/2543 |
Treatment of glioma | Active (positive) | 10 Dec 2021 |
| atrasentan EU/3/21/2542 3 clinical trials → |
Treatment of primary IgA nephropathy | Active (positive) | 10 Dec 2021 |
| crofelemer EU/3/21/2541 2 clinical trials → |
Treatment of short bowel syndrome | Active (positive) | 10 Dec 2021 |
| 3-(ethoxydifluoromethyl)-6-(5-fluoro-6-(2,2,2-trifluoroethoxy)pyridin-3-yl)-[1,2,4]triazolo[4,3-a]pyrazine EU/3/21/2540 |
Treatment of SCN8A developmental and epileptic encephalopathy | Active (positive) | 10 Dec 2021 |
| 3-(ethoxydifluoromethyl)-6-(5-fluoro-6-(2,2,2-trifluoroethoxy)pyridin-3-yl)-[1,2,4]triazolo[4,3-a]pyrazine EU/3/21/2539 |
Treatment of SCN2A developmental and epileptic encephalopathy | Active (positive) | 10 Dec 2021 |
| anti-(endothelin-1 receptor subtype A) IgG4 humanised monoclonal antibody EU/3/21/2538 |
Treatment of pulmonary arterial hypertension | Active (positive) | 10 Dec 2021 |
| retinol palmitate EU/3/21/2537 |
Prevention of bronchopulmonary dysplasia | Active (positive) | 10 Dec 2021 |
| vatiquinone EU/3/21/2536 2 clinical trials → |
Treatment of Alpers-Huttenlocher syndrome | Withdrawn | 10 Dec 2021 |
| autologous T cells ex vivo modified with a lentiviral vector encoding a chimeric antigen receptor specific for CD1a EU/3/21/2535 |
Treatment of acute lymphoblastic leukaemia | Active (positive) | 10 Dec 2021 |
| allogenic fetal mesenchymal stem cells EU/3/21/2534 |
Treatment of osteogenesis imperfecta | Active (positive) | 10 Dec 2021 |
| macitentan EU/3/21/2533 4 clinical trials → |
Treatment of chronic thromboembolic pulmonary hypertension | Withdrawn | 10 Dec 2021 |
| garadacimab EU/3/21/2532 |
Treatment of hereditary angioedema | Withdrawn | 10 Dec 2021 |
| adeno-associated viral vector serotype 9 containing the human SURF1 gene EU/3/21/2531 |
Treatment of Leigh syndrome | Withdrawn | 12 Nov 2021 |
| olverembatinib EU/3/21/2530 16 clinical trials → |
Treatment of chronic myeloid leukaemia | Active (positive) | 12 Nov 2021 |
| soticlestat EU/3/21/2529 |
Treatment of Lennox-Gastaut syndrome | Active (positive) | 12 Nov 2021 |
| D-lactic acid, glycolic acid EU/3/21/2528 |
Treatment of amyotrophic lateral sclerosis | Active (positive) | 12 Nov 2021 |
| (14S)-8-[3-(2-{dispiro[2.0.24.13]heptan-7-yl}ethoxy)-1H-pyrazol-1-yl]-12,12-dimethyl-2lamba6-thia-3,9,11,18,23-penta-azatetracyclo[17.3.1.111,14.05,10]tetracos… EU/3/21/2527 · now authorised as Alyftrek |
Treatment of cystic fibrosis | Active (positive) | 12 Nov 2021 |
| 2-[1-(3-{6-[(1E)-(hydroxyimino)methyl]-5-methyl-4-oxo-7-propyl-3H,4H-pyrrolo[2,1-f][1,2,4]triazin-2-yl}-4-propoxybenzenesulfonyl)piperidin-4-yl]ethyl nitrate EU/3/21/2526 |
Treatment of systemic sclerosis | Active (positive) | 12 Nov 2021 |
| Ibrexafungerp EU/3/21/2525 |
Treatment of invasive candidiasis | Active (positive) | 12 Nov 2021 |
| rebastinib EU/3/21/2524 |
Treatment of ovarian cancer | Active (positive) | 12 Nov 2021 |
| 5-fluoro-4-(4-fluoro-2-methoxyphenyl)-N-{4-[(S-methylsulfonimidoyl)methyl] pyridin-2-yl}pyridin-2-amine EU/3/21/2523 |
Treatment of diffuse large B-cell lymphoma | Active (positive) | 12 Nov 2021 |
| cannabidiol;Dronabinol EU/3/21/2522 16 clinical trials → |
Treatment of complex regional pain syndrome | Active (positive) | 12 Nov 2021 |
| udonitrectag lysine EU/3/21/2521 |
Treatment in solid organ transplantation | Active (positive) | 12 Nov 2021 |
| Fingolimod EU/3/21/2520 8 clinical trials → |
Treatment of adrenoleukodystrophy | Active (positive) | 12 Nov 2021 |
| autologous CD34+ enriched cells transduced with a self-inactivating lentiviral vector containing the codon-optimized RPS19 gene EU/3/21/2519 |
Treatment of Diamond-Blackfan anaemia | Active (positive) | 12 Nov 2021 |
| paclitaxel;polyoligo(ethylene glycol)methacrylate-co-poly(vinylbenzyldithiodibutyric acid-gemcitabine) EU/3/21/2518 400 clinical trials → |
Treatment of pancreatic cancer | Active (positive) | 12 Nov 2021 |
| mosunetuzumab EU/3/21/2517 · now authorised as Lunsumio 20 clinical trials → |
Treatment of follicular lymphoma | Active (positive) | 12 Nov 2021 |
| nadunolimab EU/3/21/2516 |
Treatment of pancreatic cancer | Active (positive) | 12 Nov 2021 |
| Linerixibat EU/3/21/2515 · now authorised as Lynavoy |
Treatment of primary biliary cholangitis | Active (positive) | 12 Nov 2021 |
| anti-CD38 IgG4 human monoclonal antibody EU/3/21/2514 |
Treatment of amyotrophic lateral sclerosis | Active (positive) | 12 Nov 2021 |
| psilocybine EU/3/21/2513 43 clinical trials → |
Treatment of fragile X syndrome | Active (positive) | 15 Oct 2021 |
| Idursulfase beta EU/3/21/2512 |
Treatment of mucopolysaccharidosis type II (Hunter's syndrome) | Active (positive) | 15 Oct 2021 |
| adeno-associated virus serotype rh10 containing the human GALC gene EU/3/21/2511 |
Treatment of Krabbe disease | Active (positive) | 15 Oct 2021 |
| encaleret EU/3/21/2510 3 clinical trials → |
Treatment of hypoparathyroidism | Active (positive) | 15 Oct 2021 |
| selinexor EU/3/21/2509 47 clinical trials → |
Treatment of glioma | Active (positive) | 15 Oct 2021 |
| allogeneic umbilical cord mesenchymal cells-derived extracellular vesicles EU/3/21/2508 |
Prevention of bronchopulmonary dysplasia | Active (positive) | 15 Oct 2021 |
| autologous haematopoietic stem and progenitor cell population containing CD34+ cells transduced with a lentiviral vector encoding the TCIRG1 cDNA ex vivo expan… EU/3/21/2507 |
Treatment of osteopetrosis | Active (positive) | 15 Oct 2021 |
| adeno-associated viral vector serotype 5 expressing the human Cone-Rod Homeobox gene EU/3/21/2506 |
Treatment of cone-rod dystrophy | Active (positive) | 15 Oct 2021 |
| allogeneic retinal pigment epithelial cells genetically modified with a non-viral vector to express human alpha-L-iduronidase EU/3/21/2505 |
Treatment of mucopolysaccharidosis type I | Withdrawn | 15 Oct 2021 |
| mocravimod EU/3/21/2504 |
Treatment in haematopoietic stem cell transplantation | Active (positive) | 15 Oct 2021 |
| ganaxolone EU/3/21/2503 |
Treatment of tuberous sclerosis | Active (positive) | 15 Oct 2021 |
| Humanised IgG1 monoclonal antibody against SEZ6 linked to N-acetyl-calicheamicin EU/3/21/2502 |
Treatment of small cell lung cancer | Withdrawn | 15 Oct 2021 |
| batiraxcept EU/3/21/2501 |
Treatment of ovarian cancer | Withdrawn | 15 Oct 2021 |
| Adeno-associated virus of serotype rh10 encoding Human MLC1 under the control of GFAP promoter EU/3/21/2500 |
Treatment of megalencephalic leukoencephalopathy with subcortical cysts | Active (positive) | 15 Oct 2021 |
| Devimistat EU/3/21/2499 |
Treatment of Burkitt's lymphoma | Active (positive) | 15 Oct 2021 |
| human IgG1 monoclonal antibody against sortilin EU/3/21/2498 |
Treatment of frontotemporal dementia | Withdrawn | 15 Oct 2021 |
| Glofitamab EU/3/21/2497 · now authorised as Columvi 46 clinical trials → |
Treatment of diffuse large B-cell lymphoma | Withdrawn | 15 Oct 2021 |
| autologous CD34+ cell enriched population containing haematopoietic stem and progenitor cells transduced ex vivo with a lentiviral vector encoding the human AD… EU/3/21/2496 |
Treatment of adenosine deaminase 2 deficiency (DADA2) | Active (positive) | 20 Aug 2021 |
| lurbinectedin EU/3/21/2495 17 clinical trials → |
Treatment of malignant mesothelioma | Active (positive) | 20 Aug 2021 |
| sabatolimab EU/3/21/2494 |
Treatment of myelodysplastic syndromes | Withdrawn | 20 Aug 2021 |
| autologous CD34+ haematopoietic stem and progenitor cells genetically modified with the lentiviral vector encoding for the human iduronate 2-sulfatase gene EU/3/21/2493 |
Treatment of mucopolysaccharidosis type II (Hunter's syndrome) | Active (positive) | 20 Aug 2021 |
| adeno-associated virus serotype 9 encoding human NGLY1 gene EU/3/21/2492 |
Treatment of NGLY1 deficiency | Active (positive) | 20 Aug 2021 |
| Adeno-associated viral vector serotype S3 containing codon-optimised expression cassette encoding human beta-glucocerebrosidase variant EU/3/21/2491 |
Treatment of Gaucher disease | Active (positive) | 20 Aug 2021 |
| recombinant human apolipoprotein A-I EU/3/21/2490 |
Treatment of lecithin-cholesterol acyltransferase deficiency | Active (positive) | 20 Aug 2021 |
| cemdisiran EU/3/21/2489 5 clinical trials → |
Treatment of primary IgA nephropathy | Active (positive) | 20 Aug 2021 |
| autologous CD34+ cells transfected with a lentiviral vector containing codon optimised RPS19 gene EU/3/21/2488 |
Treatment of Diamond-Blackfan anaemia | Active (positive) | 20 Aug 2021 |
| adeno-associated virus serotype PTC3 expressing the human UBE3A gene EU/3/21/2487 |
Treatment of Angelman syndrome | Active (positive) | 20 Aug 2021 |
| talquetamab EU/3/21/2486 · now authorised as Talvey 20 clinical trials → |
Treatment of multiple myeloma | Active (positive) | 20 Aug 2021 |
| Humanised IgG1 monoclonal antibody against TfR1 conjugated to double stranded siRNA oligonucleotide against DMPK via a non-cleavable linker EU/3/21/2485 |
Treatment of myotonic disorders | Active (positive) | 20 Aug 2021 |
| adeno-associated viral vector serotype 9 containing the human MECP2 gene EU/3/21/2484 |
Treatment of Rett syndrome | Active (positive) | 20 Aug 2021 |
| human keratinocytes EU/3/21/2483 |
Treatment of partial deep dermal and full thickness burns | Active (positive) | 20 Aug 2021 |
| adeno-associated viral vector serotype 9 containing the human HEXA and HEXB genes EU/3/21/2482 |
Treatment of GM2 gangliosidosis | Active (positive) | 20 Aug 2021 |
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.