Ann: FDA Notifies Clinical Data Sufficient for Refiling aGVHD BLA, page-800

I wasn't completely serious about dumping stock.

My point, which I admit was a bit rambling at 2am was simply - if MSB was unable to prove to the FDA's satisfaction that the consistency of Ryoncil wasn't in doubt in the second submission, why wouldn't the company simply ask what the difference was between the commercial stock on hand and the stock produced post-inspection in Singapore?

You stated: "Everything to do with a BLA is an application over the details of which a sponsor will spend millions and millions of dollars just so that they can have the opportunity to argue for a marketing approval from the FDA"

If again, they weren't happy - why risk moving the company forward - and not treating patients - if all they had to do was swap new stock for old.

Why argue?

Surely if all that stood between FDA approval so kids start getting treated, and not, was a doubt on 'the old stock' - then why not use new?

It can't be that simple - it makes no sense- hence my question - what CQA's were missing that upset the FDA.

It's a big turnaround from - 'go do another trial' - to 'you know what, we have had another look and the value of the drug outweighs our concerns'

You state "Instead we have to generate new data (which they haven't seen before) to close whatever their "kowledge gap". - so again I'm back at square one asking what data could the trial contain that would prompt this turnaround?

There is nothing new. No new endpoints. No new outcomes.

Either the FDA have moved the goalposts and tacitly admitted they got it wrong (privately of course) or MSB are just throwing the same stuff at them again and hoping it sticks a third time (hence my hubris comment).

I'm not being contrary - I genuinely don't understand how a twice discarded trial can now suddenly contain all the answers.

Adults and children need this drug - the results are amazing - so I hope it's a case of the FDA realising they bit their nose off to spite their own face.

Cheers

PS: Are we assuming if this submission is accepted, that the commercial aspect of the CLBP and LVHF drug manufacturing process will be a 'slam dunk' as the existing drugs for both were made by the same process as Ryoncil and newer batches will be post FDA Lonza visit?

PPS: I admit to still being concerned that the trial data is the issue - are we sure the FDA aren't saying "Show us the responding patients recieved the same dose as the non-responsive, as the data may indicate the doses were not equal"

Hence the new trial with new drugs from the post FDA inspection.

Again, not being contrary - neither the FDA nor Mesoblast have supplied definitive reasons for rejection which severely muddies the waters.

And yes, I'm rambling again, but concrete info is infuriatingly hard to find.