New England Journal of Medicine Publishes Positive Phase 3 Evinacumab Results in Patients with Severe Inherited Form of High Cholesterol

Regeneron Pharmaceuticals, Inc. today announced that the New England Journal of Medicine (NEJM) published positive results from the Phase 3 trial of evinacumab in 65 patients with homozygous familial hypercholesterolemia (HoFH). Evinacumab is an investigational medicine that binds to and blocks the function of angiopoietin-like 3 (ANGPTL3), and is the first medicine of its kind to show efficacy in patients with HoFH – including patients with little to no low-density lipoprotein (LDL) receptor function.

Patients with HoFH have severely elevated levels of bad cholesterol (low-density lipoprotein cholesterol, or LDL-C), which increases their risk for premature atherosclerotic disease and cardiac events as early as their teenage years. Treatment guidelines recommend early and intensive LDL-C lowering, but patients with HoFH are less responsive (or unresponsive) to standard lipid-lowering therapies, including statins and PCSK9 (proprotein convertase subtilisin/kexin type 9) inhibitors.

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