Sarepta to file NDA for its Duchenne muscular dystrophy compound
US RNA-based drug developer Sarepta Therapeutics (Nasdaq: SRPT) plans to submit a New Drug Application to the US Food and Drug Administration for eteplirsen, an exon skipping compound under development for the treatment of Duchenne muscular dystrophy (DMD), in the first half of 2014.
Eteplirsen is Sarepta's lead exon-skipping compound in development for the treatment of patients with DMD who have a genotype amenable to skipping of exon 51. Sarepta met with the FDA this week, to follow up the agency’s review of two recently submitted summary documents that included data on dystrophin and clinical outcomes from the existing eteplirsen studies.
The FDA stated in pre-meeting comments that it is “open to considering an NDA based on these data for filing” but requested additional information related to the methodology and verification of dystrophin quantification.
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