The FDA Approves Repatha for Children with Rare Genetic Disease

The FDA has approved Amgen’s 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).

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 can be normal weight, have a good diet, exercise enough and still have high LDL-C.

Katherine Wilemon

"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," Katherine Wilemon, founder and chief executive officer at The FH Foundation, said in a statement.

The approval is based on the HAUSER-RCT phase 3b study evaluating the safety and efficacy of Repatha in pediatric patients 10 to 17 years of age with HeFH. Monthly treatment with Repatha reduced LDL-C by a mean of 38% from baseline compared with placebo. At week 24, patients treated with Repatha had improved secondary lipid parameters from baseline in comparison with placebo, including a 35% reduction in non-high-density lipoprotein cholesterol, a 27% reduction in total cholesterol, and a 32% reduction in apolipoprotein B.

No new safety risks were identified. The most common treatment-emergent adverse events included nasopharyngitis, headache, oropharyngeal pain, influenza, and upper respiratory tract infection.

Repatha is a monoclonal antibody that is also approved to treat adults with HeFH; myocardial infarction, stroke, and coronary revascularization; and adults and children over the age of 10 to treat homozygous familial hypercholesterolemia.