Health
Sarepta Gains as FDA Official Overrules Staff to Approve Therapy
- Sarepta’s gene therapy for DMD didn’t succeed in main trial
- Decision expanding access is best possible outcome for company
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A top US Food and Drug Administration official overrode reviewers to give broad approval to Sarepta Therapeutics Inc.’s gene therapy for a rare muscle disease in children, despite a lack of data showing it actually slows overall progression of the disease.
The approval was a victory for parents who’d advocated for the controversial, $3.2 million treatment as well as for the company. The shares rose as much as 40% in New York, the most intraday in six years.