Hi
@irenekwshiu,
The exact words used by Mesoblast in the announcement were:
"
FDA ..informed (MSB) that .....clinical data appear sufficient to support the proposed BLA filing"
The first thing to note - we aren't talking Minutes here. So, whatever else, Mesoblast must be very comfortable that this statement will not embarass it before the FDA or prejudice the Minutes to be delivered in due course.
And the question then is what was '
proposed' by MSB. Obviously, I don't know, but what I expected from CEO Itescu's prior statements etc was that the submission to this meeting would likely take the form of a proposal of a filing for
accelerated approval, with standard approval as the alternative.
But I took the quote above to be intended to communicate that the GVHD-001 trial could
now (i.e. after '
further consideration') be accepted by the FDA as an adequate and well-controlled clinical trial for the purpose of its inclusion in an application for
standard marketing authorization approval. If I'm right on that, it would mean the clinical data was now considered to have the support of appropriate potency assurance. With adequate safety evidence, that would satisfy all standard approval requirements - apart from any non-potency CMC matters remaining after the PLI - and no further confirmatory trial would be required byt the FDA. There'd be no 'sword of Damocles' hanging over Remestemcel-l if & when SR-aGVHD approval were to be granted.
You'll recall that at the Feb 29 2024 Earnings CAll, CEO Itescu advised attendees:
"
And I'm pleased to say that we've achieved all of the deliverables during the first quarter of this year, and we have a number of planned activities for the rest of -- for the next quarter and the rest of the year.
...
And as you can see, in particular, with respect to Remestemcel for adult and pediatric GVHD, we achieved the expected delivery of additional potency assay data, which was provided to the FDA. We have scheduled an upcoming meeting with the FDA that will be held in March.
We've achieved completion and submission of a protocol for the adult program, and we plan to initiate enrollment in the next quarter for this adult trial...."
It seems reasonably clear all of this would have been considered by the FDA at the meeting. Of course, we'll wait for the Minutes.
But, to me, if further confirmation were required from a shareholders POV that the BLA can now proceed to be lodged as a standard approval for paediatric SR-aGVHD (and I don't consider it is), then any announcement of
ANY news about the adult program initiating enrolment or its protocol having been settled, or similar, will put that beyond doubt. Why?
Because to me the potency assurance strategy 'concerns' raised by the FDA previously with Mesoblast would have had to be agreed by the FDA to have been settled before the BMT CTN would commence to put its resources in play for that adult trial.
Hope that assists.
Cheers
GLTA(LT)H
(20min delay)