FDA Approves First MSC Therapy in Steroid-Refractory GVHD

The FDA approved remestemcel-L-rknd (Ryoncil) for the treatment of pediatric patients, aged 2 months or older, with steroid-refractory acute graft-versus-host disease (GVHD), according to a release issued by the agency.

FDA gives nod of approval to first derived mesenchymal stromal cell therapy for the treatment of pediatric patients with steroid-refractory acute GVHD.

“Ryoncil is the first FDA-approved [derived mesenchymal stromal cell (MSC)] therapy,” the FDA stated in its release.

The approval was based on data from the multicenter, prospective, single-arm MSB-GVHD001 trial (NCT02336230) of the allogeneic bone marrow-MSC therapy in 54 pediatric patients with steroid-refractory acute GVHD after allogeneic hematopoietic stem cell transplantation (HSCT).

In the trial, overall response rate at day 28 with remestemcel was 70% (95% CI, 56.4%-82.0%), which included complete and partial response rates of 30% (95% CI, 18.0%-43.6%) and 41% (95% CI, 27.6%-55.0%), respectively.

In addition, the median duration of overall response was 54 days (range 7-159+).

According to the agency, the recommended dose is 2 X 106 MSC/kg body weight per intravenous infusion, to be administered twice a week, at least 3 days apart, for 4 consecutive weeks for a total of 8 infusions.

Reference:

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