The more I think about what the FDA have done the more perplexed...

The more I think about what the FDA have done the more perplexed I am - obviously there were two sticking points in the initial CRL - potency assays and trial design. It appears MSB were able to solve one of the concerns (potency assays) but not both and here we are at this late stage because a dependency for the trial was signed off potency.

What happened I wonder to the collegial working with the FDA, surely the FDA would have known that they wanted another trial from the initial data and that the additional data wasn't enough to convince them - is there an FOI type concept in the USA where we can see any internal discussions of the FDA so we can see how they came to the decision that the 4 years of survival data wasn't enough RWD for them to at least give a conditional approval for a product with zero safety or manufacturing concerns.

The question is really why not approve for children with a confirmatory trial in adults to follow - especially as the FDA have been happy to have it offered for compassionate use.

So many questions.