Orphan status for Akcea's volanesorsen for familial chylomicronemia syndrome

Akcea Therapeutics, a wholly-owned subsidiary of Isis Pharmaceuticals (Nasdaq: ISIS), says that the US 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 II study published in the New England Journal of Medicine (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.Akcea is currently conducting an international multicenter, randomized, double-blind, placebo-controlled Phase III study in patients with FCS.

Paula Soteropoulos, president and chief executive at Akcea Therapeutics, commented: "Orphan drug designation for FCS underscores the need for improved therapies to treat patients with FCS and is an important benchmark as we complete the ongoing Phase III study in patients with FCS and prepare for commercialization." Unfortunately, she noted, current treatment options do not reduce triglyceride levels enough to reduce the risk of serious illness in patients with FCS.