Regeneron Pharmaceuticals announced that the U.S. Food and Drug Administration (FDA) has approved Evkeeza (evinacumab-dgnb), an ANGPTL3 antibody, for use in children as young as one year old with homozygous familial hypercholesterolemia (HoFH). This approval expands the drug’s indication to include patients from age 1 to less than 5 years old, as an adjunct to diet, exercise, and other lipid-lowering therapies.
Evkeeza was initially approved in 2021 for adults and adolescents aged 12 years and older with HoFH. The initial approval was based on a placebo-controlled trial which showed that adding Evkeeza to standard lipid-lowering therapies could lower low-density lipoprotein cholesterol (LDL-C) by about 50% compared to placebo. In 2023, its use was extended to children aged 5 to 11. All FDA submissions for Evkeeza have been evaluated under Priority Review due to its potential benefits in treating a serious condition.
HoFH is a rare inherited disorder that leads to extremely high LDL-C levels. It affects approximately 1,300 people in the United States and results from inheriting two copies of genes causing familial hypercholesterolemia—one from each parent. People with HoFH often experience dangerously high LDL-C levels (usually over 400 mg/dL), putting them at risk for early-onset atherosclerotic disease and cardiac events during their teenage years.
Katherine Wilemon, Founder and CEO of the Family Heart Foundation, stated: “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. Families and their medical teams will now have an effective treatment option for these very young children who are at risk of serious complications from diseased arteries and calcified valves without timely and sufficient LDL-C lowering. This development underscores the importance of, and urgency needed in, identifying children with FH through pediatric screenings in accordance with guidelines.”
The latest label extension is supported by clinical efficacy and safety data collected from six children with HoFH who participated in either the U.S. expanded access program or international compassionate use programs for Evkeeza. No new safety concerns were identified during these programs. The most common adverse reactions included nasopharyngitis, influenza-like illness, dizziness, rhinorrhea, nausea, and fatigue.
George D. Yancopoulos, M.D., Ph.D., Board co-Chair, President and Chief Scientific Officer at Regeneron said: “Evkeeza is a testament to the power of Regeneron’s science and proprietary technologies in developing first-in-class, lifechanging medicines that become standard-of-care. This label extension adds to our growing commitment to the rare disease space, which includes diverse clinical development programs in neuromuscular and genetic diseases – such as myasthenia gravis, otoferlin hearing loss, and fibrodysplasia ossificans progressiva – for which we’ve shared data.”
Regeneron offers support through its myRARE patient program providing financial assistance for eligible patients prescribed Evkeeza along with resources like product information and insurance benefit verification.
Evkeeza was developed using Regeneron’s VelocImmune technology platform—a genetically engineered mouse model designed to produce fully human antibodies optimized for therapeutic use. The company discovered the angiopoietin gene family more than twenty years ago; research published in the New England Journal of Medicine found that individuals whose ANGPTL3 gene did not function properly had significantly lower blood lipid levels—including LDL-C—and reduced risk of coronary artery disease.
Regeneron manages development and distribution of Evkeeza within the U.S., while collaborating with Ultragenyx on international efforts.
