image credit: Freepik

FDA delays decision on Duchenne gene therapy and considers narrower approval

May 24, 2023


The Food and Drug Administration has delayed its review of a closely watched gene therapy for Duchenne muscular dystrophy by one month, taking more time to consider whether approval should be initially limited to children who are most likely to benefit.

In a statement Wednesday, the treatment’s developer, Sarepta Therapeutics, said the regulator needs “modest additional time” to complete its review and will now decide whether to grant an accelerated approval by June 22. The delay will involve final negotiations around the information that would be included in the drug’s label, as well as the post-marketing commitments it will require of Sarepta. The FDA had previously set a May 29 deadline for its decision.

Read More on Biopharma Dive