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Accelerated approval will be 'the norm' for gene therapies, FDA's Peter Marks says

Zachary Brennan (Endpoints News)

February 27, 2027

The FDA will increasingly use accelerated approval for gene therapies aimed at rare conditions, according to Peter Marks, the FDA’s top official overseeing cell and gene therapies, the clearest signal yet of how widely the agency will deploy its expedited pathway.

Marks’ comments, made last week at a workshop in Washington, build on a stance he took last year backing accelerated approval for gene therapies, which often target young children with ultra-rare conditions.

“If we don’t lean into accelerated approval, we’re going to leave a lot of patients behind,” Marks said during the workshop at the Reagan-Udall Foundation in Washington last week. “Accelerated approval is going to be the norm for a lot of our initial approvals of gene therapies.”

There are more than 10,000 rare diseases, but companies often struggle to bring therapies to market for these conditions due to the small numbers of patients and financial constraints. In recent comments, Marks has indicated that he views accelerated approval as a powerful tool to speed new treatments and ensure that companies don’t give up on promising therapies.

“There are a lot of gene therapies for rare diseases that are almost there,” Marks said. “And it’s a question of, how do we get them across the finish line?”

Brennan, Z. Accelerated approval will be ‘the norm’ for gene therapies, FDA’s Peter Marks says. Endpoints News(2024). Available at: (Accessed: 1st March 2024)



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