Surrogate Endpoint, Safety Questions May Be Too Much For Waylivra To Overcome At US FDA Panel

A surrogate endpoint with uncertain clinical benefits combined with a thrombocytopenia safety signal are among the several factors that could derail the approval prospects of Akcea Therapeutics Inc.'s Waylivra (volanesorsen), which heads for an assessment by the US FDA's Endocrinologic and Metabolic Drugs Advisory Committee May 10 for the treatment of familial chylomicronemia syndrome (FCS).

A modified phosphorothioate antisense oligonucleotide (ASO), Waylivra would be the first pharmacotherapy if approved for the rare genetic syndrome, which...