Draft guidance gives recommendations for conducting one randomized controlled trial to generate the evidence for accelerated approval and confirm clinical benefit.

Any program changes would not occur until after a thorough assessment, the agency said.

Cellular, Tissue and Gene Therapies Advisory Committee will weigh whether the increase in dystrophin production seen with SRP-9001 is reasonably likely to predict clinical benefit in Duchenne muscular dystrophy, Sarepta says; agency had previously told the company a panel meeting would not be needed, but it reversed course late in the review.

Pink Sheet reporter and editors discuss the first round of mandated Medicare inflation rebates and new guidance on price negotiations, consider the VA’s decision to cover the Alzheimer’s drug Leqembi, and the US FDA continuing to search for a cell and gene therapy office leader.

The new job notice offers a top salary, but also invites questions about the candidate pool attracted by the original solicitation.

But some, including some related to inspections and master protocols, will remain in effect to allow for revisions or a transition out of the pandemic posture.