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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.

The FDA granted accelerated approval to Elevidys (formerly known as SRP-9001) on 22 June. The AAV-directed gene therapy is indicated...

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