https://www.ema.europa.eu/en/homepageOrphan designationsen-gbCopyright: (C) European Medicines AgencySat, 11 May 2024 03:02:13 +0200Thu, 11 Apr 2024 15:26:00 +0200https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-16-1714Orphan designation: 6'-(R)-methyl-5-O-(5-amino-5,6-dideoxy-α-L-talofuranosyl)-paromamine sulfate Treatment of mucopolysaccharidosis type I, 22/09/2016 Positivehttps://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-16-1714Thu, 11 Apr 2024 15:26:00 +0200Orphan designationshttps://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-14-1236Orphan designation: Diacerein Treatment of epidermolysis bullosa, 20/02/2014 Positivehttps://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-14-1236Thu, 11 Apr 2024 15:24:00 +0200Orphan designationshttps://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-23-2873Orphan designation: 3,3-Dimethyl-N-(6-methyl-5-{[2-(1-methyl-1H-pyrazol-4-yl)pyridine-4-yl]oxy}pyridine-2-yl)-2-oxopyrrolidine-1-carboxamide hydrochloride hydrate Treatment of tenosynovial giant cell tumour, localised and diffuse type, 13/12/2023 Positivehttps://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-23-2873Thu, 11 Apr 2024 15:23:00 +0200Orphan designationshttps://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-17-1861Orphan designation: (S)-8-{2-amino-6-[1-(5-chloro-biphenyl-2-yl)-(R)-2,2,2-trifluoro-ethoxy]-pyrimidin-4-yl}-2,8-diaza-spiro[4.5]decane-3-carboxylic acid ethyl ester Treatment of pulmonary arterial hypertension, 20/04/2017 Positivehttps://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-17-1861Thu, 11 Apr 2024 15:21:00 +0200Orphan designationshttps://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-22-2723Orphan designation: Upifitamab rilsodotin Treatment of ovarian cancer, 09/12/2022 Positivehttps://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-22-2723Thu, 11 Apr 2024 15:18:00 +0200Orphan designationshttps://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-20-2265Orphan designation: cusatuzumab Treatment of acute myeloid leukaemia, 22/04/2020 Positivehttps://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-20-2265Thu, 11 Apr 2024 15:16:00 +0200Orphan designationshttps://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-15-1515Orphan designation: Synthetic hypericin Treatment of cutaneous T-cell lymphoma, 28/07/2015 Positivehttps://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-15-1515Thu, 11 Apr 2024 15:15:00 +0200Orphan designationshttps://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-19-2184Orphan designation: N-((R)-2,3-dihydroxypropoxyl)-3,4-difluro-2-(2-fluoro-4-iodo-phenylamino)-benzamide Treatment of neurofibromatosis type 1, 25/07/2019 Positivehttps://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-19-2184Thu, 11 Apr 2024 15:14:00 +0200Orphan designationshttps://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-05-317Orphan designation: anti-CD3 mAb (SPV-T3a)-ricin A chain fusion protein,anti-CD7 mAb (WT1)-ricin A chain fusion protein Treatment of graft-versus-host disease, 26/08/2005 Positivehttps://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-05-317Thu, 11 Apr 2024 15:12:00 +0200Orphan designationshttps://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-10-756Orphan designation: Tranilast Prevention of scarring post glaucoma filtration surgery, 27/07/2010 Positivehttps://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-10-756Thu, 11 Apr 2024 15:08:00 +0200Orphan designationshttps://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-20-2311Orphan designation: Sodium (4-{(E)-3-(4-fluorophenyl)-3-[4-(3-morpholin-4-yl-prop1ynyl)phenyl]allyloxy}-2-methylphenoxy)acetate Treatment of mitochondrial encephalomyopathy, lactic acidosis and stroke-like episodes, 21/08/2020 Withdrawnhttps://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-20-2311Thu, 11 Apr 2024 14:59:00 +0200Orphan designationshttps://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-20-2319Orphan designation: Sodium (4-{(E)-3-(4-fluorophenyl)-3-[4-(3-morpholin-4-yl-prop1ynyl)phenyl]allyloxy}-2-methylphenoxy)acetate Treatment of long-chain 3-hydroxyacyl-coenzyme A dehydrogenase deficiency, 21/08/2020 Withdrawnhttps://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-20-2319Thu, 11 Apr 2024 14:59:00 +0200Orphan designationshttps://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-18-2010Orphan designation: branaplam Treatment of spinal muscular atrophy, 16/04/2018 Withdrawnhttps://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-18-2010Thu, 11 Apr 2024 14:54:00 +0200Orphan designationshttps://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-17-1938Orphan designation: antisense oligonucleotide targeting exon 73 in the COL7A1 gene Treatment of epidermolysis bullosa, 12/10/2017 Withdrawnhttps://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-17-1938Thu, 11 Apr 2024 14:50:00 +0200Orphan designationshttps://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-18-2080Orphan designation: Recombinant adeno-associated viral vector serotype S3 containing codon-optimised expression cassette encoding human coagulation factor IX variant Treatment of haemophilia B, 26/10/2018 Withdrawnhttps://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-18-2080Thu, 11 Apr 2024 14:49:00 +0200Orphan designationshttps://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-24-2897Orphan designation: Repagermanium Treatment of focal segmental glomerulosclerosis, 19/02/2024 Positivehttps://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-24-2897Thu, 11 Apr 2024 14:40:37 +0200Orphan designationshttps://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-24-2901Orphan designation: Andecaliximab Treatment of fibrodysplasia ossificans progressiva, 19/02/2024 Positivehttps://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-24-2901Thu, 11 Apr 2024 14:37:19 +0200Orphan designationshttps://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-24-2890Orphan designation: Raludotatug deruxtecan Treatment of ovarian cancer, 19/02/2024 Positivehttps://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-24-2890Thu, 11 Apr 2024 14:35:42 +0200Orphan designationshttps://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-24-2892Orphan designation: Treatment of small cell lung cancer, 19/02/2024 Positivehttps://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-24-2892Thu, 11 Apr 2024 14:28:33 +0200Orphan designationshttps://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-24-2891Orphan designation: carboplatin Treatment of glioma, 19/02/2024 Positivehttps://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-24-2891Thu, 11 Apr 2024 14:26:03 +0200Orphan designationshttps://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-24-2899Orphan designation: Plerixafor Treatment of acute respiratory distress syndrome (ARDS), 19/02/2024 Positivehttps://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-24-2899Thu, 11 Apr 2024 14:24:15 +0200Orphan designationshttps://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-24-2898Orphan designation: Donidalorsen Treatment of hereditary angioedema, 19/02/2024 Positivehttps://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-24-2898Thu, 11 Apr 2024 14:10:54 +0200Orphan designationshttps://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-24-2900Orphan designation: Autologous CD3-positive T-cells expressing a chimeric antigen receptor against B cell maturation agent Treatment of AL amyloidosis, 19/02/2024 Positivehttps://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-24-2900Thu, 11 Apr 2024 14:08:27 +0200Orphan designationshttps://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-24-2896Orphan designation: Human IgG1 monoclonal antibody targeting amyloid transthyretin Treatment of ATTR amyloidosis, 19/02/2024 Positivehttps://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-24-2896Thu, 11 Apr 2024 14:06:05 +0200Orphan designationshttps://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-23-2887Orphan designation: Gorilla adenovirus vector expressing HPV6 and HPV11 antigens Treatment of recurrent respiratory papillomatosis, 12/01/2024 Positivehttps://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-23-2887Thu, 11 Apr 2024 14:00:55 +0200Orphan designationshttps://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-23-2888Orphan designation: Recombinant human heparan-N-sulfatase Treatment of mucopolysaccharidosis type IIIA (Sanfilippo A syndrome), 12/01/2024 Positivehttps://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-23-2888Thu, 11 Apr 2024 13:58:46 +0200Orphan designationshttps://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-23-2881Orphan designation: Ziftomenib Treatment of acute myeloid leukaemia, 12/01/2024 Positivehttps://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-23-2881Thu, 11 Apr 2024 13:56:56 +0200Orphan designationshttps://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-23-2877Orphan designation: Apilimod dimesilate Treatment of amyotrophic lateral sclerosis, 12/01/2024 Positivehttps://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-23-2877Thu, 11 Apr 2024 13:55:24 +0200Orphan designationshttps://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-23-2884Orphan designation: Adeno-associated viral vector serotype 9 containing the human MECP2 gene, an intron encoding a miRNA generating sequence, and complementary miRNA binding sites Treatment of Rett syndrome, 12/01/2024 Positivehttps://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-23-2884Thu, 11 Apr 2024 13:52:52 +0200Orphan designationshttps://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-23-2883Orphan designation: cutamesine alpha-thalassaemia X-linked intellectual disability syndrome (due to mutations in the ATRX gene), 12/01/2024 Positivehttps://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-23-2883Thu, 11 Apr 2024 13:49:52 +0200Orphan designationshttps://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-23-2876Orphan designation: Tarlatamab Treatment of small cell lung cancer, 12/01/2024 Positivehttps://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-23-2876Thu, 11 Apr 2024 13:47:55 +0200Orphan designationshttps://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-23-2885Orphan designation: motixafortide Treatment of patients undergoing haematopoietic stem cell transplantation, 12/01/2024 Positivehttps://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-23-2885Thu, 11 Apr 2024 12:13:52 +0200Orphan designationshttps://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-23-2889Orphan designation: mitapivat sulfate Treatment of thalassaemia intermedia and major, 12/01/2024 Positivehttps://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-23-2889Thu, 11 Apr 2024 12:11:57 +0200Orphan designationshttps://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-23-2875Orphan designation: Thiophene methylimidazole pentahydrogen Diagnosis of glioma, 12/01/2024 Positivehttps://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-23-2875Thu, 11 Apr 2024 12:07:22 +0200Orphan designationshttps://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-23-2879Orphan designation: Ersodetug Treatment of insulinoma, 12/01/2024 Positivehttps://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-23-2879Thu, 11 Apr 2024 12:05:25 +0200Orphan designationshttps://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-23-2882Orphan designation: Methyl-(1-{[6-{[(1S)-1-cyclopropylethyl]amino}-2-(pyrazolo[5,1-b][1,3]thiazol-7-yl)-pyrimidin-4-yl]carbonyl}piperidin-4-yl)carbamate mono(4-methylbenzenesulfonate) Treatment of eosinophilic granulomatosis with polyangiitis, 12/01/2024 Positivehttps://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-23-2882Thu, 11 Apr 2024 12:00:42 +0200Orphan designationshttps://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-23-2878Orphan designation: golcadomide hydrochloride Treatment of diffuse large B-cell lymphoma, 12/01/2024 Positivehttps://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-23-2878Thu, 11 Apr 2024 11:58:53 +0200Orphan designationshttps://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-23-2886Orphan designation: Human coagulation factor X Treatment of acquired factor X deficiency, 12/01/2024 Positivehttps://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-23-2886Thu, 11 Apr 2024 11:52:40 +0200Orphan designationshttps://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-23-2874Orphan designation: (4R)-3-(4-fluoro-2-hydroxyphenyl)-4-methyl-4,5-dihydro-1H-pyrazole-1-carboximidamide hydrochloride Treatment of systemic sclerosis, 12/01/2024 Positivehttps://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-23-2874Thu, 11 Apr 2024 11:47:57 +0200Orphan designationshttps://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-24-2894Orphan designation: mRNA encoding the human CFTR gene Treatment of cystic fibrosis, 19/02/2024 Positivehttps://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-24-2894Thu, 11 Apr 2024 11:37:10 +0200Orphan designations