Mesoblast Files Request for Type B Meeting with FDA to Discuss Accelerated Approval Pathway for Revascor® in Ischemic Heart Failure
Mesoblast (MESO) has filed a request for a Type B meeting with the FDA to discuss the accelerated approval pathway for Revascor® in treating ischemic chronic heart failure with reduced ejection fraction (HFrEF) and inflammation. The meeting, expected this quarter, will be held under Mesoblast's Regenerative Medicines Advanced Therapy (RMAT) designation.
In a previous Type B meeting, the FDA indicated Mesoblast's eligibility to file for accelerated approval for Revascor in end-stage HFrEF patients, based on data from two randomized controlled trials. The FDA also specified the requirement for a single confirmatory trial in class II/III patients with ischemic HFrEF and inflammation post-approval.
The upcoming meeting aims to obtain FDA feedback on chemistry, manufacturing & controls (CMC), align on potency assays for commercial product release, and discuss the proposed design and primary endpoint for the confirmatory trial.
Mesoblast (MESO) ha presentato una richiesta per un incontro di Tipo B con la FDA per discutere il percorso di approvazione accelerata per Revascor® nel trattamento dell'insufficienza cardiaca ischemica cronica con frazione di eiezione ridotta (HFrEF) e infiammazione. L'incontro, previsto per questo trimestre, si terrà sotto la designazione di Regenerative Medicines Advanced Therapy (RMAT) di Mesoblast.
In un precedente incontro di Tipo B, la FDA ha indicato l'idoneità di Mesoblast a presentare una richiesta di approvazione accelerata per Revascor nei pazienti con HFrEF in fase terminale, basandosi su dati provenienti da due studi clinici controllati randomizzati. La FDA ha anche specificato il requisito di un singolo studio di conferma nei pazienti di classe II/III con HFrEF ischemica e infiammazione dopo l'approvazione.
L'incontro imminente mira a ottenere un feedback dalla FDA su chimica, produzione e controlli (CMC), allinearsi sugli assay di potenza per il rilascio del prodotto commerciale e discutere il design proposto e l'obiettivo primario per lo studio di conferma.
Mesoblast (MESO) ha presentado una solicitud para una reunión de Tipo B con la FDA para discutir la vía de aprobación acelerada para Revascor® en el tratamiento de la insuficiencia cardíaca crónica isquémica con fracción de eyección reducida (HFrEF) y la inflamación. La reunión, que se espera para este trimestre, se llevará a cabo bajo la designación de Regenerative Medicines Advanced Therapy (RMAT) de Mesoblast.
En una reunión anterior de Tipo B, la FDA indicó la elegibilidad de Mesoblast para solicitar la aprobación acelerada de Revascor en pacientes con HFrEF en etapa terminal, basándose en datos de dos ensayos controlados aleatorios. La FDA también especificó el requisito de un único ensayo de confirmación en pacientes de clase II/III con HFrEF isquémica e inflamación después de la aprobación.
La próxima reunión tiene como objetivo obtener retroalimentación de la FDA sobre química, fabricación y controles (CMC), alinearse en los ensayos de potencia para el lanzamiento del producto comercial y discutir el diseño propuesto y el objetivo primario para el ensayo de confirmación.
Mesoblast (MESO)는 FDA와의 B형 회의 요청서를 제출하여 Revascor®의 승인 가속화 경로에 대해 논의하고 있습니다. 이 약물은 감소된 박출률을 가진 허혈성 만성 심부전(HFrEF) 및 염증 치료에 사용됩니다. 이번 회의는 이번 분기 중에 열릴 예정이며, Mesoblast의 Regenerative Medicines Advanced Therapy (RMAT) 지명 아래 진행됩니다.
이전 B형 회의에서 FDA는 HFrEF 말기 환자에 대한 Revascor의 승인 가속화 요청 자격을 확인했으며, 이는 두 개의 무작위 대조 시험의 데이터를 기반으로 합니다. 또한 FDA는 승인 후 허혈성 HFrEF 및 염증이 있는 II/III 등급 환자를 대상으로 한 단일 확인 시험의 필요성을 명시했습니다.
다가오는 회의는 화학, 제조 및 품질 관리(CMC)에 대한 FDA의 피드백을 얻고, 상업적 제품 출시를 위한 효능 시험에 대해 조율하며, 확인 시험의 제안된 설계 및 주요 목표에 대해 논의하는 것을 목표로 합니다.
Mesoblast (MESO) a déposé une demande de réunion de Type B auprès de la FDA pour discuter du chemin d'approbation accélérée pour Revascor® dans le traitement de l'insuffisance cardiaque chronique ischémique avec fraction d'éjection réduite (HFrEF) et inflammation. La réunion, prévue pour ce trimestre, se tiendra sous la désignation de Regenerative Medicines Advanced Therapy (RMAT) de Mesoblast.
Lors d'une précédente réunion de Type B, la FDA a indiqué l'éligibilité de Mesoblast à demander une approbation accélérée pour Revascor chez les patients en phase terminale de HFrEF, sur la base des données de deux essais contrôlés randomisés. La FDA a également précisé l'exigence d'un essai de confirmation unique chez les patients de classe II/III avec HFrEF ischémique et inflammation après approbation.
La prochaine réunion vise à obtenir des retours de la FDA sur la chimie, la fabrication et les contrôles (CMC), à s'aligner sur les essais de puissance pour le lancement du produit commercial et à discuter du design proposé et de l'objectif principal pour l'essai de confirmation.
Mesoblast (MESO) hat einen Antrag auf ein Typ-B-Meeting bei der FDA eingereicht, um den beschleunigten Genehmigungsweg für Revascor® zur Behandlung von ischämischer chronischer Herzinsuffizienz mit reduzierter Ejektionsfraktion (HFrEF) und Entzündung zu besprechen. Das Treffen, das in diesem Quartal erwartet wird, wird unter der Regenerative Medicines Advanced Therapy (RMAT)-Bezeichnung von Mesoblast stattfinden.
In einem früheren Typ-B-Meeting hat die FDA die Berechtigung von Mesoblast zur Beantragung einer beschleunigten Genehmigung für Revascor bei Patienten mit fortgeschrittener HFrEF bestätigt, basierend auf Daten aus zwei randomisierten kontrollierten Studien. Die FDA hat auch die Anforderung an eine einzige Bestätigungsstudie bei Patienten der Klassen II/III mit ischämischer HFrEF und Entzündung nach der Genehmigung spezifiziert.
Das bevorstehende Meeting zielt darauf ab, Feedback von der FDA zu Chemie, Herstellung und Kontrollen (CMC) zu erhalten, sich auf Potenzassays für die kommerzielle Produktfreigabe abzustimmen und das vorgeschlagene Design sowie den primären Endpunkt für die Bestätigungsstudie zu besprechen.
- FDA previously confirmed eligibility for accelerated approval pathway
- RMAT designation status maintained for Revascor
- Only one confirmatory trial required post-approval
- Additional clinical trial required post-approval
- Still pending FDA feedback on manufacturing and potency assays
- Final approval timeline remains uncertain
NEW YORK, April 02, 2025 (GLOBE NEWSWIRE) -- Mesoblast (Nasdaq:MESO; ASX:MSB), global leader in allogeneic cellular medicines for inflammatory diseases, today provided an update on its plans to meet with the United States Food and Drug Administration (FDA) to discuss the accelerated approval pathway for Revascor® (rexlemestrocel-L) in the treatment of patients with ischemic chronic heart failure with reduced ejection fraction (HFrEF) and inflammation.
Mesoblast filed a request for a Type B meeting during the last quarter, in line with previous guidance, and expects to have the meeting during this quarter. The meeting will be held under Mesoblast’s Regenerative Medicines Advanced Therapy (RMAT) designation for REVASCOR.
In a Type B meeting last year, FDA provided guidance to Mesoblast that the company was eligible to file for accelerated approval of REVASCOR in patients with end-stage HFrEF based on the totality of data across two randomized controlled trials. FDA also guided that a single confirmatory trial in class II/III patients with ischemic HFrEF and inflammation will need to be completed after any accelerated approval is obtained.
The key objectives of the meeting are to obtain FDA feedback on relevant chemistry, manufacturing & controls (CMC), alignment on potency assays for commercial product release, and Mesoblast’s proposed design and primary endpoint for the confirmatory trial.
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 X: @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
What is the current FDA status of Mesoblast's Revascor for heart failure treatment?
What requirements did FDA set for Revascor's accelerated approval pathway?
What will be discussed in Mesoblast's upcoming FDA Type B meeting for Revascor?
What clinical data supports MESO's accelerated approval filing for Revascor?
