FDA Approves Ryoncil for Steroid-Refractory aGvHD in Young Children

Mesoblast Limited, global leader in allogeneic cellular medicines for inflammatory diseases, announced the Food and Drug Administration (FDA) approved Ryoncil (remestemcel-L) as the first mesenchymal stromal cell (MSC) therapy in the United States.

Ryoncil is the only MSC therapy approved in the US for any indication, and the only approved therapy for steroid-refractory acute graft versus host disease (SR-aGvHD) in children 2 months and older, including  adolescents and teenagers.

Transplant physician Dr Joanne Kurtzberg, the Jerome Harris Distinguished Professor of Pediatrics and Professor of Pathology, and Director, Marcus Center for Cellular Cures at Duke University Medical Center (DUMC), said: “Steroid-refractory acute graft versus host disease is a devastating condition with an extremely poor prognosis. From today we are able to offer Ryoncil, the first FDA-approved treatment which will be life saving for so many children and will have a great impact on their families.”

Annually in the United States approximately 10,000 patients undergo an allogeneic bone marrow transplant, 1,500 of whom are children. Approximately 50% develop aGvHD and almost half of those do not respond to steroids, the recognized first-line treatment.

In a single-arm multicenter phase 3 trial of children with SR-aGvHD, 89% of whom had high severity Grade C or Grade D disease, 70% achieved an overall response by Day 28 of treatment with Ryoncil, a measure that predicts survival in aGVHD. Ryoncil treatment was not discontinued or interrupted in any patient for any laboratory abnormality, and the full course was completed without interruption in more than 85% of patients. The full phase 3 clinical study results are available in Biology of Blood and Marrow Transplantation.

“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,” said Dr. Silviu Itescu, Chief Executive of Mesoblast. “With Ryoncil approval by FDA, Mesoblast has demonstrated the ability to bring the first MSC product to market. 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.”

Ryoncil is an allogeneic bone marrow-derived mesenchymal stromal cell (MSC) therapy indicated for the treatment of steroid-refractory acute graft versus host disease (SR-aGvHD) in paediatric atients 2 months of age and older.

The recommended dosage of Ryoncil is 2 × 106 MSC /kg body weight per intravenous infusion given twice per week for 4 consecutive weeks. Response is assessed 28 ± 2 days after the first dose and further treatment administered as appropriate.

Mesoblast is a world leader in developing allogeneic (off-the-shelf) cellular medicines for the treatment of severe and life-threatening inflammatory conditions.

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