The U.S. Food and Drug Administration (FDA) has approved Evkeeza (evinacumab-dgnb) for the treatment of children ages 1 to 5 years old with homozygous familial hypercholesterolemia (HoFH), supplemental to diet, exercise, and other lipid-lowering therapies.
HoFH is a metabolic condition characterized by very high levels of cholesterol in the blood due to genetic changes in the LDLR gene. People with HoFH have a high risk of developing coronary artery disease, as well as health problems related to the buildup of excessive cholesterol in other tissues.
Evinacumab is a fully human monoclonal antibody that binds to and blocks the function of angiopoietin-like 3, a protein that inhibits lipoprotein lipase and endothelial lipase and regulates circulating lipids. It was approved by the FDA in 2021 for adults and adolescents ages 12 years and older with HoFH based on data from a placebo-controlled trial. This trial illustrated the ability of evinacumab, when combined with standard lipid-lowering therapies, could lower lipoprotein cholesterol by about 50% compared with placebo. In 2023, it was then approved for patients ages 5 to 11 years.
This extended indication was supported by clinical efficacy and safety data from six children with HoFH who participated in the U.S. expanded access program or ex-U.S. compassionate use program. No new safety concerns have been reported and the most common adverse reactions are nasopharyngitis, influenza-like illness, dizziness, rhinorrhea, nausea, and fatigue.
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To learn more about HoFH and other rare metabolic disorders, visit https://checkrare.com/diseases/metabolic-disorders/
