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Breakthrough Therapies: Failed Study Won’t Automatically Lead To Rescinded Designation

 
• By Sue Sutter and Bridget Silverman

The decision to maintain or revoke breakthrough therapy status when trial results do not appear to support the original designation will depend on the facts specific to a development program, the US FDA says in new draft guidance. Since 2012, the agency has granted 475 breakthrough therapy designation requests and rescinded 35 designations.

Disappearing bridge
A breakthrough therapy designation won't automatically disappear if a subsequent study fails its efficacy endpoint. • Source: Shutterstock

An unsuccessful clinical trial is not automatically cause for rescinding a drug or biologic’s breakthrough therapy designation, the US Food and Drug Administration said in a new draft guidance.

Rather, the decision to maintain or revoke breakthrough therapy status when subsequent results appear not to support the evidence on which the designation was based “will depend on the

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