Mesoblast’s RYONCIL® is the First U.S. FDA-Approved Mesenchymal Stromal Cell (MSC) Therapy
Mesoblast's RYONCIL® (remestemcel-L) has received FDA approval as the first mesenchymal stromal cell (MSC) therapy in the United States. The therapy is specifically approved for children aged 2 months and older with steroid-refractory acute graft versus host disease (SR-aGvHD). Clinical trials demonstrated a 70% overall response rate by Day 28 in patients with SR-aGvHD, where 89% had high severity Grade C or Grade D disease. This breakthrough addresses a significant medical need, as approximately 50% of the 10,000 annual U.S. allogeneic bone marrow transplant patients develop aGvHD, with almost half not responding to first-line steroid treatment.
RYONCIL® di Mesoblast (remestemcel-L) ha ricevuto l'approvazione della FDA come la prima terapia con cellule stromali mesenchimali (MSC) negli Stati Uniti. La terapia è specificamente approvata per bambini di età pari o superiore a 2 mesi affetti da malattia da innesto contro l'ospite acuta refrattaria agli steroidi (SR-aGvHD). Gli studi clinici hanno dimostrato un tasso di risposta globale del 70% entro il giorno 28 nei pazienti con SR-aGvHD, dove l'89% presentava una malattia di alta gravità di Grado C o Grado D. Questa innovazione risponde a un'importante necessità medica, poiché circa il 50% dei 10.000 pazienti statunitensi che ricevono un trapianto di midollo osseo allogenico annualmente sviluppa aGvHD, con quasi la metà che non risponde al trattamento steroidale di prima linea.
RYONCIL® de Mesoblast (remestemcel-L) ha recibido la aprobación de la FDA como la primera terapia con células madre estromales mesenquimatosas (MSC) en los Estados Unidos. La terapia está específicamente aprobada para niños de 2 meses en adelante con enfermedad injerto contra huésped aguda refractaria a esteroides (SR-aGvHD). Los ensayos clínicos demostraron una tasa de respuesta general del 70% para el día 28 en pacientes con SR-aGvHD, donde el 89% tenía enfermedad de alta gravedad de Grado C o Grado D. Este avance aborda una necesidad médica significativa, ya que aproximadamente el 50% de los 10,000 pacientes anuales de trasplante de médula ósea alogénico en EE. UU. desarrollan aGvHD, y casi la mitad no responde al tratamiento esteroide de primera línea.
메조블라스트의 RYONCIL® (remestemcel-L)이 미국에서 최초의 간질세포 줄기세포(MSC) 치료제로 FDA 승인을 받았습니다. 이 치료법은 스테로이드에 내성이 있는 급성 이식편 대 숙주 질환(SR-aGvHD)으로 진단받은 2개월 이상의 어린이를 위해 특별히 승인되었습니다. 임상 시험에서 SR-aGvHD 환자에서 28일 이내에 70%의 전체 반응률이 시연되었습니다, 이 중 89%가 고도 중증인 C등급 또는 D등급 질환을 앓고 있었습니다. 이 혁신은 매년 약 10,000명의 미국 동종 골수 이식 환자 중 50%가 aGvHD를 개발하고 그 중 거의 절반이 1차 스테로이드 치료에 반응하지 않기 때문에 중요한 의학적 필요를 충족합니다.
RYONCIL® de Mesoblast (remestemcel-L) a reçu l'approbation de la FDA en tant que première thérapie par cellules stromales mésenchymateuses (MSC) aux États-Unis. Cette thérapie est spécifiquement approuvée pour les enfants de 2 mois et plus atteints de maladie du greffon contre l'hôte aiguë résistant aux stéroïdes (SR-aGvHD). Les essais cliniques ont montré un taux de réponse global de 70% au jour 28 chez les patients atteints de SR-aGvHD, dont 89% présentaient une maladie de gravité élevée de grade C ou D. Cette avancée répond à un besoin médical significatif, car environ 50% des 10 000 patients annuels de transplantation de moelle osseuse allogénique aux États-Unis développent une aGvHD, dont près de la moitié ne répondent pas au traitement stéroïdien de première ligne.
RYONCIL® von Mesoblast (remestemcel-L) hat die FDA-Zulassung als erste Therapie mit mesenchymalen Stromazellen (MSC) in den Vereinigten Staaten erhalten. Die Therapie ist speziell für Kinder ab 2 Monaten mit steroid-refraktärer akuter Transplantat-gegen-Wirt-Krankheit (SR-aGvHD) zugelassen. Klinische Studien haben eine Gesamtansprechraten von 70% bis zum Tag 28 bei Patienten mit SR-aGvHD gezeigt, wobei 89% eine hohe Schweregrad C oder D Erkrankung hatten. Dieser Fortschritt deckt einen bedeutenden medizinischen Bedarf ab, da etwa 50% der jährlich 10.000 allogen transplantierten Knochenmarkpatienten in den USA aGvHD entwickeln, von denen fast die Hälfte nicht auf die Erstlinientherapie mit Steroiden anspricht.
- First FDA-approved MSC therapy in the United States
- 70% overall response rate in Phase 3 trial for SR-aGvHD
- 85% of patients completed full treatment course without interruption
- Addresses unmet medical need in pediatric patients
- Serious adverse reactions reported including pyrexia (9%) and respiratory failure (9%)
- Treatment discontinuation in 8 patients due to adverse reactions
- to pediatric patients 2 months and older
Insights
The FDA approval of RYONCIL represents a groundbreaking milestone in cell therapy. This first-in-class MSC therapy addresses a critical unmet need in pediatric SR-aGvHD, where
The therapy's mechanism of action through immunomodulation and cytokine inhibition suggests potential applications beyond GvHD. The safety profile appears manageable, with no treatment discontinuations due to laboratory abnormalities and high treatment completion rates. This positions RYONCIL favorably for potential label expansion into other inflammatory conditions.
This approval represents a significant market opportunity for Mesoblast. With approximately 10,000 annual allogeneic bone marrow transplants in the US, including 1,500 children and considering the incidence rates of SR-aGvHD, the addressable market is substantial. Being the first FDA-approved MSC therapy provides Mesoblast with a strong competitive advantage and validates their platform technology.
The approval could accelerate the development pathway for Mesoblast's other pipeline candidates, particularly REVASCOR and rexlemestrocel-L. The company's demonstrated ability to navigate the regulatory pathway for cell therapies enhances its value proposition and could attract partnership opportunities.
The commercial launch of RYONCIL could significantly impact Mesoblast's revenue trajectory. Given the high unmet medical need and treatment options, premium pricing potential exists. The therapy's strong efficacy data and first-mover advantage in the MSC space should support favorable reimbursement discussions. The established distribution network through transplant centers provides a focused commercial strategy with lower marketing expenses.
With a market cap of
- RYONCIL (remestemcel-L) is the first MSC product approved by FDA for any indication.
- RYONCIL is the first FDA-approved therapy for children aged 2 months and older, including adolescents and teenagers, with steroid-refractory acute graft versus host disease (SR-aGvHD), a life-threatening condition with high mortality rates.
- In a single-arm, multi-center, 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’S immunomodulatory effects, including inhibition of T cell activation and secretion of pro-inflammatory cytokines, position the therapy for potential other indications in diseases with excessive inflammation.
NEW YORK, Dec. 18, 2024 (GLOBE NEWSWIRE) -- Mesoblast Limited (Nasdaq:MESO; ASX:MSB), global leader in allogeneic cellular medicines for inflammatory diseases, today 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 U.S. 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
“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 will be available in the United States at transplant centers and other treating hospitals.
Please see the full Prescribing Information at www.ryoncil.com. The FDA’s approval press release is available here.
What is RYONCIL (remestemcel-L)
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 pediatric patients 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.
Important Safety Information
Contraindications: Known hypersensitivity to dimethyl sulfoxide (DMSO) or porcine and bovine proteins.
Adverse reactions: Serious adverse reactions included pyrexia (
You may report side effects to the FDA at 1-800-FDA-1088 or www.fda.gov/medwatch. You may also report side effects to Mesoblast at toll-free phone #1-844-889-MESO (6376)
Please see the RYONCIL full Prescribing Information for additional Important Safety Information.
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 children 2 months and older is the first FDA-approved mesenchymal stromal cell (MSC) therapy.
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 provide commercial protection extending through to at least 2041 in all 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
References / Footnotes
| 1. | Rashidi A et al. Outcomes and predictors of response in steroid-refractory acute graft-versus-host disease: single-center results from a cohort of 203 patients. Biol Blood Bone Marrow Transplant 2019; 25(11):2297-2302. |
| 2. | Berger M, Pessolano R, Carraro F, Saglio F, Vassallo E, Fagioli F. Steroid-refractory acute graft-versus-host disease graded III-IV in pediatric patients. A mono-institutional experience with a long-term follow-up. Pediatric Transplantation. 2020; 24(7):e13806 |
| 3. | Biavasco F, Ihorst G, Wasch R, Wehr C, Bertz H, Finke J, Zeiser R. Therapy response of glucocorticoid-refractory acute GVHD of the lower intestinal tract. Bone Marrow Transplantation. 2022 |
| 4. | Niederwieser D, Baldomero H, Szer J. (2016) Hematopoietic stem cell transplantation activity worldwide in 2012 and a SWOT analysis of the Worldwide Network for Blood and Marrow Transplantation Group including the global survey. |
| 5. | HRSA Transplant Activity Report, CIBMTR, 2019 |
| 6. | Kurtzberg, J. et al. A Phase 3, Single-Arm, Prospective Study of Remestemcel-L, Ex Vivo Culture-Expanded Adult Human Mesenchymal Stromal Cells for the Treatment of Pediatric Patients Who Failed to Respond to Steroid Treatment for Acute Graft-versus-Host Disease. Biol Blood Marrow Transplant 26 (2020) 845-854 https://doi.org/10.1016/j.bbmt.2020.01.018 |
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 (including any future decision that the FDA in relation to remestemcel-L for with SR-aGVHD), manufacturing activities and product marketing activities, if any; the commercialization of Mesoblast’s 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.
Release authorized by the Chief Executive.
For more information, please contact:
| Corporate Communications / Investors | Media – Global |
| Paul Hughes | Allison Worldwide |
| T: +61 3 9639 6036 | Emma Neal |
| E: investors@mesoblast.com | T: +1 603 545 4843 |
| E: emma.neal@allisonworldwide.com | |
| Media – Australia | |
| Allison Worldwide | |
| Sandie Lane | |
| T: +61 401.996.361 | |
| E:sandie.lane@allisonworldwide.com | |
FAQ
What is the efficacy rate of MESO's RYONCIL in treating SR-aGvHD?
What are the main side effects of RYONCIL reported in clinical trials?
How is RYONCIL administered to patients with SR-aGvHD?
What is the target patient population for MESO's RYONCIL therapy?
What percentage of bone marrow transplant patients might need RYONCIL?
