A Phase 3b clinical trial, HAUSER-RCT, evaluating Amgen’s (NASDAQ:AMGN) Repatha (evolocumab) in pediatric patients aged 10-17 years old with heterozygous familial hypercholesterolemia (HeFH) showed that the addition of the PCSK9 inhibitor to statins and other lipid-lowering therapies significantly reduced LDL-C (“bad” cholesterol) compared to placebo. The data were virtually presented at the European Society of Cardiology Annual Meeting and published in The New England Journal of Medicine.
The study met the primary endpoint demonstrating a statistically significant 38.3% average relative reduction from baseline in LDL-C compared to control. The absolute mean reduction in LDL-C was 68.6 mg/dL (the eligibility criterion for trial was a minimum of 130 mg/dL).
The FDA approved Repatha five years ago for adults with HeFH.