In the context of approval around 90% of treatments never get there, and it's probably even higher for stem cell approvals ( it may be closer to 99% - but JB or someone else can correct that number or phrase). Statistically then, more than a 90% percent chance that MSB may never ever get Ryoncil approved which would mean waiting forever.
The light at the end of the tunnel, is the end of the tunnel and not a train at the moment. They have done 3 large, multi centre, gold standard placebo controlled trials for Rexleme, and those results are going to leave no room if repeated for anybody to say they don't work.
The facts are if FDA approve Ryoncil then FDA are saying the evidence is it works, and congradulations MSB you are one of the first. The new data was not against the Jakafi control arm, it was against Jakafi treatment. 
Look at the Severe response rate - Jakafi is at 40%, control is at 19,%, ( 180 days )
Day 100 Survival Remestemcel-L Matched Controls
Protocol 280 (pediatric), Grade B-D 79% (n=14) 54% (n=13)
Phase 3 (Study 001), Grade B-D 74% (n=54) 57%(n=30)
Expanded Access Protocol 275 66% (n=241)
Expanded Access Protocol 275, Grade D 51% (n=51) matched controls -> 31% (n=327)........ Jakafi 40.7%..... Jakafi control 19%
Jakafi was against BAT..... no doubt 275 would have also been given BAT.
Even the expanded access is over 50%, which was only in grade D, which is more comparable to the Jakafi result which was 40% for Severe... with Jakafi control arm being 19%.
Protocol 280 B-D 79%, might be comparable to Jakafi Moderate at 59% and control at 32%.
Remember the FDA were the ones trying to say it's the same disease to MSB in adults and children and therefore the results etc should be comparable, which is why they included all the old trial results which included adults.
The FDA made a mistake by including the data from old, different release criterea product - which had different outcome, and then the " Downrampers " on here blamed MSB for cherry picking patient results to try and match patients from the " old treatment " trials, with the quality of treatments from the new trials. They only did that, beasue the FDA insisted to look at the old results.
A bit like a car manufacturer taking a new model car, with a new ECU fuel map to get registration plates where it passes the emissions control standards, and then the RTA denying it because the same model car from 10 years ago failed the emmissions test, and then the manufacturer pointing out the differences and even then managing to find some old cars that actually match the new factory release criterea... and those results also pass the emissions control test.
All available indications continue to point towards the cells are not only massively better than control in the GvHD001 which we are already aware of, but using the Jakafi reach study, and several other studies that happened after the BLA was lodged, and also looking back at the 275 / 280 trials, the results still continue to show the same seperation to controls. These were prepensity matched controls... from the same hospital, at the same time MSB was holding their trial..... the only thing that was not done is that they were not included as a placebo arm.
The Downrampers on here continue banging on about " old " Failed trials"..... yet the funny thing is that once those "old", " failed trials" are also propensity matched to patients that recieved the improved treatment........ the results are consistent.
Given FDA insisted on the inclusion of the older trials, and now MSB has added the relevant results from the older trial to the IND file, as well as adding several more trial and study comparator arms, as well as the propensity matched Magic arms..... well it's going to be hard for the FDA to justify that the data is still not enough because on the face of it - another trial is simply goind to yield the same results again and cost 20 or 30 children's lives per year in order to satisfy the FDA's own science experiment.
It's not as simple as saying the old trials failed, and MSB got lucky on GvHD001..... looking at the improved product used in all the studies that it was used - it worked.
(20min delay)