PTC's Translarna: US FDA Opts For Simplicity When It Comes To Panel Questions
Executive Summary
Streamlined queries for third advisory committee review of a Duchenne muscular dystrophy drug ask only whether the data suggest ataluren is effective, may be effective but needs more study, or is not effective. In contrast, panelist had to muddle through seven discussion and voting questions for Sarepta's eteplirsen and nine for BioMarin's drisapersen.