FDA批准免疫疗法，或可使特定直肠癌患者免于化疗、放疗和手术
正文 · 中文译文
FDA 新闻发布 FDA批准一种免疫疗法，可能使某些直肠癌患者免于化疗、放疗和手术 更多新闻公告 即时发布： 2026年10月19日 美国食品药品监督管理局（FDA）今日加速批准了免疫疗法Jemperli（dostarlimab-gxly）的一项新用途，该疗法可能使一些局部晚期直肠癌患者免于那些可能对其日常生活产生持久影响的常见治疗。 局部晚期直肠癌患者通常在手术前接受化疗和放疗，这可能会影响肠道、泌尿和性功能，以及生育能力。手术还带有发生严重、有时危及生命的并发症的风险，包括肠管重新连接处的愈合不良和渗漏，以及在部分患者中需行永久性结肠造口。 “FDA致力于缩小科学进步与患者可及性之间的差距，而这一承诺不仅要在政策中体现，还必须体现在我们工作的速度上，”FDA代理局长、法学博士（J.D.）Kyle Diamantas说。“美国患者应当及时获得创新疗法，特朗普政府正在使之成为现实。提前近4个月完成本次审评，意味着一些原本可能正在准备接受手术、放疗或化疗的患者，如今或许可以与他们的医生进行一场不同的对话。” 今天的批准适用于Jemperli单药治疗既往未经治疗的II期或III期局部晚期直肠癌成人患者。在这些分期，癌症已生长至邻近组织或累及邻近淋巴结，但尚未扩散至远处器官。符合条件的要求是，患者的肿瘤必须存在错配修复缺陷（dMMR）或微卫星高度不稳定（MSI-H），且经FDA授权的检测确认。已发表的估计提示，约3%的局部晚期直肠癌为dMMR或MSI-H。 这两个术语描述的是癌细胞在复制DNA时纠正错误过程中存在的缺陷。正常情况下，一种内置的修复系统会捕获并修复这些错误。当该系统功能失常时，错误会累积并产生免疫系统可识别的蛋白质，使这些肿瘤对免疫治疗尤为敏感。 Jemperli的作用机制是阻断癌细胞利用来逃避免疫系统的一种信号。具体而言，它靶向免疫细胞上名为PD-1的蛋白。 “直肠癌的标准治疗具有重大的改变生活的影响。对于某些类型的直肠癌，此次批准代表了一种开创性的选择，可避免手术、放疗和化疗。这对患者及其家属而言是一项显著的获益，”FDA肿瘤卓越中心主任医学博士Angelo de Claro表示。“FDA正依据新兴证据尽快为患者带来新方法，同时要求进一步研究以确认这些治疗能提供持久的获益。” FDA的这项决定基于AZUR-1（NCT05723562）的中期结果，这是一项评估Jemperli单药治疗154例既往未经治疗的dMMR或MSI-H局部晚期直肠癌患者的2期研究。在该研究中，患者接受了最多九个剂量的Jemperli治疗，每3周给药一次，持续6个月。在中位随访超过2年（即至少半数患者已被监测这么长时间）后，近四分之三的患者无需接受手术、放疗或化疗。需要更长时间的随访来了解缓解持续时间以及癌症复发频率。 纪念斯隆凯特琳癌症中心开展的一项更早研究（NCT04165772）评估了Jemperli单药治疗50例既往未经治疗的dMMR或MSI-H局部晚期直肠癌患者。接受Jemperli治疗后，所有患者均未检出癌症迹象，且几乎所有患者（93%）在至少12个月内保持了无癌状态。在随访至少3年的27例患者中，仅1例癌症复发，且复发部位在原发区域。 Jemperli可能导致免疫系统攻击健康器官和组织。这些反应可能严重甚至致命。最常见的不良反应（在至少15%接受该类型直肠癌治疗的患者中报告）为疲劳、皮疹、腹泻和瘙痒。 美国食品药品监督管理局（FDA）授予Jemperli（多塔利单抗）用于治疗dMMR或MSI-H局部晚期直肠癌的申请优先审评资格，并授予其突破性疗法认定。这一举措标志着在局长国家优先券试点项目下审评的药物获得第11次批准，该项目旨在帮助加速审评解决国家公共卫生优先事项的疗法。由于这是基于中期试验结果的加速批准，申办方必须继续对患者进行随访，以证明这些缓解能带来持久的临床获益。 该批准授予葛兰素史克（GSK）。 其他资源 FDA关于Jemperli的公告 媒体： FDA征求意见 202-690-6343 消费者： 888-INFO-FDA FDA是美国卫生与公众服务部下属机构，通过确保人用和兽用药物、疫苗及其他人类用生物制品和医疗器械的安全性、有效性和可靠性来保护公众健康。该机构还负责我国食品供应、化妆品、膳食补充剂、辐射性电子产品以及烟草制品的安全和保障。 ### 当前内容截至： 2026年10月19日 受监管产品 药物 关注FDA 在X平台关注@US_FDA 在Facebook上关注FDA 在X平台关注@FDAMedia
正文 · 英文原文
FDA News Release FDA Approves Immunotherapy That May Allow Certain Patients with Rectal Cancer to Avoid Chemotherapy, Radiation and Surgery More Press Announcements For Immediate Release: October 19, 2026 The U.S. Food and Drug Administration today granted accelerated approval for a new use of the immunotherapy Jemperli (dostarlimab-gxly) that may allow some patients with locally advanced rectal cancer to avoid common treatments that can have a lasting impact on their daily life. Patients with locally advanced rectal cancer often receive chemotherapy and radiation prior to surgery, which can affect bowel, urinary, and sexual function, as well as fertility. Surgery also carries risks of serious, sometimes life-threatening complications, including poor healing and leaks where the bowel is reconnected, and in some patients, permanent colostomy. “The FDA is committed to closing the gap between scientific progress and patient access, and that commitment has to show up not just in policy, but in the pace of our work,” said Acting FDA Commissioner Kyle Diamantas, J.D. “American patients deserve timely access to innovative treatments, and the Trump Administration is making that a reality. Completing this review nearly 4 months ahead of schedule means some patients who might otherwise be preparing for surgery, radiation or chemotherapy may now have a different conversation with their doctor.” Today’s approval is for Jemperli used alone in adults with previously untreated stage II or stage III locally advanced rectal cancer. At these stages, the cancer has grown into nearby tissue or reached nearby lymph nodes but has not spread to distant organs. To be eligible, patients’ tumors must be mismatch repair deficient (dMMR) or microsatellite instability-high (MSI-H), as determined by an FDA-authorized test. Published estimates suggest that approximately 3% of locally advanced rectal cancers are dMMR or MSI-H. Both terms describe a defect in the way cancer cells correct errors when copying their DNA. Normally, a built-in repair system catches and fixes these mistakes. When the system does not work properly, errors accumulate and create proteins the immune system can recognize, making these tumors particularly vulnerable to immunotherapy. Jemperli works by blocking a signal that cancer cells use to shield themselves from the immune system. Specifically, it targets a protein called PD-1 on immune cells. “Standard treatment for rectal cancer has major life-changing effects. For certain types of rectal cancer, this approval represents a groundbreaking option that avoids surgery, radiation and chemotherapy. This represents a significant benefit for patients and their families,” said Angelo de Claro, M.D., director of the FDA’s Oncology Center of Excellence. “The FDA is acting on emerging evidence to bring new approaches to patients sooner, while requiring further studies to confirm that these treatments provide lasting benefits.” The FDA based its decision on interim results from AZUR-1 (NCT05723562), a phase 2 study evaluating Jemperli alone in 154 patients with previously untreated dMMR or MSI-H locally advanced rectal cancer. In the study, patients received up to nine doses of Jemperli, given every 3 weeks over 6 months. After a median follow-up of more than 2 years — meaning at least half of patients had been monitored for that long — almost three-fourths of patients had not needed surgery, radiotherapy or chemotherapy. Longer follow-up is needed to understand the duration of response and how often the cancer returns. An earlier study at Memorial Sloan Kettering Cancer Center (NCT04165772) evaluated Jemperli alone in 50 patients with previously untreated dMMR or MSI-H locally advanced rectal cancer. None of the patients had detectable signs of cancer after Jemperli treatment, and nearly all patients (93%) maintained that cancer-free status for at least 12 months. Of the 27 patients followed for at least 3 years, only one had their cancer return, and it recurred in the same area. Jemperli can cause the immune system to attack healthy organs and tissues. These reactions can be severe or fatal. The most common side effects, reported in at least 15% of patients treated for this type of rectal cancer, were fatigue, rash, diarrhea and itching. The FDA granted Priority Review to the application and Breakthrough Therapy designation to Jemperli for the treatment of dMMR or MSI-H locally advanced rectal cancer. This action marks the 11th approval of a drug reviewed under the Commissioner’s National Priority Voucher pilot program, which aims to help accelerate the review of therapies that address national public health priorities. Because this is an accelerated approval based on interim trial results, the sponsor must continue following patients to show these responses create lasting clinical benefit. The approval was granted to GSK. Additional Resources FDA Jemperli Announcement Media: FDA Request for Comment 202-690-6343 Consumer: 888-INFO-FDA The FDA, an agency within the U.S. Department of Health and Human Services, protects the public health by assuring the safety, effectiveness, and security of human and veterinary drugs, vaccines and other biological products for human use, and medical devices. The agency also is responsible for the safety and security of our nation’s food supply, cosmetics, dietary supplements, radiation-emitting electronic products, and for regulating tobacco products. ### Content current as of: 10/19/2026 Regulated Product(s) Drugs Follow FDA Follow @US_FDA on X Follow FDA on Facebook Follow @FDAMedia on X