Amgen (NASDAQ: AMGN) today announced positive data from the HAUSER-RCT Phase 3b 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. 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.

HeFH is an inherited, genetic condition with a prevalence of one in 250 people worldwide.1 High levels of low-density lipoprotein cholesterol (LDL-C) starting at birth accelerate the development of atherosclerotic cardiovascular disease, leading to an overall increased risk of cardiovascular events, including heart attack and other vascular conditions, and decreasing the age at which such events occur.2 Children with FH can be normal weight, have a good diet, exercise enough, and still have high LDL-C.2,3 The risk of heart disease in people with FH is about 20 times greater versus the general population.4

"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, M.D., Ph.D., 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."

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.5 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 24.5 No new safety risks were identified.5 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.5

"Amgen is dedicated to advancing the care and improving the lives of patients with cardiovascular disease. This includes investing in clinical trials and real-world evidence studies to better understand the safety and efficacy of Repatha across various patient populations and those most in need," said David M. Reese, M.D., executive vice president of research and development at Amgen. "This study increases our overall evidence base for Repatha and provides us with a better understanding of cholesterol management in children with genetically high LDL-C, bringing us one step closer to another treatment option for this historically underdiagnosed and undertreated condition."

"As a parent, it can be hard to understand that your child who looks healthy, eats well and is active, is suffering from an invisible condition that can cause an early heart attack or stroke," said Katherine Wilemon, Founder and CEO of The FH Foundation. "The good news is that with early and ongoing treatment, people with FH can greatly reduce cardiovascular risk by lowering their LDL-cholesterol. This trial data gives us hope for new, safe and effective therapies for children living with familial hypercholesteremia."