Key Highlights:
- Ryoncil® (remestemcel-L) becomes the first FDA-approved mesenchymal stromal cell (MSC) therapy in the U.S.
- Approved for children aged 2 months and older with steroid-refractory acute graft-versus-host disease (SR-aGvHD), a life-threatening condition with high mortality.
- In a Phase 3 trial, 70% of children treated with Ryoncil® achieved an overall response by Day 28, a key indicator of survival in aGVHD.
- Ryoncil® demonstrates strong immunomodulatory effects, including T-cell inhibition, paving the way for potential treatment of other inflammatory diseases.
Melbourne, Australia; December 19, 2024 – New York, USA; December 18, 2024 – Mesoblast Limited (ASX:MSB; Nasdaq:MESO), a global leader in allogeneic cellular medicines for inflammatory diseases, announced today that the U.S. Food and Drug Administration (FDA) has approved Ryoncil® (remestemcel-L), marking it as the first mesenchymal stromal cell (MSC) therapy approved in the United States.
Ryoncil® is now the only FDA-approved MSC therapy for the treatment of steroid-refractory acute graft-versus-host disease (SR-aGvHD) in children as young as 2 months old, including adolescents and teenagers. This approval represents a major milestone for the field of cellular therapies, offering a life-saving treatment for children with SR-aGvHD, a condition with a historically poor prognosis.
Dr. Joanne Kurtzberg, a leading transplant physician and director of the Marcus Center for Cellular Cures at Duke University Medical Center, commented: “Steroid-refractory acute graft-versus-host disease is one of the most devastating conditions we face in pediatric transplant care. With the approval of Ryoncil®, we can now offer a life-saving treatment that will significantly improve survival rates and the quality of life for many children and their families.”
Clinical Trial Success and Approval
Each year in the U.S., around 10,000 patients undergo an allogeneic bone marrow transplant, with 1,500 being children. Nearly 50% develop aGVHD, and for half of these patients, steroids, the standard first-line treatment, fail to work.
In a Phase 3, multi-center trial involving children with SR-aGvHD, 89% of patients had high-severity Grade C or Grade D disease, one of the most critical stages of the condition. Remarkably, 70% of patients showed an overall response by Day 28 of treatment, a result that predicts a significant improvement in survival rates for aGVHD patients. Additionally, the treatment was well-tolerated, with over 85% of patients completing the full course of treatment without any interruptions due to side effects or laboratory abnormalities.
The Potential of Ryoncil®
Ryoncil® works by harnessing the immunomodulatory power of mesenchymal stromal cells (MSCs), which have been shown to help regulate the immune system. These cells have the ability to inhibit T-cell activation, reduce inflammation, and suppress the secretion of pro-inflammatory cytokines, making Ryoncil® a highly effective therapy for conditions like SR-aGvHD.
Its approval paves the way for future applications in other inflammatory diseases, potentially extending its benefits to adults and those suffering from autoimmune and inflammatory conditions.
Mesoblast’s Commitment to the GVHD Community
Dr. Silviu Itescu, CEO of Mesoblast, expressed pride in the company’s achievement: “We are thrilled to have received FDA approval for Ryoncil®. This approval marks a pivotal moment for both Mesoblast and the GVHD community. We are dedicated to working closely with the FDA to bring other breakthrough therapies, like REVASCOR® for cardiovascular diseases and rexlemestrocel-L for inflammatory pain, to the market. Ryoncil® will also be expanded to treat both children and adults with other inflammatory conditions, potentially benefiting a broader range of patients.”
Ryoncil® Availability
Following its approval, Ryoncil® will be available at transplant centers and hospitals across the United States, offering much-needed hope to children suffering from SR-aGvHD, a condition that has, until now, left few effective treatment options.
