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Regeneron Pharma Evkeeza under USFDA priority review for children with Ultra-rare inherited form of high cholesterol
Tarrytown: Regeneron Pharmaceuticals, Inc. has announced the U.S. Food and Drug Administration (USFDA) has accepted for Priority Review the supplemental Biologics License Application (sBLA) for Evkeeza (evinacumab-dgnb) as an adjunct to other lipid-lowering therapies to treat children aged 5 to 11 years with homozygous familial hypercholesterolemia (HoFH). The FDA target action date is March 30, 2023.
HoFH is an ultra-rare inherited condition that affects approximately 1,300 patients in the U.S. and 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.
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.
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Ruchika Sharma joined Medical Dialogue as an Correspondent for the Business Section in 2019. She covers all the updates in the Pharmaceutical field, Policy, Insurance, Business Healthcare, Medical News, Health News, Pharma News, Healthcare and Investment. She has completed her B.Com from Delhi University and then pursued postgraduation in M.Com. She can be contacted at editorial@medicaldialogues.in Contact no. 011-43720751