Data Trumps Anecdotes, Emotion At Sarepta Panel
Executive Summary
At a highly charged April 25 meeting of the FDA's Peripheral and Central Nervous System (PCNS) Advisory Committee, which concluded with angry shouting from parents and patient advocates, a majority of panelists said Sarepta Therapeutics Inc. failed to provide sufficient efficacy data for its Duchenne muscular dystrophy drug eteplirsen to win US approval – accelerated or standard, although the votes were much closer on the former than on the latter question.