摘要: Givosiran (Givlaari™) is an aminolevulinate synthase 1 (ALAS1)-directed small interfering RNA (siRNA) covalently linked to a ligand enable specific delivery of the siRNA hepatocytes. This results in downregulation ALAS1 mRNA and prevents accumulation neurotoxic δ-aminolevulinic acid porphobilinogen levels that are associated with acute porphyria attacks. being developed by Alnylam Pharmaceuticals for treatment hepatic (AHP). In November 2019, givosiran was approved USA adults AHP based on positive from multinational, phase III ENVISION trial. EU, received opinion January 2020 adolescents aged 12 years older. article summarizes milestones development leading this first approval AHP.
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