FDA ODAC Meeting Material discussion analysis, page-334

@stockrock Awesome post

I think it comes down to this.

It's not that easy to clearly demonstrate the efficacy or the mechanism of action but the bottom line is a significant percentage of children are surviving this life threatening UNMET need. It's safe, fully understanding the mechanism of action may take years. Can the world wait and my NO 1 reason,

IS IT IN THE BEST INTEREST OF THE CHILDREN WITH SR-aGVHD THAT THIS PRODUCT RECEIVE A LICENSE AND APROVAL.

I think the answer is YES IT IS and Remestemcel gets approved.

Why does it get approved you say? because they have created a pathway to progress in the document. SEE BELOW.


Despite these challenges, FDA has suggested that developers of cell-based products may progress in two relevant ways.
The second approach is to use clinical performance to demonstrate potency.

1. If a product meets the primary clinical outcomes,
2. Has been extensively characterized during product development,
3. Is produced by a well-controlled manufacturing process,

The above clinical data may be considered to demonstrate potency even if the mechanism of action is not completely understood.

In this scenario OF LIFE THREATENING UNMET NEEDS IN CHILDREN, assays purporting to measure product attributes thought to be related to product potency must be sufficiently robust in terms of reproducibility and as indicators of product quality and stability.


NOTE: IT'S KNOW CERTAINTY TO GET APPROVAL AND THIS IS JUST MY OPINION DYOR