Ann: Update on BLA for Graft Versus Host Disease, page-129

Mesoblast has successfully met the pre-specified primary endpoint, prospectively agreed with FDA, of a single-arm Phase 3 trial in 54 children with SR-aGvHD. While the Oncologic Drugs Advisory Committee of FDA in August 2020 voted 9:1 in favor of remestemcel-L’s efficacy in a pediatric patient population, in September 2020 FDA recommended further steps be undertaken to obtain approval. The BLA resubmission of January 2023 included long-term follow-up data from the Phase 3 trial by the Center for International Blood and Marrow Transplant Research (CIBMTR) showing 50% survival through more than 4 years of follow-up for remestemcel-L treated patients in the Phase 3 trial for whom less than 20% survival at two years was expected based on disease severity. The resubmission also included a post-hoc propensity matched study showing 6 month survival was 67% with remestemcel-L vs 10% with other unapproved therapies in highest-risk patients as identified using the Mount Sinai Acute GVHD International Consortium (MAGIC). These pediatric data provide further support for use of remestemcel- L in the proposed study in high-risk adults with SR-aGVHD.

Fxxx the Corrupt FDA, I am finished

Last post for me, bye all
Howie