Ann: FDA Notifies Clinical Data Sufficient for Refiling aGVHD BLA, page-69

Hi Reg,

yes, you're right we're still 'not there yet'. But I'd submit the 'remaining issues' language is a reference to the outstanding CRL, which needs a formal response. In any case, I'd see it as a normal reservation to have while we are step-wise following SOPP processes, & IMO it doesn't translate necessarily as 'problem'.

I agree that, for you and me, the characterization issues that 'remain' could use more definition. But it may also simply be e.g. that Klinker doesn't want to be seen as as rubber stamp, and wants his role to be respected. OK. He comes back into the process after a CRL which listed 'product characterisation' issues as TBA. He can say 'yes, it looks good, but we need a) the formal documentation and b) time to check all the data you've provided'.

I don't see how the FDA can invite the resubmission of a BLA while these issues are so problematic that they can't be resolved during the resubmission process. Perhaps they (FDA) simply need a little more time to digest fully the data, but - on the strength of what they have seen - are prepared to do so in tandem with the preparation for filing and consideration of the resubmission of the BLA itself. Remember, the FDA's intended Review Team members would have been in attendance at this meeting, under the terms of PDUFA VII.

BTW, the signoff on potency assays and potency assurance strategies means IMO that the adult trial can now go ahead. Expect more announcements.

Think happy thoughts. If nothing else happens this year, its still going to be my shout for those oysters we're going to be consuming at the AGM after-party.

You can buy the beers.

Everyone should DYOR, of course.