Rare Disease Report

Marketing Applications Accepted for FCS Treatment in US, Canada and Europe

NOVEMBER 15, 2017
Mathew Shanley
This morning, Akcea Therapeutics, Inc. announced that its marketing applications for volanesorsen have been accepted for review in the United States, Europe and Canada.

The company has a primary focus on developing and commercializing therapeutic options for the treatment of patients with severe cardiometabolic diseases resulting from lipid disorders, and volanesorsen is intended specifically to treat familial chylomicronemia syndrome (FCS).

FCS is an autosomal recessive condition, meaning that both parents must be carriers of the genetic defect for the disease to affect a child. It is the result of a mutation in one of several proteins necessary in the gene for the body’s typical metabolism of triglycerides. The classic FCS patient has a mutation that causes the lipophilic protein not to function properly.

Per the National Institute of Health (NIH), FCS is prevalent in an estimated 1 in every 1 million individuals, and common symptoms include extreme abdominal pain, and the risk of pancreatitis. Volanesorsen would be the first approved medication intended for the treatment of the disease.

“We are committed to seeking global approvals for volanesorsen at the outset.  We are driven by the stories of people with FCS worldwide who persevere daily with a debilitating condition without an effective therapy. The acceptances of our regulatory filings in the U.S., EU and Canada are important steps forward in the global regulatory review process for volanesorsen, which brings us closer to potentially providing the first approved therapy for the treatment of people with FCS,” said Paula Soteropoulos, chief executive officer of Akcea Therapeutics in a press release. "The entire Akcea team is dedicated to working closely with each regulatory agency to support the review of volanesorsen.” 

The applications accepted for volanesorsen for the treatment of patients with FCS are based on data from the Phase 3 APPROACH and COMPASS studies, the former of which was the largest study ever conducted in the patient population. Both studies concluded that the drug exhibited positive responses and a significantly reduced rate of pancreatitis attacks.

In both APPROACH and COMPASS, the most common adverse events (AEs) were injection site reactions, which were mostly mild. Additionally, platelet count reductions were observed in many patients, however, they were not clinically significant and generally managed with proper monitoring and adjustments in dosing.

The U.S. Food and Drug Administration (FDA) has assigned a Prescription Drug User Fee Act (PDUFA) goal date of August 30, 2018.

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