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Gene Therapy: Four-Month Lag In Commercial Access Protects EMBARK Study, Sarepta Says

 
• By Sue Sutter

Company’s prediction that Duchenne muscular dystrophy patients likely would not be able to access SRP-9001 until four months after accelerated approval eased some US FDA panelists’ concerns about interference with the ongoing confirmatory trial.

Mind the gap
The gap between accelerated approval of SRP-9001 and commercial access will be at least 8 weeks and more likely about 4 months, Sarepta says. • Source: Shutterstock

Sarepta Therapeutics, Inc.’s prediction of a four-month lag in commercial access to its gene therapy SRP-9001 (delandistrogene moxeparvovec) helped sway a US Food and Drug Administration panel vote in favor of accelerated approval for the Duchenne muscular dystrophy treatment.

At their 12 May meeting, some members of the Cellular, Tissue and Gene Therapies Advisory Committee raised ethical and practical concerns about granting accelerated approval by the application’s 29 May user fee goal

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