2019年1月15日，美国食品和药物管理局（FDA）授予了一款在研的布鲁顿氏酪氨酸激酶（BTK）抑制剂泽布替尼（Zanubrutinib）突破性疗法认证，用于治疗先前接受至少一次疗法的成年套细胞淋巴瘤（MCL）患者，这也成为首个在FDA获得突破性疗法认定的中国自主研发抗癌新药。介绍了突破性疗法认证的定义，讲述了新型布鲁顿氏酪氨酸激酶抑制剂泽布替尼的特点，阐述了其对中国医药研发企业的意义。
The U.S. Food and Drug Administration (FDA) has granted the investigational BTK inhibitor Zanubrutinib (BGB-3111) a breakthrough therapy designation for the treatment of adult patients with mantle cell lymphoma (MCL) who have previously received at least 1 prior therapy, on Jan. 15th 2019. This paper introduced the definition of the breakthrough therapy designation, the breakthrough achievements of Zanubrutinib, the significance of this event to Chinese pharmaceutical R&D enterprises.
[1] Song Y, Zhou K, Zou D, et al. Safety and activity of the investigational bruton tyrosine kinase inhibitor zanubrutinib (BGB-3111) in patients with mantle cell lymphoma from a phase 2 Trial[J]. Blood, 2018, 132(Suppl 1):148.
[2] Wang M L, Rule S, Martin P, et al. Targeting BTK with ibrutinib in relapsed or refractory mantle-cell lymphoma[J]. New England Journal of Medicine, 2013, 369(6):507-516.
[3] Kaptein A, Bruin G, van Hoek M, et al. Potency and selectivity of BTK inhibitors in clinical development for B-Cell malignancies[J]. Blood, 2018, 132(Suppl 1):1871.
[4] Tam C S, Leblond V, Novotny W, et al. A head-to-head Phase Ⅲ study comparing zanubrutinib versus ibrutinib in patients with Waldenström macroglobulinemia.[J]. Hematological Oncology, 2018, 35(S2):422-423.
[5] Wang M L, Blum K A, Martin P, et al. Long-term follow-up of MCL patients treated with single-agent ibrutinib:Updated safety and efficacy results[J]. Blood, 2015, 126(6):739-745.
