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Accelerated Approval Now Starting Point For Gene Therapy Development, US FDA’s Marks Says

 
• By Derrick Gingery

Expedited approval pathway had been a secondary option when traditional approval was not possible, but the CBER director wants surrogate endpoints increasingly considered from the beginning, especially with pediatric rare disease gene therapies.

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Going forward, accelerated approval will not be an afterthought for gene therapies, Peter Marks said. • Source: Shutterstock

Gene therapy sponsors now should expect accelerated approval to be on the table from the beginning of the development process, rather than as an alternative to a traditional pathway.

Peter Marks, director of the US Food and Drug Administration’s Center for Biologics Evaluation and Research, said the change in...

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