Mesoblast launches Ryoncil in the U.S. with payer buy-in

Mesoblast's Ryoncil (remestemcel-L), the first mesenchymal stromal cell (MSC) therapy approved by the FDA for any indication, is now available for purchase in the U.S.

Ryoncil, an allogeneic bone marrow-derived MSC therapy, was approved for steroid-refractory acute graft versus host disease (SR-aGvHD) in children two months and older, including adolescents and teenagers, in December 2024. The therapy is administered via intravenous infusion given twice per week for four consecutive weeks.

Mesoblast says it has been engaging with commercial and government insurers to expedite patient access to Ryoncil. To date, nine commercial payers have published favorable medical policies representing approximately 37 million covered lives in the U.S. According to the company, it will enter into the National Drug Rebate Agreement with Medicaid and expects to be enrolled in the program “in short order.”

About 50% patients who undergo an allogeneic bone marrow transplant 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.