Listened to the CC this morning. SI sounded stunned and in a...

Listened to the CC this morning. SI sounded stunned and in a state of shock. Obviously, like all SHs, he never saw this coming.

I’ve openly expressed a view that I considered conditional approval with a confirmatory phase IV in adults as highly likely. I can’t get my head around why this never happened. The FDA want additional data having accepted the potency assays so surely this could be obtained by treating these children whilst conducting the additional trial. It makes no sense at all. They are happy to let it be used under the EAP so what is the difference?

There is going to need to be some serious tightening of the belt and a lot of refinancing or the company will be in serious trouble. Personally, I don’t see much else happening with other trials and I got the impression from the CC that everything else was now going to be on the back burner.

The Type A meeting is going to be critical and the finer details will be so important in determining this company’s future.

Also, talk of legal action is a non starter. The evidence has always been that the FDA want another trial. MSB thought that additional data would negate this need. The FDA said no and have remained constant on what they want. Good luck winning any legal battle with that context.

Finally, all those gloating, and I don’t mean @DocMcstuffins and @whytee, need to have a serious think about their behaviour. Appalling is the polite word, I’ll leave it at that.

I wish everyone well and stay strong, hopefully there will be better times ahead of us.