Keep up Doc - it is common knowledge that Mesoblast submitted data from 241 patients from the EAP
Additionally we've generated data from the expanded access program, EAP 275 that followed 241 children over more than 10 years, who -- where Remestemcel was used as salvage therapy. ............................. So these new data will all be part of the BLA that's submitted, resubmitted to the FDA. And we expect to complete that by the end of this quarter.
https://seekingalpha.com/article/4538049-mesoblast-limited-meso-q4-2022-earnings-call-transcript
Go to next slide please. So what is our plan for the BLA resubmission for Steroid-refractory Graft versus Host Disease? In response to questions raised by the FDA, we have evaluated our potency assays that were in place and that were prospectively analyzed against clinical outcomes at the time of the Phase 3 trial. And we believe that the key potent test measuring T-cell activation helps establish a clear relationship between potency and survival based on the Phase 3 trial outcomes, and helps establish a clear understanding of the mechanism of action by which Remestemcel improves GVHD outcomes in particular survival, since we understand that T-cells are the basis of the disease itself.
Additionally we've generated data from the expanded access program, EAP 275 that followed 241 children over more than 10 years, who -- where Remestemcel was used as salvage therapy. And in that program, the existing potency assay targeting T cells demonstrating how Remestemcel targets T cell activation has shown that changes in manufacturing, which resulted in enhanced bio activity of the product could be predicted by the assay as could improvements in outcomes, notably survival.
So these new data will all be part of the BLA that's submitted, resubmitted to the FDA. And we expect to complete that by the end of this quarter.
https://www.fda.gov/science-research/science-and-research-special-topics/real-world-evidence
Real-World Evidence
FDA has a long history of using what we currently call real-world data (RWD) and real-world evidence (RWE) to monitor and evaluate the postmarket safety of approved drugs. RWE has also been used historically to support effectiveness, but on a more limited basis. Advances in
the availability and analysis of RWD have increased the potential for generating robust RWE to support FDA regulatory decisions.
FDA is committed to realizing the full potential of fit-for-purpose RWD to generate RWE that will advance the development of therapeutic products and strengthen regulatory oversight of medical products across their lifecycle.
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