It depends entirely, doesn't it, on what the FDA commits to (if...

It depends entirely, doesn't it, on what the FDA commits to (if it will "commit" to anything) via the proposed "salvage therapy" trial on adults as a result of the Type A meeting?

The proposed trial is indeed clearly NOT an RCT, and if that's the only thing that the FDA will accept in order to obtain approval for Rem-L in respect of AGVHD, then why bother running it at all? It would seem to make more sense to just head straight to an RCT on those same patients.

Or would it be, a'la Southoz's comment, that they are really only at P2 with this new salvage therapy trial on adults given their limited data in that group and thus couldn't run an approved P3 RCT until that first trial is completed regardless?

While it seems somewhat unfair that the FDA wouldn't approve in the face of ludicrously improved survival outcomes in these worst-of-the-worst cases using generally accepted mortality rates, they rejected essentially identical data in respect of these kids in the most recently rejected application and for much longer time periods.

It will be very interesting to see what SI can get from the FDA in writing.