Regeneron’s Evkeeza Receives FDA Approval to Treat HoFH in Children Aged 1 to Less Than 5

Regeneron Pharmaceuticals Inc. (NASDAQ:REGN) is one of the best NASDAQ stocks to buy according to hedge funds. On September 26, Evkeeza (evinacumab-dgnb), an ANGPTL3 antibody developed by Regeneron Pharmaceuticals, received an extended indication approval from the US FDA for the treatment of children from age 1 to less than 5 years old with homozygous familial hypercholesterolemia/HoFH.

Evkeeza is used as an adjunct to diet, exercise, and other lipid-lowering therapies. HoFH is an ultra-rare, inherited condition and the most severe form of familial hypercholesterolemia, affecting ~1,300 people in the US. It results in dangerously high levels of low-density lipoprotein cholesterol/LDL-C, usually >400 mg/dL, putting patients at risk for premature atherosclerotic disease and cardiac events even in their teenage years.

Regeneron's Evkeeza Receives FDA Approval to Treat HoFH in Children Aged 1 to Less Than 5

Evkeeza was initially approved in 2021 for adults and adolescents aged 12 years and older with HoFH, based on a placebo-controlled trial showing it could lower LDL-C by about 50% compared to placebo when added to standard lipid-lowering therapies. The indication was subsequently extended in 2023 for children aged 5 to 11. All FDA submissions for Evkeeza were evaluated under Priority Review.

Regeneron Pharmaceuticals Inc. discovers, invents, develops, manufactures, and commercializes medicines for treating various diseases worldwide.

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This article is originally published at Insider Monkey.