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Gene Therapies: US FDA Has Used Accelerated Approval Four Times Based On Intermediate Clinical Endpoints

 
• By Sue Sutter

Three of the accelerated approvals were for CAR-T products, a class that the agency considers to be gene therapies. The FDA’s September approval of bluebird bio’s Skysona for cerebral adrenoleukodystrophy was the lone non-cancer product in the group. Pink Sheet infographic tracks the required confirmatory studies and timelines for all four products.

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Accelerated approval has been used a total of four times for novel gene therapies and supplemental indications. • Source: Shutterstock

Sarepta Therapeutics, Inc.’s Duchenne muscular dystrophy gene therapy SRP-9001 is heading into a high-profile advisory committee meeting on 12 May where accelerated approval based on micro-dystrophin as a surrogate endpoint reasonably likely to predict clinical benefit will be up for debate.

The product’s regulatory path through the US Food and Drug Administration is being closely watched not only by those in...

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