Mesoblast’s Ryoncil Generates US$13.2M in First Quarter Post FDA Launch
Mesoblast has reported a strong commercial debut for Ryoncil, the first FDA-approved mesenchymal stromal cell therapy for pediatric steroid-refractory acute graft-versus-host disease, generating US$13.2 million in gross revenue in its initial quarter. The company is advancing regulatory and clinical milestones while expanding insurance coverage across the US.
- US$13.2 million gross revenue from Ryoncil sales in first quarter post-launch
- Onboarding of 25+ transplant centers, targeting 45 priority centers in US
- Mandatory Medicaid coverage effective July 1, expanding patient access
- Seven years orphan drug exclusivity and biologic exclusivity until 2036 secured
- US$162 million cash on hand with US$16.6 million net operating cash spend
Strong Commercial Launch of Ryoncil
Mesoblast Limited has marked a significant milestone with the successful commercial launch of Ryoncil (remestemcel-L-rknd), the first and only FDA-approved mesenchymal stromal cell (MSC) therapy for pediatric steroid-refractory acute graft-versus-host disease (SR-aGvHD). Since becoming commercially available on March 28, 2025, the therapy generated US$13.2 million in gross revenue through to June 30, underscoring early market acceptance in a critical niche.
CEO Dr. Silviu Itescu highlighted the progress in onboarding over 25 of the 45 priority US transplant centers that account for approximately 80% of pediatric transplants. The company expects to complete this onboarding process in the current quarter, which is essential for scaling sales and patient access.
Expanding Insurance Coverage and Patient Access
Access to Ryoncil has been bolstered by insurance coverage expansions, with over 250 million US lives now insured by commercial and government payers. Notably, mandatory fee-for-service Medicaid coverage across all US states became effective on July 1, 2025, removing a significant barrier for patients requiring this life-saving therapy.
To further support patients and healthcare providers, Mesoblast has launched the MyMesoblast™ patient access hub, facilitating insurance navigation, financial assistance, and ordering processes. This initiative aims to ensure no eligible patient is left behind due to coverage complexities.
Robust Intellectual Property and Regulatory Exclusivity
Ryoncil benefits from seven years of orphan drug exclusivity granted by the FDA, preventing approval of competing MSC products for pediatric SR-aGvHD during this period. Additionally, biologic exclusivity extends until December 2036, barring biosimilar market entry. Mesoblast’s extensive patent portfolio further protects its technology until at least 2044, creating a formidable commercial moat.
Regulatory momentum continues with a recent FDA Type B meeting to discuss a pivotal trial aiming to extend Ryoncil’s label to adult SR-aGvHD patients. This trial, to be conducted with the NIH-funded Bone Marrow Transplant Clinical Trials Network, could significantly broaden the therapy’s market.
Pipeline Progress and Financial Position
Beyond Ryoncil, Mesoblast is advancing clinical trials for rexlemestrocel-L, targeting chronic inflammatory heart failure and chronic low back pain. The company is aligned with FDA on requirements for a biologics license application for Revascor, its heart failure candidate, and is actively enrolling patients in a Phase 3 trial for low back pain.
Financially, Mesoblast reported a net operating cash spend of US$16.6 million for the quarter, supported by a strong cash position of US$162 million. The company maintains secured loan facilities totaling US$80 million and continues cost management measures, including voluntary salary reductions by directors.
Bottom Line?
Mesoblast’s initial commercial traction with Ryoncil sets a strong foundation, but upcoming clinical trials and label expansions will be critical to sustaining growth and market leadership.
Questions in the middle?
- How quickly will Mesoblast complete onboarding of all priority transplant centers?
- What is the timeline and likelihood for FDA approval to extend Ryoncil’s indication to adults?
- How will reimbursement dynamics evolve as Ryoncil gains broader Medicaid and commercial coverage?
