ISCT MSC委员会关于美国FDA批准同种异体骨髓间充质基质细胞的声明。

IF 3.7 3区医学 Q2 BIOTECHNOLOGY & APPLIED MICROBIOLOGY

Cytotherapy Pub Date : 2025-01-17 DOI:10.1016/j.jcyt.2025.01.005

Katarina Le Blanc , Francesco Dazzi , Karen English , Dominique Farge , Jacques Galipeau , Edwin M. Horwitz , Nadir Kadri , Mauro Krampera , Manoj Mathew Lalu , Jan Nolta , Nikita M. Patel , Yufang Shi , Daniel J. Weiss , Sowmya Viswanathan

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引用次数: 0

摘要

2024年12月，美国食品和药物管理局（FDA）批准了Mesoblast公司的Ryoncil (remestemcell -l - rkind)-同种异体骨髓间充质基质细胞（MSC(M)）治疗儿科急性类固醇难治性移植物抗宿主病，最终结束了美国MSC临床产品的长期干旱。虽然包括欧洲、日本、印度和韩国在内的其他司法管辖区已经销售了自体或同种异体MSC产品，但美国在批准方面却落后了。发起人的重大努力和投资，与FDA密切合作，通过跨越数年的迭代过程解决有关临床疗效和一致的MSC效力的问题，获得了这一里程碑式的批准。这一批准将重振对MSC产品的投资和热情，进一步在主要市场获得批准，并将继续预示着MSC作为一种药物的长期成功。

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ISCT MSC committee statement on the US FDA approval of allogenic bone-marrow mesenchymal stromal cells

The December 2024 US Food and Drug Administration (FDA) approval of Mesoblast's Ryoncil (remestemcel-L-rknd)—allogeneic bone marrow mesenchymal stromal cell (MSC(M)) therapy—in pediatric acute steroid-refractory graft-versus-host-disease finally ended a long-lasting drought on approved MSC clinical products in the United States. While other jurisdictions—including Europe, Japan, India, and South Korea—have marketed autologous or allogeneic MSC products, the United States has lagged in its approval. The sponsor's significant efforts and investments, working closely with the FDA addressing concerns regarding clinical efficacy and consistent MSC potency through an iterative process that spanned several years, was rewarded with this landmark approval. This approval will revive investment and enthusiasm in MSC products, further approvals in major markets, and will continue to foreshadow the long-predicted success of MSCs as a pharmaceutical.

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来源期刊

Cytotherapy 医学-生物工程与应用微生物

CiteScore

6.30

自引率

4.40%

发文量

683

审稿时长

49 days

期刊介绍： The journal brings readers the latest developments in the fast moving field of cellular therapy in man. This includes cell therapy for cancer, immune disorders, inherited diseases, tissue repair and regenerative medicine. The journal covers the science, translational development and treatment with variety of cell types including hematopoietic stem cells, immune cells (dendritic cells, NK, cells, T cells, antigen presenting cells) mesenchymal stromal cells, adipose cells, nerve, muscle, vascular and endothelial cells, and induced pluripotential stem cells. We also welcome manuscripts on subcellular derivatives such as exosomes. A specific focus is on translational research that brings cell therapy to the clinic. Cytotherapy publishes original papers, reviews, position papers editorials, commentaries and letters to the editor. We welcome "Protocols in Cytotherapy" bringing standard operating procedure for production specific cell types for clinical use within the reach of the readership.