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A Duchenne Breakthrough Awaits In 2023 - But Risks Remain For Sarepta And US FDA

Gene Therapy Could Be Launched Mid-2023

 
• By Andrew McConaghie

The FDA and Sarepta have had a controversial history with the accelerated approvals of the firm’s earlier drugs for Duchenne muscular dystrophy – but the company’s investigational gene therapy, SRP-9001, now under a priority review, could be a genuine breakthrough.

Sarepta Building 2

The US Food and Drug Administration is to decide by 29 May 2023 on whether or not to approve SRP-9001, Sarepta Therapeutics’s gene therapy for Duchenne, which many hope will be a breakthrough against the debilitating and eventually fatal inherited muscle wasting disease.

The FDA has decided to review the drug’s data early via a priority review, well before Sarepta’s Phase III placebo-controlled EMBARK

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