Product Quality ‘Wild Card’? Sarepta’s Gene Therapy Manufacturing Process Change Means More Empty Capsids
SRP-9001 intended for commercialization is not analytically comparable to the product used in earlier clinical studies; FDA CMC reviewer said agency does not mandate the extent of empty or full capsids but, instead, looks at the totality of data to assess safety and efficacy.
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With characterization of cell and gene therapies still in its infancy, the FDA shares advice on how to demonstrate comparability between the versions manufactured for clinical trials and the versions to be produced for commercial use. Starting earlier with scalable manufacturing processes helps. Limited additional clinical studies may be needed in some cases.
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.
FDA clinical, clinical pharmacology and statistical review teams did not recommend approval of Elevidys, but the CBER director concluded that randomized data in a subgroup of patients ages four and five years old were ‘compelling,’ justifying accelerated approval; expansion beyond this age-restricted subgroup will hinge on data from the EMBARK confirmatory trial.