Ryoncil, a mesenchymal stromal cell therapy derived from bone marrow, has received FDA approval for the treatment of steroid-refractory acute graft-vs-host disease (SR-aGVHD) in children aged 2 months or older. The approval marks a significant advancement in cell-based therapies for life-threatening diseases. In a phase 3 study, Ryoncil demonstrated effectiveness in inducing responses in children with SR-aGVHD.
, an allogeneic bone marrow‒derived mesenchymal stromal cell therapy for steroid-refractory acute graft-vs-host disease in children 2 months of age or older. According to a new release from the company, roughly 10,000 patients undergo an allogeneic bone marrow transplant each year in the United States, 1500 of whom are children. About half develop aGVHD, and almost half of those do not respond to steroids.
The safety and effectiveness of Ryoncil were evaluated in a multicenter, single-arm phase 3 study of 54 children with SR-aGVHD after undergoing allogeneic They were evaluated at baseline using the International Blood and Marrow Transplantation Registry Severity Index Criteria to evaluate which organs have been affected and the overall severity of the disease. Most had high-severity Grade C or D disease.
Sixteen children had a complete response to treatment 28 days after receiving Ryoncil, while 22 had a partial response.
FDA Approval Stem Cell Therapy Graft-Vs-Host Disease Pediatrics Clinical Trials
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