Conditional approval of Waylivra to treat genetically confirmed familial chylomicronemia syndrome

Akcea Therapeutics, Inc. an affiliate of Ionis Pharmaceuticals, Inc., and Ionis Pharmaceuticals, Inc. announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has adopted a positive opinion recommending conditional marketing authorization for Waylivra as an adjunct to diet in adult patients with genetically confirmed familial chylomicronemia syndrome (FCS) who are at high risk for pancreatitis, in whom response to diet and triglyceride lowering therapy has been inadequate.

Waylivra’s Marketing Authorization Application (MAA) is based on results from the Phase III APPROACH study and the ongoing APPROACH Open Label Extension study and supported by results from the Phase III COMPASS study. Results from the Phase III APPROACH trial, the largest study ever conducted in patients with FCS, show that in comparison to placebo, treatment with Waylivra reduced triglycerides by 77 percent after 3 months of treatment. The most common adverse events in the APPROACH study were injection site reactions and reductions in platelet levels. In addition to the OLE study, there are also ongoing global Early Access Programs for Waylivra.