FDA approves Evkeeza for young children with ultra-rare form of high cholesterol

The U.S. Food and Drug Administration has extended the approval of evinacumab-dgnb (Evkeeza; Regeneron) as an adjunct to other lipid-lowering therapies to treat children aged 5 to 11 with homozygous familial hypercholesterolemia (HoFH), according to a press release.

Evinacumab was approved in February 2021 as an adjunct to other lipid-lowering therapies in those aged 12 years and older with HoFH.

“Since it was first approved, Evkeeza has become the standard of care for homozygous familial hypercholesterolemia in those aged 12 years or older. We’re gratified that now children as young as 5 years old have the potential to benefit from this treatment,” said George D. Yancopoulos, MD, PhD., President and Chief Scientific Officer at Regeneron, in a press release. “As a first-in-class medicine for this relentless disease, Evkeeza exemplifies the promise of genetics-based research to transform treatment paradigms. Evkeeza’s journey from target discovery to treatment innovation was only made possible due to our long-term investment in genetics research and monoclonal antibody technologies, and this remains a central tenet of our science-first approach to this day.”

Read the full press release here.