FDA批准首个用于治疗某些BCG无反应性膀胱癌患者的疗法
正文 · 中文译文
FDA新闻发布 FDA批准首款治疗特定BCG无应答膀胱癌患者的药物 Anktiva的扩大批准标志着FDA在密切相关的膀胱癌类型中使用证据的新方法 更多新闻公告 立即发布： 2026年10月14日 美国食品药品监督管理局（FDA）今日扩大了Anktiva（nogapendekin alfa inbakicept-pmln）的批准用途，该药物与卡介苗（BCG）联合使用，用于治疗尚未侵犯膀胱肌层且对BCG治疗无应答的高级别纯乳头状膀胱癌成人患者。Anktiva是FDA批准的首款专门针对BCG无应答、高级别纯乳头状病变患者的治疗方案。 此次批准标志着FDA在评估BCG无应答非肌层浸润性膀胱癌（NMIBC）治疗方法时采用了新方法。该机构基于支持先前批准Anktiva用于另一种NMIBC类型（即伴或不伴乳头状肿瘤的原位癌（CIS））成人患者的证据，以及CIS与高级别纯乳头状病变之间的科学相似性，作出了这一决定。 “我们倾听患者、临床医生和研究人员的声音，当科学支持新方法时我们采取行动，”FDA肿瘤卓越中心主任R. Angelo de Claro医学博士表示。“对于Anktiva，我们通过对这些癌症之间相似性的审查，得以在CIS患者的结果基础上进一步扩展，为仅患高级别乳头状肿瘤的患者提供更多治疗选择。这一方法也可能有助于指导未来治疗方法的开发。” 非肌层浸润性膀胱癌（NMIBC）是指尚未侵犯膀胱肌层的膀胱癌，可包括原位癌（CIS）和乳头状肿瘤。原位癌沿膀胱内壁形成扁平斑块，而乳头状肿瘤则从内壁向膀胱腔内生长，呈小的指状突起。原位癌始终为高级别，而乳头状肿瘤可为低级别或高级别。高级别癌症比低级别癌症更可能生长和扩散。 高危非肌层浸润性膀胱癌的治疗通常从卡介苗（BCG）开始，该药可激活膀胱内的免疫系统以对抗癌症。然而，有时癌症会持续存在或复发。加入Anktiva为免疫系统提供了另一种对抗癌症的途径，这种联合治疗可帮助一些仅用卡介苗无法控制癌症的患者。 Anktiva获批用于原位癌的依据是一项试验（NCT03022825），该试验评估了该治疗在77名成人中的有效性，其中部分患者同时伴有乳头状肿瘤。62%的患者达到完全缓解，即在随访评估期间未检测到高级别癌症；在这些患者中，58%维持该缓解至少12个月，40%维持至少24个月。 对于此次扩大批准，FDA在考虑这些结果的同时，也结合了临床和实验室证据，这些证据表明原位癌（CIS）和高级别乳头状肿瘤具有重要共同特征。它们常同时出现，在显微镜下可能难以区分，并且在卡介苗（BCG）治疗不再有效时，具有较高的复发或进展风险。这些相似性支持将Anktiva在CIS患者中的有效性证据应用于仅患有高级别乳头状病变的患者。 为帮助开发者应用这一方法，FDA正在努力敲定其草案指南《卡介苗无反应性非肌层浸润性膀胱癌：开发治疗用药物和生物制品》。该指南旨在包括讨论利用CIS患者研究中的证据，以支持针对BCG无反应性、仅高级别乳头状NMIBC的治疗获批。 Anktiva通过导管与BCG一起直接注入膀胱。每周给药一次，持续六周，后续治疗根据癌症反应而定。治疗可持续长达37个月。如果在第二个六周疗程后癌症仍持续存在、复发或进展，或副作用变得不可接受，则应停止治疗。 最常见的不良反应和实验室异常包括排尿疼痛或尿频、尿急、尿中带血、尿路感染、肌肉或关节疼痛、寒战、发热，以及血肌酐和血钾水平升高。Anktiva的处方信息警告称，延迟切除全部或部分膀胱的手术可能导致癌症扩散至身体其他部位，这可能是致命的。 该批准授予Altor BioScience, LLC。 其他资源 FDA关于Anktiva批准的公告 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。该机构还负责美国食品供应、化妆品、膳食补充剂、放射性电子产品的安全与保障，并监管烟草制品。 ### 内容当前截至： 2026年10月14日 受监管产品 药物 关注FDA 在X上关注@US_FDA 在Facebook上关注FDA 在X上关注@FDAMedia
正文 · 英文原文
FDA News Release FDA Approves First Treatment for Certain People With BCG-Unresponsive Bladder Cancer Expanded approval of Anktiva marks a new FDA approach to using evidence across closely related forms of bladder cancer More Press Announcements For Immediate Release: October 14, 2026 The U.S. Food and Drug Administration today expanded the approved use of Anktiva (nogapendekin alfa inbakicept-pmln), given with Bacillus Calmette-Guérin (BCG), to include adults with high-grade papillary-only bladder cancer that has not spread into the bladder muscle and has not responded to BCG treatment. Anktiva is the first FDA-approved treatment specifically for patients with BCG-unresponsive, high-grade papillary-only disease. The approval marks a new FDA approach to evaluating treatments for BCG-unresponsive non-muscle invasive bladder cancer (NMIBC). The agency based its decision on evidence supporting an earlier approval of Anktiva for adults with another form of NMIBC, carcinoma in situ (CIS), with or without papillary tumors, and scientific similarities between CIS and high-grade papillary-only disease. “We listen to patients, clinicians, and researchers, and we act when the science supports a new approach,” said R. Angelo de Claro, M.D., Director of the FDA’s Oncology Center of Excellence. “For Anktiva, our review of the similarities between these cancers allowed us to build on the results in patients with CIS and expand treatment options for people with high-grade papillary tumors alone. This approach may also help guide the development of future treatments.” NMIBC is bladder cancer that has not grown into the bladder’s muscle layer and can include CIS and papillary tumors. CIS forms flat patches along the bladder’s lining, while papillary tumors grow from the lining into the bladder as small, finger-like projections. CIS is always high-grade, while papillary tumors can be low-grade or high-grade. High-grade cancers are more likely to grow and spread than low-grade cancers. Treatment for high-risk NMIBC often starts with BCG, which activates the immune system inside the bladder to fight cancer. Sometimes, however, the cancer persists or returns. Adding Anktiva gives the immune system another way to fight the cancer, and the combination can help some people whose cancer was not controlled by BCG alone. Anktiva’s CIS approval was supported by a trial (NCT03022825) that evaluated the treatment’s effectiveness in 77 adults, some of whom also had papillary tumors. Sixty-two percent achieved a complete response, meaning no high-grade cancer was detected during follow-up assessments, and of those, 58% maintained that response for at least 12 months and 40% for at least 24 months. For today’s expanded approval, the FDA considered these results alongside clinical and laboratory evidence showing that CIS and high-grade papillary tumors share important features. They often occur together, can be difficult to distinguish under a microscope and carry a high risk of returning or progressing when BCG treatment no longer works. These similarities supported applying the evidence of Anktiva’s effectiveness in people with CIS to those with high-grade papillary-only disease. To help developers apply this approach, the FDA is working toward finalizing its draft guidance, “Bacillus Calmette-Guérin-Unresponsive Nonmuscle Invasive Bladder Cancer: Developing Drugs and Biological Products for Treatment.” The guidance is intended to include discussion of using evidence from studies in patients with CIS to support approval of treatments for BCG-unresponsive, high-grade papillary-only NMIBC. Anktiva is given with BCG directly into the bladder through a catheter. It is given once a week for six weeks, with additional treatment based on how the cancer responds. Treatment may continue for up to 37 months. It should be stopped if the cancer persists after a second six-week course, returns or progresses, or side effects become unacceptable. The most common side effects and laboratory abnormalities included painful or frequent urination, an urgent need to urinate, blood in the urine, urinary tract infections, muscle or joint pain, chills, fever, and increased blood levels of creatinine and potassium. Anktiva’s prescribing information warns that delaying surgery to remove all or part of the bladder can allow the cancer to spread to other parts of the body, which can be fatal. The approval was granted to Altor BioScience, LLC. Additional Resources FDA Anktiva Approval 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: 10/14/2026 Regulated Product(s) Drugs Follow FDA Follow @US_FDA on X Follow FDA on Facebook Follow @FDAMedia on X