Press release
Evkeeza® (evinacumab-dgnb) ANGPTL3 Antibody Approved in the U.S. for Children as Young as 1 Year Old with Ultra-Rare Form of High Cholesterol
FDA approval extends the indication of Evkeeza to treat younger patients with homozygous hypercholesterolemia (HoFH) HoFH is an inherited condition
About this update from Regeneron Pharmaceuticals, Inc.
[{"type":"text","content":"FDA approval extends the indication of Evkeeza to treat younger patients with homozygous hypercholesterolemia (HoFH) HoFH is an inherited condition characterized by extremely high levels of low-density lipoprotein cholesterol (LDL-C) Initial Evkeeza approval based on 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 TARRYTOWN, N.Y., Sept. 26, 2025 (GLOBE NEWSWIRE) -- Regeneron Pharmaceuticals, Inc. (NASDAQ: REGN) today announced that the U.S. Food and Drug Administration (FDA) has approved 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. It was then approved for children aged 5 to 11 in 2023. All Evkeeza FDA submissions were evaluated under Priority Review, which is reserved for medicines that represent potentially significant improvements in efficacy or safety in treating serious conditions. HoFH is the most severe form of familial hypercholesterolemia (FH), and affects approximately 1,300 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 (usually >400 mg/dL) of low-density lipoprotein cholesterol (LDL-C). Those living with HoFH are at risk for premature atherosclerotic disease and cardiac events even in their teenage years. Many patients are not diagnosed or are only diagnosed later in life. “The approval of Evkeeza for children as young as 1 year of age addresses a critical unmet need for those with homozygous familial hypercholesterolemia, a life-threatening condition that causes extraordinarily high LDL-C levels from birth,” said Katherine Wilemon, Founder and CEO of the Family Heart Foundation. “Families and their medical teams will now have an effective treatment option for these very young children who are at risk of serious complications f...