FDA Approves Evkeeza for Young Children with Ultra-Rare Form of High Cholesterol

Regeneron Pharmaceuticals announced on March 22, 2023 that FDA has extended the approval of Evkeeza (evinacumab-dgnb) as an adjunct to other lipid-lowering therapies to treat children aged five to 11 with homozygous familial hypercholesterolemia (HoFH), an inherited condition characterized by extremely high low-density lipoprotein cholesterol (LDL-C). The treatment was initially approved in February 2021 as an adjunct to other lipid-lowering therapies in those aged 12 years and older with HoFH.

HoFH is an ultra-rare inherited condition that affects approximately 1300 people in the United States and is the most severe form of familial hypercholesterolemia, according to a company press release. Those living with HoFH are at risk for premature atherosclerotic disease and cardiac events even in their teenage years.

“At the Family Heart Foundation, we know that children with homozygous familial hypercholesterolemia, and those caring for them, often live in fear of what the future holds as they contend with the dangerously high levels of bad cholesterol, or LDL-C, caused by this genetic disorder,” said Mary McGowan, chief medical officer of the Family Heart Foundation, in the release. “Only 5% of rare diseases actually have an FDA-approved treatment. With this FDA approval, the HoFH community now has a much-needed treatment for young children, potentially making it possible for many to achieve recommended LDL-C levels much earlier in the course of this rare disease. This is a hopeful development for those living with HoFH.”
Source: Regeneron