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250 results
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Orphan designation: Ivosidenib for: Treatment of biliary tract cancer (updated)
Date of designation: 21/03/2018, Positive, Last updated: 12/05/2023 -
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Orphan designation: Ivosidenib for: Treatment of acute myeloid leukaemia (updated)
Date of designation: 12/12/2016, Positive, Last updated: 12/05/2023 -
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Orphan designation: 1-[(3S)-3-{4-amino-3-[(3,5-dimethoxyphenyl)ethynyl]-1H-pyrazolo[3,4-d]pyrimidin-1-yl}pyrrolidin-1-yl]-2-propen-1-one (futibatinib) for: Treatment of biliary tract cancer (updated)
Date of designation: 01/04/2019, Positive, Last updated: 08/05/2023 -
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Orphan designation: 2-hydroxy-6-((2-(1-isopropyl-1H-pyrazol-5-yl)pyridin-3-yl)methoxy)benzaldehyde (voxelotor) for: Treatment of sickle cell disease (updated)
Date of designation: 18/11/2016, Positive, Last updated: 04/05/2023 -
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Orphan designation: Gallium (68Ga)-edotreotide for: Diagnosis of gastro-entero-pancreatic neuroendocrine tumours (updated)
Date of designation: 19/03/2015, Positive, Last updated: 03/05/2023 -
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Orphan designation: Lutetium (177Lu)-N-[(4,7,10-Tricarboxymethyl-1,4,7,10-tetraazacyclododec-1-yl)acetyl]-D-phenylalanyl-L-cysteinyl-L-tyrosyl-D-tryptophanyl-L-lysyl-L-threoninyl-L-cysteinyl-L-threonine-cyclic(2-7)disulfide (lutetium (177Lu) oxodotreotide) for: Treatment of gastro-entero-pancreatic neuroendocrine tumours (updated)
Date of designation: 31/01/2008, Positive, Last updated: 03/05/2023 -
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Orphan designation: Fenfluramine hydrochloride for: Treatment of Lennox-Gastaut syndrome
Date of designation: 27/02/2017, Positive, Last updated: 25/04/2023 -
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Orphan designation: Fenfluramine hydrochloride for: Treatment of Dravet syndrome
Date of designation: 18/12/2013, Positive, Last updated: 25/04/2023 -
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Orphan designation: arimoclomol citrate for: Treatment of Niemann-Pick's disease, type C
Date of designation: 19/11/2014, Positive, Last updated: 11/04/2023 -
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Orphan designation: Tobramycin for: Treatment of Pseudomonas aeruginosa lung infection in cystic fibrosis
Date of designation: 17/03/2003, Positive, Last updated: 03/04/2023 -
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Orphan designation: Recombinant fusion protein consisting of a modified form of the extracellular domain of human activin receptor IIB linked to the human IgG1 Fc domain (luspatercept) for: Treatment of beta thalassaemia intermedia and major
Date of designation: 29/07/2014, Positive, Last updated: 21/03/2023 -
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Orphan designation: Panobinostat for: Treatment of multiple myeloma
Date of designation: 08/11/2012, Positive, Last updated: 13/03/2023 -
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Orphan designation: Treprostinil sodium for: Treatment of pulmonary arterial hypertension and chronic thromboembolic pulmonary hypertension
Date of designation: 14/04/2004, Positive, Last updated: 13/02/2023 -
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Orphan designation: Phosphorothioate oligonucleotide targeted to apolipoprotein C-III (volanesorsen) for: Treatment of familial chylomicronaemia syndrome
Date of designation: 19/02/2014, Positive, Last updated: 02/02/2023 -
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Orphan designation: Allogeneic Epstein-Barr virus specific cytotoxic T lymphocytes (Tabelecleucel) for: Treatment of post-transplantation lymphoproliferative disorders
Date of designation: 21/03/2016, Positive, Last updated: 30/01/2023 -
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Orphan designation: Bardoxolone methyl for: Treatment of Alport syndrome
Date of designation: 25/05/2018, Positive, Last updated: 30/01/2023 -
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Orphan designation: Autologous T cells transduced with retroviral vector encoding an anti-CD19 CD28/CD3 zeta chimeric antigen receptor (axicabtagene ciloleucel) for: Treatment of diffuse large B-cell lymphoma
Date of designation: 16/12/2014, Positive, Last updated: 16/12/2022 -
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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
Date of designation: 18/12/2013, Positive, Last updated: 14/12/2022 -
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Orphan designation: mitapivat sulfate for: Treatment of pyruvate kinase deficiency
Date of designation: 22/04/2020, Positive, Last updated: 07/12/2022 -
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Orphan designation: Autologous peripheral blood T cells CD4 and CD8 selected and CD3 and CD28 activated transduced with retroviral vector expressing anti-CD19 CD28/CD3-zeta chimeric antigen receptor and cultured (brexucabtagene autoleucel) for: Treatment of acute lymphoblastic leukaemia
Date of designation: 19/10/2020, Positive, Last updated: 02/12/2022 -
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Orphan designation: Maribavir for: Treatment of cytomegalovirus disease in patients with impaired cell-mediated immunity
Date of designation: 07/06/2013, Positive, Last updated: 24/11/2022 -
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Orphan designation: Maribavir for: Prevention of cytomegalovirus disease in patients with impaired cell-mediated immunity
Date of designation: 18/12/2007, Positive, Last updated: 24/11/2022 -
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Orphan designation: 6alpha-ethyl-chenodeoxycholic acid for: Treatment of primary biliary cirrhosis
Date of designation: 27/07/2010, Positive, Last updated: 21/11/2022 -
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Orphan designation: Cyclic pyranopterin monophosphate (fosdenopterin) for: Treatment of molybdenum-cofactor deficiency type A
Date of designation: 20/09/2010, Positive, Last updated: 18/11/2022 -
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Orphan designation: Humanised IgG4 monoclonal antibody against total complement component 1, subcomponent s (sutimlimab) for: Treatment of autoimmune haemolytic anaemia
Date of designation: 17/02/2016, Positive, Last updated: 18/11/2022