Volanesorsen is filed (NDA) at FDA for the treatment of familial chylomicronemia syndrome.-Akcea Therapeutics.

Akcea Therapeutics, Inc., an affiliate of Ionis Pharmaceuticals, Inc. focused on developing and commercializing drugs to treat patients with serious cardiometabolic diseases caused by lipid disorders, announced the submission of a New Drug Application (NDA) to the FDA for volanesorsen, an investigational medicine for the treatment of familial chylomicronemia syndrome (FCS).The submission of volanesorsen for the treatment of FCS is based on data from the Phase III APPROACH and COMPASS studies.

The pivotal APPROACH study, a one-year, randomized, placebo-controlled study in 66 patients with FCS (average baseline triglycerides of 2,209 mg/dL, or 25.0 mmol/L), achieved its primary endpoint of reduction in triglycerides at three months, with a 77% mean reduction in triglycerides, which translated into a 1,712 mg/dL (19.3 mmol/L) mean absolute triglyceride reduction in volanesorsen-treated patients. The treatment difference is 94% compared to an 18% increase for placebo. In addition, in the APPROACH study, treatment with volanesorsen was associated with a statistically significant reduced rate of on-study pancreatitis attacks in the group of patients who had multiple pancreatitis events during the 5 years prior to screening and reduced abdominal pain in patients reporting pain during the screening period.

The COMPASS study, a six-month randomized placebo-controlled study in 113 patients with very high triglycerides (>500 mg/dL), also achieved its primary endpoint of reduction in triglycerides at three months, with a 71% mean reduction in triglycerides. In the COMPASS study, treatment with volanesorsen was associated with a statistically significant reduction in on-study pancreatitis attacks.

Comment: Following the results of COMPASS no drug available today has demonstrated the magnitude of the triglyceride reductions observed with volanesorsen..