Sarepta's shares jump as FDA seeks more data on eteplirsen filing

Sarepta Therapeutics said that the FDA requested dystrophin data, as measured by western blot, from biopsies already obtained from an ongoing confirmatory study of eteplirsen as part of its ongoing evaluation of the drug for the treatment of Duchenne muscular dystrophy. Shares in the company jumped as much as 31 percent on the news.

Last month, Sarepta indicated that the FDA would not be able to complete its review of eteplirsen by the target goal date of May 26, raising the possibility that the regulator could approve the treatment despite an earlier negative recommendation from an advisory panel. Sarepta said that it plans to submit data from 13 patient biopsy samples, at baseline and week 48, to the regulator over the coming weeks.

Recently, shares in Sarepta declined after the FDA unveiled changes to the process used by doctors to request expanded access to investigational drugs and biologics. The announcement led to speculation that the regulator might want Sarepta to provide eteplirsen on a "compassionate use" basis instead of granting accelerated approval. For related analysis, see ViewPoints: Regulatory delay for Sarepta's eteplirsen provides hope for patients but sharpens scrutiny of the FDA.

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