STAT+: What to know about a pivotal FDA hearing on Sarepta’s gene therapy for Duchenne
On Friday, a committee of advisers to the Food and Drug Administration will meet to discuss Sarepta Therapeutics’ closely watched experimental gene therapy for Duchenne muscular dystrophy.
It will be the first FDA advisory panel hearing for a Duchenne drug from Sarepta since 2016, when hundreds of patients and family members traveled to the FDA campus in Maryland to plead with experts to authorize an earlier therapy from the company despite limited evidence. That led to one of the most contentious approvals in recent memory, as the agency overruled its committee’s recommendations, leading to celebrations from families and outcry from senior agency officials.
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