CBER’s Marks Calls for Accelerated Approval in Gene Therapies for Rare Diseases

Peter Marks, director of CBER, is pushing to get more gene therapies targeting rare diseases to apply for accelerated approval (AA) to give the promising space a leg up in development.

During Tuesday’s Alliance for a Stronger FDA webinar on CBER’s budget hopes for FY 25 and the office’s plans for calendar year 2024, Marks pointed out that large randomized clinical trials are often not possible in small, rare disease populations.

And though 18 gene therapies have been approved over the last two years, venture funding in the space has slowed down because the development process is so slow, partially due to the lack of randomized trials.

Marks said CBER also wants to use the money to modernize its systems and shore up its efforts to monitor the supply chain.

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