FDA Approves Evkeeza for Kids With Ultra-Rare Cholesterol Disorder

The FDA has expanded Regeneron’s Evkeeza (evinacumab-dgnb) approval to include treatment of children aged five years old to 11 suffering from homozygous familial hypercholesterolemia (HoFH), an ultra-rare form of high cholesterol.

Evkeeza, an angiopoietin-like protein 3 inhibitor, was originally approved in February 2021 as an adjunct to other lipid-lowering drugs in patients 12 years and older with HoFH.

The approval was supported by a pediatric study of Evkeeza in patients previously treated with lipid-lowering therapies that demonstrated an average reduction of 48 percent in low-density lipoprotein cholesterol (LDL-C) at week 24.

HoFH, the most severe manifestation of familial hypercholesterolemia, afflicts an estimated 1,300 people in the U.S., and is caused by two copies of the FH-inducing gene being inherited from each parent which results in dangerous levels of LDL-C.