Regeneron Pharmaceuticals has achieved a significant regulatory milestone, receiving FDA approval to administer its cholesterol-lowering drug Evkeeza to children as young as one year old. This authorization represents a crucial expansion into a previously underserved patient population and marks the latest step in the therapy’s progressive label extension.
Addressing Rare Genetic Condition in Youngest Patients
The U.S. Food and Drug Administration has cleared Evkeeza for treating children aged 1-5 years who suffer from homozygous familial hypercholesterolemia, an extremely rare inherited disorder. This decision follows earlier approvals for adults and adolescents from 12 years upward, then from 5 years upward, demonstrating continued confidence in the ANGPTL3 antibody therapy. Clinical evidence indicates that Evkeeza, when combined with standard treatments, can reduce LDL cholesterol levels by approximately 50 percent. For pediatric patients, this early intervention opportunity could prevent life-threatening cardiovascular complications.
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Financial Foundation Amid Market Challenges
Despite facing stock market pressure throughout the year, Regeneron continues to demonstrate financial resilience. The biotechnology firm reported second-quarter 2025 revenue of $3.68 billion, representing a 4 percent increase, while adjusted earnings per share jumped 12 percent to $12.89. Evkeeza contributed $41 million to U.S. product revenue during this period, establishing a growing presence in the rare disease market. This solid operational performance supports an ambitious development pipeline featuring approximately 45 product candidates in clinical trials.
Market Impact Considerations
While the expanded approval undoubtedly strengthens Regeneron’s position within the lucrative rare disease sector, questions remain about its ability to reverse the stock’s downward trajectory. Although accessing new patient groups offers long-term potential, the extreme rarity of the treated condition suggests immediate revenue impact may be modest. Market observers will be watching closely to determine whether additional pipeline successes will be necessary to generate sustained momentum for Regeneron’s shares.
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