Mesoblast RYONCIL becomes first FDA-approved mesenchymal stromal cell therapy

The company states: “Mesoblast (MESO) announced the FDA approved Ryoncil, remestemcel-L, as the first mesenchymal stromal cell, MSC, therapy in the United States. RYONCIL is the only MSC therapy approved in the U.S. for any indication, and the only approved therapy for steroid-refractory acute graft versus host disease in children 2 months and older, including adolescents and teenagers. Annually in the United States approximately 10,000 patients undergo an allogeneic bone marrow transplant, 1,500 of whom are children. Approximately 50% develop aGvHD – acute graft versus host disease – and almost half of those do not respond to steroids, the recognized first-line treatment. In a single-arm multi-center Phase 3 trial of children with SR-aGvHD, 89% of whom had high severity Grade C or Grade D disease, 70% achieved an overall response by Day 28 of treatment with RYONCIL, a measure that predicts survival in aGVHD. RYONCIL treatment was not discontinued or interrupted in any patient for any laboratory abnormality, and the full course was completed without interruption in more than 85% of patients.”