trending Market Intelligence /marketintelligence/en/news-insights/trending/y_cvubsvdkretvcwlvakdw2 content esgSubNav
In This List

Akcea gets US FDA advisory committee recommendation for genetic disease drug

Blog

Gauging Supply Chain Risk In Volatile Times

Blog

Insight Weekly: Banks' efficiency push; vacuuming carbon; Big Pharma diversity goals

Blog

The Future of Risk Management Digitization in Credit Risk Management

Blog

Climate Credit Analytics: Diving into the model


Akcea gets US FDA advisory committee recommendation for genetic disease drug

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.