Heterozygous familial hypercholesterolemia is a disorder characterized by an increased level of LDL cholesterol, resulting in an increased risk of premature atherosclerotic cardiovascular disease. Inclisiran is an experimental drug for the treatment of atherosclerotic cardiovascular disease and heterozygous familial hypercholesterolemia, but its efficacy is not well established. This study aims to evaluate the effects of inclisiran in patients with heterozygous familial hypercholesterolemia.

This randomized, double-blind, phase-3 trial included a total of 482 patients (mean age 56 years, 47% men) with heterozygous familial hypercholesterolemia. The patients were randomly assigned in a 1:1 ratio to receive subcutaneous injections of inclisiran sodium (300 mg) or matching placebo. The primary outcome of the study was the percentage change in LDL cholesterol level from baseline (153 mg per dL).

At a follow-up of 510 days, the percent reduction in LDL cholesterol from baseline was 39.7% in the inclisiran group, as compared with an 8.2% increase in the placebo group. The time-adjusted percentage change from baseline between day 90 and day 540 was a 38.1% reduction in the inclisiran group and a 6.2% increase in the placebo group.

The research concluded that inclisiran was associated with reduced LDL cholesterol levels from baseline in patients with heterozygous familial hypercholesterolemia.

Ref: https://www.nejm.org/doi/full/10.1056/NEJMoa1913805