Positive topline data from a phase 3b study evaluating evolocumab (Repatha) for the treatment of heterozygous familial hypercholesterolemia (HeFH) in pediatric patients were presented at the European Society of Cardiology (ESC) Congress 2020.

The multicenter, double-blind, placebo-controlled HAUSER-RCT study assessed the efficacy and safety of evolocumab in 157 patients aged 10 to 17 years with HeFH on an approved statin therapy. Patients were randomized 2:1 to receive either evolocumab 420mg subcutaneously once monthly (n=104) or placebo (n=53) for 24 weeks. The primary end point was the percent change in low-density lipoprotein cholesterol (LDL-C) from baseline to week 24.

Results showed that treatment with evolocumab reduced LDL-C by 38.3% compared with placebo, and was associated with a mean absolute reduction in LDL-C of 68.6mg/dL. Moreover, evolocumab demonstrated improvements in secondary end points, including a 42.1% reduction in mean LDL-C from weeks 22 to 24, a 35% 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 a 36.4% reduction in ApoB/apolipoprotein A1 (ApoA1) ratio at week 24.

As for safety, there were no new safety risks identified. The most common treatment-emergent adverse events (>2%) proportionally higher (>1%) in the evolocumab arm compared with placebo were headache, oropharyngeal pain, influenza, influenza-type illness, upper respiratory tract infection and constipation.

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“Pediatric patients with FH are at increased risk for cardiovascular events from a very early age, making effective management of LDL-C levels in children with HeFH so important,” said Daniel Gaudet, MD, PhD, from the Department of Medicine at the Université de Montréal and senior author of the Hauser-RCT study. “This study shows the potential that Repatha offers as a safe and effective treatment option in pediatric HeFH patients already on lipid-lowering therapies who need further LDL-C reduction.”

Repatha® (Amgen), a proprotein convertase subtilisin kexin type 9 (PCSK9) inhibitor, is currently indicated to reduce the risk of myocardial infarction, stroke, and coronary revascularization in adults with established cardiovascular disease. It is also approved as an adjunct to diet, alone or in combination with other lipid-lowering therapies (eg, statins, ezetimibe) for the treatment of adults with primary hyperlipidemia (including HeFH) to reduce LDL-C; and as an adjunct to diet and other LDL-lowering therapies (eg, statins, ezetimibe, LDL apheresis) in patients with homozygous familial hypercholesterolemia who require additional lowering of LDL-C.

For more information visit amgen.com.


Amgen announces positive data from phase 3B study of Repatha® (evolocumab) In pediatric patients with heterozygous familial hypercholesterolemia at ESC Congress 2020. https://www.prnewswire.com/news-releases/amgen-announces-positive-data-from-phase-3b-study-of-repatha-evolocumab-in-pediatric-patients-with-heterozygous-familial-hypercholesterolemia-at-esc-congress-2020-301120643.html. Accessed August 31, 2020.