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.