Yes,
to Clarify these are the numbers
11,000 transplants completed annually, along with baseline immunosuppresants administered.
5,500 of those ( about 50% ), develop aGvHD, with around 1200 being children.
First line approved treatment
Adults recieve "optimised" - ( More ) steroids and immunosupressants.
Children have nothing approved, but may get the same as the adults seeing as nothing is approved.
Second line treatments
Adults recieve anything ( these are not approved either ) , there is not " standard" second line. It could be MSC, different steroids, green tea, Reg's poems, anything.
After completeing the first / second line treatment lines approximately 600 children, and 3000 adults remain as non responders. ( Nothing has worked ).
These are in my opinion the most needy candidates for off label, as without some treatment that works - death will occur.
That would equate to around 2 Billion USD in sales if all 3600 were treated with the pricing assumptions of 400,000 child, 600,000 adult.
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