Expert Committee Finds Akcea’s NDA for Rare Genetic Disease is Safe and Effective

An FDA advisory committee voted 12 to 8 Thursday that Akcea Therapeutics’ NDA for treatment of a rare genetic disease demonstrated safety and efficacy.

Members of the Endocrinologic and Metabolic Drugs Advisory Committee voted that the data supported approval of the NDA for volanesorsen solution, indicated as an adjunct to diet for the treatment of patients with familial chylomicronemia syndrome, a rare genetic disease.

Committee members noted that the data for the NDA demonstrated three-month efficacy on triglycerides, but not clinical benefits. However, most members agreed that clinical benefit could be inferred.