What is the mechanism of action of Lumasiran?
Lumasiran is a subcutaneously administered small interfering RNA targeting the mRNA for hydroxyacid oxidase 1 gene that is used for the treatment of primary hyperoxaluria type 1 (PH1). By silencing the gene encoding glycolate oxidase, lumasiran depletes glycolate oxidase and thereby inhibits the synthesis of oxalate, which is the toxic metabolite that is directly associated with the clinical manifestations of PH1.