TARRYTOWN, N.Y., Feb. 11, 2021 /PRNewswire/ —

Homozygous familial hypercholesterolemia (HoFH) is an ultra-rare inherited condition that affects approximately 1,300 patients in the U.S. and is characterized by extremely high low-density lipoprotein cholesterol (LDL-C)

In pivotal Phase 3 HoFH trial, adding Evkeeza to standard lipid-lowering therapies reduced LDL-C by nearly half at 24 weeks, compared to placebo

Regeneron Pharmaceuticals, Inc. (NASDAQ: REGN) today announced that the U.S. Food and Drug Administration (FDA) approved Evkeeza^TM (evinacumab-dgnb) as an adjunct to other low-density lipoprotein cholesterol (LDL-C) lowering therapies to treat adult and pediatric patients aged 12 years and older with homozygous familial hypercholesterolemia (HoFH). Evkeeza is the first FDA-approved treatment that binds to and blocks the function of angiopoietin-like 3 (ANGPTL3), a protein that plays a key role in lipid metabolism.

“The FDA’s approval of Evkeeza is a watershed moment for individuals born with HoFH, a severe form of familial hypercholesterolemia,” said Katherine A. Wilemon, Founder and CEO of the FH Foundation. “Those living with HoFH have faced devastatingly high LDL-C levels and an uncertain future. Evkeeza significantly lowered LDL-C levels in clinical trials and this new treatment offers an important new option for people living with HoFH.”

HoFH, also known as homozygous FH, is an ultra-rare inherited condition that affects approximately 1,300 patients in the U.S. HoFH occurs when two copies of the familial hypercholesterolemia (FH)-causing genes are inherited, one from each parent, resulting in dangerously high levels (>400 mg/dL) of LDL-C (bad cholesterol). Patients with HoFH are at risk for premature atherosclerotic disease and cardiac events as early as their teenage years.