Oct. 25 at 12:39 AM
$CAPR
Part-3
4. And finally...PUL 2.0 did pass the initial futility analysis - which means the study monitors were noticing a difference at 3 months - which is expected to broaden at 12 months.
The other aspect is what happens if PUL 2.0 is a marginal miss? For me in this situation, the expectation is that they will be able to respond to the CRL with LVEF "additional data" and restart the BLA. It will result in a narrower label, but it will likely lead to an approval. From what I'm told, there's language in the type-A meeting minutes talking about "regulatory flexibility" - and I expect this to show up just like it did for
$REPL earlier this month. The reason for this is not just a change of heart for Dr. Vinay Prasad - its a situation where he's feeling the pressure from multiple sources and he needs to demonstrate this "flexibility" just to stay on the job. I'm also told that in this scenario -
$CAPR will not only PR the results, but till also PR their intent to respond to the CRL.