The Food and Drug Administration’s surprise decision to reject the company’s Duchenne drug Vyondys 53 on Monday is another twist in the crazy, never-boring Sarepta Therapeutics story.
You can read more about the FDA’s denial of Vyondys 53 here, but read on for some additional thoughts, analyses, and lingering questions for investors trying to figure out what it all means for Sarepta moving forward.
No one really believes Sarepta’s stated reasons for the rejection.
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Sarepta’s lack of foresight in planning for needing more data since 2016 is going to majorly impact this company’s ability to perform. Not only are they behind on follow up data with Exondys 51, they also had the hubris to think they could get accelerated approval with less data with Vyondys 53? It’s no wonder FDA rejected this drug.
If what Adam says is right, they now have to rely on their internal pipeline almost completely for growth. No company with late phase assets is going to want to do a deal with these guys for bio bucks, so they have to rely on their current pipeline to get anything done or pay through the nose upfront for anything later stage.
All around bad for investors and especially bad for patients and their families. This company should have been much more responsible with the resources it was given over the years to build a credible rare disease franchise.