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Akcea Waylivra's Surrogate Endpoint Gains Support At US FDA Advisory Committee

 
• By Michael Cipriano

In wide-ranging meeting, majority of panelists believe volanesorsen's surrogate marker will correlate to clinical outcomes such as reduced pancreatitis. 

The US FDA's Endocrinologic and Metabolic Drugs Advisory Committee showed a degree of faith in the surrogate endpoint of percent change in fasting triglyceride (TG) at three months for Akcea Therapeutics Inc.'s Waylivra (volanesorsen) with a 12 to 8 vote in favor of approval for the treatment of familial chylomicronemia syndrome (FCS).

Panelists who voted "yes" during the May 10 meeting generally felt that the surrogate would correlate to clinical outcomes of reductions in abdominal pain and pancreatitis, despite the pivotal Phase III CS6 trial not being powered to measure these endpoints due to a small sample size

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