美国食品药品监督管理局（FDA）于周三加速批准了首款针对转移性胰腺癌的创新靶向药物daraxonrasib（商品名Rasonque）123。该药物由Revolution Medicines销售，是一种每日口服一次的RAS抑制剂123。它通过阻断在超过90%的胰腺癌病例中驱动肿瘤生长的突变蛋白来发挥作用12。该药适用于既往接受过至少一次系统治疗，或不适合多药联合系统治疗的成人转移性胰腺导管腺癌患者3。
临床试验结果显示，接受daraxonrasib治疗的转移性胰腺癌患者中位总生存期从标准化疗组的6.7个月显著提升至13.2个月，且严重副作用更少123。这项研究共招募了500名转移性胰腺癌患者12。由于该药在高度未满足需求领域展示了前所未有的结果，FDA授予了其突破性疗法和孤儿药资格，并给予优先审评3。最终，FDA比原定目标日期提前了六个多月（具体为6.5个月）批准了该药物123。
胰腺癌五年总体生存率仅为13%，美国癌症协会估计今年美国将诊断约67000例新病例，超过52000人将死于该疾病12。其中90%至95%的病例为胰腺导管腺癌3。FDA代理局长Kyle Diamantas表示：“我们的基本职责是尽快为患者提供更多治愈方法和有意义的治疗”12。他同时强调，该批准为面临极难治疗癌症的患者提供了关键新选择3。FDA肿瘤卓越中心主任Angelo de Claro也称赞该药在高度未满足需求领域展示了前所未有的结果3。
On Wednesday, the U.S. Food and Drug Administration (FDA) approved Rasonque (daraxonrasib), a first-in-class targeted therapy for metastatic pancreatic ductal adenocarcinoma 13. The oral, once-daily RAS inhibitor is the first innovative drug designed to block the mutated protein that drives tumor growth in over 90% of pancreatic cancer cases 123. Marketed by Revolution Medicines 123, the medication was also granted breakthrough therapy and orphan drug designations, along with priority review 3.
In a clinical trial involving 500 patients with metastatic pancreatic cancer, Rasonque extended the median overall survival to 13.2 months, compared to 6.7 months for the standard chemotherapy group, while also resulting in fewer severe side effects 123. The FDA approved the drug 6.5 months ahead of its user fee deadline, which is more than six months earlier than the original target date 123. The treatment is specifically indicated for adult patients who have previously received at least one systemic treatment or who are not candidates for multi-agent systemic therapy 3.
Pancreatic cancer has a five-year overall survival rate of just 13%, with the American Cancer Society estimating approximately 67,000 new diagnoses and over 52,000 deaths in the U.S. this year 123. Of these cases, 90% to 95% are pancreatic ductal adenocarcinoma 3. FDA Acting Director Kyle Diamantas stated, "Our fundamental duty is to provide patients with more cures and meaningful treatments as quickly as possible," and added that the approval provides a critical new option for patients facing a very hard-to-treat cancer 13. Additionally, Angelo de Claro, Director of the FDA's Oncology Center of Excellence, highlighted that the drug has demonstrated unprecedented results in an area of high unmet need 3.
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