Rare Disease Report

FDA Votes in Favor of FCS Drug

MAY 10, 2018
Mathew Shanley
Today, the Endocrinologic and Metabolic Drugs Advisory Committee (EMDAC) voted 12-8 in favor of approval of volanesorsen (Waylivra) for the treatment of familial chylomicronemia syndrome (FCS).

The EMDAC of the U.S. Food and Drug Administration (FDA) met to review data supporting the new drug application (NDA) for the drug, a therapy in development at Akcea Therapeutics for the rare lipid disorder. A Prescription Drug User Fee Act (PDUFA) goal date of August 30, 2018 was set for the completion of its review.

“We thank all the members of the Committee for their time in conducting a thorough and thoughtful review of Waylivaratm, a new potential first and only therapy for people with familial chylomicronemia syndrome, or FCS,” said Paula Soteropoulos, CEO of Akcea. “FCS is ultra-rare, severe and potentially fatal with no therapeutic options. The data, including results from two phase three clinical trials, demonstrate clear improvement on several important measures of disease in these patients. We believe that these results provide strong support to make WAYLIVRA available to treat people with FCS.”

Earlier this week, Akcea announced that it would be expanding its global early access program (EAP) for volanesorsen to the United States.

Volanesorsen is being developed specially to decrease the production of ApoC-III, a protein produced in the liver that plays a central role in the regulation of plasma triglycerides and has the potential to affect other metabolic parameters. In a pair of recent Phase 3 studies – APPROACH and COMPASS – treatment with the drug demonstrated the ability to lessen triglycerides by 77% and by 71%, respectively.

In the pair of studies, incidence of pancreatitis was significantly reduced in patients treated with the drug, and treatment with volanesorsen had a mostly well-tolerated safety profile.

“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 to people suffering with this devastating disease.”

If approved, volanesorsen would be the first treatment option indicated specifically for people within this indication. Currently, the only available therapy for patients who are looking to manage symptoms associated with the condition is to follow an exceptionally restrictive, very low-fat diet (≤20 g daily), limit simple carbohydrates, and abstain from alcohol. Even with strict adherence, triglycerides have been proven in studies to remain high, leading to patients remaining at risk for acute pancreatitis.

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