BioPharma Dive May 24, 2024
Ben Fidler

“In rare diseases, we need something now, not later,” said Peter Marks, head of the FDA office that reviews gene therapies, at a conference held Friday.

A top Food and Drug Administration official on Friday again advocated for the speedy approval of gene therapies for rare diseases. But he didn’t drop any clues on where the agency stands on a coming decision to possibly broaden use of one of them, a Duchenne muscular dystrophy treatment the regulator cleared last year.

At a meeting hosted by the patient advocacy group CureDuchenne, Peter Marks, head of the FDA office that reviews gene therapies, said the agency’s thinking has changed in recent years to become more patient focused. That mindset has led it...

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Topics: Biotechnology, FDA, Govt Agencies, Pharma / Biotech
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