banter and General Discussion, page-8253

Hi @Lopez


that's exactly what I'm thinking as far as the improved (Potency 1b) assay data from runs vs trial stock goes. And I'm dead certain that if I was BMT CTN that's what I'd be telling the FDA I wanted on paper before getting going (because of FDA's 'form' on potency assays of RYONCIL (cf. PROCHYMAL)).

But IMO the connection to the actual paediatic indication approval doesn't need to be made a 'black & white' pre-condition for BMT CTN to start the adult trial - and that would be improper as I see it (and I'm sure the FDA would see it that way too). So, I don't think they did that. It's enough that the FDA admits that the product to be used in the trial will be sufficiently characterised for Potency 1b to be a 'suitable' potency assay for the upcoming adult trial. That's when the die is cast.

And that's where the real beauty of this approach begins to flower, in my mind.

All that I think MSB needed to get things going in the right direction was the FDA's signoff on Potency 1b as a validated assay i.e. capable of supporting release should efficacy/ safety etc be demonstrated in the adult trial with BMT CTN (in due course).

Then I think MSB took that Potency 1b assay and is now collating data from what's, in effect, a backward-looking comparability study against the assay in place during GVHD-001 i.e. Potency 1a. If the data is there to show that Potency 1a is 'very similar' i.e. comparable with Potency 1b, this will enable pooling of data forward across the adult trial datapoints but, more importantly, it will enable IMO an immediate relodging of the paediatric BLA with a 'suitable' i.e. fit-for-purpose potency assay. More about this later...

There's more than one way to skin a cat.

Have a good one
Cheers