Ryoncil® is Now Available for Purchase in the United States
Mesoblast (MESO) has announced that Ryoncil® (remestemcel-L), the first FDA-approved mesenchymal stromal cell (MSC) therapy, is now commercially available in the United States. The therapy is approved for treating pediatric patients aged 2 months and older with steroid-refractory acute graft versus host disease (SR-aGvHD).
The recommended dosage is 2×106 MSC/kg body weight per intravenous infusion, administered twice weekly for 4 weeks. Mesoblast has established MyMesoblast™, a patient access hub, to facilitate insurance coverage and financial assistance. Nine commercial payers, covering approximately 37 million lives in the US, have published favorable medical policies. The company is also in process of entering the National Drug Rebate Agreement with Medicaid.
Mesoblast (MESO) ha annunciato che Ryoncil® (remestemcel-L), la prima terapia con cellule stromali mesenchimali (MSC) approvata dalla FDA, è ora disponibile commercialmente negli Stati Uniti. La terapia è approvata per il trattamento di pazienti pediatrici di età pari o superiore a 2 mesi con malattia acuta da rigetto del trapianto resistente agli steroidi (SR-aGvHD).
Il dosaggio raccomandato è di 2×106 MSC/kg di peso corporeo per infusione endovenosa, somministrato due volte a settimana per 4 settimane. Mesoblast ha istituito MyMesoblast™, un hub di accesso per i pazienti, per facilitare la copertura assicurativa e l'assistenza finanziaria. Nove pagatori commerciali, che coprono circa 37 milioni di vite negli Stati Uniti, hanno pubblicato politiche mediche favorevoli. L'azienda è anche in procinto di entrare nell'Accordo Nazionale sui Rimborsi dei Farmaci con Medicaid.
Mesoblast (MESO) ha anunciado que Ryoncil® (remestemcel-L), la primera terapia con células madre estromales mesenquimatosas (MSC) aprobada por la FDA, ya está disponible comercialmente en los Estados Unidos. La terapia está aprobada para tratar a pacientes pediátricos de 2 meses en adelante con enfermedad injerto contra huésped aguda refractaria a esteroides (SR-aGvHD).
La dosis recomendada es de 2×106 MSC/kg de peso corporal por infusión intravenosa, administrada dos veces por semana durante 4 semanas. Mesoblast ha establecido MyMesoblast™, un centro de acceso para pacientes, para facilitar la cobertura del seguro y la asistencia financiera. Nueve pagadores comerciales, que cubren aproximadamente 37 millones de vidas en los EE. UU., han publicado políticas médicas favorables. La empresa también está en proceso de entrar en el Acuerdo Nacional de Reembolso de Medicamentos con Medicaid.
Mesoblast (MESO)는 Ryoncil® (remestemcel-L)이 FDA 승인 최초의 중간엽 줄기세포(MSC) 치료제로서 미국에서 상업적으로 이용 가능하다고 발표했습니다. 이 치료법은 스테로이드 내성 급성 이식편대숙주병(SR-aGvHD)으로 고통받는 2개월 이상의 소아 환자를 치료하는 데 승인되었습니다.
권장 용량은 체중 kg당 2×106 MSC로, 정맥 주사로 주 2회, 4주 동안 투여됩니다. Mesoblast는 보험 보장 및 재정 지원을 용이하게 하기 위해 MyMesoblast™라는 환자 접근 허브를 설립했습니다. 미국에서 약 3,700만 명의 생명을 보장하는 아홉 개의 상업적 보험자가 유리한 의료 정책을 발표했습니다. 이 회사는 Medicaid와의 국가 약물 환급 계약에 들어가는 과정에 있습니다.
Mesoblast (MESO) a annoncé que Ryoncil® (remestemcel-L), la première thérapie approuvée par la FDA utilisant des cellules souches stromales mésenchymateuses (MSC), est désormais disponible commercialement aux États-Unis. La thérapie est approuvée pour traiter les patients pédiatriques âgés de 2 mois et plus atteints de maladie du greffon contre l'hôte aiguë réfractaire aux stéroïdes (SR-aGvHD).
La posologie recommandée est de 2×106 MSC/kg de poids corporel par infusion intraveineuse, administrée deux fois par semaine pendant 4 semaines. Mesoblast a mis en place MyMesoblast™, un hub d'accès pour les patients, afin de faciliter la couverture d'assurance et l'aide financière. Neuf payeurs commerciaux, couvrant environ 37 millions de vies aux États-Unis, ont publié des politiques médicales favorables. L'entreprise est également en train de rejoindre l'accord national de remboursement des médicaments avec Medicaid.
Mesoblast (MESO) hat bekannt gegeben, dass Ryoncil® (remestemcel-L), die erste von der FDA zugelassene Therapie mit mesenchymalen Stammzellen (MSC), nun kommerziell in den Vereinigten Staaten erhältlich ist. Die Therapie ist zur Behandlung von pädiatrischen Patienten ab 2 Monaten mit steroid-refraktärer akuter Graft-versus-Host-Krankheit (SR-aGvHD) zugelassen.
Die empfohlene Dosierung beträgt 2×106 MSC/kg Körpergewicht pro intravenöser Infusion, die zweimal wöchentlich über 4 Wochen verabreicht wird. Mesoblast hat MyMesoblast™ eingerichtet, ein Zugangszentrum für Patienten, um die Versicherungsschutz und finanzielle Unterstützung zu erleichtern. Neun kommerzielle Kostenträger, die etwa 37 Millionen Leben in den USA abdecken, haben günstige medizinische Richtlinien veröffentlicht. Das Unternehmen ist auch dabei, dem National Drug Rebate Agreement mit Medicaid beizutreten.
- First FDA-approved MSC therapy now commercially available in US
- Nine commercial payers covering 37M lives have approved favorable policies
- Successful commercial launch within 3 months of FDA approval
- Patient access hub established to facilitate insurance coverage
- None.
Insights
Mesoblast's announcement that Ryoncil® is now commercially available marks a pivotal transition from development-stage to commercial-stage company - a fundamental milestone in biotech evolution. The three-month timeline from FDA approval to market availability demonstrates efficient operational execution for a complex cell therapy product.
The commercial infrastructure appears well-established with the MyMesoblast™ patient access hub addressing critical reimbursement challenges typical of advanced therapies. The favorable coverage policies from nine commercial payers representing approximately 37 million covered lives indicates successful initial market access efforts, though this represents only a portion of the US insured population.
As the first FDA-approved mesenchymal stromal cell therapy for any indication, Ryoncil establishes Mesoblast's leadership in the cellular medicine space. While pediatric steroid-refractory acute GvHD represents a specialized market with patient numbers, successful commercialization validates the company's platform technology and manufacturing capabilities.
The pending Medicaid enrollment through the National Drug Rebate Agreement will expand access to additional patient populations. The lack of pricing details or revenue projections in this announcement is typical at this stage, with commercial performance metrics likely to emerge in future quarterly reports.
The commercial launch of Ryoncil® represents significant progress in Mesoblast's reimbursement strategy. Securing favorable medical policies from nine commercial payers covering 37 million lives this early in the launch cycle suggests successful payer engagement, particularly noteworthy for a novel cell therapy where reimbursement hurdles are typically substantial.
The establishment of the MyMesoblast™ patient access hub indicates the company has implemented sophisticated patient support infrastructure - a critical success factor for specialized therapies with complex administration requirements. The hub's focus on insurance coverage assistance and financial support programs addresses the practical barriers that often limit uptake of advanced therapies.
The ongoing Medicaid enrollment process through the National Drug Rebate Agreement will be crucial for enabling access for economically disadvantaged patients. While the timeline is described only as "in short order," government program enrollment typically takes 1-2 quarters to fully implement.
For steroid-refractory acute GvHD patients who face high mortality rates, the availability of this therapy addresses a significant unmet medical need. The twice-weekly administration for four weeks represents a defined treatment course, which is favorable from both a clinical and reimbursement perspective compared to open-ended treatment regimens.
NEW YORK, March 26, 2025 (GLOBE NEWSWIRE) -- Mesoblast Limited (Nasdaq:MESO; ASX:MSB), global leader in allogeneic cellular medicines for inflammatory diseases, today announced that Ryoncil® (remestemcel-L) the first mesenchymal stromal cell (MSC) therapy approved by U.S. Food and Drug Administration (FDA) for any indication, is now available for purchase in the United States.
Ryoncil® is approved for treatment of pediatric patients 2 months and older, including adolescents and teenagers, with steroid-refractory acute graft versus host disease (SR-aGvHD), a condition with high mortality rates. The recommended dosage of Ryoncil® for treatment of pediatric SR-aGvHD is 2×106 MSC/kg body weight per intravenous infusion given twice per week for 4 consecutive weeks.
To assist patients and institutions with insurance coverage, financial assistance, and access programs, ensuring that no patient is left behind in receiving this potentially life-saving therapy, Mesoblast has established a patient access hub termed MyMesoblast™, where Ryoncil® is now available for ordering. Additional information is available on ryoncil.com, where valuable resources for healthcare providers, patients and caregivers can be found.
We have been engaging with commercial and government insurers to expedite patient access to therapy. To date nine commercial payers have published favorable medical policies representing approximately 37 million covered lives in the United States. We continue to broaden the commercial payer access landscape on a daily basis. The Company will enter into the National Drug Rebate Agreement with Medicaid and expects to be enrolled in the program in short order.
“The commercial availability of RYONCIL is a significant milestone in our mission to bring innovative cellular medicines to patients in need,” said Dr. Silviu Itescu, CEO of Mesoblast. “We have made RYONCIL available three months after receiving FDA approval, a significant commercial achievement and a reflection of our team and partners who are driven by the overwhelming desire to help children and their families faced with this devasting 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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