Ann: FDA Advisory Committee Sets Review Date for Remestemcel-L, page-705

I’ve hitched my wagon to a few underperforming biotech nags over the years, so until recently, I wasn’t too familiar with the FDA’s approval processes. To be honest I was an ODAC virgin until 6 weeks ago, (happy ending).

For that reason I haven’t posted predictions on likely FDA outcomes or share price implications, much as I appreciate everyone else’s. But with the critical decision looming, I’m tempted to cross the line.

At a roulette wheel, after a few reds in a row, watch the money pile on black. Why? Because the punters figure there’s more chance of a black result next. Wrong. Every spin is an independent event (if the wheel is legit). It doesn’t matter what’s come before. Apart from zero, 50/50 each spin.

So are FDA decisions also independent events? Is every application considered independently on its merits? You’d hope generally yes. But does that apply in this case?

Given the unmet need, efficacy and unquestioned safety profile data of remestemcel-l for pediatric SR-aGVHD, I think the odds strongly favour approval. If the FDA were to knock back the BLA, the only grounds I can see could be batch consistency/QA, that they hammered at ODAC. (And I suspect MSB and the FDA would be in earnest discussion on any issues).

My suspicion is, worst case, a conditional approval with a confirmatory Phase 4 adult aGVHD trial. IMO this is almost certainly what the lawyers at ODAC were trying to avoid, putting other adult treatments under threat.

After some questionable FDA Covid-19 therapeutic approvals, and given the looming proximity of the U.S. election, if the BLA was rejected on QA grounds, and Mesoblast’s ARDs and MIS-c trials then come up trumps, I can’t see how the FDA could then give approval for these conditions.

So back to the roulette analogy. I don’t think MSB’s spins of the FDA wheel are independent. If the FDA approves for SR-aGVHD this week, imo it considerably improves the chances of subsequent approvals.

In the age of Covid-19, promising therapeutics and vaccines are in the spotlight. While I think the focus on MSB up to now has been underwhelming to say the least, that could all change very quickly.

Hopefully it all starts this week.

Usual bias. Herro