Vorasidenib (VORANIGO®；施维雅) 是一种口服、一流、高度脑渗透性的突变异柠檬酸脱氢酶 1 和 2 (IDH1/2) 双重抑制剂，正在开发用于 IDH 突变弥漫性神经胶质瘤。 Vorasidenib 于 2024 年 8 月 6 日在美国首次获得批准，用于治疗手术（包括活检、次全切除）后患有易感 IDH1 或 IDH2 突变的 2 级星形细胞瘤或少突胶质细胞瘤的成人和 12 岁以上儿童患者，或全切除。批准基于多国 III 期 INDIGO 试验的结果，其中与安慰剂相比，vorasidenib 显着改善了无进展生存期和下次抗癌干预的时间。在欧盟和世界其他国家，针对 IDH 突变神经胶质瘤的 vorasidenib 的监管审查目前正在进行中。本文总结了 vorasidenib 开发过程中的里程碑，导致首次批准用于神经胶质瘤。© 2024。作者，获得 Springer Nature Switzerland AG 的独家许可。

Vorasidenib (VORANIGO®; Servier) is an orally administered, first-in-class, highly brain-penetrant dual inhibitor of mutant isocitrate dehydrogenase 1 and 2 (IDH1/2) enzymes being developed for use in IDH-mutant diffuse glioma. Vorasidenib received its first approval on 6 August 2024, in the USA, for the treatment of adult and pediatric patients aged 12 years and older with grade 2 astrocytoma or oligodendroglioma with a susceptible IDH1 or IDH2 mutation following surgery, including biopsy, sub-total resection, or gross total resection. Approval was based on results from the multinational phase III INDIGO trial, in which vorasidenib significantly improved progression-free survival and time to the next anticancer intervention relative to placebo. In the EU and other countries worldwide, regulatory review of vorasidenib in IDH-mutant glioma is currently underway. This article summarizes the milestones in the development of vorasidenib leading to this first approval for glioma.© 2024. The Author(s), under exclusive licence to Springer Nature Switzerland AG.