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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.

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‘Pipeline In A Pill’ Or Pipe Dream? US FDA’s April Goal Dates Test Expansion Strategies

 
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