Mesoblast Secures FDA Approval for Ryoncil, First Mesenchymal Stromal Cell Therapy for Pediatric Steroid-Refractory Acute Graft-Versus-Host Disease

Approval Date:
The FDA approved Ryoncil on December 19, 2024, for the treatment of steroid-refractory acute graft-versus-host disease (SR-aGVHD) in pediatric patients aged 2 months and older234.

Therapy Type:
Ryoncil is the first FDA-approved mesenchymal stromal cell (MSC) therapy, derived from the bone marrow of unrelated donors34.

Efficacy:
The approval is based on a Phase III single-arm study that showed a 70% overall response rate and a 30% complete response rate at 28 days in pediatric patients with SR-aGVHD234.

Regulatory Path:
Mesoblast faced two complete response letters from the FDA in 2020 and 2023 before securing approval, highlighting the challenging regulatory journey for Ryoncil23.

Future Plans:
Mesoblast plans to initiate a study in steroid and Jakafi-refractory adults with SR-aGVHD, to be conducted by the Blood and Marrow Transplant Clinical Trials Network2.

Safety:
Common adverse reactions include infections, fever, hemorrhage, edema, abdominal pain, and hypertension. Patients should be monitored for hypersensitivity reactions and other complications4.

Designations:
Ryoncil received Orphan Drug, Fast Track, and Priority Review designations from the FDA4.

Sources:

2. https://www.oncologypipeline.com/apexonco/third-time-lucky-mesoblast

3. https://www.biospace.com/fda/mesoblast-wins-first-mesenchymal-stromal-cell-therapy-fda-approval-for-gvhd

4. https://www.fda.gov/news-events/press-announcements/fda-approves-first-mesenchymal-stromal-cell-therapy-treat-steroid-refractory-acute-graft-versus-host