The BCMA-targeted chimeric antigen receptor (CAR) T cell therapy ciltacabtagene autoleucel (cilta-cel) has demonstrated exceptional efficacy in studies conducted worldwide, which has resulted in regulatory approvals in >40 countries. Herein, we examine the regulatory pathways that led to its approval in different regions, focus on challenges in clinical development and regulatory submission, and provide insight into strategies for advancing innovative cell therapies.
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Acknowledgements
The authors sincerely thank the teams from the Center for Drug Evaluation at the NMPA for their valuable input in discussing the regulatory aspects of CAR T cell therapies in China. The authors also thank the teams from Legend Biotech and Johnson & Johnson, who helped to ensure the accuracy of the content regarding the development process and the timeline of the regulatory submission history. We also thank Pharmcube for providing access to their database, which was instrumental in gathering relevant product information.
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Ge, C., Yin, C. & Chen, X. International approvals of cilta-cel: a lens on CAR T cell regulation. Nat Rev Clin Oncol 22, 233–234 (2025). https://doi.org/10.1038/s41571-025-00996-1
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DOI: https://doi.org/10.1038/s41571-025-00996-1
