Sarepta readies FDA pitch after a small study spotlights 1% dystrophin add for golodirsen in Duchenne MD
After winning an FDA approval for Exondys51 as a new therapy for Duchenne muscular dystrophy without having to actually produce data to prove it worked, Sarepta $SRPT followed up this new morning with a fresh set of data from a small study underscoring the ability of its experimental followup exon-skipping drug golodirsen to trigger added production of dystrophin.
The numbers it cites, though, are tiny. Despite that, Sarepta is telling analysts today that it plans to see if the FDA could be willing to give it an accelerated green light.
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