FDA approves Repatha for the treatment of pediatric patients with heterozygous familial hypercholesterolemia .-Amgen

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 is based on the HAUSER-RCT Phase IIIb 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 24.5 No new safety risks were identified. The most common treatment-emergent adverse events (greater than 5% of patients treated with Repatha and occurring more frequently than placebo) included nasopharyngitis, headache, oropharyngeal pain, influenza and upper respiratory tract infection..