CBER Director Marks’ Intervention On Sarepta Gene Therapy Filing Decision Appears To Backfire
Former OTAT Director Wilson Bryan says Peter Marks’ order to file Sarepta’s DMD gene therapy application was mainly a public relations move, which does not seem to have worked in the FDA’s favor. Upcoming advisory committee could now involve as much scrutiny of the agency as the application itself.
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Sarepta’s DMD Gene Therapy: Approval Delayed And Indication Narrowed, But Still On Track
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.
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.
Slim Adcomm Majority Boosts Sarepta’s Gene Therapy In Duchenne Muscular Dystrophy
Accelerated approval seems imminent after a majority of the US FDA’s Cellular, Tissue and Gene Therapies Advisory Committee voted the risk-benefit balance supported it.