Journal Scan

Positive Phase 3 results of investigation medicine in patients with severe inherited form of high cholesterol published

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Positive results from the Phase 3 trial of evinacumab in 65 patients with homozygous familial hypercholesterolemia (HoFH), were published in the New England Journal of Medicine.

The investigational medicine, which binds to and blocks the function of angiopoietin-like 3 (ANGPTL3), is the first treatment to show efficacy in patients with HoFH, a form of early cardiovascular disease that leads to severely elevated levels of bad cholesterol.

In the trial, adding evinacumab to other lipid-lowering therapies reduced their LDL-C by 49%.

“The vast majority of my patients with HoFH never reach their target LDL-C despite taking multiple lipid-lowering therapies, and they remain at increased risk of premature heart disease because of their persistently high LDL-C levels,” said Professor Derick J. Raal, MMED, PhD, principal investigator and Professor & Head, Division of Endocrinology & Metabolism at the University of the Witwatersrand, South Africa in a press release. “If approved, evinacumab will provide a major step forward for the treatment of patients with HoFH who have significant unmet needs.”

The Biologics License Application (BLA) for evinacumab is currently under Priority Review with the U.S. Food and Drug Administration, with a target action date of February 11, 2021.

Read more here.