First Three Children to Commence Treatment With Ryoncil®
Mesoblast (MESO) has entered into the Medicaid National Drug Rebate Agreement with U.S. Centers for Medicare & Medicaid Services for Ryoncil®, the first FDA-approved mesenchymal stromal cell therapy. This agreement ensures treatment access for approximately 40% of U.S. children covered by Medicaid who have steroid-refractory acute graft versus host disease (SR-aGvHD), with the remaining coverage through private insurance.
States can immediately opt to cover Ryoncil®, with mandatory coverage starting July 1, 2025. The first three children with SR-aGvHD will begin treatment this week. Ryoncil® is approved for pediatric patients 2 months and older, with a recommended course of 2×106 MSC/kg body weight per intravenous infusion, administered twice weekly for 4 consecutive weeks.
Mesoblast (MESO) ha stipulato un accordo con i Centri statunitensi per Medicare e Medicaid per il Ryoncil®, la prima terapia con cellule stromali mesenchimali approvata dalla FDA. Questo accordo garantisce l'accesso al trattamento per circa il 40% dei bambini statunitensi coperti da Medicaid che soffrono di malattia acuta da trapianto contro ospite resistente agli steroidi (SR-aGvHD), con la restante copertura tramite assicurazione privata.
Gli stati possono immediatamente scegliere di coprire il Ryoncil®, con copertura obbligatoria a partire dal 1 luglio 2025. I primi tre bambini con SR-aGvHD inizieranno il trattamento questa settimana. Il Ryoncil® è approvato per pazienti pediatrici di età pari o superiore a 2 mesi, con un corso raccomandato di 2×10^6 MSC/kg di peso corporeo per infusione endovenosa, somministrato due volte a settimana per 4 settimane consecutive.
Mesoblast (MESO) ha firmado un acuerdo con los Centros de Medicare y Medicaid de EE. UU. para el Ryoncil®, la primera terapia con células madre mesenquimatosas aprobada por la FDA. Este acuerdo garantiza el acceso al tratamiento para aproximadamente el 40% de los niños en EE. UU. cubiertos por Medicaid que padecen enfermedad injerto contra huésped aguda resistente a esteroides (SR-aGvHD), con la cobertura restante a través de seguros privados.
Los estados pueden optar por cubrir Ryoncil® de inmediato, con cobertura obligatoria a partir del 1 de julio de 2025. Los primeros tres niños con SR-aGvHD comenzarán el tratamiento esta semana. Ryoncil® está aprobado para pacientes pediátricos de 2 meses o más, con un curso recomendado de 2×10^6 MSC/kg de peso corporal por infusión intravenosa, administrado dos veces por semana durante 4 semanas consecutivas.
Mesoblast (MESO)는 미국 메디케어 및 메디케이드 서비스 센터와 Ryoncil®에 대한 메디케이드 국가 약품 리베이트 계약을 체결했습니다. 이 계약은 스테로이드 내성 급성 이식편 대 숙주 질환(SR-aGvHD)을 앓고 있는 메디케이드에 가입된 미국 어린이의 약 40%가 치료를 받을 수 있도록 보장합니다. 나머지 보장은 민간 보험을 통해 이루어집니다.
주에서는 즉시 Ryoncil®의 보장을 선택할 수 있으며, 2025년 7월 1일부터 의무 보장이 시작됩니다. SR-aGvHD를 앓고 있는 첫 세 명의 어린이가 이번 주에 치료를 시작합니다. Ryoncil®은 2개월 이상의 소아 환자에게 승인되었으며, 권장 치료 용량은 체중 kg당 2×10^6 MSC의 정맥 주사로, 주 2회 4주 연속으로 투여됩니다.
Mesoblast (MESO) a conclu un accord avec les centres américains Medicare et Medicaid pour le Ryoncil®, la première thérapie par cellules souches mésenchymateuses approuvée par la FDA. Cet accord garantit l'accès au traitement pour environ 40 % des enfants américains couverts par Medicaid souffrant de la maladie du greffon contre l'hôte aiguë résistante aux stéroïdes (SR-aGvHD), le reste étant couvert par une assurance privée.
Les États peuvent immédiatement choisir de couvrir le Ryoncil®, avec une couverture obligatoire à partir du 1er juillet 2025. Les trois premiers enfants atteints de SR-aGvHD commenceront leur traitement cette semaine. Le Ryoncil® est approuvé pour les patients pédiatriques âgés de 2 mois et plus, avec un traitement recommandé de 2×10^6 MSC/kg de poids corporel par infusion intraveineuse, administré deux fois par semaine pendant 4 semaines consécutives.
Mesoblast (MESO) hat einen Vertrag mit den US-Centern für Medicare und Medicaid über Ryoncil® abgeschlossen, die erste von der FDA genehmigte Therapie mit mesenchymalen Stammzellen. Dieser Vertrag stellt sicher, dass etwa 40 % der in den USA durch Medicaid versicherten Kinder mit steroid-refraktärer akuter Graft-versus-Host-Krankheit (SR-aGvHD) Zugang zur Behandlung haben, während die restliche Abdeckung durch private Versicherungen erfolgt.
Die Bundesstaaten können sofort entscheiden, Ryoncil® zu übernehmen, wobei die obligatorische Abdeckung am 1. Juli 2025 beginnt. Die ersten drei Kinder mit SR-aGvHD werden diese Woche mit der Behandlung beginnen. Ryoncil® ist für pädiatrische Patienten ab 2 Monaten zugelassen, mit einem empfohlenen Verlauf von 2×10^6 MSC/kg Körpergewicht pro intravenöser Infusion, die zweimal wöchentlich über 4 aufeinanderfolgende Wochen verabreicht wird.
- Secured Medicaid coverage agreement, expanding access to approximately 40% of eligible U.S. children
- First FDA-approved mesenchymal stromal cell therapy in any indication
- Treatment initiation for first three patients, marking commercial launch
- Mandatory coverage ensures consistent revenue stream starting July 2025
- Delayed mandatory coverage implementation until July 2025
- State-by-state optional coverage may result in inconsistent initial market penetration
Insights
Mesoblast's announcement represents a significant commercial milestone that materially improves market access for Ryoncil. The Medicaid National Drug Rebate Agreement with CMS guarantees reimbursement for approximately 40% of potential pediatric patients with steroid-refractory acute graft versus host disease (SR-aGvHD) in the US who are covered by Medicaid.
The reimbursement landscape is now complete, with mandatory Medicaid coverage beginning July 1, 2025 (with optional immediate coverage) and private insurance already covering the remaining patients. Removing this critical access barrier significantly de-risks Ryoncil's commercial launch by ensuring payment for the full treatment course across all insurance types.
The commencement of treatment for the first three patients this week marks the transition from regulatory approval to commercial execution. SR-aGvHD is characterized by high mortality rates, and as the first FDA-approved MSC therapy for any indication, Ryoncil addresses a high unmet medical need with a novel therapeutic modality.
The recommended treatment course of 8 infusions over 4 weeks suggests a substantial per-patient revenue opportunity. While not explicitly quantified, securing government-mandated reimbursement substantially strengthens Mesoblast's near-term revenue prospects and establishes commercial validation for their broader cell therapy platform.
This CMS agreement represents a landmark reimbursement milestone for advanced cell therapies in the US healthcare system. The Medicaid National Drug Rebate Agreement effectively mandates coverage for an innovative cellular therapy across all state Medicaid programs – which is particularly significant since pediatric patients constitute a higher proportion of Medicaid beneficiaries compared to private insurance.
The phased implementation approach – allowing immediate optional coverage with mandatory coverage by July 1, 2025 – follows standard CMS practice for newly approved therapies but creates a clear timeline for nationwide access. This structured reimbursement framework removes the typical state-by-state variability in Medicaid coverage that often creates treatment disparities.
For a rare condition like steroid-refractory acute GvHD with treatment options, this agreement establishes an important precedent for how novel cell therapies can achieve broad reimbursement status. The timing between FDA approval and securing this agreement appears relatively efficient compared to historical timelines for complex biologics.
This development signals CMS's willingness to support access to innovative cell therapies that address high-mortality conditions, potentially setting a favorable reimbursement pathway for future cellular medicines targeting severe inflammatory diseases with treatment alternatives.
United States Centers for Medicare and Medicaid Services (CMS) Mandates Ryoncil® Coverage
NEW YORK, March 30, 2025 (GLOBE NEWSWIRE) -- Mesoblast (Nasdaq:MESO; ASX:MSB), global leader in allogeneic cellular medicines for inflammatory diseases, today announced that it has entered into the Medicaid National Drug Rebate Agreement (NDRA) with the U.S. Centers for Medicare & Medicaid Services (CMS), Department of Health and Human Services, for Ryoncil® (remestemcel-L), the first mesenchymal stromal cell (MSC) therapy approved by U.S. Food and Drug Administration (FDA) for any indication. The NDRA agreement with Mesoblast means that the U.S. Government now provides inpatient and outpatient access for a treatment course of Ryoncil® to the approximately
The first three children with SR-aGvHD will receive a course of Ryoncil® commencing this week. Ryoncil® is an allogeneic MSC therapy for treatment of pediatric patients 2 months and older, including adolescents and teenagers, with SR-aGvHD, a condition with high mortality rates. The recommended course of Ryoncil® for pediatric SR-aGvHD is 2×106 MSC/kg body weight per intravenous infusion given twice per week for 4 consecutive weeks. Additional information is available on ryoncil.com, where valuable resources for healthcare providers, patients and caregivers can be found.
Mesoblast Chief Executive Dr. Silviu Itescu said: “We are delighted to be commencing treatment with Ryoncil® for children suffering with SR-aGvHD and are proud that the product is available to all children in the U.S. irrespective whether they have private insurance or Medicaid. This is a significant commercial achievement by our team and partners who are driven by an overwhelming desire to help children and their families faced with this devastating disease.”
About Mesoblast
Mesoblast (the Company) is a world leader in developing allogeneic (off-the-shelf) cellular medicines for the treatment of severe and life-threatening inflammatory conditions. The therapies from the Company’s proprietary mesenchymal lineage cell therapy technology platform respond to severe inflammation by releasing anti-inflammatory factors that counter and modulate multiple effector arms of the immune system, resulting in significant reduction of the damaging inflammatory process.
Mesoblast’s RYONCIL® (remestemcel-L) for the treatment of steroid-refractory acute graft versus host disease (SR-aGvHD) in pediatric patients 2 months and older is the first FDA approved mesenchymal stromal cell (MSC) therapy. Please see the full Prescribing Information at www.ryoncil.com.
Mesoblast is committed to developing additional cell therapies for distinct indications based on its remestemcel-L and rexlemestrocel-L allogeneic stromal cell technology platforms. RYONCIL is being developed for additional inflammatory diseases including SR-aGvHD in adults and biologic-resistant inflammatory bowel disease. Rexlemestrocel-L is being developed for heart failure and chronic low back pain. The Company has established commercial partnerships in Japan, Europe and China.
About Mesoblast intellectual property: Mesoblast has a strong and extensive global intellectual property portfolio, with over 1,000 granted patents or patent applications covering mesenchymal stromal cell compositions of matter, methods of manufacturing and indications. These granted patents and patent applications are expected to provide commercial protection extending through to at least 2041 in major markets.
About Mesoblast manufacturing: The Company’s proprietary manufacturing processes yield industrial-scale, cryopreserved, off-the-shelf, cellular medicines. These cell therapies, with defined pharmaceutical release criteria, are planned to be readily available to patients worldwide.
Mesoblast has locations in Australia, the United States and Singapore and is listed on the Australian Securities Exchange (MSB) and on the Nasdaq (MESO). For more information, please see www.mesoblast.com, LinkedIn: Mesoblast Limited and Twitter: @Mesoblast
Forward-Looking Statements
This press release includes forward-looking statements that relate to future events or our future financial performance and involve known and unknown risks, uncertainties and other factors that may cause our actual results, levels of activity, performance or achievements to differ materially from any future results, levels of activity, performance or achievements expressed or implied by these forward-looking statements. We make such forward-looking statements pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995 and other federal securities laws. Forward-looking statements should not be read as a guarantee of future performance or results, and actual results may differ from the results anticipated in these forward-looking statements, and the differences may be material and adverse. Forward-looking statements include, but are not limited to, statements about: the initiation, timing, progress and results of Mesoblast’s preclinical and clinical studies, and Mesoblast’s research and development programs; Mesoblast’s ability to advance product candidates into, enroll and successfully complete, clinical studies, including multi-national clinical trials; Mesoblast’s ability to advance its manufacturing capabilities; the timing or likelihood of regulatory filings and approvals, manufacturing activities and product marketing activities, if any; the commercialization of Mesoblast’s RYONCIL for pediatric SR-aGVHD and any other product candidates, if approved; regulatory or public perceptions and market acceptance surrounding the use of stem-cell based therapies; the potential for Mesoblast’s product candidates, if any are approved, to be withdrawn from the market due to patient adverse events or deaths; the potential benefits of strategic collaboration agreements and Mesoblast’s ability to enter into and maintain established strategic collaborations; Mesoblast’s ability to establish and maintain intellectual property on its product candidates and Mesoblast’s ability to successfully defend these in cases of alleged infringement; the scope of protection Mesoblast is able to establish and maintain for intellectual property rights covering its product candidates and technology; estimates of Mesoblast’s expenses, future revenues, capital requirements and its needs for additional financing; Mesoblast’s financial performance; developments relating to Mesoblast’s competitors and industry; and the pricing and reimbursement of Mesoblast’s product candidates, if approved. You should read this press release together with our risk factors, in our most recently filed reports with the SEC or on our website. Uncertainties and risks that may cause Mesoblast’s actual results, performance or achievements to be materially different from those which may be expressed or implied by such statements, and accordingly, you should not place undue reliance on these forward-looking statements. We do not undertake any obligations to publicly update or revise any forward-looking statements, whether as a result of new information, future developments or otherwise.
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FAQ
When does mandatory Medicaid coverage for Ryoncil (MESO) begin?
What is the recommended dosage for Ryoncil in treating SR-aGvHD?
What percentage of U.S. children with SR-aGvHD will be covered by Medicaid for Ryoncil treatment?
What age group is eligible for Ryoncil treatment for SR-aGvHD?
