banter and General Discussion, page-10493

This is the latest on the FDA Rare Disease Pilot Program that kicked off in June. I mentioned it in a previous post way back but it is finally up and running. Understanding the well articulated philosophy of the FDA behind this initiative will help us put some meat to the bones for our contention that the chance of approval for Ryoncil for SR aGVHD for kids is good. It was too late to get onto the program, but an approval, if it happens, just can’t come soon enough for this rare disease.

https://www.fda.gov/drugs/our-perspective/fda-opens-doors-more-treatments-rare-diseases-through-new-start-pilot-program#:~:text=FDA's%20Center%20for%20Biologics%20Evaluation,life%2Dsaving%20therapies%20for%20rare