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Orphan designation: Rozanolixizumab for: Treatment of myasthenia gravis
Date of designation: 22/04/2020, Positive, Last updated: 13/08/2020 -
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Orphan designation: cusatuzumab for: Treatment of acute myeloid leukaemia
Date of designation: 22/04/2020, Positive, Last updated: 11/08/2020 -
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Orphan designation: DNA plasmid encoding IL-12 p35 and p40 genes for: Treatment of ovarian cancer
Date of designation: 22/04/2020, Positive, Last updated: 11/08/2020 -
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Orphan designation: Adeno-associated viral vector serotype 3B encoding human multidrug resistance protein 3A for: Treatment of progressive familial intrahepatic cholestasis
Date of designation: 22/04/2020, Positive, Last updated: 11/08/2020 -
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Orphan designation: florbetaben (18F) for: Diagnosis of AL amyloidosis
Date of designation: 22/04/2020, Positive, Last updated: 12/08/2020 -
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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: haematopoietic stem cells and blood progenitors umbilical cord-derived expanded with (1R, 4R)-N1-(2-benzyl-7-(2-methyl-2H-tetrazol-5-yl)-9H-pyrimido[4,5-b]indol-4-yl)cyclohexane-1,4-diamine dihydrobromide dihydrate for: Treatment in haematopoietic stem cell transplantation
Date of designation: 20/04/2020, Positive, Last updated: 12/08/2020 -
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Orphan designation: anti-(integrin beta-3) human monoclonal antibody for: Prevention of fetal and neonatal alloimmune thrombocytopenia due to human platelet antigen-1a incompatibility
Date of designation: 22/04/2020, Positive, Last updated: 13/08/2020 -
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Orphan designation: adeno-associated virus vector serotype hu37 encoding human factor VIII for: Treatment of haemophilia A
Date of designation: 22/04/2020, Positive, Last updated: 13/08/2020 -
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Orphan designation: Glucagon analogue linked to a human immunoglobulin Fc fragment for: Treatment of insulin autoimmune syndrome
Date of designation: 22/04/2020, Positive, Last updated: 13/08/2020