美国、欧盟和日本对淋巴系统肿瘤 CAR-T 细胞与双特异性抗体产品的监管批准

Chimeric antigen receptor (CAR)-T cell and bispecific antibodies (BsAb) have substantially improved outcomes for lymphoid neoplasms (LN); however, a comprehensive analysis regarding the regulatory approval for these products is not available; therefore, we aimed to address this research gap.

We identified all indications for CAR-T cell and BsAb products for LN approved in the United States, European Union, or Japan between January 2010 and September 2023 using public databases. The United States was the most frequent region of first approval for both CAR-T (11/12 [92%]) and BsAb groups (6/9 [67%]). Regulatory incentives, such as orphan designation and expedited reviews, were more common in the CAR-T group than in the BsAb group in the United States, the European Union, and Japan.

Pivotal trials for approval were generally identical in the United States and the European Union, and all indications, excluding the two indications for CAR-T cell products, were approved based on single-arm trials. The proportion of regular approvals was higher in the CAR-T group than in the BsAb group in both the United States (75% vs. 11%) and the European Union (67% vs. 25%). Unlike in the United States and European Union, all indications were granted regular approval in Japan.

In conclusion, most indications for CAR-T cell and BsAb products were approved based on single-arm studies in the United States, European Union, and Japan; however, regulatory incentives and regular approvals were more frequently granted for CAR-T cell products than for BsAb products.