Therapeutics confirms FDA Advisory Committee votes in favor of WAYLIVRA for treatment of familial chylomicronemia syndrome
- Akcea Therapeutics, Inc. (AKCA), an affiliate of Ionis (IONS), announced that the FDA Division of Metabolism and Endocrinology Products Advisory Committee voted 12-8 to support approval of WAYLIVRATM (volanesorsen) for the treatment of people with familial chylomicronemia syndrome. The committee's non-binding recommendation will be considered by the FDA in its review of Akcea's New Drug Application for WAYLIVRA. The PDUFA date for completion of the review of WAYLIVRA is August 30, 2018.
- The Advisory Committee reviewed data from two Phase 3 clinical trials, APPROACH and COMPASS, as well as the ongoing APPROACH Open Label study for WAYLIVRA. Results from the phase 3 APPROACH trial, the largest study ever conducted in patients with FCS, show that patients with FCS treated with WAYLIVRA achieved a statistically significant mean reduction in triglycerides of 77% from baseline and decreased risk of pancreatitis. The most common adverse events in the APPROACH study were injection site reactions and platelet declines. The Committee's input will be considered by the FDA in its review of the New Drug Application for WAYLIVRA. The FDA is not bound by the Committee's guidance, but takes its advice into consideration when reviewing investigational medicines. WAYLIVRA is also under regulatory review in the European Union and Canada.