Mesoblast has announced that the US Food and Drug Administration (FDA) has approved Ryoncil (remestemcel-L) as the first mesenchymal stromal cell (MSC) therapy in the US.
Ryoncil is an allogeneic bone marrow-derived MSC therapy. It is now authorised for the treatment of steroid-refractory acute graft-versus-host disease (SR-aGvHD) in children aged two months and older, including adolescents and teenagers.
According to Mesoblast, Ryoncil is the only MSC therapy approved for any indication in the US and is the only approved treatment for SR-aGvHD.
Mesoblast CEO Silviu Itescu said: “We are very pleased that the FDA has granted approval of Ryoncil and are proud of the company’s commitment to the GVHD community in bringing this important new treatment to children and families with no other acceptable options.
“With Ryoncil approval by FDA, Mesoblast has demonstrated the ability to bring the first MSC product to market.”
The safety and effectiveness of Ryoncil were evaluated in a multicentre, single-arm study involving 54 paediatric participants with SR-aGvHD following allogeneic haematopoietic stem cell transplantation (allo-HSCT).
Participants received intravenous Ryoncil infusions twice weekly for four weeks, totalling eight infusions.
Each study participant’s baseline conditions were assessed using the International Blood and Marrow Transplantation Registry Severity Index Criteria (IBMTR) to evaluate organ involvement and disease severity.
Ryoncil’s effectiveness was primarily measured by the rate and duration of response 28 days after treatment initiation.
Participants with partial or mixed responses continued infusions with weekly infusions for an additional four weeks.
After 28 days, 16 participants achieved a complete response, while 22 participants had a partial response.
FDA Center for Biologics Evaluation and Research (CBER) director Peter Marks said: “This first mesenchymal stromal cell therapy approval demonstrates the FDA’s commitment to supporting the development of safe and effective products that could improve the quality of life for patients with symptoms that are unresponsive to other therapies.”
Australia-based Mesoblast is also advancing additional cell therapies for various indications using its remestemcel-L and rexlemestrocel-L allogeneic stromal cell technology platforms.
Ryoncil is being developed for further inflammatory diseases, including SR-aGvHD in adults and biologic-resistant inflammatory bowel disease.
Rexlemestrocel-L is in development for heart failure and chronic lower back pain.
Mesoblast has established commercial partnerships in Japan, Europe, and China to expand its global reach.
In December 2021, Novartis cancelled its agreement with Mesoblast to develop and market remestemcel-L. The deal, signed in November 2020, included a $50m upfront payment, including $25m in equity.
