FDA still feel they are missing the MOA and its relationship...

FDA still feel they are missing the MOA and its relationship with bio markers. The adult trial will track a lot more biomarkers and MSB will also need to track more on-vitro assay properties. Although this issue seemed to be played down as a by the way comment in the announcement, I feel there is more to it.

If Rem-L had another approval under its belt which demonstrated efficacy via an RCT, like Jakafi, then they would have had a better shot using the 001 trial data. FDA even stated, somewhat unfairly, a number of studies even outside aGVHD that alogenic MSC's had not shown efficacy. So from their 'scientific' perspective they have no large scale RCTs which have ever shown efficacy to predefined endpoints for any indication.

We really need the ARDS trial to address these gaps. This trial did track in-vivo markers and hopefully the current CQA's will match up with efficacy. Ironically, because of the tighter control in manufacturing on these CQA's we probably won't have the variability in them to show they correspond to efficacy. A point which SI turned black and blue trying explain and re-explain to the FDA dummies and some ODAC members. The old and new manufacturing were the perfect unintentional control for identifying and proving the CQA's.