ICER pushes back on FDA's potential conversion of Sarepta's gene therapy to full approval
The integrity of the FDA’s accelerated approval process should be called into question if the agency grants full approval to treatments that have failed their required confirmatory trials, the chief medical officer at the drug pricing watchdog ICER wrote in a new viewpoint.
Published in the Journal of the American Medical Association on Wednesday, ICER’s David Rind put the FDA’s accelerated approval of Sarepta’s Elevidys, a gene therapy for Duchenne muscular dystrophy, under the microscope, taking issue with the surrogate endpoint used in the approval, and the potential for it to be granted full approval next month. He also argues in the piece that the therapy’s high cost — $3.2 million — is hard to justify, contrasting it with other expensive treatments that have shown curative benefit.
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