Regeneron Pharma Evkeeza under USFDA priority review for children with Ultra-rare inherited form of high cholesterol

The sBLA is supported by data from a three-part trial evaluating Evkeeza in children aged 5 to 11 years with HoFH. Efficacy was assessed in 14 children enrolled in the Part B portion of the trial. Despite treatment with other lipid-lowering therapies, these children entered the trial with an average LDL-C level of 264 mg/dL, more than twice the target (<110 mg/dL) for pediatric patients with HoFH. The trial met its primary endpoint, showing children who added Evkeeza to other lipid-lowering therapies reduced their LDL-C by 48% at week 24 on average. Furthermore, 79% (n=11) saw their LDL-C reduced by at least half at 24 weeks following Evkeeza treatment, with an average absolute reduction in LDL-C from baseline of 132 mg/dL.

Evkeeza is the first angiopoietin-like 3 (ANGPTL3) targeted therapy approved by the FDA, European Commission, and the United Kingdom's Medicines and Healthcare products Regulatory Agency as an adjunct therapy for patients aged 12 years and older with HoFH. The potential use of Evkeeza in HoFH patients aged 5 to 11 years has not been fully evaluated by any regulatory authority.