Complete Response Letter for Waylivra, a drug to treat Familial chylomicronemia syndrome. - Ionis Pharma + Akcea Therapeutics.

Akcea Therapeutics, Inc an affiliate of Ionis Pharmaceuticals, Inc., and Ionis Pharmaceuticals, Inc. announced that they received a Complete Response Letter (CRL) from the Division of Metabolism and Endocrinology Products of the FDA regarding the New Drug Application (NDA) for Waylivra (volanesorsen).

Familial chylomicronemia syndrome (FCS) is an ultra-rare, devastating hereditary disease that causes unpredictable and potentially fatal acute pancreatitis, chronic complications due to permanent organ damage, and a severe impact on daily living. The hallmark of FCS is extremely elevated triglycerides. Results from the Phase 3 APPROACH trial, the largest study ever conducted in patients with FCS, show that in comparison to placebo, treatment with Waylivra reduced triglycerides by 77% (-94% when compared to placebo). The Endocrine Society and current clinical practice guidelines recommend triglyceride reduction as the goal of treatment for FCS. The most common adverse events in the APPROACH study were injection site reactions and reductions in platelet levels.

Comment: On May 10, an FDA advisory committee voted 12-8 in favor of approving Waylivra. Though the FDA tends to follow such suggestions, safety concerns appear to haveaffected the drug's approval chances.