In weighing presentations from Akcea Therapeutics Inc. and the FDA on the risk/benefit profile of the antisense oligonucleotide (ASO) volanesorsen (Waylivra) in familial chylomicronemia syndrome (FCS), members of the Endocrinologic and Metabolic Drugs Advisory Committee (EMDAC) came down on the side of the drug in a 12-8 vote, with no abstentions. In comments following the vote late Thursday, several EMDAC members said they gave more weight to the unmet need in FCS, an orphan disease with no approved treatments, than to concerns about adequate characterization of the patient population and the number and severity of adverse events (AEs). A few openly acknowledged they were swayed by comments during the open public hearing, when 13 individuals – patients, family caregivers and physicians – all spoke in favor of approval.