Sarepta Data Disappoints Wall Street As Expert Sees A Sea Change At FDA

It may be one of the most-watched small studies in biotech history. This morning, of Cambridge, Mass., released 144-week data from its study of eteplirsen, for Duchenne muscular dystrophy, in 12 boys. Sarepta — as well as its investors and a vocal group of families who are facing Duchenne — have always hoped that this tiny study would be enough to get eteplirsen approved by the Food and Drug Administration.