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Sarepta’s DMD Gene Therapy: Approval Delayed And Indication Narrowed, But Still On Track

 
• By Derrick Gingery

As Sarepta again brings a product application from the brink of rejection to the cusp of approval, the one-month review delay creates more assurance the confirmatory EMBARK trial will be completed as well as providing more time for label negotiations with the US FDA.

delay sign
Accelerated approval is expected in boys age four to five at first, but could grow pending the results of the EMBARK trial. • Source: Shutterstock

Sarepta Therapeutics, Inc. must wait another month for the likely accelerated approval of its proposed gene therapy for Duchenne muscular dystrophy, as well as deal with a restricted patient population, at least at the start of marketing.

Key Takeaways

  • The FDA needs more time to complete labeling negotiations and postmarketing discussions

  • SRP-9001 is expected to be limited to DMD patients age four and five pending the completion of the EMBARK trial

The company announced on 24 May that the US Food and Drug Administration needed more time to complete its review of the pending biologics license application for SRP-9001 (delandistrogene moxeparvovec), including final label

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