Sarepta Therapeutics (SRPT) shares cratered in November after the FDA told the company not to seek accelerated approval for its Duchenne muscular dystrophy drug eteplirsen.
But what if FDA is changing its mind?
FDA official Catherine Chew is sending a very interesting letter to people who write the agency in support of eteplirsen's early approval.
I've reprinted the full text of the letter below, but check out this paragraph:
We understand that you feel that eteplirsen is highly effective, and may be confused by what you have read or heard about FDA's actions on eteplirsen. Unfortunately, the information reported in the press or discussed in blogs does not necessarily reflect FDA's position. FDA has reached no conclusions about the possibility of using accelerated approval for any new drug for the treatment of Duchenne muscular dystrophy, and for eteplirsen in particular. [Emphasis added.]
Now, let's not get too excited. There has been no indication from Sarepta about a change in regulatory strategy, meaning the company is still focused on finalizing the design of the required eteplirsen phase III study. An agreement with FDA on that front is expected later this quarter.
However, Chew's letter, suggests some wiggle room for the possibility that FDA would... might... possibly... be willing to review eteplirsen based on the existing phase II data.
In fact, the same could be said for Prosensa (RNA) and GlaxoSmithKline's (GSK) drisapersen, if the companies decide to submit data from the failed phase III study.
Sarepta shares are down 10% to $18.33 today because Citibank analyst Yaron Werber downgraded the stock to a sell. He doesn't believe eteplirsen has a chance in hell of receiving an early FDA review. In fact, he doesn't expect eteplirsen to be approved before 2017.
Werber's view is close to consensus. But then there's this FDA letter making the rounds, so maybe Sarepta's door isn't shut entirely.
The full text of Chew's letter is below: