FDA Approves Evolocumab for Patients Ages 10 and Older With Heterozygous Familial Hypercholesterolemia

The FDA has approved evolocumab (Repatha; Amgen) for the treatment of pediatric patients 10 years of age and older with heterozygous familial hypercholesterolemia (HeFH) to reduce low-density lipoprotein cholesterol (LDL-C).

HeFH is a genetic condition affecting 1 in 250 people worldwide, according to an Amgen press release. It is estimated that 1 million people in the United States have familial hypercholesterolemia (FH), although fewer than 10% are diagnosed, according to the press release. It is characterized by high LDL-C levels starting at birth, which can accelerate the development of atherosclerotic cardiovascular disease. This can lead to an increased risk of cardiovascular events at an early age, including heart attack and other vascular conditions.

According to the press release, children with FH can have a normal weight, a good diet, and adequate exercise, and can still have LDL-C. The drug is approved as an adjunct to diet and other LDL-C-lowering therapies.

“The approval of Repatha for pediatric patients with FH represents a much-needed adjunct treatment option for these children with genetically high cholesterol who are unable to manage their high LDL-C with other lipid-lowering agents alone,” said David M. Reese, MD, executive vice president of research and development at Amgen, in the press release.

The approval is based on data from the HAUSER-RCT phase 3b study, which evaluated the safety and efficacy of evolocumab in pediatric patients between 10 and 17 years of age who were diagnosed with HeFH. According to the press release, 53 patients received a placebo and 104 patients received 420 mg of evolocumab.

Patients were required to be on a low-fat diet and must have been receiving optimized background lipid-lowering therapy. According to the press release, monthly treatment with evolocumab reduced LDL-C by a mean of 38% from baseline compared to placebo, which showed that the study met its primary endpoint.

LDL-C reductions were also observed by the first post-baseline assessment at the week 12 time point and were maintained throughout the trial. Patients who received evolocumab had improved secondary lipid parameters from baseline compared to placebo, including a 35% reduction in non-high-density lipoprotein cholesterol (non-HDL-C), a 27% reduction in total cholesterol, and a 32% reduction in apolipoprotein B at week 24.

No new safety risks were identified in the trial. The most common treatment-emergent adverse events included nasopharyngitis, headache, oropharyngeal pain, influenza, and upper respiratory tract infection, according to the press release.

“As pediatric FH is an under-recognized condition that can lead to premature coronary artery disease, it’s critically important to have additional treatments that can significantly lower cholesterol,” said Katherine Wilemon, founder and chief executive officer at The FH Foundation, in the press release.

REFERENCE

FDA Approves Repatha (evolocumab) In Pediatric Patients Age 10 And Older With Heterozygous Familial Hypercholesterolemia. News release. Amgen; September 24, 2021. Accessed September 27, 2021. https://www.amgen.com/newsroom/press-releases/2021/09/fda-approves-repatha-evolocumab-in-pediatric-patients-age-10-and-older-with-heterozygous-familial-hypercholesterolemia