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129 results
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Orphan designation: Talquetamab for: Treatment of multiple myeloma (updated)
Date of designation: 20/08/2021, Positive, Last updated: 21/09/2023 -
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Orphan designation: Ganaxolone for: Treatment of CDKL5 deficiency disorder
Date of designation: 13/11/2019, Positive, Last updated: 31/07/2023 -
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Orphan designation: Glofitamab for: Treatment of diffuse large B-cell lymphoma
Date of designation: 15/10/2021, Positive, Last updated: 18/07/2023 -
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Orphan designation: Recombinant human acid sphingomyelinase (also known as olipudase alfa) for: Treatment of Niemann-Pick disease
Date of designation: 24/06/2022, Positive, Last updated: 18/07/2023 -
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Orphan designation: (1R,2R)-octanoic acid[2-(2',3'-dihydro-benzo[1,4] dioxin-6'-yl)-2-hydroxy-1-pyrrolidin-1-ylmethyl-ethyl]-amide-L-tartaric acid salt (eliglustat) for: Treatment of Gaucher disease
Date of designation: 04/12/2007, Positive, Last updated: 18/07/2023 -
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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: 18/07/2023 -
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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: 17/07/2023 -
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Orphan designation: Autologous CD34+ cells transfected with retroviral vector containing adenosine deaminase gene for: Treatment of severe combined immunodeficiency (SCID) due to adenosine deaminase (ADA) deficiency
Date of designation: 26/08/2005, Positive, Last updated: 27/06/2023 -
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Orphan designation: Ivosidenib for: Treatment of biliary tract cancer
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
Date of designation: 12/12/2016, Positive, Last updated: 12/05/2023 -
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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
Date of designation: 18/11/2016, Positive, Last updated: 04/05/2023 -
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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: 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: 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: 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: cholic acid for: Treatment of inborn errors in primary bile acid synthesis
Date of designation: 18/12/2002, Positive, Last updated: 11/11/2022 -
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Orphan designation: Burosumab for: Treatment of phosphaturic mesenchymal tumour
Date of designation: 16/04/2018, Positive, Last updated: 08/11/2022