Ann: FDA Notifies Clinical Data Sufficient for Refiling aGVHD BLA, page-473

Scribbled this down from Webinar from 14:40 for fun to put some colour around Si's perspective
on FDA behaviour vis a vis assays. Couldn't harvest the cc so, um, transcribed it.
Si's response to punter's question at 17:40
Apologies if already posted or if transcript available elsewhere.

https://www.youtube.com/watch?v=HBGR6ZAQPPA

Q: With regards to the Ryoncil why did the FDA request additional data from the second potency assay and was the additional data in line with the original potency assay?

Si: Yeah..look... it's the first product of its kind, first in class and ah, you know, we've suffered a couple of years of delays and ongoing requests for more data by the FDA ah even though we were successful and even though we had an advisory committee meeting that voted 9:1 advising the FDA to approve the product, uhm, you know FDA want more, bit more clarity and a bit more certainty that every vial that will go into the market to treat children and adults is identical and meets stringent potency requirements so this 2nd potency assay I think addresses the FDA's concerns and is very much in line with the first assay and in fact was, ah, in place even in the phase 3 trial so we actually had more than one assay in place we've just, uhm, we've just re-analysed the data in a more ah I would say more robust way and we've provided all the data to the FDA and we think more than meets their, um, their criteria.

But I think you know you need to appreciate the product even when its approved for children will be available for hospitals and users for much more, much wider use and I think the FDA want to be certain that when they release this product on the market its widespread use will come with potency assays and disclaimers that allow it be used safely."