Majority of FDA advisory panel back approval of Akcea, Ionis' Waylivra for familial chylomicronaemia syndrome

Akcea Therapeutics and Ionis Pharmaceuticals announced that an FDA advisory committee voted 12 to 8 in support of approval of Waylivra (volanesorsen) for the treatment of people with familial chylomicronaemia syndrome. The agency is scheduled to make a decision on clearance of the antisense therapy by August 30.

The advisory panel reviewed data from two Phase III studies, with results from the APPROACH trial showing that Waylivra achieved a significant mean reduction in triglycerides of 77 percent from baseline and decreased risk of pancreatitis. The most common adverse events in the APPROACH study were injection site reactions, which led to five patients stopping treatment, and platelet declines, which also caused five patients to exit the trial. However, Akcea and Ionis noted that once platelet monitoring was fully implemented in the trial, no patients discontinued treatment.

Brett Monia, chief operating officer at Ionis, remarked "Waylive illustrates how our antisense technology can create targeted drugs for people living with severe diseases who currently have no available therapeutic options." The therapy, which is designed to reduce the production of ApoC-III, a protein produced in the liver that plays a central role in the regulation of plasma triglycerides, is also under regulatory review in the EU and Canada.

For related analysis, see ViewPoints: FDA presents a tough case for Akcea's first commercial foray.

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