On November 7, 2025, the National Medical Products Administration (NMPA) officially approved Puzolcabtagene Autoleucel Injection, an independently developed Class I biological drug, for marketing in China for the treatment for children and adolescents with B-cell acute lymphoblastic leukemia (B-ALL).
This approval marks a historic milestone — the first CAR-T cell therapy in China specifically designed for children and adolescents with relapsed or refractory B-cell acute lymphoblastic leukemia (r/r B-ALL). The therapy employs a fully humanized CD19 single-chain variable fragment (scFv), making it the only humanized CD19 CAR-T product globally approved for pediatric and adolescent use.
Puzolcabtagene Autoleucel not only fills a long-standing clinical gap in China’s pediatric leukemia treatment landscape but also represents a “China solution” to global challenges in childhood cancer care.
Rigorous Clinical Development
The therapy’s clinical program followed a single-arm, open-label design.
The Phase I trial was led by Professor Jianfeng Zhou of Tongji Hospital, Tongji Medical College, Huazhong University of Science and Technology.
The Phase II pivotal trial was co-led by Professor Tianyou Wang from Beijing Children’s Hospital, Capital Medical University, and Professor Yicheng Zhang from Tongji Hospital, with participation from more than ten leading hospitals nationwide.
Humanized Design Enhances Safety and Reduces Immunogenicity
Unlike earlier-generation CAR-T products utilizing murine-derived anti-CD19 scFv, Puzolcabtagene Autoleucel applies humanized antibody engineering optimized for the Chinese population’s MHC reactivity.
This innovation greatly reduces immunogenicity and the risk of anti-drug antibody (ADA) formation, effectively preventing side effects associated with the human anti-mouse antibody (HAMA) response, and extending the therapy’s durability and safety profile — crucial for pediatric patients.
Outstanding Clinical Efficacy
The pivotal Phase II study demonstrated remarkable efficacy in treating pediatric and adolescent r/r B-ALL patients.
As of April 18, 2024, the study included 64 participants with a median follow-up of 211 days. The patient group was characterized by highly refractory and high-risk disease features:
Median age: 11 years (range 3–21)
75% carried at least one high-risk genetic abnormality
56.25% had high tumor burden (≥50% bone marrow blasts)
Despite these challenging conditions, the outcomes were impressive:
Best CR/CRi rate within 3 months: 90.63% (58/64)
Complete remission (CR): 78.13%
CR with incomplete recovery (CRi): 12.5%
MRD-negative rate among CR/CRi patients: 98.27%
3-month sustained CR/CRi rate: 76.56%
Median overall survival (OS): 23.92 months (95% CI: 9.86–not reached)
Expert Perspectives
Professor Tianyou Wang
Principal Investigator, Beijing Children’s Hospital
“For children and adolescents who have exhausted multiple treatment options, Puzolcabtagene Autoleucel opens a new window of life. Its remarkable clinical efficacy gives renewed hope to patients and families facing relapsed leukemia.”
Professor Cheng Qian
Chairman and Chief Scientific Officer of the research team
“The approval of Puzolcabtagene Autoleucel marks a major leap forward for China in the field of advanced cell therapy. This success is the result of years of scientific dedication and innovation, reflecting China’s growing leadership in global biotechnology.”
“With over 300 million RMB invested and the effort of more than 300 researchers, the development journey overcame numerous technological challenges and optimized manufacturing processes thousands of times. We will continue advancing innovative cellular therapies for both hematologic and solid tumors, contributing to the vision of a Healthy China and bringing new hope to patients worldwide.”
A New Era for Pediatric CAR-T Therapy
The approval of Puzolcabtagene Autoleucel represents a turning point in pediatric oncology — demonstrating China’s capability to independently develop world-class cell therapies. With its innovative humanized design, superior safety, and high remission rates, this therapy establishes a new benchmark for treating childhood leukemia and paves the way for future breakthroughs in cellular immunotherapy.
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