FDA grants breakthrough designation to hypercholesterolemia medication
The FDA has granted a breakthrough therapy designation to evinacumab to treat hypercholesterolemia in patients with Homozygous Familial Hypercholesterolemia (HoFH).
Regeneron Pharmaceuticals, which manufactures evinacumab, announced the news on April 6.
The FDA has not approved evinacumab, which is an investigational monoclonal antibody to angiopoietin-like protein 3. The agency gives breakthrough therapy designations to medications “that are intended to treat a serious condition and preliminary clinical evidence indicates that the drug may demonstrate substantial improvement over available therapy on a clinically significant endpoint(s).”
In May, Regeneron announced an interim analysis of a phase 2, proof-of-concept trial that found four patients with HoFH who received evinacumab plus their current lipid-lowering therapy had a mean 55 percent reduction in low-density lipoprotein (LDL) cholesterol at four weeks. The LDL cholesterol reduction ranged from 25 percent to 90 percent.
The phase 2 study is ongoing and expects to enroll eight patients. Regeneron plans on initiating a phase 3 trial, as well.
Approximately one to two people million have HoFH, which is the most severe form of hypercholesterolemia. If people with HoFH do not receive treatment, they can have a LDL cholesterol level from 500 mg/dL to 1,000 mg/dl and have an elevated risk of cardiovascular disease. Normal LDL cholesterol levels are typically less than 130 mg/dL.
Executive Editor
Tim Casey joined TriMed Media Group in 2015 as Executive Editor. For the previous four years, he worked as an editor and writer for HMP Communications, primarily focused on covering managed care issues and reporting from medical and health care conferences. He was also a staff reporter at the Sacramento Bee for more than four years covering professional, college and high school sports. He earned his undergraduate degree in psychology from the University of Notre Dame and his MBA degree from Georgetown University.