Center for Drug Evaluation and Research, U.S. Food and Drug Administration, Silver Spring, Maryland, USA.
Oncology Center of Excellence, U.S. Food and Drug Administration, Silver Spring, Maryland, USA.
Pediatr Blood Cancer. 2022 Aug;69(8):e29602. doi: 10.1002/pbc.29602. Epub 2022 May 13.
In January 2021, the U.S. Food and Drug Administration (FDA) approved crizotinib for pediatric patients 1 year and older and young adults with relapsed or refractory systemic anaplastic large cell lymphoma (sALCL). This is the first approval for pediatric sALCL. Approval was based on a single-arm trial of crizotinib monotherapy that included 26 patients, aged 1-20 years, with previously treated sALCL. Efficacy was based on centrally assessed objective response rate (88%) and duration of response. Herein, we highlight unique aspects of the regulatory review, including extension of the indication to young adults, postmarketing safety, and dose optimization strategies.
2021 年 1 月，美国食品和药物管理局 (FDA) 批准克唑替尼用于 1 岁及以上儿科患者和复发或难治性系统性间变性大细胞淋巴瘤 (sALCL) 的年轻成人。这是儿科 sALCL 的首次批准。批准基于克唑替尼单药治疗的单臂试验，该试验纳入了 26 名年龄在 1-20 岁、先前接受过 sALCL 治疗的患者。疗效基于中心评估的客观缓解率（88%）和缓解持续时间。在此，我们重点介绍监管审查的独特方面，包括将适应症扩展至年轻成人、上市后安全性和剂量优化策略。
