Ann: Mesoblast Type A Meeting with FDA, page-53

Contrary to many of the posts above I believe there is actually some real inconsistency here. Krause very clearly said in the recent post CRL conference that the FDA did not question efficacy:

"one of the significant positive indicators from the CRL - is that the CRL did not question the efficacy of the product as demonstrated in GvHD001, (which was the case for the previous CRL )" (Krause, 31 Aug 2023)

But then in this update:

"FDA noted that the lack of a suitable potency assay for the RYONCIL product used during the Phase 3 trial MSB-GVHD001 for the pediatric acute GVHD indication has prevented the trial from being considered an adequate study for the purpose of demonstrating substantial evidence of effectiveness required for a marketing approval."

If they didn't accept the potency assay, then they didn't in fact accept the efficacy as demonstrated by GVHD0001.

Perhaps I am wrong, but I think Silviu and Krause, are loudly here setting out the inconsistencies they are dealing with from the FDA themselves, for all too see, and showing all how they are arguing with the FDA on the validity of the potency assay and adding ever more data to back their claim and refute the contentions of the FDA, which they are showing as inconsistent and are picking away at.

"FDA indicated its willingness to consider Mesoblast’s proposed registrational trial design in adults, subject to agreement on the suitability of the potency assay for the product to be used in adults."

Clearly they are making some difficult progress on the potency assays with the FDA, both past used and those going forward and can't jeopardize that too much by too loudly pointing out FDA inconsistency, which has been shown innumerable times in other treatment decisions/processing by the FDA.

(happy be corrected by more informed MSB posters - not the peanut brigade)