Blanc Katarina Le, Dazzi Francesco, English Karen, Farge Dominique, Galipeau Jacques, Horwitz Edwin M, Kadri Nadir, Krampera Mauro, Lalu Manoj Mathew, Nolta Jan, Patel Nikita M, Shi Yufang, Weiss Daniel J, Viswanathan Sowmya
Department of Laboratory Medicine, Karolinska Institutet, Stockholm, Sweden; Department of Cell Therapies and Allogeneic Stem Cell Transplantation, Karolinska University Hospital, Stockholm, Sweden.
School of Cardiovascular and Metabolic Medicine and Sciences, King's College London, London, United Kingdom; AstraZeneca Biopharma R&D, Cambridge, United Kingdom.
Cytotherapy. 2025 Apr;27(4):413-416. doi: 10.1016/j.jcyt.2025.01.005. Epub 2025 Jan 17.
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.
2024年12月，美国食品药品监督管理局（FDA）批准了Mesoblast公司的Ryoncil（remestemcel-L-rknd）——同种异体骨髓间充质基质细胞（MSC(M)）疗法——用于治疗儿童急性类固醇难治性移植物抗宿主病，这终于结束了美国批准的MSC临床产品长期匮乏的局面。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将自体或同种异体MSC产品推向市场，但美国在批准方面一直滞后。通过长达数年的反复过程，申办方与FDA密切合作，解决了有关临床疗效和MSC效力一致性的问题，并付出了巨大努力和投资，最终获得了这一具有里程碑意义的批准。这一批准将重振对MSC产品的投资和热情，推动在主要市场获得更多批准，并将继续预示着MSC作为一种药物长期以来被预测的成功。
