Regeneron's Evkeeza Reduces Bad Cholesterol By 48% In Children Aged 5-11 Years

  • Regeneron Pharmaceuticals Inc REGN announced positive results from a Phase 3 trial evaluating Evkeeza (evinacumab) in children aged 5 to 11 with homozygous familial hypercholesterolemia (HoFH). 
  • HoFH is a disorder where it is hard for the body to remove LDL "bad" cholesterol from the blood, raising the chances of a heart attack at an early age.
  • The trial met its primary endpoint. It showed that children who added Evkeeza to other lipid-lowering therapies reduced their low-density lipoprotein-cholesterol (LDL-C) by 48% at week 24 on average
  • Regulatory submission to the FDA is expected later this year.
  • Despite treatment with other lipid-lowering therapies, children (n=14) entered the trial with an average LDL-C level of 264 mg/dL, more than twice the target (<130 mg/dL) for pediatric patients with HoFH. 
  • After 24 weeks of Evkeeza treatment, 79% of patients reduced their LDL-C by at least half. On average, an absolute 132 mg/dL reduction in LDL-C from baseline.
  • Evkeeza was generally well-tolerated, with all patients completing the trial. The most common adverse events (AEs) were throat pain (21%) as well as upper abdominal pain, diarrhea, headache, and nasopharyngitis (all 14%). 
  • Two severe AEs (aortic stenosis and tonsilitis) were reported, both considered unrelated to treatment.
  • Evkeeza is approved in the U.S. and European Commission as an adjunct therapy for certain patients aged 12 years and older with HoFH. 
  • Regeneron is responsible for developing and distributing Evkeeza in the U.S. and is collaborating with Ultragenyx Pharmaceutical Inc RARE to clinically develop, commercialize, and distribute Evkeeza outside of the U.S.
  • Price Action: REGN shares closed higher by 0.61% at $661.14 on Friday.
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