FDA Approves Ryoncil for SR-aGvHD Treatment
Mesoblast Limited announced the FDA approved Ryoncil (remestemcel-L) as the first mesenchymal stromal cell (MSC) therapy in the United States. Rynocil 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 (SR-aGvHD) in children 2 months and older, including adolescents and teenagers.
Transplant physician Dr Joanne Kurtzberg, the Jerome Harris Distinguished Professor of Pediatrics and Professor of Pathology, and Director, Marcus Center for Cellular Cures at Duke University Medical Center (DUMC), said: “Steroid-refractory acute graft versus host disease is a devastating condition with an extremely poor prognosis. From today we are able to offer Rynocil, the first FDA-approved treatment which will be life saving for so many children and will have a great impact on their families.”
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 and almost half of those do not respond to steroids, the recognized first-line treatment.1-5 In a single-arm multicenter Phase III 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 Rynocil, a measure that predicts survival in aGVHD. Rynocil 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. The full Phase III clinical study results are available in Biology of Blood and Marrow Transplantation
Citation: Kurtzberg, J. et al. "A Phase III, Single-Arm, Prospective Study of Remestemcel-L, Ex Vivo Culture-Expanded Adult Human Mesenchymal Stromal Cells for the Treatment of Pediatric Patients Who Failed to Respond to Steroid Treatment for Acute Graft-versus-Host Disease" Biol Blood Marrow Transplant 26 (2020) 845-854 https://doi.org/10.1016/j.bbmt.2020.01.018
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