Monoclonal antibody to ANGPTL3 receives FDA Breakthrough Therapy designation for HoFH

Evinacumab is directed against angiopoietin-like protein 3, which acts as an inhibitor of lipoprotein lipase and endothelial lipase. Positive interim phase 2 results with evinacumab have been reported in HoFH.

The U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation status to evinacumab for the treatment of hypercholesterolemia in patients with Homozygous Familial Hypercholesterolemia (HoFH).

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.

Breakthrough Therapy designation was created to expedite the development and review of drugs that target serious or life-threatening conditions. A Breakthrough Therapy drug must show preliminary clinical evidence of a substantial improvement on a clinically significant endpoint over available therapies, or over placebo if there is no available therapy.

Press release Regeneron April 6, 2017