The EMBARK trial’s availability to more patients likely contributed to the much quicker enrollment than confirmatory trials for Exondys 51, Vyondys 53 and Amondys 45, which also saw enrollment targets and eligibility ages change over the years.

The Duchenne muscular dystrophy candidate should be approved because it will help grow development in the space, patient groups argue, not just because of the value the gene therapy could provide to those receiving the treatment itself.

Sandoz has struck a deal with Adalvo that will give the company rights to six products in the US – four of which are potential first-to-market opportunities – targeting markets that are collectively worth around $3bn.

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.