美国食品和药物管理局批准摘要:Selpercatinib用于治疗高级RET融合阳性固体瘤
Elizabeth S Duke1, Diana Bradford1, Michelle Marcovitz1
1Center for Drug Evaluation and Research, U.S. Food and Drug Administration, Silver Spring, Maryland.
概括
美国食品和药物管理局批准了selpercatinib用于具有RET基因融合的先进固体瘤,标志着这些罕见癌症的第一个组织不可知疗法. 这种向治疗显示,除肺癌和甲状腺癌外,在各种瘤类型中,反应率为44%.
科学领域:
- 在瘤学瘤学.
- 遗传学 遗传学 是一个
- 药理学 药理学是指药理学的学科.
背景情况:
- 在转染过程中重新排列的 (RET) 基因融合是各种固体瘤中的致癌驱动因素.
- 针对RET改变的癌症的向疗法已经显示出希望,但广泛的适用性仍然是一个研究领域.
研究的目的:
- 评估塞尔珀卡提尼布在患有RET基因融合的高级固体瘤的成年患者中的疗效和安全性.
- 评估selpercatinib在一系列瘤类型的活性,超出此前批准的适用性.
主要方法:
- 研究LOXO-RET-17001 (LIBRETTO-001) 是一个国际性的,非随机的,多队列的临床试验.
- 患有局部晚期或转移性固体瘤与RET基因融合的患者接受了selpercatinib.
- 瘤类型包括非小细胞肺癌,甲状腺癌和其他罕见的固体瘤.
主要成果:
- 塞尔珀卡提尼布获得了FDA的加速批准,用于先进或转移的RET融合阳性固体瘤的成年患者,这些瘤在先前的治疗中进展.
- 在RET融合阳性固体瘤患者中 (不包括NSCLC和甲状腺癌),整体应答率为44% (95% CI,28-60%).
- 在14种瘤类型中的10种,包括胰腺,结直肠,唾液和卵巢癌,观察到客观反应,反应的中位持续时间为24.5个月.
结论:
- 塞尔珀卡提尼布代表了FDA批准的第一个组织不可知,RET导向的向疗法.
- 这项批准扩大了患有广泛罕见RET融合阳性固体瘤的患者的治疗选择.
- 常见的不良反应包括,腹,疲劳和高血压.
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