Sarepta Therapeutics anticipates completing a rolling submission of its new-drug application for Duchenne muscular dystrophy drug candidate golodirsen by the end of the year. The company obtained positive Phase I/II study results in which the exon 53-skipping treatment increased mean dystrophin protein levels to 1.019% of normal at week 48 compared with 0.095% of normal at baseline in Western blot measurement, and will request accelerated approval for the filing based on an increase in dystrophin protein as a surrogate endpoint.
Sarepta to file NDA for Duchenne muscular dystrophy drug by year-end
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