Posted by Michael Wonder on 12 Aug 2020
FDA accepts evinacumab biologics license application for priority review as a treatment for patients with HoFH, an ultra-rare inherited form of high cholesterol
12 August 2020 - Homozygous familial hypercholesterolaemia affects approximately 1,300 patients in the U.S., the vast majority of whom are unable to reach target LDL-C levels with currently available therapies.
Regeneron Pharmaceuticals today announced that the U.S. Food and Drug Administration (FDA) has accepted for Priority Review a Biologics License Application (BLA) for evinacumab as an adjunct to other lipid-lowering therapies in patients with homozygous familial hypercholesterolemia (HoFH). Evinacumab is the first investigational medicine of its kind to show efficacy in patients with HoFH – including patients with little to no low-density lipoprotein (LDL) receptor function – by binding to and blocking the function of angiopoietin-like 3 (ANGPTL3).
The target action date for the FDA decision is 11 February 2021. The FDA granted evinacumab breakthrough therapy designation in 2017 for the treatment of hypercholesterolaemia in patients with HoFH.
