RARE Daily

FDA Grants Priority Review to Regeneron’s Evkeeza for Kids with Ultra-Rare Genetic Form of High Cholesterol

December 1, 2022

The U.S. Food and Drug Administration granted Regeneron Pharmaceuticals Priority Review for its supplemental Biologics License Application for Evkeeza as an adjunct to other lipid-lowering therapies to treat children aged 5 to 11 years with homozygous familial hypercholesterolemia.

Priority Review shortens the time the agency takes to act on an application to six months from 10 months. The FDA is expected to act on the application by March 30, 2023.

Homozygous familial hypercholesterolemia (HoFH) is an ultra-rare inherited condition that affects approximately 1,300 patients in the United States. It is the most severe form of familial hypercholesterolemia (FH). The disease occurs when two copies of the FH-causing genes are inherited, one from each parent, resulting in dangerously high levels (usually >400 mg/dL) of low-density lipoprotein-cholesterol (LDL-C or bad cholesterol). Those living with HoFH are at risk for premature atherosclerotic disease and cardiac events even in their teenage years.

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 application to expand the use of Evkeeza 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 percent at week 24 on average. Furthermore, 79 percent (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.

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 occurring in ≥15 percent of patients included COVID-19, pyrexia, headache, throat pain, as well as upper abdominal pain, diarrhea, vomiting, fatigue, nasopharyngitis, rhinitis and cough. Most reported adverse events 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.

Author: Rare Daily Staff

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