Akcea Therapeutics Receives Orphan Drug Designation from the US FDA for Volanesorsen (ISIS-APOCIII Rx) for the Treatment of Familial Chylomicronemia Syndrome

BOSTON, July 6, 2015 /PRNewswire/ -- Akcea Therapeutics, a wholly-owned subsidiary of Isis Pharmaceuticals, Inc. (NASDAQ: ISIS), announced today that the U.S. Food and Drug Administration has granted Orphan Drug Designation to volanesorsen (ISIS-APOCIIIRx) for the treatment of patients with Familial Chylomicronemia Syndrome (FCS). FCS is a rare genetic disease characterized by extremely high triglyceride levels and risk of pancreatitis. In a Phase 2 study published in the NEJM in December 2014, patients with FCS treated with volanesorsen achieved substantial reductions in apoC-III, triglycerides, chylomicrons and apoC-III-associated very low density lipoprotein-cholesterol (VLDL-C) particles.1 Akcea is currently conducting an international multi-center, randomized, double-blind, placebo-controlled Phase 3 study in patients with FCS.

The release as fetched from its publisher. ionis.com ↗