Ann: FDA Notifies Clinical Data Sufficient for Refiling aGVHD BLA, page-788

FDA clarified that the key remaining issue for pediatric approval is providing further evidence that the potency assay will assure the consistent efficacy of commercial product.

Will someone brighter than me show where the available clinical data from its Phase 3 study MSB-GVHD001 (that appears sufficient to support submission of the proposed Biologics License Application (BLA) for remestemcel-L according to Mesoblast) goes in any way toward confirming the MSB-GVHD001 Potency Assay is consistent with the standard required for FDA approval.


I know they went to Singapore for a plant inspection and found no issues - so that's good.




However the FDA appear to be saying - OK, great trial, but prove the good responses weren't from a higher dose inadvertently produced from poor batch control or inconsistent production leading to those results?

The FDA can test batches at Singapore until Christmas but how do they test vials given to children in the trial from 2020?

My question therefore is simple:

What new information has the FDA gleaned from the 2020 trial that addresses the concern about dosage consistency?






Are they saying there is inconsistency with the results in the children due to the way the trial was run (endpoints, days / dates of comparison with control group ie: 28 days, 6 months etc etc)

OR

There is inconsistency with the results due to the way the drug was produced and administered - the doses and amounts were inconsistent.

If anyone can answer this in a simple way - I'd appreciate it - and show me what in the trial that is prompting the FDA to apparently do a 180 degree about face using 4 year old data.

Thanks very much.