Slim Adcomm Majority Boosts Sarepta’s Gene Therapy In Duchenne Muscular Dystrophy
Accelerated approval seems imminent after a majority of the US FDA’s Cellular, Tissue and Gene Therapies Advisory Committee voted the risk-benefit balance supported it.
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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.
FDA team held several internal meetings on the gene therapy after the 12 May advisory committee, culminating in a teleconference with Sarepta in which the CBER director outlined his accelerated approval decision and reiterated recommendations to modify the ongoing EMBARK trial to better ensure confirmation of benefit. Pink Sheet's Drug Review Profile dives into the story behind the Elevidys review.
US FDA Center Directors Should Not Influence Adcomm Votes Or Overrule Scientific Staff, Coalition Says
In a letter to Commissioner Robert Califf, the Patient, Consumer and Public Health Coalition says medical product center directors and other senior staff should refrain from trying to sway advisory committee votes and be reminded that overruling decisions of their own scientific staff undermines the public trust.