A Small Interfering RNA Drug for LDL Management

Inclisiran — a non–FDA-approved, long-acting, synthetic, small interfering RNA (siRNA) molecule — disrupts hepatic production of proprotein convertase subtilisin–kexin type 9 (PCSK9), an enzyme that binds to LDL particles. In a previous phase 2, manufacturer-sponsored, randomized, double-blind, placebo-controlled trial in 501 people, inclisiran was associated at 6 months with LDL reductions of almost 40% after a single 300-mg dose and 52% after two 300-mg doses (NEJM JW Cardiol May 2017 and N Engl J Med 2017; 376:1430). Now, investigators report the 1-year follow-up results.

The participants had a history of either of the following, despite taking lipid-lowering therapy: atherosclerotic cardiovascular disease and LDL ≥70 mg/dL or type 2 diab…