Keam Susan J
Springer Nature, Private Bag 65901, Mairangi Bay, Auckland, 65901, New Zealand.
Paediatr Drugs. 2023 Jan;25(1):127-133. doi: 10.1007/s40272-022-00543-w.
Ibrutinib (IMBRUVICA), a small molecule inhibitor of Bruton's tyrosine kinase (BTK) developed by Pharmacyclics, Inc. and Janssen Pharmaceutical, is well established as a treatment for B-cell malignancies and is also approved in the USA in adult patients with chronic graft-versus-host disease (cGVHD) and in Japan in adults and adolescents aged ≥ 12 years with cGVHD. Ibrutinib was approved in August 2022 in the USA for use in pediatric patients with cGVHD after failure of one or more lines of systemic therapy and is the first treatment approved for use in this group of patients aged < 12 years (ibrutinib is now indicated for the treatment of adult and pediatric patients aged 1 year and older with cGVHD after failure of one or more lines of systemic therapy). This article summarizes the milestones in the development of ibrutinib leading to this pediatric first approval for the treatment of patients with cGVHD.
伊布替尼（IMBRUVICA）由Pharmacyclics公司和杨森制药公司研发，是一种布鲁顿酪氨酸激酶（BTK）小分子抑制剂，已被广泛确立为B细胞恶性肿瘤的治疗药物，在美国也被批准用于治疗慢性移植物抗宿主病（cGVHD）的成年患者，在日本则被批准用于年龄≥12岁的成年和青少年cGVHD患者。2022年8月，伊布替尼在美国被批准用于一线或多线全身治疗失败后的儿童cGVHD患者，是首个被批准用于这组年龄<12岁患者的治疗药物（伊布替尼现被批准用于一线或多线全身治疗失败后年龄≥1岁的成年和儿童cGVHD患者）。本文总结了伊布替尼研发过程中的里程碑事件，这些事件促成了其在儿科首次获批用于治疗cGVHD患者。
