Ann: Update on BLA for Graft Versus Host Disease, page-339

“Now why could they not have done things in reverse order >

1) Conditional approval for kids to commence immediately then,

2) require adult trial

Of course, the MSB price does not get smashed doing things in that order.

I will be very suspicious if the kids end up getting accelerated approval at the meeting - no reason it could not have been done in the first place.”


You are so correct. And listening to Si on the CC tells me that he is also baffled by the FDA’s logic.
The previous CRL recommended another trial on adults and/or children. It wasn’t going to happen on children for reasons of ethics, leaving just an adult trial. But that wasn’t going to happen until the assays were approved as part of the new submission. So approval was never going to happen.
I am sure the delay in the announcement was because Mesoblast were furious and disgusted with the twisted logic of the FDA. As am I.
Perhaps there will be an AA for paediatric use, but I will believe it only when I see it.