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2427 results
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Orphan designation: Three human monoclonal antibodies against the EBOV glycoprotein for: Treatment of Ebola virus disease (updated)
Date of designation: 25/05/2018, Positive, Last updated: 07/10/2021 -
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Orphan designation: human monoclonal antibody against activin A for: Treatment of fibrodysplasia ossificans progressiva (updated)
Date of designation: 18/11/2016, Positive, Last updated: 07/10/2021 -
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Orphan designation: Single-chain urokinase plasminogen activator for: Treatment of pleural empyema (updated)
Date of designation: 16/12/2014, Positive, Last updated: 27/09/2021 -
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Orphan designation: velaglucerase alfa for: Treatment of Gaucher disease (updated)
Date of designation: 06/06/2010, Positive, Last updated: 24/09/2021 -
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Orphan designation: Recombinant human parathyroid hormone for: Treatment of hypoparathyroidism (updated)
Date of designation: 16/01/2014, Positive, Last updated: 24/09/2021 -
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Orphan designation: Maralixibat chloride for: Treatment of biliary atresia (updated)
Date of designation: 27/07/2020, Positive, Last updated: 24/09/2021 -
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Orphan designation: (4R,5R)-1-[[4-[[4-[3,3-dibutyl-7-(dimethylamino)-2,3,4,5- tetrahydro-4-hydroxy-1,1-dioxido-1-benzothiepin-5-yl]phenoxy]methyl]phenyl]methyl]-4-aza-1-azoniabicyclo[2.2.2]octane chloride (maralixibat chloride) for: Treatment of primary sclerosing cholangitis (updated)
Date of designation: 16/01/2014, Positive, Last updated: 24/09/2021 -
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Orphan designation: (4R,5R)-1-[[4-[[4-[3,3-dibutyl-7-(dimethylamino)-2,3,4,5- tetrahydro-4-hydroxy-1,1-dioxido-1-benzothiepin-5-yl]phenoxy]methyl]phenyl]methyl]-4-aza-1-azoniabicyclo[2.2.2]octane chloride (maralixibat chloride) for: Treatment of primary biliary cirrhosis (updated)
Date of designation: 16/01/2014, Positive, Last updated: 24/09/2021 -
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Orphan designation: (4R,5R)-1-[[4-[[4-[3,3-dibutyl-7-(dimethylamino)-2,3,4,5- tetrahydro-4-hydroxy-1,1-dioxido-1-benzothiepin-5-yl]phenoxy]methyl]phenyl]methyl]-4-aza-1-azoniabicyclo[2.2.2]octane chloride (maralixibat chloride) for: Treatment of Alagille syndrome (updated)
Date of designation: 16/01/2014, Positive, Last updated: 24/09/2021 -
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Orphan designation: humanised fusion protein consisting of extracellular domain of CD24 linked to IgG1 Fc domain for: Prevention of graft-versus-host disease (updated)
Date of designation: 11/11/2015, Positive, Last updated: 24/09/2021 -
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Orphan designation: eptacog alfa (activated) for: Treatment of diffuse alveolar haemorrhage (updated)
Date of designation: 14/12/2005, Withdrawn, Last updated: 21/09/2021 -
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Orphan designation: drotrecogin alfa (activated) for: Treatment of acute respiratory distress syndrome (ARDS) (updated)
Date of designation: 22/09/2008, Withdrawn, Last updated: 21/09/2021 -
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Orphan designation: 1-(2-isopropoxyethyl)-2-thioxo-1,2,3,5-tetrahydro-pyrrolo[3,2-d] pyrimidin-4-one (verdiperstat) for: Treatment of multiple system atrophy (updated)
Date of designation: 16/12/2014, Positive, Last updated: 21/09/2021 -
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Orphan designation: Infigratinib for: Treatment of cholangiocarcinoma (updated)
Date of designation: 21/08/2020, Positive, Last updated: 21/09/2021 -
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Orphan designation: Recombinant human IgG1 kappa light chain monoclonal antibody targeting plasma kallikrein (lanadelumab) for: Treatment of hereditary angioedema (updated)
Date of designation: 09/10/2015, Positive, Last updated: 20/09/2021 -
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Orphan designation: Allogeneic CD4+ and CD25+ T lymphocytes ex vivo incubated with GP120 for: Treatment in haematopoietic stem cell transplantation (updated)
Date of designation: 22/02/2018, Positive, Last updated: 17/09/2021 -
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Orphan designation: Maribavir for: Prevention of cytomegalovirus disease in patients with impaired cell-mediated immunity (updated)
Date of designation: 18/12/2007, Positive, Last updated: 17/09/2021 -
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Orphan designation: Maribavir for: Treatment of cytomegalovirus disease in patients with impaired cell-mediated immunity (updated)
Date of designation: 07/06/2013, 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-polycythaemia vera myelofibrosis (updated)
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 (updated)
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 (updated)
Date of designation: 01/10/2010, Positive, Last updated: 17/09/2021 -
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Orphan designation: perflubron for: Treatment of respiratory distress syndrome (updated)
Date of designation: 09/12/2020, Positive, Last updated: 16/09/2021 -
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Orphan designation: Perflubron for: Treatment of congenital pulmonary hypoplasia (updated)
Date of designation: 13/11/2020, Positive, Last updated: 16/09/2021 -
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Orphan designation: paclitaxel for: Treatment of soft tissue sarcoma (updated)
Date of designation: 17/10/2019, Positive, Last updated: 16/09/2021 -
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Orphan designation: 4-oxo-4H-chromene-2-carboxylic acid (2-(2-4-(2-(6,7-dimethoxy-3,4-dihydro-1H-isoquinolin-2-yl)-ethyl)-phenyl-2H-tetrazol-5-yl)-4,5-dimethoxy-phenyl)-amide (encequidar) for: Treatment of soft tissue sarcoma (updated)
Date of designation: 17/10/2019, Positive, Last updated: 16/09/2021