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Accelerated Approval: Gene Therapy Confirmatory Trial Timelines Can Stretch

 
• By Sue Sutter and Nancy Pham

Pink Sheet infographic looks at the nascent space of gene therapy accelerated approvals, where confirmatory trial timelines can run many years beyond approval due to disease rarity and a focus on durability of response.

Years down the road
Confirmatin of benefit for accelerated approval gene therapies may be years down the road. • Source: Shutterstock

The US Food and Drug Administration has granted accelerated approval to four gene therapies to date, including three anti-cancer CAR-T products and bluebird bio’s cerebral adrenoleukodystrophy treatment Skysona (elivaldogene autotemcel).

New confirmatory trials were required for all of them. In the case of Skysona, the last study report is not...

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