FDA approval for PCSK9 siRNA to lower LDL-cNews - Dec. 23, 2021
The FDA has approved the small interfering RNA (siRNA) PCSK9 therapy inclisiran to lower LDL-c with two doses a year, after a first dose and one at 3 months.
Inclisiran is indicated in the US as adjunct to diet and maximally tolerated statins for the treatment of adults with clinical ASCVD or heterozygous familial hypercholesterolemia (HeFH).
“As a first-of-its-kind siRNA therapy, inclisiran works differently than other cholesterol treatments, with twice-yearly dosing that makes it a compelling option for the millions of people with ASCVD already on cholesterol-lowering medications struggling to reach their LDL-C target.” said Norman Lepor, MD, a cardiologist and a clinical investigator in the Phase III clinical program for inclisiran.
The FDA approval was based on results form the phase III ORION-9, -10, and -11 trials. In the phase III trials, inclisiran resulted in effective and sustained LDL-c reduction up to 52% after 17 months, compared to placebo in participants with ASCVD or HeFH. It was well-tolerated with a safety profile similar to placebo.
Clinical trials are ongoing to assess the effect of inclisiran on CV morbidity and mortality.