Let’s Go To The Video: Recordings Of DMD Patients After Elevidys Treatment ‘Compelling’ But Not Substantial Evidence

CBER Director Peter Marks urged the review teams for Sarepta’s gene therapy to consider pre- and post-treatment videos showing improved physical function, but clinical and statistical reviewers cited limitations in the patient videos and said they failed to inform whether micro-dystrophin is a suitable surrogate endpoint for accelerated approval.