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.
I've been wondering where things stand as far as the phase 3 trial (regardless of accelerated approval). Seems that if it needs to be a large trial, most of the boys who can benefit from it would be enrolled in the trial anyway…though a placebo arm is scary for the parents in kids involved.