Gene Therapy: Four-Month Lag In Commercial Access Protects EMBARK Study, Sarepta Says
Executive Summary
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.
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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.
Unpredictable DMD Progression Complicates Sarepta’s Gene Therapy Efficacy Claims
As with many rare diseases, a clear natural history would make deciphering the data easier, but for now what Sarepta characterizes as stabilization is seen by FDA reviewers as expected improvement based on previous measures of disease progression at younger ages.
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.