Ann: Update on BLA for Graft Versus Host Disease, page-256

over sold of course,
having trouble digesting all this, but also thinking upside,

MSB looking to get approval re kids, but then translate to adult,
the FDA have melded the kids effort to being part of same in Adults, again as other have said too...it was suppose to be the avenue for KIDs approval. (a result adult confirmatory trial out that)

so by the the FDA melded this comming trial... what about the kids trial and approval...is that now base on Adult tiral... teh wording was unclear to me...perhaps the call conference will explain....
did it actually say the kids approval was denied...as it seems focussed on the Adult outcome?

it seem the compassoinate grounds use, éxpanded access under US terms will continue?! and it would seem data to date from that has been shelved by FDA.

and because the FDA has melded...approval will be for a range of treatment outcomes...
how long a trial...and how fast can we get a result.... if set up, we should get a result for overwhelming effeciency within 9 months.... as 80% sadly die, ..then if 60% survive treatment that wouldseem good enough...but if the FDA endpioints look at 12 or 24 months we are ??
as discussed over time.... this will leave door open to other treatments?
please explain Si

folks these past call conferneces seems stacked with journo that have 'been selective' but if folks are able to sumbit question please please do....
it almost a must.
Si will put a positve view. on it all, but preferr he should have in the past had a clear kick at the FDA ie we follow what you wanted by now nothings...
and Si and the team should have fulll foreseen what was to come 3 years ago,..and did the trial regardless, to fully support any submission or even give patient a better chance at living. its all part of the R&D anyway, while it cost heaps to do,
it has gone very wrong in vision to execute
Si can see the end goal and truely believes but the path to it is somewhat filled with large rocks to stumble over, and Si and teh team have tripped many times. with vision to avoid or mitigate