FDA Approves Mesoblast's Ryoncil for GvHD

The FDA has granted approval to Mesoblast Limited's Ryoncil (remestemcel-L), marking it as the first mesenchymal stromal cell therapy available in the U.S. for treating steroid-refractory acute graft-versus-host disease (SR-aGvHD) in children aged two months and older. This therapy, derived from bone marrow MSCs, addresses a critical need as about 50% of patients undergoing allogeneic stem cell transplants develop aGvHD, with many not responding to standard steroid treatments. In clinical trials, Ryoncil demonstrated an overall response rate of 70% within 28 days among participants, with 30% achieving complete responses. The approval follows two previous rejections and is expected to significantly impact the lives of affected children and their families. Mesoblast aims to explore further applications of Ryoncil and other therapies targeting inflammatory diseases.