The US Food and Drug Administration (FDA) has approved Amgen’s Repatha (evolocumab) to treat paediatric patients aged ten years and above with heterozygous familial hypercholesterolemia (HeFH).
The drug received approval as an adjunct to diet and other low-density lipoprotein cholesterol (LDL-C)-lowering treatment to decrease LDL-C in this patient population.
Increased LDL-C is an abnormality of cholesterol and/or fats in the blood and familial hypercholesterolemia (FH) is an inherited ailment that leads to high LDL-C levels at an early age.
High levels of LDL-C that begin at birth speed up atherosclerotic cardiovascular disease development, which will increase risk of cardiovascular events such as heart attack and other vascular conditions.
Repatha, a human monoclonal antibody, inhibits proprotein convertase subtilisin/kexin type 9 (PCSK9).
Amgen Research and Development executive vice-president David Reese said: “The approval of Repatha for paediatric 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.
“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 FDA approval is based on the multi-centre, randomised, double-blind, placebo-controlled Phase IIIb HAUSER-RCT trial, which assessed the efficacy, safety and tolerability of Repatha in paediatric patients aged ten to 17 years with HeFH.
Data showed that the monthly injections of Repatha reduced LDL-C by mean 38% and improved secondary lipid parameters compared to placebo.
It also showed a 35% decrease in non-high-density lipoprotein cholesterol (non-HDL-C), a 27% reduction in complete cholesterol and a 32% lowering of apolipoprotein B (ApoB) at week 24.
Amgen stated that new safety risks were not identified when using Repatha in the trial.
The most common adverse reactions include influenza, headache, nasopharyngitis, oropharyngeal pain and upper respiratory tract infection.