15 November 2017 - FDA Prescription Drug User Fee Act goal date set for 30 August 2018.
Akcea Therapeutics, an affiliate of Ionis Pharmaceuticals, focused on developing and commercialising drugs to treat patients with serious cardiometabolic diseases caused by lipid disorders, today announced that all of its marketing applications for volanesorsen have been accepted for review in the U.S., EU and Canada for the treatment of patients with familial chylomicronemia syndrome (FCS).
In the U.S., the FDA assigned a Prescription Drug User Fee Act goal date of 30 August 2018. The new drug submission has passed screening by Health Canada and was granted priority review. The EMA has validated the volanesorsen marketing authorisation application. Volanesorsen has been granted a Promising Innovative Medicine Designation by the United Kingdom’s Medicines and Healthcare products Regulatory Agency for the treatment of people with FCS.
The applications for volanesorsen for the treatment of people with FCS are based on data from the Phase 3 APPROACH and COMPASS studies. The pivotal APPROACH study was the largest study ever conducted in patients with FCS. APPROACH, 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 seen in the study was 77% compared to an 18% increase for placebo.