CHF Accelerated Approval !, page-86

"with potentially serious outcome for which confirmation of the result with a second trial would be practically or ethically impossible."

Because we aced a reduction in MACE, and there is an unmet need in this respect, the sticking point for us IMO regarding the outcome for a single trial approval may well come down to the question of ethics. Because we are dealing with Major Adverse Cardiac Events, and because this type of trial must measure actual incidences of these events, the trial obviously requires time to see incidences of heart attacks etc. So the FDA will likely be giving serious consideration to the risks of running another trial (i.e during a reasonable period of time it takes to run another Phase III, the data/results indicate that there will likely be a significant number of ischemics and diabetics with CHF that will die if Rex-L was not approved on a single trial basis). They may consider this unethical and instead ask Mesoblast to submit an application for approval for ischemics and diabetics and consider approving for this patient population and likely ask for a confirmatory Phase IV trial in this group to confirm our Phase III results.

Ethical question may go either way and of course there are other factors to consider. But as I have said before, IMO there is still a chance that we will may be encouraged to formally submit an application for approval based on above groups.