Akcea Therapeutics, Inc. (NASDAQ: AKCA) rose in Friday after the company disclosed that the FDA Advisory Committee voted in favor of WAYLIVRA for the treatment of familial chylomicronemia syndrome.
Akcea, an affiliate of Ionis Pharmaceuticals, Inc., (NASDAQ: IONS) and Ionis, the parent company, announced that the U.S. Food and Drug Administration’s (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 (FCS).
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.
"We thank the committee members for their time and their comments today. The Committee’s majority vote in favor of approval is an important positive step to bring WAYLIVRA to people with FCS who have no adequate treatment options, said Paula Soteropoulos, chief executive officer of Akcea Therapeutics.
"We look forward to working with the FDA to complete the final stages of regulatory review for WAYLIVRA. We are committed to the FCS community and will continue to focus on bringing WAYLIVRA, potentially the first and only treatment, to people living with this serious and potentially fatal disease."
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.
Shares rocketed $3.17, or 15.3%, to $23.83
