Regeneron's Evkeeza Wins Expanded FDA Nod for Children

Regeneron Pharmaceuticals (REGN) has secured an expanded U.S. Food and Drug Administration (FDA) approval for its cholesterol-lowering drug, Evkeeza, now cleared to treat children between the ages of five and 11. The decision addresses a critical unmet need for young patients with homozygous familial hypercholesterolemia (HoFH), a rare and life-threatening genetic condition.

HoFH causes dangerously high levels of low-density lipoprotein (LDL) cholesterol from birth, leading to aggressive and premature cardiovascular disease. This expanded approval provides a crucial new treatment option for a vulnerable population at high risk for early cardiac events. The in pediatric patients, which demonstrated the drug's ability to significantly lower LDL-C when added to other lipid-lowering therapies.

First approved in 2021 for patients 12 and older, Evkeeza is the first and only FDA-approved treatment that works by binding to and blocking the function of angiopoietin-like 3 (ANGPTL3), a protein that plays a key role in lipid metabolism. For Regeneron, the expanded label strengthens Evkeeza's market position and opens a new, albeit niche, revenue stream within its cardiovascular and rare disease franchise.

"This approval for a younger age group is a significant milestone for families affected by HoFH," commented a leading pediatric cardiologist. "Effectively managing LDL cholesterol from the earliest possible age is critical to slowing the progression of cardiovascular damage."

While the patient population for HoFH is small, treatments for such often command high prices due to the significant research and development investment. The highlights the serious nature of the condition and the drug's potential to provide a meaningful therapeutic benefit where few options exist. This strategic win reinforces Regeneron's focus on developing therapies for severe diseases with limited or no available treatments.