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.

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. (NASDAQ:REGN) discovers, invents, develops, manufactures, and commercializes medicines for treating various diseases worldwide.

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Disclosure: None. This article is originally published at Insider Monkey.