Evinacumab-dgnb is the first FDA-approved treatment that binds to and blocks the function of angiopoietin-like 3, a protein that plays a key role in lipid metabolism.
Evinacumab-dgnb (Evkeeza; Regeneron Pharmaceuticals, Inc.) was approved earlier this year as an adjunct to other low-density lipoprotein cholesterol (LDL-C) lowering therapies to treat patients aged 12 years and older with homozygous familial hypercholesterolemia (HoFH). According to Regeneron, evinacumab-dgnb 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.
HoFH, also known as homozygous FH, is an ultra-rare inherited condition that affects approximately 1300 patients in the United States HoFH occurs when 2 copies of the familial hypercholesterolemia (FH)-causing genes are inherited, 1 from each parent, resulting in dangerously high levels (>400 mg/dL) of LDL-C. Patients with HoFH are at risk for premature atherosclerotic disease and cardiac events as early as their teenage years.