Ann: Annual Financial Results Presentation, page-141

So tell us why FDA gave the company a green light for BLA resubmission after looking at the additional data on the potency assay. If what you are saying is true, FDA would still insist on a new trial for validation of the potency assay, but it’s clear that is not the case any more.

No its not clear.

To me its clear that you understand BLA submission processes less well than say LED or even Reg now. Maybe they like you enough to point the the relevant SOPPs out to you.

There is a difference between the form and the substance of an application - and applications can be made by anyone anytime no green light is necessary - you are using Silviu's dumbing down of processes as though the actual processes aren't public domain documents.

If an application doesn't address each item in a CRL - it can be rejected in 14 calendar days. But if it appears to address each item -then the form will be adequate a PDUFA date will be advised and the review proper of the substance of the submission goes ahead.


I find your questions tangential and annoying like regs styles of asking whytee but if and if and if - what they whytee. He wants whoever he is trying to persuade to spend their time on his points of minor interest and to overlook what to them are the points of major interest and relevance.

Holders believing everything Silviu says to be objective truth (including about the FDA's actual statements and position) are almost impossible to have reasonable discussions with. They want unreasonable amounts of suspension of disbelief from the person they want to have a discussion with - and they want to crack jokes that aren't self deprecating but that are shots at the person whose opinion they pretend to want.

LED and reg have both posted useful and informative links about a company (whose name I can't currently recall) that was an example of the FDA reversing its position. It was for a muscular dystropy gene therapy from memory. I read some of the links from the links of a post LED made and got more of the back story - yeah the FDA really did have internal conflicts and disagreements about how to handle that company's application - resignations were submitted - ODAC recommendations (negative ones) were over-ruled - first line investigators concerns were over-ruled. There is precedent for internal disagreement in the FDA - its possible Silviu spoke with someone at a higher level than Matt Klinker and someone might be a career politician or a subjective nuff nuff like Kurtzberg who can't remember that the pain of the patient in front of her is not more real than the pain of the many patients not in front of her if she doesn't get her science head on.

So its possible in my mind Silviu got something from some FDA officer at some level that amounts to bad science from a weak person - but - the science will have to come out eventually - if someone approves crap - that crap will come back on them - and I'd expect to see Klinker tear strips off weak science (and though I wouldn't expect to see it - I'd hope he and a bunch of other professionals with integrity would fight fiercely inside the FDA for good medicines and good proofs of effectiveness) because I know he knows what weak science looks like as he has correctly identified it before - he has seen MSBs submissions before.

If the science is somehow - which I can't explain - because MSB isn't putting up science - its not answering questions like I asked of boomer55 for instance - it should have people that could - people like Paul Simmons for instance - then the FDA may find a BLA can be granted on the merits and no cheating or weak persons playing politics will be needed. IF.

But secret science is bs - its not science.

There has been no statements that MSB has a validated potency assay since the last CRL.
There has been no statements that Klinker has changed his mind. There has been noting in the patent material I have seen - and I'm willing to look at whatever even someone like Paul Simmons could point to - my mind is genuinely open - to show any of the holes in the reasoning that existed before have been closed. Silviu hasn't identified anything in scientifically specific terms that can explain what is different now to before. He should be able to - he needs to establish priority to patent anything anyway/