An amended version of an earlier post: On the FDA website, they...

An amended version of an earlier post:

On the FDA website, they indicate a desire “to encourage innovation so that the medical industry can properly harness the potential of stem cell products.”

https://beta.regulations.gov/document/FDA-2020-N-1539-0038

If the FDA are genuinely concerned to "encourage innovation", a positive gesture would be to allow MSB a reasonable middle ground outcome at the Type A meeting, (assuming a meeting is granted).

The industry will be watching.

If an ethical first mover like MSB is seen to be repeatedly stonewalled by FDA bureaucracy, the innovation the FDA professes to encourage will be stifled, and the investment capital stem cell industry players rely on, so much harder to find.

And who would win from that? Certainly not patients. The obvious answer is drug manufacturers for the conditions MSB is targeting.

The FDA has an opportunity here to back words with action. A conditional tentative or accelerated paediatric SR-aGVHD approval, pending a confirmatory trial in adults, is the middle ground I'd be hoping for. An adult trial would then be money well spent, because it offers potential access to a much larger market.

I wouldn’t consider an outcome such as an ongoing EAP for paediatric SR-aGVHD to be adequate. Yes, an EAP is a clear concession to needy patients, but also an initially unrewarded drain on MSB’s inventory and funds.

Perhaps more significantly, should the FDA use its previous arguments about critical quality attributes or verifiable efficacy to further block approval, they could be painting themselves into a very tight corner.

I strongly suspect that at some point, the ARDS trial will confirm efficacy.

After some dubious Covid drug approvals already this year, a positive ARDS result would inevitably provoke another embarrassing (for the FDA) backflip, forcing them to grant Remestemcel-l approval as the ARDS therapeutic of last resort, (and a rethink on GVHD). The FDA would have no choice imo; political and public pressure will force their hand. Too many lives depend on it.

As some have suggested, yesterdays announcement hints that the FDA might be stalling on GVHD, pending the ARDS result, and may perhaps approve remestemcel-l for ARDS and GVHD at the same time.

MSB have multiple shots on goal over the next few months. I remain cautiously optimistic.

Herro