FDA Approves Remestemcel-L-rknd for Pediatric Steroid-Refractory Acute Graft-Versus-Host Disease

Remestemcel-L-rknd (Ryoncil; Mesoblast), the first allogeneic bone marrow–derived mesenchymal stromal cell therapy, has been FDA-approved for steroid-refractory acute graft-versus-host disease (SR-aGVHD) in pediatric patients aged ≥2 months.¹

The multicenter, prospective, single-arm MSB-GVHD001 (NCT02336230) study evaluated 54 pediatric patients with SR-aGVHD after allogeneic hematopoietic stem-cell transplantation.

Overall response rate at day 28 was 70% (95% confidence interval [CI], 56.4-82.0), including a complete response rate of 30% (95% CI, 18.0-43.6) and a partial response rate of 41% (95% CI, 27.6-55.0). The median duration of response, calculated from response at day 28 to either progression, new systemic therapy for aGVHD, or any-cause death, was 54 days (range, 7-159+).

The most common nonlaboratory adverse reactions (incidence ≥20%) were viral infectious disorders, bacterial infectious disorders, unspecified infection, pyrexia, hemorrhage, edema, abdominal pain, and hypertension.

Reference

1. FDA. FDA approves remestemcel-L-rknd for steroid-refractory acute graft versus host disease in pediatric patients. December 18, 2024. Accessed February 10, 2025. www.fda.gov/drugs/resources-information-approved-drugs/fda-approves-remestemcel-l-rknd-steroid-refractory-acute-graft-versus-host-disease-pediatric