Regeneron Pharmaceuticals announced today the extended FDA approval of Evkeeza (evinacumab-dgnb) for the treatment of homozygous familial hypercholesterolemia (HoFH) in patients from age one to less than 5 years old, according to a news release .

Evkeeza is an ANGPTL3 antibody inhibitor, initially approved by the FDA in 2021 for adults and pediatric HoFH patients ages 12 and older. In 2023, the indication expanded to include children as young as 5 years old. It works by binding to and blocking the function of angiopoietin-like 3, a protein that regulates circulating lipids.

HoFH is the most severe form of familial hypercholesterolemia (FH), a genetic disorder that impacts how the body recycles low-density lipoprotein cholesterol (LDL-C), sometimes referred to as “bad cholesterol.

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