A U.S. Food and Drug Administration advisory committee recommended the approval of Akcea Therapeutics Inc.'s Waylivra.
Waylivra is meant to treat familial chylomicronemia syndrome, or FCS, a rare genetic disease characterized by a patient's inability to break down fat. There are no approved therapies for FCS, which is often misdiagnosed or goes undiagnosed, according to Akcea.
The recommendation from the FDA's Division of Metabolism and Endocrinology Products Advisory Committee will be taken into consideration alongside Akcea's new drug application for Waylivra, which has an Aug. 30 target action date.
The company's regulatory submission features data from two phase 3 clinical trials, as well as findings from an ongoing study. In one phase 3 trial, called Approach, Waylivra delivered a 77% mean reduction in triglycerides, a type of fat in the blood, and decreased risk of pancreatitis, which FCS patients often experience.
Akcea is unit of Ionis Pharmaceuticals Inc.