Posted by Michael Wonder on 06 Apr 2017
Regeneron announces evinacumab has received FDA breakthrough therapy designation for homozygous familial hypercholesterolaemia
6 April 2017 - Regeneron Pharmaceuticals today announced that the U.S. FDA has granted breakthrough therapy designation status to evinacumab for the treatment of hypercholesterolemia in patients with homozygous familial hypercholesterolemia (HoFH), an inherited disorder that can lead to premature cardiovascular disease due to very high levels of LDL cholesterol.
Evinacumab is an investigational monoclonal antibody to angiopoietin-like protein 3 (ANGPTL3). ANGPTL3 acts as an inhibitor of lipoprotein lipase and endothelial lipase, and appears to play a central role in lipoprotein metabolism.
Regeneron previously reported positive interim phase 2 results for evinacumab in HoFH patients and is currently planning a phase 3 trial.
