Amgen's Repatha Scores FDA Nod For Pediatric Patients Over 10 Years With Inherited Heart Disease

  • The FDA has approved Amgen Inc's (NASDAQ:AMGN) Repatha (evolocumab) as an adjunct for pediatric patients aged 10 years and older with heterozygous familial hypercholesterolemia (HeFH) to reduce LDL-C.
  • Repatha is approved as an add-on to diet and other low-density lipoprotein cholesterol (LDL-C)-lowering therapies.
  • Related Content: Amgen's Repatha Improves Plaque Stability In Heart Patients
  • HeFH is an inherited genetic condition characterized by high levels of LDL-C starting at birth that accelerate the development of atherosclerotic cardiovascular disease at an earlier age.
  • The approval is based on the HAUSER-RCT Phase 3b study. Monthly treatment with Repatha reduced LDL-C by a mean of 38% from baseline compared to placebo, meeting its primary endpoint.
  • Repatha had improved secondary lipid parameters from baseline compared to the placebo.
  • The FDA also approved Repatha as an adjunct to other LDL-C lowering therapies to treat homozygous familial hypercholesterolemia (HoFH) for younger pediatric patients. Repatha was already approved for HoFH patients aged 13 and older.
  • Related content: Benzinga's Full FDA Calendar.
  • Price Action: AMGN stock closed 0.67% lower at $213.61 on Friday.
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