International + Font Resize - Mesoblast submits clinical efficacy and safety data to US FDA in rolling BLA for Remestemcel-L to treat children with SR-aGVHD Melbourne, Australia Thursday, January 2, 2020, 17:00 Hrs [IST] Mesoblast Limited, global leader in cellular medicines for inflammatory diseases, has received a confirmed receipt from the United States Food and Drug Administration (FDA) for filing of its clinical efficacy and safety data for Remestemcel-L in its rolling Biologics Licence Application (BLA) for the treatment of children with steroid-refactory acute versus host disease (SR-aGVHD). The final module will be filed during January and Mesoblast will request an accelerated FDA review of the BLA under the product candidate's existing fast track designation. The clinical submission included analyses of 309 children with SR-aGVHD who have received Remestemcel-L across three separate studies. The results of the comparative analysis between Mesoblast's open-label phase 3 study and contemporaneous controls receiving institutional standard of care demonstrate the effectiveness of Remestemcel-L in this patient population. These conclusions are supported by prior results from an expanded access program in 241 children where Remestemcel-L was used as salvage theory after failure of steroids and other agents. In Mesoblast’s open-label phase 3 trial of of Remestemcel-L in 55 children with SR-aGVHD the primary endpoint of day 28 overall response in those exposed with Remestemcel-L was achieved in 70 per cent and day 100 survival was 75 per cent. Mesoblast's Remestemcel-L is an investigational therpay comprising of culture-expanded mesenchymal stem cells derived from the bone marrow of an unrelated donor. "We are pleased to have submitted to the FDA clinical efficacy and safety data for Remestemcel-L, as well as comparative clinical outcome data from contemporaneous controls.We are working closely with the FDA to make our cellular medicine available and improve outcomes in children with this devestating condition," chief medical officer Dr Fred Grossman commented. Acute GVHD is a potentially life-threatening condition which occurs in about 50 per cent of patients who receive an allogenic bone marrow transplant.