FDA Approves Remestemcel-L in Pediatric SR-aGVHD

Findings from the MSB-GVHD001 study support the FDA approval of remestemcel-L in this population.

The FDA has approved remestemcel-L-rknd (Ryoncil) for pediatric patients 2 months and older with steroid-refractory acute graft versus host disease (SR-aGVHD), according to a news release from the agency.¹

Supporting data for the approval came from the single-arm phase 3 MSB-GVHD001 trial (NCT02652130). Among 54 evaluable patients with SR-aGVHD following allogeneic hematopoietic stem cell transplantation (HSCT), the overall response rate (ORR) at day 28 was 70% (95% CI, 56.4%-82.0%). Additionally, data showed a complete response (CR) rate of 30% (95% CI, 18.0%-43.6%) and a partial response (PR) rate of 41% (95% CI, 27.6%-55.0%) .

The median duration of response (DOR) from day 28 to death, disease progression, or additional line of therapy was 54 days (range, 7 to 159+).

Common adverse effects (AEs) associated with the agent included viral and bacterial infectious disorders, infection, pyrexia, edema, hemorrhage, hypertension, and abdominal pain.

Patients enrolled on the single-arm, prospective phase 3 trial were assigned to receive remestemcel-L intravenously at 2 x 10⁶ mesenchymal stem cells (MSC)/kg twice per week for 4 consecutive weeks.²

The trial’s primary end point was ORR 28 days after the initiation of the study treatment. Secondary end points included overall survival at 100 days and ORR at 56 days and 100 days after the start of therapy.

Patients 2 months to 17 years old with grade B to D acute GVHD who required treatment with corticosteroid systemic therapy were eligible for enrollment on the trial. Having a Karnofsky or Lanksy performance status of 30 or higher, adequate renal function, and progression on prior steroid treatment were additional requirements for enrollment. Those with grade B aGVHD with involvement only in the skin, prior second-line therapy for aGVHD, or prior systemic treatment apart from steroids and prophylactic agents were ineligible to enroll on the trial.

References

1. FDA approves remestemcel-L-rknd for steroid-refractory acute graft versus host disease in pediatric patients. News release. FDA. December 18, 2024. Accessed December 18, 2024. https://tinyurl.com/3mzae43x
2. A prospective study of remestemcel-L, ex-vivo cultured adult human mesenchymal stromal cells, for the treatment of pediatric participants who have failed to respond to steroid treatment for acute graft-versus-host disease (aGVHD). ClinicalTrials.gov. Accessed December 18, 2024. https://tinyurl.com/yc3eena4