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FDA Accepts Evinacumab Biologics License Application for Priority Review as a Treatment for Patients with HoFH, an Ultra-rare Inherited Form of High Cholesterol
TARRYTOWN, N.Y., Aug. 12, 2020 /PRNewswire/ -- Homozygous familial hypercholesterolemia affects approximately 1,300 patients in the U.S., the vast majority of
About this update from Regeneron Pharmaceuticals, Inc.
[{"type":"text","content":"TARRYTOWN, N.Y., Aug. 12, 2020 /PRNewswire/ --\nHomozygous familial hypercholesterolemia affects approximately 1,300 patients in the U.S., the vast majority of whom are unable to reach target LDL-C levels with currently available therapies\nRegeneron Pharmaceuticals, Inc. (NASDAQ: REGN) today announced that the U.S. Food and Drug Administration (FDA) has accepted for Priority Review a Biologics License Application (BLA) for evinacumab as an adjunct to other lipid-lowering therapies in patients with homozygous familial hypercholesterolemia (HoFH). Evinacumab is the first investigational medicine of its kind to show efficacy in patients with HoFH – including patients with little to no low-density lipoprotein (LDL) receptor function – by binding to and blocking the function of angiopoietin-like 3 (ANGPTL3). \nThe target action date for the FDA decision is February 11, 2021. The FDA granted evinacumab Breakthrough Therapy designation in 2017 for the treatment of hypercholesterolemia in patients with HoFH.\nHoFH is an ultra-rare inherited disease that affects approximately 1,300 patients in the U.S. 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. \nThe BLA was supported by a pivotal Phase 3 trial evaluating the efficacy and safety of evinacumab in patients with HoFH. Results from this trial were presented as a late-breaking presentation at the American College of Cardiology's Annual Scientific Session together with World Congress of Cardiology (ACC.20) in March 2020.\nPrevious research published in NEJM in 2017 by the Regeneron Genetics Center found that patients with loss-of-function mutations in their ANGPTL3 gene have significantly lower levels of key blood lipids, including LDL-C. By blocking the ANGPTL3 protein, evinacumab was designed to replicate this loss-of-function mutation effect to lower LDL-C in patients with HoFH. \nRegulatory submissions for evinacumab are also progressing in the Europea...