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Amgen Announces Positive Data From Phase 3B Study Of Repatha® (Evolocumab) In Pediatric Patients With Heterozygous Familial Hypercholesterolemia At ESC Congress 2020
First Phase 3 Trial Evaluating a PCSK9 Inhibitor When Added to Lipid-Lowering Therapy in Pediatric HeFH Population Repatha Significantly Reduced LDL-C and
About this update from Amgen Inc.
[{"type":"text","content":"First Phase 3 Trial Evaluating a PCSK9 Inhibitor When Added to Lipid-Lowering Therapy in Pediatric HeFH Population\n Repatha Significantly Reduced LDL-C and Improved All Secondary Lipid Parameters\n\n\nTHOUSAND OAKS, Calif., Aug. 29, 2020 /PRNewswire/ -- Amgen (NASDAQ:AMGN) today announced positive data from the HAUSER-RCT Phase 3b study evaluating the safety and efficacy of Repatha® (evolocumab) in pediatric patients, 10-17 years of age, with heterozygous familial hypercholesterolemia (HeFH). The study showed that Repatha, in combination with statins and other lipid-lowering therapies, significantly reduced low-density lipoprotein cholesterol (LDL-C) compared to placebo. These data are being presented during an oral presentation at ESC 2020 – The Digital Experience, organized by the European Society of Cardiology, Aug. 29–Sept. 1 and simultaneously published in The New England Journal of Medicine. \nHeFH is an inherited, genetic condition with a prevalence of one in 250 people worldwide.1 High levels of low-density lipoprotein cholesterol (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, and decreasing the age at which such events occur.2 Children with FH can be normal weight, have a good diet, exercise enough, and still have high LDL-C.2,3 The risk of heart disease in people with FH is about 20 times greater versus the general population.4 \n\"Pediatric patients with FH are at increased risk for cardiovascular events from a very early age, making effective management of LDL-C levels in children with HeFH so important,\" said Daniel Gaudet, M.D., Ph.D., from the Department of Medicine at the Université de Montréal and senior author of the Hauser-RCT study. \"This study shows the potential that Repatha offers as a safe and effective treatment option in pediatric HeFH patients already on lipid-lowering therapies who need further LDL-C reduction.\" \nResults from the randomized, double-blind 24-week study show that in pediatric patients with HeFH, monthly treatment with Repatha reduced LDL-C by mean 38.3% from baseline compared to placebo, and absolute reduction in LDL-C was 68.6 mg/dL (mean absolute reduction) meeting its primary endpoint and showing superiority of evol...