Givosiran sodiumProduct ingredient for Givosiran

Givosiran is a small interfering RNA (siRNA) directed towards 5-aminolevulinic acid synthase, a critical enzyme in the heme biosynthesis pathway.⁵ It is manufactured by Alnylam Pharmaceuticals and was first approved for use in the United States in November 2019 for the treatment of adults with acute hepatic porphyria, a genetic disorder in which the overproduction of toxic heme intermediates leads to neuro-, nephro-, and gastrotoxicity.⁵ Givosiran represents an important step forward in the treatment of acute hepatic porphyria as it is the first approved pharmacotherapy for the prevention of acute attacks - previous strategies involved non-therapeutic measures (e.g. trigger avoidance), intravenous hemin for the treatment of attacks, and liver transplantation in refractory cases.³ Givosiran is the second-ever FDA-approved member of the siRNA drug class (the first being patisiran), a new class of drugs promising an important and exciting step forward in the treatment of genetic disorders.