Amgen has obtained the US Food and Drug Administration (FDA) approval for Repatha (evolocumab) to treat heterozygous familial hypercholesterolemia (HeFH) in children aged 10 years and above.
The drug was indicated as an adjunct to diet and other low-density lipoprotein cholesterol (LDL-C)-lowering therapies to reduce LDL-C in paediatric patients with HeFH.
Repatha is a human monoclonal antibody that inhibits proprotein convertase subtilisin or kexin type 9 (PCSK9) from binding to the low-density lipoprotein (LDL) receptor (LDLR).
It prevents the PCSK9-mediated LDLR degradation, allowing LDLR to recycle back to the liver cell surface, reducing the levels of LDL-C in the blood.
The drug was previously approved in the US, in addition to other LDL-C lowering therapies, to treat homozygous familial hypercholesterolemia (HoFH) in paediatric patients.
Repatha is approved in 75 countries, including the US, Japan, China and the European Union (EU) member states, with regulatory applications under review in other countries.
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 Phase 3b HAUSER-RCT trial, which assessed the safety, efficacy, and tolerability of Repatha in children with HeFH, aged 10 to 17 years.
In the 24-week study, the monthly subcutaneous injections of Repatha 420mg showed reduced LDL-C 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.
Also, patients receiving Repatha showed reduced secondary lipids compared to placebo, with no new safety risks identified.
The treatment reduced non-high-density lipoprotein cholesterol (non-HDL-C) by 35%, the total cholesterol by 27%, and apolipoprotein B (ApoB) by 32%, at week 24.
Nasopharyngitis, headache, oropharyngeal pain, influenza and upper respiratory tract infection include the most common treatment-emergent adverse events, said the biotechnology company.
HeFH is an inherited, genetic condition, characterised by elevated levels of LDL-C, an abnormality of cholesterol in the blood, at an early age.
The disease advances to the development of cardiovascular disease, leading to an overall increased risk of cardiovascular events and other vascular conditions.
