The U.S. Food and Drug Administration (FDA) on Friday approved Regeneron Pharmaceuticals, Inc.’s (NASDAQ: REGN) Evkeeza (evinacumab-dgnb) ANGPTL3 antibody as an adjunct to diet and exercise and other lipid-lowering therapies for the treatment of children from age 1 to less than 5 years old with homozygous familial hypercholesterolemia (HoFH).
Evkeeza was initially approved in 2021 for adults and adolescents aged 12 years and older with HoFH based on a placebo-controlled trial showing Evkeeza, when added to standard lipid-lowering therapies, could lower LDL-C by about 50% compared to placebo in this high unmet need population.
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It was then approved for children aged 5 to 11 in 2023.
HoFH is the most severe form of familial hypercholesterolemia (FH), and affects approximately 1.3 million people in the U.S.
It occurs when two copies of the FH-causing genes are inherited, one from each parent, resulting in dangerously high levels of low-density lipoprotein cholesterol (LDL-C).
The extended indication for Evkeeza was supported by clinical efficacy and safety data among six children living with HoFH.
No new safety concerns have been identified in the compassionate use program.
The most common adverse reactions (≥5%) of Evkeeza include nasopharyngitis, influenza-like illness, dizziness, rhinorrhea, nausea, and fatigue.
Price Action: REGN stock is down 0.76% at $551.28 at the last check on Friday.
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