Approval Based on HAUSER-RCT Study Demonstrating a Significant Reduction in LDL-C
Amgen today announced that the U.S. Food and Drug Administration (FDA) has approved Repatha® (evolocumab) as an adjunct to diet and other LDL cholesterol (LDL-C)-lowering therapies for the treatment of pediatric patients aged 10 years and older with heterozygous hypercholesterolemia (HeFH) to scale back LDL-C.
HeFH is an inherited, genetic condition with a prevalence of 1 in 250 people worldwide.1 High levels of LDL-C starting at birth accelerate the event of atherosclerotic disorder , resulting in an overall increased risk of cardiovascular events, including attack and other vascular conditions, at an earlier age.2 Children with hypercholesterolemia (FH) are often normal weight, have an honest diet, exercise enough and still have high LDL-C.2,3
“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,” said David M. Reese, M.D., executive vice chairman of Research and Development at Amgen. “This milestone further reinforces the security profile of Repatha and aligns with Amgen’s commitment to addressing the unmet needs of the high-risk cardiovascular community.”
The approval is predicated on the HAUSER-RCT Phase 3b study evaluating the security and efficacy of Repatha in pediatric patients, 10 – 17 years aged , 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.4 Reductions in LDL-C were observed by the primary post-baseline assessment at the Week 12 time point and were maintained throughout the trial.4 Patients treated with Repatha had improved secondary lipid parameters from baseline as compared 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.5 the foremost common treatment-emergent adverse events (>5% of patients treated with Repatha and occurring more frequently than placebo) included nasopharyngitis, headache, oropharyngeal pain, influenza and upper tract infection.5
“As pediatric FH is an under-recognized condition which will cause premature arteria coronaria disease, it’s critically important to possess additional treatments which will significantly lower cholesterol,” said Katherine Wilemon, founder and chief military officer at The FH Foundation.
The FDA also approved Repatha as an adjunct to other LDL-C lowering therapies for the treatment of homozygous hypercholesterolemia (HoFH) for younger pediatric patients. Repatha was already approved for treatment in HoFH patients aged 13 and older and is now available as a treatment for patients aged 10 and older.
About hypercholesterolemia
Elevated LDL cholesterol (LDL-C) is an abnormality of cholesterol and/or fats within the blood.5,6 hypercholesterolemia (FH) is an inherited condition that causes high levels of LDL-C at an early age7. it’s estimated that 1 million people within the U.S. have FH (heterozygous and homozygous forms), yet but 10% are diagnosed.8 Heterozygous FH (HeFH) is that the more common sort of FH and occurs globally in approximately 1 in 250.9 People with HeFH have a 50% chance of passing the condition to their children.8
About HAUSER-RCT Study Design
HAUSER-RCT was a Phase 3b, multicenter, randomized (2:1), double-blind, placebo-controlled study evaluating the efficacy, safety, and tolerability of 24 weeks of monthly subcutaneous injections of Repatha® (evolocumab) 420 mg (n = 104) versus placebo (n = 53) in patients 10 to 17 years aged with heterozygous familial hypercholesteremia, or HeFH. Randomization was stratified by LDL-C
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