Regeneron wins FDA priority review for label expansion of dyslipidemia therapy
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Regeneron (NASDAQ:REGN) announced Wednesday that the FDA accepted under its priority review a supplemental Biologics License Application (sBLA) for Evkeeza to expand its existing indication to children with homozygous familial hypercholesterolemia (HoFH).
An ultra-rare genetically driven condition, HoFH affects about 1,300 patients in the U.S. With the sBLA Regeneron (REGN) seeks FDA approval for Evkeeza as an adjunct to other lipid-lowering agents in children aged 5 to 11 years with HoFH.

The company backs the sBLA with data from a three-part trial which met the primary endpoint indicating a 48% average reduction in LDL-C (bad cholesterol) at week 24 in HoFH children treated with Evkeeza. The FDA has issued Mar. 30, 2023, as the target action date for its decision.
In 2021, the FDA approved Evkeeza for HoFH patients aged 12 years and older as an adjunct to other lipid-lowering therapies.

In January, Regeneron (REGN) announced a partnership with Ultragenyx Pharmaceutical (RARE) to commercialize and distribute Evkeeza in overseas markets.