REGN 1500 filed with the FDA for homozygous familial hypercholesterolemia.- Regeneron Pharmaceuticals

Regeneron Pharmaceuticals announced that the FDA has accepted for Priority Review a Biologics License Application (BLA) for REGN 1500 (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 February 11, 2021. The FDA granted evinacumab Breakthrough Therapy designation in 2017 for the treatment of hypercholesterolemia in patients with HoFH.The BLA was supported by a pivotal Phase III trial evaluating the efficacy and safety of evinacumab in patients with HoFH. Results from this trial were presented as a late-breaking presentation at the American College of Cardiology's Annual Scientific Session together with World Congress of Cardiology (ACC.20) in March 2020.