MSB to dispute FDA finding in Type A meeting, page-303

Only my take but incl some links.
From ODAC meeting, plans already underway, pre-empted.
There was also discussion by the panel of wanting to see a randomisedcontrolled trial against best available treatment in the adult setting, where thereis already an approved drug Jakafi. Panel asked MSB about their plans around thatand MSB confirmed that they are committed to running a post marketing study in Adultswith Severe SR-aGvHD. They had already convened an advisory board of GvHDexperts to weigh in on this trial on 1st Aug’20. They will be looking to run a superioritytrial to show improved response rate and survival in high risk adults (high unmet needdespite approved therapies or those that do not respond to existing therapies). In thistrial the optimised remestemcel-L product will be used.

From Mesoblast August product updates:
Remestemcel-L for Adults With SR-aGVHD
Beyond pediatric SR-aGVHD, Mesoblast will seek to obtain approval for RYONCIL in adults with the
most severe forms of SR-aGVHD. In an earlier randomized placebo-controlled Phase 3 trial, a post-hoc
analysis showed that remestemcel-L was associated with an increased Day 28 overall response in
steroid-refractory patients with Grade C/D disease. This patient population continues to represent a
high-risk population with poor overall survival, and in August 2020 Mesoblast convened an advisory
meeting with key opinion leaders to develop a clinical trial design for a post-market study evaluating
remestemcel-L in this patient population.

I am a little confused that MSB stated above that Phase 3 already showed results post hoc analysis?? Yet why reference a pediatric study as part of the case for Adults yet to be trialled at P3 level.
If anyone can find a results readout for this P1/2 adult clinical trial.....good luck

Edisons October '19
The much larger market is adult use. Mesoblast has not run trials in adults so this indication
depends on gaining a label extension, which may require further adult trials. In Europe, this might
be a conditional approval, which is a full approval requiring further data and a subsequent review;
Zalmoxis was EMA approved on this basis. There was a 244-patient, Phase III randomised,
placebo-controlled study run by Osiris (before the product was acquired by Mesoblast in 2013).
Data were released in 2009 but the trial failed to meet the primary endpoint, although there was a
response in adult subgroups with liver (p<0.05) and gut (p<0.05) involvement15 and the paediatric
subset.

Phase 3 for Adults was always coming - because Product candidate table shows only Phase 1/2 stage for Remestemcell-L link: https://www.mesoblast.com/product-candidates/product-candidates-overview
If anyone can find a results readout for this adult clinical trial.....good luck
FDA has always used randomized placebo controlled studies - Mesoblast chose single arm study for pediatric AGVHD, something the FDA would be unlikely to accept because it is not their design for a clinical study.Was always going to be a sticking point.

https://www.sciencedirect.com/science/article/pii/S1083879120300513 as a reference to the current submission.