FDA Approves Repatha ® (evolocumab) in Pediatric Patients Age 10 And Older With Heterozygous Familial Hypercholesterolemia
Amgen (NASDAQ:AMGN) today announced that the U.S. Food and Drug Administration (FDA) has approved Repatha ® (evolocumab) as an adjunct to diet and other low-density lipoprotein cholesterol (LDL-C)-lowering therapies for the treatment of pediatric patients aged 10 years and older with heterozygous familial hypercholesterolemia (HeFH) to reduce LDL-C.
HeFH is an inherited, genetic condition with a prevalence of one in 250 people worldwide. High levels of 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, at an earlier age. Children with familial hypercholesterolemia (FH) can be normal weight, have a good diet, exercise enough and still have high LDL-C.
“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, M.D., executive vice president of Research and Development at Amgen. “This milestone further reinforces the safety profile of Repatha and aligns with Amgen’s commitment to addressing the unmet needs of the high-risk cardiovascular community.”
The approval is based on the HAUSER-RCT Phase 3b study evaluating the safety and efficacy of Repatha in pediatric patients, 10 – 17 years of age, with HeFH. Monthly treatment with Repatha reduced LDL-C by mean 38% (95% CI: 45%, 31%; p < 0.0001) from baseline compared to placebo, meeting its primary endpoint. Reductions in LDL-C were observed by the first post-baseline assessment at the Week 12 time point and were maintained throughout the trial. Patients treated with Repatha had improved secondary lipid parameters from baseline in comparison to placebo, including a 35% (CI: 42%, 28%) reduction in non-high-density lipoprotein cholesterol (non-HDL-C) at week 24, a 27% (CI: 32%, 21%) reduction in total cholesterol at week 24 and a 32% (CI: 39%, 26%) reduction in apolipoprotein B (ApoB) at week 24. No new safety risks were identified. The most common treatment-emergent adverse events (>5% of patients treated with Repatha and occurring more frequently than placebo) included nasopharyngitis, headache, oropharyngeal pain, influenza and upper respiratory tract infection.
“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.
The FDA also approved Repatha as an adjunct to other LDL-C lowering therapies for the treatment of homozygous familial hypercholesterolemia (HoFH) for younger pediatric patients. Repatha was already approved for treatment in HoFH patients aged 13 and older and is now available as a treatment for patients aged 10 and older.