Anecdotal evidence and urgent need sway FDA panel toward Sarepta’s DMD therapy

The urgency of getting a potentially disease-modifying treatment to Duchenne muscular dystrophy patients again outweighed a litany of concerns about safety and efficacy, as an FDA advisory committee voted — narrowly — in favor of granting accelerated approval to Sarepta’s gene therapy.

The most immediate question now is how FDA will use the opinions gathered from the discussion in its quickly approaching decision, which has a May 29 target date. FDA’s reviewers raised substantial concerns about the data during the meeting, which seemed to suggest the agency has a negative view of the application. It’s unclear how a split committee vote this late in the review process will influence the agency’s thinking. ...