27 August 2018 - Akcea Therapeutics today announced that they received a complete response letter from the Division of Metabolism and Endocrinology Products of the U.S. FDA regarding the new drug application for Waylivra (volanesorsen).

“We are extremely disappointed with the FDA’s decision. FCS is an ultra-rare and debilitating disease. Our disappointment extends to the patient and physician community who currently do not have a treatment available to them. We continue to feel strongly that Waylivra demonstrates a favourable benefit/risk profile in people with FCS as was reflected in the positive outcome from our Advisory Committee hearing in May. We will continue to work with the FDA to confirm the path forward,” said Paula Soteropoulos, chief executive officer of Akcea Therapeutics.

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).

Read Akcea Therapeutics press release