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ISCT Hails Landmark US FDA Approval of RYONCIL as Major Milestone for MSC Field

ISCT Hails Landmark US FDA Approval of RYONCIL as Major Milestone for MSC Field

ISCt 歡迎美國 FDA 批准 RYONCIL,認爲這是 MSC 領域的重要里程碑
PR Newswire ·  2024/12/20 19:00

VANCOUVER, BC, Dec. 20, 2024 /PRNewswire/ - The International Society for Cell & Gene Therapy (ISCT) celebrates the US Food and Drug Administration's (FDA) landmark decision to approve Mesoblast Limited's allogenic, bone marrow-derived mesenchymal stromal cell (MSC) product, RYONCIL (remestemcel-L) for pediatric, steroid-refractory, acute Graft-versus-Host-Disease (GvHD). This decision is a watershed moment for the MSC field, marking the first approval of an MSC therapy in the US.
Around 50% of pediatric patients receiving an allogeneic bone marrow transplant develop acute GvHD, where transplanted cells recognize the host body as foreign and attack the patient's tissues and organs. Half of these patients do not respond to first-line steroid treatment. RYONCIL will now provide a clinically effective option to meet the significant mortality and life-changing impacts of steroid-refractory acute GvHD.

溫哥華,BC,2024年12月20日 /PRNewswire/ - 國際電芯與基因治療學會(ISCT)慶祝美國食品藥品監督管理局(FDA)做出歷史性決定,批准Mesoblast Limited的同種異體、骨髓來源的間充質基質細胞(MSC)產品RYONCIL(remestemcel-L)用於兒童類固醇耐藥型急性移植物抗宿主病(GvHD)。這一決定標誌着MSC領域的一個重要時刻,標誌着在美國首次批准MSC治療。
約50%的接受同種異體骨髓移植的兒童患者會發展爲急性GvHD,在這種情況下,移植的細胞將宿主身體視爲外來物並攻擊患者的組織和器官。這些患者中有一半對一線類固醇治療沒有反應。RYONCIL現在將提供一種臨牀有效的選擇,以應對類固醇耐藥型急性GvHD所導致的重大死亡率和生活變化影響。

Sowmya Viswanathan, PhD, ISCT MSC Committee Chair, spoke to the patient perspective, "The approval of RYONCIL addresses an urgent and unmet need for pediatric patients. Acute GvHD is a painful condition, and as organ failure spreads, quality of life deteriorates rapidly. This approval is a victory for patients and families who can now look towards a viable treatment option against a debilitating condition."

Sowmya Viswanathan博士,ISCT MSC委員會主席,談到了患者的觀點,"RYONCIL的批准回應了兒童患者迫切的未滿足需求。急性GvHD是一種痛苦的病症,隨着器官衰竭的加劇,生活質量迅速惡化。這一批准是患者和家庭的勝利,他們現在可以期待一種有效的治療方案來應對這一致殘病症。"

Mesoblast Limited secured the approval on their third Biologics License Application (BLA) resubmission, working closely with the US FDA to address concerns after initial submissions were rejected in 2020 and 2023 respectively. Clinical efficacy was based on a single-arm, 54 patient, Phase III trial (NCT02336230) using a day 28 overall response, which was 70.4% compared to preset control group of 45%.

Mesoblast Limited在提交其第三份生物製品許可證申請(BLA)時獲得了批准,與美國FDA密切合作以解決2020年和2023年初始提交被拒後的疑慮。臨牀療效基於一項單臂、54名患者、III期試驗(NCT02336230),使用第28天的總體反應,70.4%對比預設對照組的45%。

"While MSC products first arrived to market over twelve years ago in other jurisdictions, this first approval in the United States is of historical importance," noted Jacques Galipeau, MD, ISCT Immediate Past President, "it rewards the work of researchers, clinicians, and innovators around the globe who have dedicated their careers to this field."

"雖然MSC產品在其他司法轄區早在十二年前就已上市,但在美國的首次批准具有歷史意義," ISCT往屆主席Jacques Galipeau博士指出,"這證明了全球研究人員、臨牀醫生和創新者在這一領域辛勤工作的成果。"

Speaking on this important approval, Miguel Forte, MD, PhD, ISCT President, stated "ISCT commends the resilient efforts that the team at Mesoblast has put into acquiring regulatory approval through hard-won proof of clinical efficacy. This is a victory for the field, as enthusiasm and investment towards research and development of MSC therapies will be reignited. We may look back on this approval as a pivotal moment in the history of medicine, shaping the future of therapeutics."

在談論這一重要的批准時,Miguel Forte醫學****,表示:「ISCt讚揚Mesoblast團隊在通過艱苦的臨牀有效性證明獲得監管批准方面所付出的堅韌努力。這對該領域來說是一個勝利,因爲對MSC治療研究和開發的熱情與投資將重新燃起。我們將可能把這一批准視爲醫學歷史上的一個關鍵時刻,塑造未來的治療手段。」

About the International Society for Cell & Gene Therapy

關於國際細胞與基因治療學會

Established in 1992, the International Society for Cell & Gene Therapy (ISCT) is a global society of clinicians, regulators, researchers, technologists, and industry partners with a shared vision to translate cell and gene therapy into safe and effective treatments to improve lives worldwide.

國際細胞與基因治療學會(ISCT)成立於1992年,是一個全球性的學會,彙集了臨牀醫生、監管機構、研究人員、技術人員和行業合作伙伴,共同致力於將細胞和基因治療轉化爲安全有效的治療方法,以改善全球人們的生活。

ISCT is the global leader focused on pre-clinical and translational aspects of developing cell and gene-based therapeutics, thereby advancing scientific research into innovative treatments for patients. ISCT offers a unique collaborative environment that addresses three critical areas of translation: Academia, Regulatory, and Commercialization. Through solid relationships with global regulatory agencies, academic institutions, and industry partners, ISCT drives the advancement of research into a standard of care.

ISCt是全球領先的機構,專注於開發基於細胞和基因的治療方案的前臨牀和轉化方面,從而推動科學研究向創新患者治療的進展。ISCt提供獨特的協作環境,解決轉化的三個關鍵領域:學術、監管和商業化。通過與全球監管機構、學術機構和行業合作伙伴建立牢固的關係,ISCt推動研究進展到護理標準。

Comprising over 4,000 cell and gene therapy experts across five geographic regions and with representation from over 60 countries, ISCT members are part of a global community of peers, thought leaders, and organizations invested in cell and gene therapy translation. For more information about the society, key initiatives, and upcoming meetings, please visit , @ISCTglobal.

ISCt成員由來自五個地理區域的4,000多名細胞和基因治療專家組成,覆蓋60多個國家的代表,是一個投資於細胞和基因治療轉化的全球社區,成員包括同行、思想領袖和組織。有關該學會、關鍵倡議和即將召開的會議的更多信息,請訪問, @ISCTglobal.

SOURCE International Society for Cell & Gene Therapy

來源:國際細胞與基因治療學會

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