FDA加速批准Jemperli用于特定直肠癌 部分患者有望免化疗放疗与手术
华盛顿——美国食品药品监督管理局（FDA）10月19日授予 Jemperli（dostarlimab-gxly）加速批准，作为单药用于既往未经治疗的 II 期或 III 期局部晚期直肠癌成人患者，肿瘤须经 FDA 授权检测确认为错配修复缺陷（dMMR）或微卫星高度不稳定（MSI-H）。FDA 表示，这是首个获批用于该人群的免疫疗法，部分患者有望免于化疗、放疗与手术。
批准主要依据 II 期 AZUR-1 试验（NCT05723562）的中期结果：154 名患者接受最长 9 个周期、为期约 6 个月的 Jemperli 治疗。经独立中心评审，71% 的患者仅接受 Jemperli 治疗后无须再接受其他治疗且无疾病进展，持续 12 个月临床完全缓解率（cCR12）为 47%；研究者评估显示，68% 的可评估患者达到 cCR12，探索性分析显示 76% 的可评估患者在治疗一年后无癌症迹象，应答者中位随访逾两年尚未报告疾病复发。GSK 表示，完整数据将于 10 月 25 日在马德里举行的欧洲肿瘤内科学会（ESMO）年会上以最新突破摘要形式公布。
FDA 称，该申请获得优先审评与突破性疗法认定，并通过局长国家优先凭证试点项目审评，审评完成时间较原定目标日期提前近 4 个月。代理局长 Kyle Diamantas 表示，提前近 4 个月完成审评意味着，一些原本可能准备接受手术、放疗或化疗的患者，现在可以与医生展开不同的讨论。FDA 肿瘤卓越中心主任 Angelo de Claro 表示，直肠癌的标准治疗会产生改变人生的影响，对特定类型的直肠癌而言，这项批准提供了避免手术、放疗与化疗的突破性选择；FDA 依据新出现的证据采取行动、让新方法更早抵达患者，同时要求进一步研究确认这些治疗能带来持久获益。
在纪念斯隆凯特琳癌症中心进行的一项更早研究（NCT04165772）中，50 名患者在 Jemperli 治疗后获得有意义且持续的 12 个月临床完全缓解，93% 的患者至少 12 个月保持无癌状态。该中心结直肠癌科主任 Andrea Cercek 表示，AZUR-1 的结果显示，在这一生物标志物界定的人群中，治疗路径有可能发生根本改变。
约 5%–10% 的直肠癌属于 MSI-H/dMMR 肿瘤。目前局部晚期直肠癌的标准治疗通常结合化疗、放疗与手术，可能影响肠道、膀胱与性功能，部分患者需要永久性造口。FDA 提醒，Jemperli 可能引起免疫系统攻击健康器官与组织，常见不良反应包括疲劳、皮疹、腹泻与瘙痒；此次为加速批准，该适应证的持续批准可能取决于后续确证性试验的结果。
FDA Grants Accelerated Approval to Jemperli for Certain Rectal Cancers, Offering Some Patients a Path to Avoid Chemotherapy, Radiation and Surgery
WASHINGTON — The U.S. Food and Drug Administration on October 19 granted accelerated approval to Jemperli (dostarlimab-gxly), used alone, for adults with previously untreated stage II or III locally advanced rectal cancer whose tumors are mismatch repair deficient (dMMR) or microsatellite instability-high (MSI-H), as determined by an FDA-authorized test. The FDA said it is the first immunotherapy approved for this population, and that some patients may be able to avoid chemotherapy, radiation and surgery.
The approval rests mainly on interim results from the phase 2 AZUR-1 trial (NCT05723562), in which 154 patients received up to nine doses of Jemperli over about six months. By independent central review, 71% of patients received dostarlimab alone with no additional treatment and no disease progression, and the sustained 12-month clinical complete response rate (cCR12) was 47%. Investigator assessment showed 68% of evaluable patients achieved cCR12, and an exploratory analysis found 76% of evaluable patients had no evidence of cancer one year or more after treatment; no disease recurrences have been reported among responders after a median follow-up of more than two years. GSK said full interim data will be presented as a late-breaking abstract at the ESMO Congress 2026 in Madrid on October 25.
The application received Priority Review and Breakthrough Therapy designation and was reviewed under the Commissioner's National Priority Voucher pilot, finishing nearly four months ahead of schedule. Acting FDA Commissioner Kyle Diamantas, J.D., said completing the review early means some patients who might otherwise be preparing for surgery, radiation or chemotherapy may now have a different conversation with their doctor. Angelo de Claro, M.D., director of the FDA's Oncology Center of Excellence, said standard treatment for rectal cancer has major life-changing effects, and that for certain types of rectal cancer the approval is a groundbreaking option that avoids surgery, radiation and chemotherapy, adding that the FDA is acting on emerging evidence while requiring further studies to confirm lasting benefit.
In an earlier Memorial Sloan Kettering study (NCT04165772) of 50 patients, dostarlimab produced meaningful and sustained clinical complete responses at 12 months, with 93% of patients maintaining cancer-free status for at least 12 months. Andrea Cercek, M.D., of Memorial Sloan Kettering, said the AZUR-1 results demonstrate the potential for a fundamentally different treatment approach in this biomarker-defined population.
About 5% to 10% of rectal cancers are MSI-H/dMMR tumors. Standard treatment for locally advanced rectal cancer often combines chemotherapy, radiation and surgery, which can affect bowel, bladder and sexual function and sometimes requires a permanent ostomy. The FDA noted that Jemperli can cause the immune system to attack healthy organs and tissues, and that common adverse effects include fatigue, rash, diarrhea and itching. Because this is an accelerated approval, continued approval may depend on confirmatory trial results.
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图片来源：美国食品药品监督管理局（FDA）紧急行动中心（资料照片）。美国食品药品监督管理局摄，公共领域图片，经 Wikimedia Commons 发布。
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