Mesoblast prepares to launch FDA-approved therapy for Pediatric Steroid-Refractory aGVHD

Proactive - Interviews for investors

Mesoblast Limited CEO Silviu Itescu joined Steve Darling from Proactive to share news the company has announced the U.S. Food and Drug Administration has approved remestemcel-L, now marketed under the brand name Ryoncil, as the first and only therapy for steroid-refractory acute graft-versus-host disease (SR-aGVHD) in children aged two months and older, including adolescents. This milestone FDA approval is based on results from a phase III clinical trial that demonstrated the efficacy of Ryoncil in treating SR-aGVHD. Among children with high-severity Grade C or Grade D disease, 70% achieved an overall response by Day 28, a critical measure strongly associated with improved survival outcomes in aGVHD. The approval marks a significant breakthrough for this devastating condition, which has an extremely poor prognosis and limited treatment options. Each year, approximately 10,000 patients in the U.S., including 1,500 children, undergo allogeneic bone marrow transplants, with nearly half developing aGVHD. Of those cases, around 50% become steroid-resistant, leaving patients with few effective therapeutic options. The company is actively preparing for the U.S. market launch of Ryoncil, focusing on ensuring that children and adolescents battling this life-threatening disease have access to the first approved therapy of its kind. Mesoblast has two more products in its pipeline nearing FDA approval: a cardiovascular treatment, REVASCOR, which showed significant reductions in heart attacks and mortality, and a back pain therapy that provides long-lasting relief from a single injection. Both products represent multi-billion dollar opportunities. #proactiveinvestors #mesoblastlimited #asx #msb #nasdaq #meso FDAApproval #Ryoncil #MSCtherapy #BiotechInnovation #GvHD #MedicalBreakthrough #InflammatoryConditions #ProactiveInvestors #CellTherapy