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.
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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.
As Sarepta again brings a product application from the brink of rejection to the cusp of approval, the one-month review delay creates more assurance the confirmatory EMBARK trial will be completed as well as providing more time for label negotiations with the US FDA.
Company’s prediction that Duchenne muscular dystrophy patients likely would not be able to access SRP-9001 until four months after accelerated approval eased some US FDA panelists’ concerns about interference with the ongoing confirmatory trial.