Lumasiran, an RNAi therapeutic for primary hyperoxaluria type 1
New England Journal of Medicine Apr 07, 2021
Garrelfs SF, Frishberg Y, Hulton SA, et al. - Given that lumasiran [an investigational RNA interference therapeutic agent] decreases hepatic oxalate generation by targeting glycolate oxidase, so, researchers tested lumasiran among patients (6 years of age or older) suffering from primary hyperoxaluria type 1 (PH1) in this double-blind, phase 3 trial. They randomized patients (in a 2:1 ratio) to receive subcutaneous lumasiran or placebo for 6 months (with doses given at baseline and at months 1, 2, 3, and 6). Findings revealed that decrease in urinary oxalate excretion (which is the cause of progressive renal failure in PH1) was conferred by treatment with lumasiran in patients with PH1. Following 6 months of treatment with lumasiran, most of the patients exhibited normal or near-normal levels.
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