2021年12月10日，美国食品药品监督管理局 (FDA) 扩大了利博西尼的适应症，将其用于治疗激素受体 (HR) 阳性、HER2阴性的晚期或转移性乳腺癌的男性患者。现在，利博西尼以与芳香化酶抑制剂 (AI) 联合应用作为成人患者的初始内分泌治疗，或与富勒酮一起用作初始内分泌治疗，或在内分泌治疗 (ET) 疾病进展后使用。该适应症扩展于绝经后妇女或男性患者。利博西尼+AI治疗男性患者的疗效主要基于前述 MONALEESA-2 和 MONALEESA-7 试验对利博西尼的有利利益-风险评估，并得到 COMPLEEMENT-1 的支持，后者是一项开放标签的、单臂的、多中心临床试验，共有39名男性患者 (n = 3,246名总患者) 接受了利博西尼+来曲唑+高斯莱琳/鹿地肽治疗。基线可测疾病男性患者的确诊反应率 (ORR) 为46.9% (95% CI: 29.1, 65.3)，与总体人群中的 ORR 43.6% (95% CI: 41.5, 45.8) 一致。总体而言，与利博西尼+ET治疗的女性患者相比，治疗男性患者的不良反应相似。利博西尼+富勒酮对男性患者的疗效主要基于 MONALEESA-3 试验有利利益-风险评估的前期发现，并得到FDA对在临床实践中接受利博西尼+富勒酮治疗的少数男性患者的临床数据审核的支持。已知的作用机制、生物学依据和临床信息充分证明利博西尼+AI /富勒酮在男性和女性患者中的疗效和安全性相似。本文总结了FDA针对乳腺癌男性患者批准利博西尼的决策和支持该决策的数据，并讨论了监管的见解。

On December 10, 2021, the FDA expanded the indications for ribociclib to include male patients for the treatment of hormone receptor (HR)-positive, HER2-negative advanced or metastatic breast cancer. Ribociclib is now indicated in combination with an aromatase inhibitor (AI) as initial endocrine-based therapy in adult patients, or with fulvestrant as initial endocrine-based therapy or following disease progression on endocrine therapy (ET), in postmenopausal women or in men. The efficacy of ribociclib+AI for male patients was primarily based on previous favorable benefit-risk assessments of ribociclib from MONALEESA-2 and MONALEESA-7 trials, and supported by COMPLEEMENT-1, an open-label, single arm, multicenter clinical trial, in which 39 male patients (n=3,246 total patients) received ribociclib+letrozole+goserelin/leuprolide. The ORR based on confirmed responses in male patients with measurable disease at baseline was 46.9% (95% CI: 29.1, 65.3), consistent with an ORR 43.6% (95% CI: 41.5, 45.8) in the overall population. Overall, adverse reactions occurring in male patients were similar to those occurring in female patients treated with ribociclib+ET. The efficacy of ribociclib+fulvestrant for male patients was primarily based on the previous findings of a favorable benefit-risk assessment from the MONALEESA-3 trial, supported by FDA review of clinical data of a limited number of male patients treated in clinical practice receiving ribociclib+fulvestrant. The known mechanism of action, biologic rationale, and clinical information available adequately demonstrate that the efficacy and safety of ribociclib+AI/fulvestrant are similar in male and female patients. This article summarizes the FDA's decision-making and data supporting the approval of ribociclib in male patients with breast cancer, and discusses regulatory insights.