FDA Breakthrough status for evinacumab in HoFH

The US Food and Drug Administration 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 low-density lipoprotein (LDL) cholesterol.

Evinacumab is an investigational monoclonal antibody to angiopoietin-like protein 3 (ANGPTL3) under development by US biotech firm Regeneron Pharmaceuticals (Nasdaq: REGN). ANGPTL3 acts as an inhibitor of lipoprotein lipase and endothelial lipase, and appears to play a central role in lipoprotein metabolism. Breakthrough Therapy designation was created to expedite the development and review of drugs that target serious or life-threatening conditions.

Regeneron previously reported positive interim Phase II results for evinacumab in HoFH patients and is currently planning a Phase III trial. Evinacumab is similar to Regeneron’s alirocumab, partnered with Sanofi under the trade name Praluent, but is designed to work slightly differently, targeting elevated triglycerides in the blood as well as other lipids such as LDL cholesterol.