Come on you guys … Once something is approved it is difficult to...

Come on you guys … Once something is approved it is difficult to withdraw it.

MSB are arguing for approval now … then it will do the adult study. The FDA say do the adult study then we will see.

MSB has never met a primary endpoint in a RCT in 17 years and over a dozen trials. So yes there is time and money … but big risk.

QRX was in the exact same boat. SPL is in it right now. Two years ago SPL received a CRL. It claimed a new trial would be quick and easy. But they wanted to argue the case with the FDA. Two years later … black hole; no new trial - no nothing.

That is unfortunately the most likely outcome here; in two years time people will wondering why MSB just didn’t get on with the job and do the adult study.

In terms of reading the tea leaves about what went on here with the BLA and Ad Com meeting?

I don’t think the FDA changed their minds about anything.

Right at the start MSB were told a single arm trial was potentially approvable … but the design had to be reviewed before being conducted.

MSB failed to have the design reviewed by the FDA and made a blunder with how it derived the historical control group estimate.

MSB were asked to provide further justification for the control group estimate and so they conducted the Mt Sinai study.

The FDA then rejected this. In the eyes of the FDA MSB had done little more than find someone who agreed with their original estimate.

What the FDA wanted was a case-control study with matching at the individual level (not at the group level as MSB had done). Confirmatory – with a proper pre-specified hypothesis that could be tested in a framework that enabled replication.

This misunderstanding about what exactly the FDA wanted by way of further justification from the Mt Sinai study was not picked up because MSB failed to lodge the SAP with the FDA for review.

So yep … that’s the FDA definitely not liking something.

Why would MSB request an Ad Com meeting if it had such a weak case? MSB believed it had a good case. And they were right in a sense - they convinced 9 members of the jury. MSB would have been hopeful that this would sway the judge. As we know it didn’t.

In terms of a sponsor requesting an Ad Com meeting as part of a rolling BLA dispute - someone will have to google it.

But look all imo … and this is water under the bridge – it was months ago and other events have overtaken it somewhat.

And theres another little fish on the fry pan that is distracting me … where I need to jot a few things out. Hopefully in a more coherent fashion.