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.