Cell Therapy News/Articles, page-6985

I would imagine that MSB is paying serious money for experienced lawyers in helping them navigate this process. Unfortunately, they aren't going to make us privy to their precise strategy, but the work that we are doing to crowdsource what may potentially be going on with their thinking is red meat to experienced FDA practitioners.

I assume there is a clear method to MSB's madness - as well as to the FDA's madness, too.

I know ARDS isn't AGVHD, but I would NOT be surprised if the FDA was really just buying time with the CRL to let the ARDS trial work its way through to (early, hopefully) completion. It seems logical that it will be easier (and less controversial) for the FDA to agree to a conditional approval for pediatric AGVHD in exchange for a required RCT in adults...where the ARDS trial has resoundingly proved efficaciousness AND effectiveness for ARDS.

Indeed, who's to say that the CRL wasn't a RESULT of the ongoing ARDS trial - where results there are just around the corner, so rather than be seen to be going out on a limb with a standalone approval for AGVHD (as they may have done if not for the 300-person RCT), the FDA decided it better to wait for something really compelling as proof of concept. In that regard, combine the single-arm pediatric AGVHD trial with the (hopefully successful) results of the ARDS trial and it's not difficult to extrapolate out to likely success in an adult AGVHD trial.

And if the ARDS trial flops...well, the FDA won't have egg on their face from having given an approval for something not demonstrated by an RCT.