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Orphan designation: Dersimelagon for: Treatment of erythropoietic protoporphyria
Date of designation: 16/03/2022, Positive, Last updated: 21/07/2023 -
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Orphan designation: edaravone for: Treatment of amyotrophic lateral sclerosis
Date of designation: 19/06/2015, Positive, Last updated: 21/07/2023 -
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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
Date of designation: 01/04/2019, Positive, Last updated: 18/07/2023 -
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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 saltĀ (eliglustat) for: Treatment of Gaucher disease
Date of designation: 04/12/2007, Positive, Last updated: 18/07/2023 -
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Orphan designation: Synthetic double-stranded siRNA oligonucleotide directed against antithrombin mRNA and covalently linked to a ligand containing three N-acetylgalactosamine residues (fitusiran) for: Treatment of haemophilia A
Date of designation: 29/07/2014, Positive, Last updated: 18/07/2023 -
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Orphan designation: Synthetic double-stranded siRNA oligonucleotide directed against antithrombin mRNA and covalently linked to a ligand containing three N-acetylgalactosamine residues (fitusiran) for: Treatment of haemophilia B
Date of designation: 29/07/2014, Positive, Last updated: 18/07/2023 -
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Orphan designation: for: Treatment of Fabry disease
Date of designation: 22/08/2014, Positive, Last updated: 18/07/2023 -
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Orphan designation: (3S)-1-azabicyclo[2.2.2]oct-3-yl{2-[2-(4-fluorophenyl)-1,3-thiazol-4-yl]propan-2-yl}carbaNmate (venglustat) for: Treatment of Gaucher disease
Date of designation: 19/11/2014, Positive, Last updated: 18/07/2023 -
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Orphan designation: Humanised IgG4 monoclonal antibody against total complement componentĀ 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: sutimlimab for: Treatment of immune thrombocytopenia
Date of designation: 28/02/2020, Positive, Last updated: 18/07/2023 -
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Orphan designation: Rilzabrutinib for: Treatment of immune thrombocytopenia
Date of designation: 04/06/2020, Positive, Last updated: 18/07/2023 -
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Orphan designation: Venglustat for: Treatment of GM2 gangliosidosis
Date of designation: 21/08/2020, Positive, Last updated: 18/07/2023 -
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Orphan designation: Humanised monoclonal antibody derivative against fibroblast growth factor receptor 3 for: Treatment of achondroplasia
Date of designation: 21/06/2021, Positive, Last updated: 18/07/2023 -
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Orphan designation: Humanised IgG4 monoclonal antibody against active complement component 1, subcomponent s for: Treatment of autoimmune haemolytic anaemia
Date of designation: 18/07/2022, 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: tasimelteon for: Treatment of non-24-hour sleep-wake disorders in blind people with no light perception
Date of designation: 23/02/2011, Positive, Last updated: 30/06/2023 -
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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: (S)-N-(5-(4-(1-(benzo[d][1,3]dioxol-5-yl)ethyl)piperazin-1-yl)-1,3,4-thiadiazol-2-yl)acetamide, hydrochloride salt for: Treatment of progressive supranuclear palsy
Date of designation: 09/12/2020, Positive, Last updated: 27/06/2023 -
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Orphan designation: Recombinant adeno-associated viral vector expressing the human CNGA3 gene for: Treatment of achromatopsia caused by mutations in the CNGA3 gene
Date of designation: 09/10/2015, Positive, Last updated: 27/06/2023 -
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Orphan designation: Zanidatamab for: Treatment of gastric cancer
Date of designation: 13/11/2020, Positive, Last updated: 27/06/2023 -
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Orphan designation: 6-Amino-5-chloro-N-((1R)-1-(5-(((5-chloro-4-(trifluoromethyl)-2-pyridinyl)amino)carbonyl)-2-thiazolyl)ethyl)-4-pyrimidinecarboxamide for: Treatment of glioma
Date of designation: 20/05/2021, Positive, Last updated: 27/06/2023 -
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Orphan designation: Zanidatamab for: Treatment of biliary tract cancer
Date of designation: 19/07/2021, Positive, Last updated: 27/06/2023 -
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Orphan designation: Recombinant human elafin for: Treatment of oesophagus carcinoma
Date of designation: 28/01/2010, Positive, Last updated: 20/06/2023