HAUSER-RCT Phase IIIb study of Repatha in heterozygous familial hypercholesterolemia published in NEJM.- Amgen

Amgen announced positive data from the HAUSER-RCT Phase IIIb study evaluating the safety and efficacy of Repatha (evolocumab) in pediatric patients, 10-17 years of age, with heterozygous familial hypercholesterolemia (HeFH). The study showed that Repatha, in combination with statins and other lipid-lowering therapies, significantly reduced low-density lipoprotein cholesterol (LDL-C) compared to placebo. Results from the randomized, double-blind 24-week study show that in pediatric patients with HeFH, monthly treatment with Repatha reduced LDL-C by mean 38.3% from baseline compared to placebo, and absolute reduction in LDL-C was 68.6 mg/dL (mean absolute reduction) meeting its primary endpoint and showing superiority of evolocumab administered on top of statins. Patients treated with Repatha had improved secondary lipid parameters from baseline in comparison to placebo, including a 42.1% reduction in mean LDL-C from weeks 22-24, a 35.0% reduction in non-high-density lipoprotein cholesterol (non-HDL-C) at week 24, a 32.5% reduction in apolipoprotein B (ApoB) at week 24, and 36.4% reduction in ApoB/apolipoprotein A1 (ApoA1) ratio at week 24. No new safety risks were identified. The most common treatment-emergent adverse events (>2%) proportionally higher (>1%) in the Repatha group compared with placebo were headache, oropharyngeal pain, influenza, influenza-type illness, upper respiratory tract infection and constipation. These data are being presented during an oral presentation at ESC 2020 – The Digital Experience, organized by the European Society of Cardiology, Aug. 29–Sept. 1 and simultaneously published in The New England Journal of Medicine. See: "Evolocumab in Pediatric Heterozygous Familial Hypercholesterolemia." Raul D. Santos et al. NEJM August 29, 2020 DOI: 10.1056/NEJMoa2019910