近日，由再生元制药研发的Libtayo（cemiplimab-rwlc）静脉注射液获FDA批准用于转移性皮肤鳞状细胞癌（CSCC）或局部晚期CSCC治疗，适用于不能实施治愈性手术或治愈性放疗的患者。这是FDA批准的首个专门用于晚期CSCC的药物。Libtayo通过靶向细胞通路PD-1（在人体免疫细胞及某些癌细胞发现的蛋白质）而发挥作用。该药物通过阻断这一通路，可以帮助人体免疫系统抗击癌细胞。 FDA药物评价与研究中心血液及肿瘤产品办公室代理主任兼FDA肿瘤优化中心主任Pazdur称：我们将继续看到肿瘤学领域朝着识别和开发针对特定分子靶点的药物转变。随着Libtayo的获批，FDA已经批准了6款靶向PD-1/PD-L1通路的免疫检查点抑制剂，用于治疗从膀胱癌到头颈癌等各种肿瘤，到现在可以治疗CSCC。这种肿瘤到了晚期可能难以有效地治疗，重要的是我们继续为患者带来了新的治疗方案。 批准日期：2018年9月29日 公司：Regeneron LIBTAYO（cemiplimab-rwlc）注射液，用于静脉注射 美国最初批准：09/2018 作用机制 PD-1配体PD-L1和PD-L2与T细胞上发现的PD-1受体的结合抑制T细胞增殖和细胞因子产生。PD-1配体的上调在一些肿瘤中发生，并且通过该途径的信号传导可以有助于抑制肿瘤的活性T细胞免疫监视. Cemiplimab-rwlc是一种重组人免疫球蛋白G4（IgG4）单克隆抗体，可与PD-1结合并阻断其与PD-L1和PD-L2的相互作用，释放PD-1通路介导的免疫应答抑制，包括抗肿瘤免疫应答。 Insyngeneic小鼠肿瘤模型，阻止PD-1活性导致肿瘤生长减少。 适应症和用法 LIBTAYO是一种程序性死亡受体-1（PD-1）阻断抗体，用于治疗转移性皮肤鳞状细胞癌患者细胞癌（CSCC）或局部晚期CSCC，不适合手术或治疗性放射治疗。 剂量和给药 LIBTAYO的推荐剂量为350mg静脉输注，每3周30分钟。 剂量形式和强度 注射：350mg/7mL（50mg/mL）溶液在单剂量小瓶中。 禁忌症 没有。 警告和注意事项 严重和致命的免疫介导的不良反应：免疫介导的不良反应可发生在任何器官系统或组织，包括以下：免疫介导的肺炎，免疫介导的结肠炎，免疫介导的肝炎，免疫介导的内分泌疾病，免疫介导的皮肤病学不良反应和免疫介导的肾炎和肾功能不全。 监测免疫介导的不良反应的症状和体征。在治疗期间基线和周期评估临床化学，包括肝脏和甲状腺功能。扣留或永久停用LIBTAYO并根据反应的严重程度给予皮质类固醇。 输注相关反应：中断，减慢输注速度或根据反应的严重程度永久停药。 胚胎-胎儿毒性：可能导致胎儿伤害。建议女性对胎儿的潜在风险和使用有效的感染具有生殖潜力。 不良反应 最常见的不良反应（发生率≥20％）是疲劳，皮疹和腹泻。 用于特定人群 哺乳期： 建议不要母乳喂养。 包装提供/存储和处理 LIBTAYO（cemiplimab-rwlc）注射液是一种透明至微乳白色，无色至淡黄色溶液，可能含有微量半透明至白色颗粒。 它由一个装有1个单剂量小瓶的纸盒供应： 250mg/5mL（50mg/mL）（NDC 61755-007-01） 350mg/7mL（50mg/mL）（NDC 61755-008-01） 储存在原装纸箱中，温度为2°C至8°C（36°F至46°F）的冰箱中。 避光。 不要冷冻或摇晃。 完整资料附件：https://www.accessdata.fda.gov/drugsatfda_docs/label/2018/761097s000lbl.pdf First Treatment for Advanced Cutaneous Squamous Cell Carcinoma FDA-Approved The Food and Drug Administration (FDA) has approved Libtayo (cemiplimab-rwlc; Sanofi and Regeneron), a programmed death receptor-1 (PD-1) blocking antibody, for the treatment of patients with metastatic cutaneous squamous cell carcinoma (CSCC) or locally advanced CSCC who are not candidates for curative surgery or curative radiation. This marks the first FDA approval of a drug specifically indicated for advanced CSCC. The approval was based on data from 2 open-label, non-randomized studies where patients with metastatic (nodal or distant) CSCC or locally advanced CSCC who were not candidates for curative surgery or curative radiation received Libtayo 3mg/kg intravenously every 2 weeks for up to 48 weeks (Study 1423) or up to 96 weeks (Study 1540) until disease progression, unacceptable toxicity, or completion of planned treatment. The primary efficacy outcome measures were confirmed objective response rate (ORR; percentage of patients who experienced partial shrinkage or complete disappearance of their tumor(s) after treatment), as assessed by independent central review (ICR) and ICR-assessed duration of response. Results showed that the confirmed ORR was 47.2% in the combined CSCC group (N=108; 75 with metastatic disease and 33 with locally-advanced disease); 61% of these patients (N=31) had a duration of response ≥6 months. The most common adverse reactions associated with therapy included fatigue, rash, and diarrhea. "With the Libtayo approval, the FDA has approved 6 immune checkpoint inhibitors targeting the PD-1 / PD-L1 pathway for treating a variety of tumors, from bladder to head and neck cancer, and now advanced CSCC,” said Richard Pazdur, MD, director of the FDA's Oncology Center of Excellence and acting director of the Office of Hematology and Oncology Products in the FDA's Center for Drug Evaluation and Research. “This type of cancer can be difficult to treat effectively when it is advanced and it is important that we continue to bring new treatment options to patients.” Libtayo will be supplied as a 350mg/7mL (50mg/mL) solution in a single-dose vial.