Mesoblast resubmits BLA to FDA for remestemcel-L

Mesoblast Limited announced it has resubmitted to the U.S. Food and Drug Administration its Biologics License Application for approval of remestemcel-L in the treatment of children with steroid-refractory acute graft versus host disease. Survival outcomes have not improved over the past two decades for the most severe forms of SR-aGVHD, a life-threatening complication of an allogeneic bone marrow transplant following treatment for blood cancers and other conditions.1-3 The lack of any approved treatments for children under 12 means that there is an urgent need for a therapy that improves the dismal survival outcomes. If remestemcel-L receives FDA approval, it will be the first allogeneic "off-the-shelf" cellular medicine to be approved in the United States, and the first therapy for children under 12 years old with SR-aGVHD. The resubmission contains substantial new information as required by FDA in the Complete Response Letter received in September 2020 to the BLA for remestemcel-L. Mesoblast has maintained an active dialogue with FDA since receiving the CRL and in October 2022 provided a high-level synopsis of the substantial new information under its Investigational New Drug application for remestemcel-L. FDA granted remestemcel-L Fast Track designation, a process to facilitate the development and expedited review of therapies for serious conditions that fill unmet medical needs, and Priority Review designation, which is given to drugs that treat a serious condition and provide a significant improvement in safety or effectiveness over existing treatments. The BLA resubmission will have a review period up to six months from filing upon acceptance by FDA.