Videos aimed at showing improved function among Duchenne muscular dystrophy patients after receiving Sarepta Therapeutics, Inc.’s gene therapy Elevidys (delandistrogene moxeparvovec-rokl) are compelling but do not provide the substantial evidence needed to support an approval action, a US Food and Drug Administration clinical reviewer said.
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.
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