Eteplirsen NDA Submission Plans for Rare Neuromuscular Disorder

Sarepta Therapeutics has announced its plans to submit a New Drug Application (NDA), by the end of 2014 for eteplirsen for the treatment of Duchenne muscular dystrophy (DMD), an X-linked rare degenerative neuromuscular disorder. Eteplirsen is an exon-skipping drug candidate in development for the treatment of patients with DMD who have a genotype amenable to skipping of exon 51.

The plan is based on a FDA guidance letter that proposed a strategy to submit a NDA for eteplirsen under a potential Accelerated Approval pathway. The FDA stated that, “with additional data to support the efficacy and safety of eteplirsen for the treatment of DMD, an NDA should be fileable.” Examples were also outlined for additional data and analysis that, if positive, will be important to enhance the acceptability of an NDA filing by addressing areas of ongoing concern in the existing dataset.

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The FDA provided clear guidance on an open-label, historically controlled confirmatory study of eteplirsen, as well as initial guidance on a placebo-controlled study of one or more follow-on DMD drug candidates, which, like the open-label study, could also be considered an acceptable confirmatory study to verify the clinical benefit of eteplirsen in the event of an accelerated approval.

Sarepta plans to initiate several additional clinical studies with eteplirsen later this year in exon-51 amenable genotypes. These studies will include a clinical trial with predefined efficacy endpoints for ambulatory patients between the ages of 7–16 years who can walk a minimum distance. Two other clinical trials will also be initiated that will evaluate safety and biomarkers in DMD patients younger than 7 years and DMD patients who have advanced in their disease progression to a point they cannot walk a minimum distance or have become non-ambulant. Additionally, Sarepta plans to initiate a placebo-controlled study with one or more of its follow-on DMD exon-skipping drug candidates by the end of the year.

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