Sarepta Enters Uncharted Territory with Adcomm Approval

On May 12, an FDA advisory committee recommended the regulator approve Sarepta Therapeutics’ gene therapy, SRP-9001, to treat Duchenne muscular dystrophy. While the FDA has yet to decide whether or not to approve SRP-9001 based on priority review, Sarepta successfully convinced 8 of 14 advisers that it could address multiple outstanding issues raised by the agency.

However, the discussion highlighted issues developers of future gene therapies need to consider.