Sarepta’s DMD Gene Therapy: FDA Says Concluding Effectiveness Or Ineffectiveness Is ‘Challenging’
Concerns about the clinical trials, the manufacturing, and the preclusive nature of Sarepta's gene therapy for Duchenne muscular dystrophy raise questions ahead of the upcoming US FDA advisory committee meeting.
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CBER director does not want to hold back potentially beneficial products, but also warned that patients will have difficult treatment decisions to make since an immune response might prevent a patient from receiving another gene therapy using the same vector ever again.
Cell/Gene Therapies: US FDA Wants ‘Quantitative Correlation' Between Surrogate And Clinical Outcome, Former Reviewer Says
When weighing accelerated approval, the FDA wants to see that ‘X change in the surrogate translates into Y change in the clinically meaningful outcome,’ Parexel’s Steve Winitsky says. CBER Director Peter Marks opines on the types of therapeutic settings where accelerated approval is best suited, and what happens when a confirmatory trial disappoints.
Sarepta’s Elevidys: Evidence For Micro-Dystrophin Surrogate Endpoint In Young Patients Insufficient, FDA Reviewers Said
Clinical, statistical and clinical pharmacology review teams recommended a complete response letter, concluding exploratory subgroup analyses of data in four and five year-old DMD patients were insufficient; their views were overridden by CBER Director Peter Marks, who granted accelerated approval for the age-restricted subgroup.