Mesoblast Limited (ASX: MSB; Nasdaq: MESO), a global leader in cellular medicines for inflammatory diseases, has achieved a groundbreaking milestone with FDA approval of RYONCIL® (remestemcel-L). This is the first mesenchymal stromal cell (MSC) therapy approved in the U.S. and the only treatment for steroid-refractory acute graft-versus-host disease (SR-aGvHD) in children aged 2 months and older.
Key Highlights
- First FDA-Approved MSC Therapy:
- RYONCIL is the first-ever MSC product to gain FDA approval, marking a significant advancement in cellular therapies.
- It is the first therapy available for children with SR-aGvHD, a life-threatening condition associated with high mortality rates.
- Phase 3 Clinical Trial Results:
- In a multi-center Phase 3 trial of pediatric patients with SR-aGvHD, including 89% with severe Grade C or D disease:
- 70% achieved an overall response by Day 28, a key measure predicting survival.
- Treatment was completed in more than 85% of patients without interruption, and no patients discontinued due to lab abnormalities.
- In a multi-center Phase 3 trial of pediatric patients with SR-aGvHD, including 89% with severe Grade C or D disease:
- Life-Saving Potential:
- SR-aGvHD affects nearly half of all bone marrow transplant patients who develop graft-versus-host disease and fail first-line steroid treatments.
- Approximately 1,500 children annually in the U.S. face this condition, and RYONCIL offers new hope for these patients and their families.
What is RYONCIL?
RYONCIL is an allogeneic mesenchymal stromal cell therapy with immunomodulatory properties. It works by:
- Inhibiting T-cell activation and pro-inflammatory cytokine secretion, addressing the excessive inflammation associated with SR-aGvHD.
- Potentially expanding to treat other inflammatory diseases with unmet needs.
Expert and Industry Perspectives
Dr. Joanne Kurtzberg, Director at Duke University Medical Center, highlighted:
“Steroid-refractory acute graft-versus-host disease is devastating, and RYONCIL will save lives and bring hope to countless families.”
Dr. Silviu Itescu, CEO of Mesoblast, stated:
“FDA approval of RYONCIL is a testament to our commitment to the GVHD community. We aim to expand its indications to both children and adults with inflammatory conditions while progressing our late-stage products for cardiovascular and pain indications.”
Looking Ahead: Expanding MSC Therapy Applications
Mesoblast plans to make RYONCIL widely available across transplant centers and treating hospitals in the U.S. Future developments include:
- FDA approvals for other late-stage products like REVASCOR® (cardiovascular diseases) and rexlemestrocel-L (inflammatory pain).
- Exploring expanded indications for RYONCIL in broader pediatric and adult populations.
This milestone positions Mesoblast as a leader in MSC-based therapies and underscores the transformative potential of cellular medicine in treating severe inflammatory conditions.
