The Food and Drug Administration (FDA) approved Ryoncil (remestemcel-L-rknd) for the treatment of steroid-refractory acute graft versus host disease in children aged two months and older.
This marks the first FDA-approved mesenchymal stromal cell therapy, according to an alert from the agency.
The approval was based on findings from the MSB-GVHD001 study, which analyzed data from 54 children with steroid-refractory acute graft versus host disease. In particular, participants enrolled on the study had grade B to D (moderate to very severe) steroid-refractory acute graft versus host disease. For purposes of this study, steroid-refractory acute graft versus host disease was defined as advanced graft versus host disease that progressed within three days or that has not improved within seven days of treatment with methylprednisolone, a corticosteroid medication.
In the study, the researchers measured overall response rate at Day 28, which included complete and partial response rates, and duration of overall response, according to the alert. The median duration of response was calculated from response at Day 28 to either new systemic therapy for advanced graft versus host disease, progression or all-cause death.
At Day 28, the overall response rate was 70%, including a complete response rate of 30% and a partial response rate of 41%. The median duration of response was 54 days, and ranged from seven days to at least 159 days, the FDA noted
The most common side effects, which occurred in at least 20% of patients, included bacterial infectious disorders, viral infectious disorders, fever, infection with an unidentified pathogen, edema, bleeding, high blood pressure and abdominal pain.
The FDA explained that the recommended dose of Ryoncil is 2 x 106 MSC/kg body weight, to be administered intravenously twice a week for four consecutive weeks, totaling eight infusions. Of note, the infusions are delivered at least three days apart. Patients may continue treatment with Ryoncil based on the disease’s response at 28 days after initial treatment.
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