Regeneron's Evkeeza Wins FDA Nod for Young Children with Rare Cholesterol Disorder
Label expansion for the ANGPTL3 inhibitor now includes patients as young as one year old, broadening the addressable market for this ultra-rare disease treatment.
Regeneron Pharmaceuticals, Inc. (NASDAQ: REGN) announced it has secured a significant label expansion from the U.S. Food and Drug Administration (FDA) for its cholesterol-lowering drug, Evkeeza® (evinacumab-dgnb). The approval now extends the drug's use to children as young as one year old suffering from homozygous familial hypercholesterolemia (HoFH), an ultra-rare and life-threatening genetic disorder.
This latest regulatory milestone marks a critical advancement for a small, vulnerable patient population. HoFH is characterized by dangerously high levels of low-density lipoprotein cholesterol (LDL-C), which can lead to premature and aggressive cardiovascular disease. for the estimated 1,300 people in the U.S. affected by the condition, offering a new therapeutic option for the youngest patients who previously had limited choices.
Originally approved in 2021 for adults and adolescents, Evkeeza's indication was first expanded in March 2023 to children aged five to eleven. This new approval for toddlers and young children was granted under Priority Review, a designation reserved for medicines that may offer significant improvements in treating serious conditions. The decision was supported by efficacy and safety data from a study of six children with HoFH, where .
Wall Street has reacted positively to the news, viewing the label expansion as a strategic win that strengthens Regeneron's position in the lucrative rare disease market. While the patient population for HoFH is small, the high price of orphan drugs can translate into significant revenue. , with some projecting targets as high as $831 per share, reflecting confidence in the company's growth trajectory and robust pipeline.
Evkeeza is an antibody that works by inhibiting angiopoietin-like 3 (ANGPTL3), a protein that plays a key role in lipid metabolism. In clinical trials, the drug has been shown to reduce LDL-C levels by approximately 50% in patients who are already receiving other lipid-lowering therapies. The expanded approval for Evkeeza not only provides a much-needed treatment for a devastating pediatric disease but also reinforces for patients with high unmet medical needs.