Abstract
Primary hyperoxaluria type 1 (PH1) is a rare genetic disorder characterized by hepatic overproduction of oxalate. Excretion of excess oxalate by the kidneys leads to recurrent kidney stones, nephrocalcinosis, progressive kidney disease, and multiorgan damage from systemic oxalosis. Lumasiran is a subcutaneously administered, liver-directed RNA interference (RNAi) therapeutic to reduce urinary oxalate (UOx) excretion in patients with PH1. Treatment with lumasiran demonstrated substantial reductions in UOx excretion and an acceptable safety profile in pediatric and adult patients with PH1.
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