By: Adam Feuerstein | 04/08/14 - 02:34 PM EDT
It's time to prep for more
Sarepta executives met again with FDA officials in March to nail down a regulatory path for the Duchenne muscular dystrophy drug eteplirsen. The company has promised to update investors about the outcome of the FDA meeting, but without saying exactly when. This week, Sarepta cancelled a presentation at an investor conference, which of course, has everyone speculating about an imminent eteplirsen announcement. What does imminent mean? Tonight? Tomorrow? Who knows, but let's hope Sarepta tells us something before April rolls out.
My Sarepta crystal ball cracked last year, but let's work through the scenarios:
1. FDA does a total 180 and agrees to let Sarepta file for approval of eteplirsen based on the phase 2 study and the subsequent supportive, follow-up data. Remember this FDA letter? Grand slam home run!
2. FDA doubles down on being a total hard-ass, insisting Sarepta run a phase III study enrolling a large number of DMD boys, some of whom will be forced into a placebo control arm. Ouch.
3. Sarepta and FDA reach a compromise. A phase III study is required but it can be on the smallish side and use historical controls or boys with non-exon 51 skips as the comparator. The most expected outcome.
4. There is no decision on an eteplirsen regulatory path yet. Sarepta and FDA continue to negotiate and will meet again soon. Ugh. Torture.
5. Sarepta disagree with FDA's position (whatever that is) and decides to submit an eteplirsen approval filing anyway -- essentially daring regulators to reject the drug. Wow! Gutsy but perhaps incredibly foolish.
[Thanks to biotech trader Dan Rosenblum and Baird analyst Brian Skorney for helping me work through the eteplirsen scenarios.]
Meantime, investors and everyone else waits for Sarepta to say something.
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