FDA Approves Amgen’s Repatha for Autosomal Dominant Disease

FDA recently approved Amgen’s Repatha to treat pediatric patients ten and older with autosomal dominant disease, heterozygous familial hypercholesterolemia (HeFH), to reduce low-density lipoprotein cholesterol (LDL-C). 

Specifically, the agency approved the therapy 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,” David M. Reese, MD, executive vice president of research and development at Amgen, said in the announcement. 

“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,” Reese continued. 

The FDA based its approval of Repatha on the randomized, double-blind HAUSER-RCT Phase 3b study evaluating the safety and efficacy of Repatha in pediatric patients 10 to 17 years of age with HeFH. 

In the trial, Repatha reduced LCL-C by 38 percent from baseline compared to placebo.

Repatha also improved lipid parameters from baseline compared to placebo, including a 35 percent reduction in total cholesterol at week 24 and a 32 percent reduction in apolipoprotein B (ApoB) at week 24.

Notably, no safety risks were identified. 

HeFH is an inherited genetic condition that affects just one in 250 people globally. High levels of LDL-C accelerate the development of atherosclerotic cardiovascular disease, leading to an overall increased risk of cardiovascular events. 

Dietary and lifestyle modifications are the main treatment options for LDL‐C lowering in patients with HeFH. But multidrug treatment is often required to achieve adequate LDL‐C levels.

Atherosclerotic cardiovascular disease risk factors and comorbidities, such as hypertension and diabetes mellitus, should also be treated.  

Repatha is also currently approved as an adjunct to other LDL-C lowering therapies to treat homozygous familial hypercholesterolemia (HoFH) for pediatric patients. And the therapy is now available as a treatment for patients ten years of age and older as well. 

“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.