STAT+: Top FDA official: Agency needs to start using accelerated approval for gene therapies
An FDA official says accelerated approval should be extended to gene therapies for rare diseases — comments that come as the agency considers a gene therapy for Duchenne muscular dystrophy.
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.”
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