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Sarepta’s DMD Gene Therapy Helped Across Accelerated Approval Finish Line By CBER’s Peter Marks

 
• By Sue Sutter

FDA clinical, clinical pharmacology and statistical review teams did not recommend approval of Elevidys, but the CBER director concluded that randomized data in a subgroup of patients ages four and five years old were ‘compelling,’ justifying accelerated approval; expansion beyond this age-restricted subgroup will hinge on data from the EMBARK confirmatory trial.

Finish line
Elevidys crossed the finish line for a smaller population of patients than Sarepta would have liked. • Source: Shutterstock

Sarepta Therapeutics, Inc.’s gene therapy Elevidys (delandistrogene moxeparvovec-rokl) won US Food and Drug Administration accelerated approval in an age-restricted population of Duchenne muscular dystrophy patients thanks to Center for Biologics Evaluation and Research Director Peter Marks, who overruled objections from various review disciplines.

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