Regeneron Announces FDA Approval of Evkeeza for Pediatric HoFH Patients Aged 5–11

On March 30, Regeneron announced that the FDA had approved Evkeeza (evinacumab) as an adjunctive therapy to other lipid-lowering drugs for pediatric patients aged 5-11 with homozygous familial hypercholesterolemia (HoFH). The drug was previously approved by the FDA in February 2021 for use in individuals aged 12 and above.

HofH is an extremely rare genetic disorder, affecting approximately 1,300 patients in the United States, and it represents the most severe form of familial hypercholesterolemia (FH). HoFH occurs when two copies of the gene responsible for causing FH are inherited, leading to elevated levels of low-density lipoprotein cholesterol (LDL-C) or harmful cholesterol (typically >400 mg/dL). Individuals with HoFH are at risk of early atherosclerotic disease and cardiac events, even during adolescence.

Evkeeza, developed by Regeneron, is a drug targeting angiopoietin-like protein 3 (ANGPTL3). It was granted Breakthrough Therapy designation by the FDA for the treatment of HoFH in 2017. In February 2021, the drug was launched in the United States as an adjunctive therapy to other lipid-lowering medications for pediatric patients aged 12 years and older and adults with homozygous familial hypercholesterolemia (HoFH), becoming the first ANGPTL3 monoclonal antibody approved by the FDA.

In May this year, Regeneron announced that the Phase III clinical trial of evinacumab for 5-11-year-old HoFH pediatric patients who had received other lipid-lowering drug treatments met the primary endpoint. The results showed that after 24 weeks of treatment, the average LDL-C level in these patients decreased by 48%.

The average LDL-C level of the pediatric subjects enrolled in the study was 264 mg/dL, more than twice the target value (<130 mg/dL). After 24 weeks of Evkeeza treatment (15mg/kg intravenous infusion every 4 weeks), the trial met its primary endpoint. The results are as follows:

• 79% of patients had their LDL-C reduced by at least half;

• Compared with baseline, LDL-C was reduced by an average of 132 mg/dL;

• All lipid endpoint parameters evaluated, such as apolipoprotein B, non-high-density lipoprotein cholesterol, lipoprotein(a), and total cholesterol, showed decreased levels within the first 8 weeks of treatment.

In terms of safety, subjects generally tolerated Evkeeza well. The most common adverse events (AEs) were sore throat, upper abdominal pain, diarrhea, headache, and nasopharyngitis. Additionally, there were two serious AEs (aortic valve stenosis and tonsillitis), which were unrelated to the treatment.

Regeneron Chief Scientific Officer George D. Yancopoulos said, "Since its initial approval, Evkeeza has become the standard therapy for treating HoFH patients aged 12 and above. We are pleased that patients as young as 5 years old can now benefit from it. As an innovative drug for HoFH, Evkeeza demonstrates the promising potential of genetics-based research to transform treatment paradigms."

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