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.

Rare disease advocates push for National Academies of Sciences, Engineering and Medicine study on how or whether ultra-rare should be defined, which could create legislative pressure. In an interview, Ultragenyx CEO Emil Kakkis suggests patient thresholds, accelerated approval triggers.

A better description of the US FDA’s regulatory flexibility may be necessary, but tinkering with the foundational law for rare disease drug development could open the door for more substantial and unwanted changes.