Loving seeing the 99% likelihood of success being thrown around...

Loving seeing the 99% likelihood of success being thrown around here.

Wish I shared the confidence.

My concern is that when you look at the specific recommendation by the FDA and why they rejected the initial BLA application, MSB never actually addressed the main problem - the FDA wanting a randomised control trial.

I understand the ethical implications of using a control group in these kids - but these studies allow for early termination in the setting of overwhelming efficacy - and if that's what you expect, then why wouldn't you go for it? Right now, you have no kids getting this therapy, whereas during the trial 50% would be.

Which is less ethical?

Sure they've got some longer term data - but from the same study that was initially criticised by the FDA. They have said that they've come up with a solution to the potency assay issue, and hopefully the FDA finds that convincing.

I worry that the other trial failures, which had not occurred by the time of the initial BLA issues, won't weigh on the FDAs mind regarding the efficacy of these cells.

It may get approval. But I am not nearly as optimistic as some here.

We will know soon.