![]() Questions raised about confirmatory study completion There were also concerns about safety, including some cardiac and liver reactions. And how to know which patients it might help, and which it wouldn't." "Additional uncertainties have to do with whether of expression of Sarepta's micro-dystrophin is suitable as a surrogate endpoint considered reasonably likely to predict clinical benefit. "Others involve the nonclinical data, the results from animal studies," he added. A process change led to reduced purity for the gene therapy in later production. "Some have to do with the manufacturing," he said. Mike Singer summarized some of the agency's concerns. Tempering these stories, FDA scientists spent more than an hour raising questions about the evidence submitted by Sarepta for the gene therapy.įDA scientist Dr. We cried nearly a quadrillion tears of joy." Hopping up off the ground after sitting or laying on the couch. Racing up stairs, climbing indoor playground equipment, running, jumping. ![]() ![]() Check this out,' showing me live footage of Andrew doing the unexpected. "Or we'd connect over FaceTime and she'd very discreetly whisper, 'Nate, Nate. "Sarah often opened with her excitedly exclaiming: 'You're never going to believe what Andrew just did today,' " he said. Craig McDonald from the University of California, Davis, who testified on behalf of the company, said, "We cannot afford to delay access to this transformational treatment." He said there's evidence micro-dystrophin levels after treatment are a good measure of its effectiveness, and that Sarepta does have preliminary evidence the treatment is, in fact, helpful.ĭuring testimony from the public, Nathan Plasman described calls he'd get from his wife after their son was treated as part of a Sarepta study. ![]() Sarepta presented a complicated mix of results from animal and human studies in support of its application for an accelerated approval of SRP-9001. The company's request for approval rested mainly on how much micro-dystrophin the treatment produces in patients' muscles instead of waiting for clear, real-world evidence that it's actually helping patients. ![]()
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