FDA批准Komzifti治疗NPM1突变复发难治急性髓系白血病，KOMET-001试验完全缓解率21.4%
Kura Oncology与协和麒麟（Kyowa Kirin）11月13日宣布，美国食品药品管理局（FDA）已完全批准Komzifti（ziftomenib），用于治疗带有易感NPM1突变、无满意替代治疗方案的复发或难治性急性髓系白血病（AML）成人患者。该药是首个且唯一获批的每日一次口服menin抑制剂。
批准依据为关键性KOMET-001试验数据。该试验共纳入112名复发或难治性NPM1突变AML患者，完全缓解（CR）加部分血液学恢复的完全缓解（CRh）率为21.4%（95%置信区间：14.2至30.2），CR/CRh缓解中位持续时间为5个月。两家公司表示，此次批准未附带与QTc延长或尖端扭转型室性心动过速相关的黑框警告。
Komzifti现已在美国上市，可通过有限的特药药房和经销网络购买。相关数据此前已发表于《临床肿瘤学杂志》。
FDA Approves Komzifti for NPM1-Mutated Relapsed or Refractory AML; KOMET-001 CR/CRh Rate 21.4%
Kura Oncology and Kyowa Kirin announced on November 13 that the U.S. Food and Drug Administration (FDA) has granted full approval of Komzifti (ziftomenib) for adult patients with relapsed or refractory acute myeloid leukemia (AML) with a susceptible NPM1 mutation who have no satisfactory alternative treatment options. It is the first and only once-daily oral menin inhibitor approved for this population.
The approval is supported by the pivotal KOMET-001 trial, which enrolled 112 patients with relapsed or refractory NPM1-mutated AML. The rate of complete remission (CR) plus CR with partial hematologic recovery (CRh) was 21.4% (95% CI: 14.2, 30.2), and the median duration of CR/CRh response was 5 months. The companies said the approval carries no boxed warning related to QTc prolongation or Torsades de Pointes.
Komzifti is now commercially available in the United States through a limited network of specialty pharmacies and distributors. The supporting data were previously published in the Journal of Clinical Oncology.
来源：Kura Oncology与协和麒麟
发布：GSM·美国（发布链接）
图片来源：图中是美国食品药品管理局（FDA）位于马里兰州银泉市白橡树园区的62号楼外景。来源：Wikimedia Commons, Public domain, The U.S. Food and Drug Administration。
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