Study: Evolocumab Significantly Reduces LDL Cholesterol in Pediatric Patients With Heterozygous Familial Hypercholesterolemia

Evolocumab in combination with statins and other lipid-lowering therapies was found to significantly reduce low-density lipoprotein cholesterol in pediatric patients with heterozygous familial hypercholesterolemia.

According to positive results from the recent HAUSER-RCT phase 3b study, evolocumab (Repatha, Amgen) used in combination with statins and other lipid-lowering therapies was able to significantly reduce low-density lipoprotein cholesterol (LDL-C) in pediatric patients between 10 and 17 years of age with heterozygous familial hypercholesterolemia (HeFH).

HeFH is an inherited, genetic condition that affects 1 in 250 people globally. Pediatric patients with high levels of LDL-C can be normal weight, follow a healthy diet, exercise regularly, and still have high LDL-C.

High LDL-C can enhance the development of atherosclerotic cardiovascular disease (CVD) and increase the risk of CVD events, such as heart attack and other vascular conditions. These CVD events are also more likely to occur at a younger age for patients with HeFH than the general public, with the risk of heart disease also becoming 20 times more likely.

"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 senior author Daniel Gaudet, MD, PhD, from the Department of Medicine at the Université de Montréal, in a press release. "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."

The results from the randomized, double-blind 24-week study demonstrated that monthly treatment with evolocumab reduced LDL-C in pediatric patients on average by 38.3% from baseline compared with the placebo. Additionally, the average absolute reduction met its primary endpoint by reducing LDL-C by 68.6 mg/dL.

Specifically, from weeks 22 to 24, there was a 42.1% reduction in mean LDL-C. At week 24, there was a 35.0% reduction in non-high-density lipoprotein cholesterol, a 32.5% reduction in apolipoprotein B (ApoB), and a 36.4% reduction in ApoB/apolipoprotein A1 ratio.

In the trial, the researchers did not identify any new safety risks, with the most common adverse events (AEs) appearing in less than 2% of patients. The AEs included headache, oropharyngeal pain, influenza, influenza-type illness, upper respiratory tract infection, and constipation.

"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, in a press release. "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."


Amgen Announces Positive Data From Phase 3B Study Of Repatha® (Evolocumab) In Pediatric Patients With Heterozygous Familial Hypercholesterolemia At ESC Congress 2020. Thousand Oaks, CA: Amgen; August 29, 2020. Accessed August 31, 2020.

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