STAT+: Pharmalittle: FDA official wants to use accelerated approval for gene therapies; pharma asks Biden for tax breaks in the semiconductor package
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A top U.S. Food and Drug Administration official says the agency needs to start using accelerated approval, a much-debated path commonly used for advancing cancer drugs, to advance gene therapies for rare diseases, STAT reports. In a speech at an industry conference, Marks largely focused on 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 a Sarepta Therapeutics gene therapy for Duchenne muscular dystrophy, a muscle-wasting disease that takes many patients’ lives in their 20s. This is the first time the agency will consider using an accelerated approval for gene therapy.
