CHF, CLBP and ARDS all appear to offer paths to approval. They clearly work in particular doses on particular sets of patients with particular criteria. The question is whether with the current Phase III data it will be enough to win an approval for ANY of them in less than, let's say, 3-4 years.
For CHF and CLBP in the US, it seems we won't know anything about the path to approval MSB is choosing to pursue until after they meet with the FDA to obtain guidance on their view, the FDA's potential openness to accepting secondary endpoints from those trials in spite of failed primary endpoints, clear effectiveness in respect of certain subgroups, etc. If the FDA gives the thumbs down to the results, we will likely be right back in trial mode on both conditions, with no hope of interim approval in spite of SI's initial bullishness on such pathways to approval (though admittedly, approvals without further trials were never really viewed as likely to be obtained for these treatments in any event).
MSB is likely still slicing and dicing the data from those trials to see what the best way is to frame the data and make their pitch to the FDA. I haven't heard anyone mention timing for those initial FDA meetings, but I would imagine they are likely a few months off at least, and with all that's going on what ARDS, maybe longer?
For CLBP in the EU, I would HOPE that Grunenthal is already working with MSB to put together an application to the EU authority for an EU trial. The initial data that was released certainly seemed compelling for opioid use reduction and pain reduction in patients that had been suffering with CLBP for less than 5 years. But was Grunenthal actually convinced? I would guess that they and MSB are carefully reviewing that data to agree on whether the conditions precedent for Grunenthal to apply for/undertake such a trial have been met through the CLBP results. We haven't heard a peep about Grunenthal's reaction to the data, however, let alone whether they do indeed intend to apply for an EU trial and when such an application might be made. Likely more months of waiting here, then.
For COVID ARDS in the US, I would expect that MSB and Novartis are huddling over the data to agree on the best path forward (and again, to agree on whether any conditions precedent have been met, or if they haven't, whether Novartis still wishes to proceed).
Given the high priority of COVID-related treatments, I would also expect that there is a active back channel between NIH and MSB, and quite likely the FDA already, too (particularly if the results of the trial are as clear cut re: effectiveness for under 65s as MSB indicated in the announcement). If Novartis remains in, I would expect it to slide directly into the driver's seat for any EUA application with the FDA, if it's not already heavily involved.
If Novartis bails, then it's going to be MSB going back to the well with the FDA on their own to make any EUA pitch. Oh, and if Novartis bails, that's likely a very good indication that the results from the COVID ARDS trial were NOT as clear cut and overwhelming as MSB made them sound in their announcement, which may not bode very well for an EUA in any event...
Postively, though, you'd expect that given their involvement, the recent announcement would have been crafted in conjunction with Novartis, or at least been run past them for comments/suggestions. Same with SI's comments about the Novartis deal and the timing of a "Yes" or a "No" re: the contract. He's not making such comments in a vacuum. Here's hoping his giving a FIRM date ("within the next two weeks") was done with Novartis' blessing. Will be good to just have that question answered one way or another, as the uncertainty is NOT helping things.
For COVID ARDS plans for any other market? Remember that there was early talk of trials in the EU and the UK, and locations in Oz were added to the trial, but there has been zero information released in respect of any other possible plays in other jurisdictions. The India via Japan play really does sound like a no-brainer to me, but what the heck do I know?
Finally, I think we can expect that the AGVHD appeal application will be quickly updated with any positive data that can be gleaned from the COVID ARDS trial, particularly in respect of bio-markers that were measured. SI was bullish that this new information was going to be persuasive to the FDA, and other folks on this board and elsewhere have similarly voiced views that this information and other compelling data (along with a groundswell of discontent from pracitioners to the FDA's heavy-handed rejection of the 9-1 ODAC vote) could be what finally gives the FDA a good basis to reverse its decision. It would be great to get a more detailed update on the status of the dispute resolution process, but MSB has put out VERY LITTLE on this point. Still, maybe we get an update in the next 4-6 months?
So where does this put us now? Waiting, waiting, waiting, and forced to read the tea leaves of any announcement that comes out. And a LONG way to ANY approvals, outside of AGVHD or COVID ARDS in the US (where Novartis being "IN" will be a huge tick of approval and speak volumes about just how compelling and convincing the ARDS data REALLY is, and could drag the FDA kicking and scream to both an EUA and AGVHD approval).
SI, you really had better make good on your promise of news on Novartis in "two weeks"...
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