Thanks
@col69 - so a few very interesting responses.
"Sure. I will make a quick point here and that is the CRL had, although, of course, the disappointment of being a CRL, but had two positive indicators in it as well or two significant positive indicators. One of course was the favorable results of the inspection, but the other important thing to take note of is that, the
CRL did not question the efficiency of the product as was demonstrated in GVHD001, as had been the case in the previous CRL.
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. So, maybe I will stop there, but could take a follow-up to previous one."
I'll take it that the above should have been translated was wrongly translated as "efficiency" when it should have been efficacy - right?
Anyway, the CRL is definitely all about the potency assay and the last paragraph is what has to be done if Mesoblast cannot demonstrate the "the characterization and standardization of product" - so it's not about more data for efficacy, but more data to confirm the consistency of the product.
That is the big difference to me!
GL MSB'ers - let's hear what the FDA has to say from the Type A meeting, but I'm feeling a lot better now.
I can appreciate why there are people eager to buy on market now, e.g. Gregory George
(20min delay)