Not so fast

FDA documents concerning the review of Exondys 51 eteplirsen to treat DMD show that the chorus keening that the approval opens the floodgates for companies to combine shoddy, underpowered studies with patient activism to gain easy approvals and quick riches has misinterpreted the episode.

The Duchenne muscular dystrophy community's interaction with FDA had been hailed as a model for patient-focused drug development, and the accelerated approval of Sarepta Therapeutics Inc.'s NDA was a resounding victory for DMD advocates...