Ryoncil (remestemcel-L-rknd) has been granted approval by the FDA for the treatment of steroid-refractory acute graft-versus-host-disease (SR-aGVHD) in pediatric patients 2 months of age and older. This decision makes Ryoncil the first mesenchymal stromal cell (MSC) therapy to ever receive FDA approval.
Developed by Australian cell therapy specialist Mesoblast, Ryoncil is an allogeneic therapy that contains mesenchymal stromal cells, a type of cell that can differentiate into many other cell types. The cells used in this treatment are obtained from the bone marrow of healthy donors.
“Today’s decision marks an important milestone in the use of innovative cell-based therapies to treat life-threatening diseases with devastating impacts on patients, including children,” said Peter Marks, director of the FDA’s Center for Biologics Evaluation and Research (CBER). “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.”
SR-aGVHD is a life-threatening condition that can arise as a complication of allogeneic hematopoietic stem cell transplantation, also known as bone marrow transplants. It is estimated that approximately 50% of patients receiving this type of transplant develop acute graft-versus-host disease (aGVHD), where the immune cells produced by the donor blood marrow react against their host.
The standard first-line treatment for aGVHD is systemic steroid therapy, but up to 50% of patients become refractory to this treatment, which significantly increases their mortality rate and lowers quality of life.
“Steroid-refractory acute graft-versus-host disease can have significant, wide-ranging health consequences, including damage to multiple organs, reduced quality of life and risk of death in affected patients,” said Nicole Verdun, director of the Office of Therapeutic Products at CBER. “The FDA remains dedicated to helping address the urgent unmet needs of individuals with debilitating and deadly diseases, and today’s approval is an important step in that effort.”
In addition to being the first MSC therapy to receive FDA approval, Ryoncil is now also the only approved therapy for SR-aGVHD in children and teenagers.
“Steroid-refractory acute graft versus host disease is a devastating condition with an extremely poor prognosis,” said Joanne Kurtzberg, Director of the Marcus Center for Cellular Cures at Duke University Medical Center. “From today we are able to offer Ryoncil, which will be life saving for so many children and will have a great impact on their families.”
The approval of Ryoncil is based on data from a phase 3 clinical trial that enrolled 54 pediatric patients with SR-aVVHD. After four weeks of treatment, during which Ryoncil was delivered through intravenous infusion twice weekly, 30% of patients achieved a complete response and 41% had a partial response.
The immunomodulatory effects of Ryoncil, which include the inhibition of T cell activation and the secretion of pro-inflammatory cytokines, makes the therapy a promising candidate to treat other inflammatory indications, including acute respiratory distress syndrome (ARDS) and inflammatory bowel disease (IBD). Mesoblast’s cell therapy pipeline also includes an experimental treatment for heart failure and a therapy for chronic low back pain.
“With Ryoncil’s approval by the FDA, Mesoblast has demonstrated the ability to bring the first MSC product to market,” said Silviu Itescu, Chief Executive of Mesoblast. “We will continue to work closely with FDA to obtain approval of our other late-stage products, including Revascor for cardiovascular diseases and rexlemestrocel-L for inflammatory pain indications, as well as expanding the indications for Ryoncil in both children and adults with inflammatory conditions.”
