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DALLAS — A top Food and Drug Administration official said Monday that the agency needs to start using accelerated approval, a much-debated path commonly used for advancing cancer drugs, to advance gene therapies for rare disease.

“We always are going to keep safety, front and foremost,” Peter Marks, head of the Center for Biologics Evaluation and Research, told attendees at the annual meeting of the Muscular Dystrophy Association. “But I think the issue here is we can’t be so careful about our approvals under accelerated approval that we prevent potentially lifesaving therapies from getting to market in a timely manner.”


Marks geared much of his speech around the rarest of diseases, those that affect fewer than 100 patients per year. But his remarks came as the FDA considers granting accelerated approval for Sarepta’s gene therapy for Duchenne muscular dystrophy, a muscle-wasting disease that takes many patients’ lives in their 20s. 

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