Thanks for that link
@Bazsa .
Yes the patent was filed in 2022.
The Type A meeting was held in 2023. FDA said go and generate the data we need. Company supplied that data to the FDA in January.
note I deduced that from Ask Cameron's article where it is written we went back to the FDA in January but the permission to refile was at a type C in March.
Pure speculation- but it fits unlike OJ's glove.
My take is that the company had not used the IL-2Ra assay in two prior submissions. But have included it in the recent resubmission- after getting the thumbs up from the FDA-
There was a significant relationship between % IL-2Ra inhibition in vitro as a measure of a lot’s potency and reduction in CD3+CD4+CD25+HLA-DR+ activated T cell phenotype, from screening to Day 28 (p=0.009).
The FDA has requested to refile- they have not followed established procedures and waited for the company to resubmit in their own time.
Can any scientific types please provide some feedback on the IL-2Ra assay's potential to have swayed the FDA?
@JB1975 am interested in your take.
Is this the Golden Egg?
[176] Product lot potency(inhibitory activity on IL-2Ra expression on activated PBMC in vitro), correlated with progressively decreasing inflammatory biomarkers in vivo. This was most evident in those children with highest baseline levels of inflammatory biomarkers, defined prospectively on the basis of having high MAP scores (Figure 6). There was a significant relationship between % IL-2Ra inhibition in vitro as a measure of a lot’s potency and reduction in CD3+CD4+CD25+HLA-DR+ activated T cell phenotype, from screening to Day 28 (p=0.009).
[177] The inhibitory activity of stem cell product on IL-2Ra levels in cultures of activated PBMC correlates with in vivo bioactivity in pediatric aGVHD as indicated by decreases in inflammatory biomarkers, reductions in circulating levels of inflammatory biomarkers and activated T cells and improved survival.
ANNUAL REPORT
Notably, at the recent Type A meeting with FDA we presented data that RYONCIL product made with the validated manufacturing process proposed for commercial release resulted in similar consistently high survival rates whether used in children with SR-aGVHD in the Phase 3 trial
or under an Emergency Investigational New Drug (EIND) protocol through 2023, as well as in adults with SR-aGVHD
who failed ruxolitinib or other second-line agents. Further, to support approval for the pediatric indication,
we are currently generating additional data using the IL-2R alpha inhibition potency assay which was in place
during the pediatric Phase 3 trial to provide the needed link assuring consistency between the RYONCIL product
that was used in the pediatric Phase 3 trial and available commercial inventory
Reg
(20min delay)