The U.S. Food and Drug Administration (FDA) recently approved remestemcel-L-rknd (Ryoncil), an allogeneic bone marrow–derived mesenchymal stromal cell therapy for steroid-refractory acute graft-vs-host disease (GVHD) in pediatric patients 2 months of age and older. The application received Orphan Drug, Fast Track, and Priority Review designations by the FDA.
This first FDA-approved mesenchymal stromal cell therapy contains mesenchymal stromal cells that are isolated from the bone marrow of healthy adult human donors. Steroid-refractory acute GVHD is a serious and life-threatening condition that can occur as a complication of allogeneic hematopoietic stem cell transplantation (allo-HSCT).
Single-Arm Study
The safety and effectiveness of remestemcel-L-rknd were evaluated in a multicenter, single-arm study of 54 pediatric participants with steroid-refractory acute GVHD after undergoing allo-HSCT. They received intravenous infusion of the therapy twice weekly for 4 consecutive weeks, for a total of eight infusions. The condition of study participants was analyzed at baseline via the International Blood and Marrow Transplantation Registry (IBMTR) Severity Index Criteria.
The effectiveness of remestemcel-L-rknd was based primarily on the rate and duration of response to treatment 28 days after initiating therapy. Study participants who had a partial or mixed response to treatment received additional infusions once weekly for an additional 4 weeks. A total of 16 study participants (30%) had a complete response to treatment 28 days after receiving therapy, and 22 study participants (41%) had a partial response.
Treatment Considerations
Infusion of remestemcel-L-rknd should be monitored by the treating physician, and the infusion should be discontinued if there is any evidence of a reaction that may include dyspnea, hypotension, fever, tachypnea, cyanosis, and hypoxia. The most common adverse reactions in study participants who received remestemcel-L-rknd were infections, fever, hemorrhage, edema, abdominal pain, and hypertension. Complications such as hypersensitivity and acute infusion reactions, transmission of infectious disease or agents, and ectopic tissue formation may occur following treatment with remestemcel-L-rknd.
Patients should be premedicated with corticosteroids and antihistamines prior to infusion with remestemcel-L-rknd and monitored for hypersensitivity reactions during treatment.
