Ann: FDA Accepts Mesoblast's BLA Resubmission for Remestemcel-L, page-310

Hi @Fastback6666

I share a sense of relief. When I examine it a little more closely, some of the key reasons seem to be:

- firstly, that yesterday's announcement was of action by the FDA & not Mesoblast. Because it was a tangible step in the administrative process of the BLA & outside our control (i.e. MSB's control) it is more "real" & objectively certain than things we hear the company saying it has done on its own. We could go & search the FDA's registers and check it out further if we wanted, but there's no obvious need. It's more 'legit' (as Prof. Kurtzberg might say).

- secondly, also because it was an action by a 3rd party, it indirectly validates what we have been told by MSB that it was doing to progress the BLA resubmission to this point i.e. to prepare it in readiness for FDA consideration and substantive review. FDA accepted it for review, therefore it was ready to be accepted for review. That's nice.

- thirdly, on my own analysis the market may not yet have given Mesoblast credit for what this acceptance achieves. There are those downrampers and short-selling promoters who say this was only ever a 'tick-the-box' exercise. What a pathetic joke. Nothing could be further from the truth.

Consider:

A. FDA has created a whole new set of legal & regulatory obligations for itself, despite scarce CBER administrative funds & other resources. If it wanted to merely buy time, it could have delivered a refusal to file note to MSB. But instead FDA has created an entire new tiemframe-driven process review for itself & its live now;

B. The key process review that FDA has undertaken voluntarily relates to what is likely to become the first allogeneic cellular therapy approved in teh United States. That first-in-class status will necessarily attract extra attention and resources. Whole new sub-procedures will be tested for the first time. FDA has now committed to doing this, despite the time pressures. It's a big deal.

C. Mesoblast has been given a PDUFA date. This is a highly significant, almost-tangible legal and regulatory commitment by the FDA. In addition to CBER's internal policy-driven timeframes, it has the effect of a promise against which the FDA is willing to hold itself accountable to the US Congress and pharma industry stakeholders. That, in itself, is a valuable asset granted to MSB (and, indirectly, MSB holders).

D. The FDA reviewers (see link: SOPP 8405.1), especially the Regulatory Project Manager and at least one of the Review Committee members, have conducted not one, but at least two, initial reviews to ensure firstly that all data has been entered into the appropriate regulatory system, including assignment of a third level STN (i.e. a SubmissionTracking Number), if appropriate, secondly to confer about the RPM's initial decision that the resubmission looks like a complete response, and then thirdly, to decide which Classification is appropriate. These can be very time-consuming matters. Especially the sign-off on the data formatting of the entirety of the data accompanying the submission - e.g. making sure every cross-referenced data item links appropriately. That data will include the off-site data referenced in the submission.

Not 'tick-the-box' at all is it @whytee.

PS: The FDA likes to use the same Regulatory Project Manager throughout the entire process. Think about reasons why MSB may have delayed it's submission. Interesting, but I digress.

E. Lastly, as a comforting thought to those who think we've cast our dice & now have to just sit on our hands 'in the lap of the gods': even though Mesoblast has lodged the requisite documents for CBER review, Mesoblast still retains the regulatory right to make a major amendment to its BLA Resubmission with limited downside - see, for example, Section V.C of SOPP 8405.1, extracted below:




So, even though MSB will reserve the right to make a major amendment prior to the PDUFA date, that amendment will only extend the review clock by 3 months maximum.

This is a hell of a deal IMO.

As I see it, any other cell therapy biotech in the world would cut their CEO's left arm off to be in this position.

So, yes @Fastback6666 I feel the approval seems a bit easier from here.

Cheers all MSB holders.