Good morning @JB1975 ,Your comments- How can MSB possibly have...

Good morning @JB1975 ,

Your comments-
How can MSB possibly have at least one adequate and well controlled trial on the data they have provided? I say - they can't or at least I can't see any way they can without putting new batches of product into new patients

My opinion is that you are focused on the 001 trial and not the 2 CRL's.

CRL 1 questioned potency and efficacy

CRL 2 questioned potency of current stock intended for sale , matching 001 trial stock

Phil Krause

But the CRL did continue to question and raise questions about the potency assay. And specifically, the characterization and standardization of product that went into GVHD001, and then the ability then to make future product that was similar to that, which went into GVHD001.

And so, this leaves open the real possibility that by using exactly the same assays that were used to characterize the product going into GVHD001, for a new commercial product that it would be possible to

show that the new commercial product is similar enough to that, which was shown to be effective in GVHD001

. Now, the CRL then went on to say that, if that can't be accomplished, in other words, this demonstration, then the only way forward would then be another clinical trial in either adults or pediatrics, which would then allow this connection between potency assay and clinical outcomes to be made, and thus allow future product to be related to product that was shown to be effective in a clinical.

Left E over the ditch proposed this to OP

@OP, 1) the failed Osiris GvHD trial might have a paradoxical benefit. In order to prove that a chosen CQA correlates with potency, one needs to test it across a range from low to high and show that when it's low patients don't respond.... and when it's high patients do respond. Unlike some on HC, I suspect he's using the same two CQA's put forth with BLA 1, namely TNFR levels on the infused MSC's (quantitative) and IL-2R inhibition on host T cells (qualitative). They could retro-test frozen batches from the Osiris trial for those two thereby explaining why a significant number of patients failed there compared to later patients receiving the "new improved" Ryoncil.

My view is that the FDA is satisfied with the new data presented- there was no other way forward- either they were satisfied or a new trial had to be conducted.

Ryoncil works- they have established efficacy in a clinical trial- they have provided data that appears sufficient to get approval.

Your point was
- I say - they can't or at least I can't see any way they can without putting new batches of product into new patients

It appears they are going to be putting the same batch of product into new patients very soon - this time via sales and not emergency access protocols.

Prochymal did not work - Ryoncil for the win

Reg