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140 results
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Orphan designation: Glasdegib maleate for: Treatment of acute myeloid leukaemia
Date of designation: 16/10/2017, Positive, Last updated: 09/01/2018 -
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Orphan designation: recombinant human interleukin-3 truncated diphtheria toxin fusion protein for: Treatment of blastic plasmacytoid dendritic cell neoplasm
Date of designation: 11/11/2015, Positive, Last updated: 25/01/2021 -
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Orphan designation: (R)-2-Methyl-6-nitro-2-{4-[4-(4-trifluoromethoxyphenoxy)piperidin-1-yl]phenoxymethyl}-2,3-dihydroimidazo[2,1-b]oxazole (delamanid) for: Treatment of tuberculosis
Date of designation: 02/02/2008, Positive, Last updated: 18/06/2014 -
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Orphan designation: ivacaftor, tezacaftor for: Treatment of cystic fibrosis
Date of designation: 27/02/2017, Positive, Last updated: 25/01/2022 -
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Orphan designation: carfilzomib for: Treatment of multiple myeloma
Date of designation: 03/06/2008, Positive, Last updated: 02/12/2015 -
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Orphan designation: Autologous CD34+ cells transfected with lentiviral vector containing the human arylsulfatase A cDNA for: treatment of metachromatic leukodystrophy
Date of designation: 13/04/2007, Positive, Last updated: 14/07/2022 -
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Orphan designation: Synthetic double-stranded siRNA oligonucleotide directed against hydroxyacid oxidase 1 mRNA and covalently linked to a ligand containing three N-acetylgalactosamine residues (lumasiran) for: Treatment of primary hyperoxaluria
Date of designation: 21/03/2016, Positive, Last updated: 25/11/2020 -
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Orphan designation: blinatumomab for: Treatment of acute lymphoblastic leukaemia
Date of designation: 24/07/2009, Positive, Last updated: 08/12/2015 -
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Orphan designation: (S)-2-nitro-6-(4-trifluoromethoxy)benzyloxy)-6,7-dihydro-5H-imidazo[2,1-b] [1,3] oxazine (pretomanid) for: Treatment of tuberculosis
Date of designation: 29/11/2007, Positive, Last updated: 09/12/2020 -
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Orphan designation: Recombinant IgG degrading enzyme of Streptococcus pyogenes (imlifidase) for: Prevention of graft rejection following solid organ transplantation
Date of designation: 12/01/2017, Positive, Last updated: 28/08/2020 -
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Orphan designation: [5-(5-chloro-1H-pyrrolo[2,3-b]pyridin-3-ylmethyl)-pyridin-2-yl]-(6-trifluoromethyl-pyridin-3-ylmethyl)-amine hydrochloride for: Treatment of tenosynovial giant cell tumour, localised and diffuse type
Date of designation: 19/03/2015, Withdrawn, Last updated: 02/02/2022 -
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Orphan designation: Humanised monoclonal antibody against P-selectin (crizanlizumab) for: Treatment of sickle cell disease
Date of designation: 09/08/2012, Positive, Last updated: 24/11/2020 -
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Orphan designation: (S)-1-(4-fluorophenyl)-1-(2-(4-(6-(1-methyl-1H-pyrazol-4-yl)pyrrolo[2,1-f][1,2,4]triazin-4-yl)piperazin-yl)pyrimidin-5-yl)ethan-1-amine for: Treatment of gastrointestinal stromal tumours
Date of designation: 17/07/2017, Positive, Last updated: 22/05/2019 -
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Orphan designation: Humanised monoclonal antibody targeting B-cell maturation antigen conjugated with maleimidocaproyl monomethyl auristatin F for: Treatment of multiple myeloma
Date of designation: 16/10/2017, Positive, Last updated: 17/09/2020 -
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Orphan designation: N-tert-butyl-3-[(5-methyl-2-{[4-(2-pyrrolidin-1-ylethoxy)phenyl]amino}pyrimidin-4-yl)amino] benzenesulfonamide dihydrochloride monohydrate (fedratinib) for: Treatment of post-polycythaemia vera myelofibrosis
Date of designation: 26/11/2010, Positive, Last updated: 17/09/2021 -
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Orphan designation: N-tert-butyl-3-[(5-methyl-2-{[4-(2-pyrrolidin-1-ylethoxy)phenyl]amino}pyrimidin-4-yl)amino] benzenesulfonamide dihydrochloride monohydrate (fedratinib) for: Treatment of post-essential thrombocythaemia myelofibrosis
Date of designation: 26/11/2010, Positive, Last updated: 17/09/2021 -
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Orphan designation: N-tert-butyl-3-[(5-methyl-2-{[4-(2-pyrrolidin-1-ylethoxy)phenyl]amino}pyrimidin-4-yl)amino] benzenesulfonamide dihydrochloride monohydrate (fedratinib) for: Treatment of primary myelofibrosis
Date of designation: 01/10/2010, Positive, Last updated: 17/09/2021 -
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Orphan designation: (1R,2S) 6-bromo-alpha-[2-(dimethylamino)ethyl]-2-methoxy-alpha-(1-naphthyl)-beta-phenyl-3-quinolineethanol bedaquiline for: Treatment of tuberculosis
Date of designation: 26/08/2005, Positive, Last updated: 01/04/2014 -
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Orphan designation: Ivacaftor, N-(1,3-dimethyl-1H-pyrazole-4-sulfonyl)-6-[3-(3,3,3-trifluoro-2,2-dimethylpropoxy)-1H-pyrazol-1-yl]-2-[(4S)-2,2,4-trimethylpyrrolidin-1-yl]pyridine-3-carboxamide, tezacaftor (ivacaftor / tezacaftor / elexacaftor) for: Treatment of cystic fibrosis
Date of designation: 14/12/2018, Positive, Last updated: 25/01/2022 -
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Orphan designation: Selumetinib for: Treatment of neurofibromatosis type 1
Date of designation: 31/07/2018, Positive, Last updated: 15/09/2021 -
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Orphan designation: Daratumumab for: Treatment of plasma-cell myeloma
Date of designation: 17/07/2013, Positive, Last updated: 02/10/2018 -
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Orphan designation: Autologous T lymphocyte-enriched population of cells transduced with a lentiviral vector encoding a chimeric antigen receptor targeting human B cell maturation antigen with 4-1BB and CD3-zeta intracellular signalling domains (idecabtagene vicleucel) for: Treatment of multiple myeloma
Date of designation: 20/04/2017, Positive, Last updated: 03/12/2021 -
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Orphan designation: Humanised Fc engineered monoclonal antibody against CD19 (tafasitamab) for: Treatment of diffuse large B-cell lymphoma
Date of designation: 15/01/2015, Positive, Last updated: 08/09/2021 -
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Orphan designation: 1-Deoxygalactonojirimycin hydrochloride for: Treatment of Fabry disease
Date of designation: 22/05/2006, Positive, Last updated: 21/05/2019 -
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Orphan designation: artesunate for: Treatment of malaria
Date of designation: 28/02/2020, Positive, Last updated: 13/01/2022