Akcea Therapeutics, a wholly owned subsidiary of Ionis Pharmaceuticals (IONS), announced positive results from the pivotal Phase 3 APPROACH study of the drug volanesorsen. The results demonstrate that the drug met its primary endpoint of reducing triglyceride levels in patients with familial chylomicronemia syndrome (FCS).
The Disease
familial chylomicronemia syndrome (FCS) is a severe, rare genetic metabolic disorder characterized by an inability of the body to store fat in normal locations. These results in a tremendous elevation in the level of triglycerides in the bloodstream, in abnormal fat distribution around . . .