Regeneron Pharmaceuticals announced that the FDA has granted Breakthrough Therapy Designation status to evinacumab, a potentially first-in-class drug designed to treat an inherited disorder that causes high levels of LDL cholesterol.
Evinacumab is an investigational monoclonal antibody designed 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 may be helpful for patients with cholesterol levels that are unresponsive to standard treaments, such as Regeneron and Sanofi's Praluent.
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.
Read the press release