Sarepta Therapeutics, Inc.’s Duchenne muscular dystrophy gene therapy SRP-9001 is heading into a high-profile advisory committee meeting on 12 May where accelerated approval based on micro-dystrophin as a surrogate endpoint reasonably likely to predict clinical benefit will be up for debate.
The product’s regulatory path through the US Food and Drug Administration is being closely watched not only by those in the DMD community eager to see new treatments as quickly as possible for the devastating neurological disease that strikes young males, but also by