I believe some of the reasons a drug can fail approval is not meeting statistical significance or having a bad side-effect profile. I suspect its a cost-benefit approach. If the benefit isn't great, and the costs (side-effects) are significant, or the underlying disease being treated isn't that serious it may not pass i.e. FDA doesn't want the cure to be worse than the disease (remember Thalidomide).
Looked at from this perspective, Retts is about as serious a disease as you can have, and Trof showed both strong statistical significance and a low side-effect profile. The disease also has a well defined diagnosis and strong advocacy groups. You'd have to think Trof has already passed all its hurdles for approval with room to spare.
It's hard to see any hook by which it could be refused approval. More likely, I'd think, would be some delay while FDA request clarification on something. My understanding is its not uncommon for some drugs to go for approval a few times before they get through. A bit like one of Neuren's original Phase 2 trials was sent back to re-specify before it was approved.
I don't think a delay like this is likely, but if any negative outcome is likely, its probably this one, not an outright fail.
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