FDA has accepted for Priority Review the supplemental Biologics License Application for Evkeeza in homozygous familial hypercholesterolemia

The FDA target action date is March 30, 2023.

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.></110>

Among 20 children evaluated for long-term safety across Parts A, B and C of the trial (mean exposure: 52 weeks, range: 42-64 weeks), the most common adverse events (AEs) occurring in ?15% of patients included COVID-19 (n=15), pyrexia (n=5), headache (n=4), throat pain (oropharyngeal pain, n=4) as well as upper abdominal pain, diarrhea, vomiting, fatigue, nasopharyngitis, rhinitis and cough (all n=3). Most reported AEs were mild or moderate, and none led to study discontinuation. The safety profile of Evkeeza observed in these patients was generally consistent to those seen in adults and pediatric patients aged 12 years and older.