Inclisiran for the Treatment of Heterozygous Familial Hypercholesterolemia

This phase 3, double-blind, randomized trial evaluated the efficacy and safety of inclisiran a small interfering RNA targeting hepatic PCSK9 synthesis in 482 adults with heterozygous familial hypercholesterolemia. Inclisiran, administered subcutaneously on days 1, 90, 270, and 450, led to a 47.9 percentage point greater reduction in LDL cholesterol at day 510 compared with placebo. Time-averaged LDL reductions between days 90 and 540 were 44.3 percentage points greater in the treatment group. Reductions were consistent across all genotypes and were accompanied by favorable changes in PCSK9, total cholesterol, apolipoprotein B, and non-HDL cholesterol. Adverse events were similar between groups, with mild injection-site reactions more common in the treatment arm. The study concludes that inclisiran offers substantial and sustained LDL reduction with infrequent dosing in patients with familial hypercholesterolemia.

This phase 3, double-blind, randomized trial evaluated the efficacy and safety of inclisiran a small interfering RNA targeting hepatic PCSK9 synthesis in 482 adults with heterozygous familial hypercholesterolemia. Inclisiran, administered subcutaneously on days 1, 90, 270, and 450, led to a 47.9 percentage point greater reduction in LDL cholesterol at day 510 compared with placebo. Time-averaged LDL reductions between days 90 and 540 were 44.3 percentage points greater in the treatment group. Reductions were consistent across all genotypes and were accompanied by favorable changes in PCSK9, total cholesterol, apolipoprotein B, and non-HDL cholesterol. Adverse events were similar between groups, with mild injection-site reactions more common in the treatment arm. The study concludes that inclisiran offers substantial and sustained LDL reduction with infrequent dosing in patients with familial hypercholesterolemia.