AstraZeneca’s Imfinzi (durvalumab) in combination with gemcitabine and cisplatin as neoadjuvant treatment, followed by Imfinzi as adjuvant monotherapy after radical cystectomy (surgery to remove the bladder) has been approved in the US for the treatment of adult patients with muscle-invasive bladder cancer (MIBC).

阿斯利康的Imfinzi（durvalumab）联合吉西他滨和顺铂作为新辅助治疗，随后以Imfinzi作为 根治性膀胱切除术（切除膀胱的手术）后的辅助单药治疗，已在美国获得批准用于治疗肌层浸润性膀胱癌 (MIBC) 的成年患者。

The approval was granted by the Food and Drug Administration (FDA) after securing Priority Review and was based on results from the NIAGARA Phase III trial which were presented during a Presidential Symposium at the 2024 European Society for Medical Oncology (ESMO) Congress and simultaneously published in The New England Journal of Medicine.

该批准是由美国食品药品管理局（FDA）在获得优先审查后授予的，其基于 NIAGARA 第三阶段试验的结果，该结果在 2024 年欧洲肿瘤内科学会（ESMO）大会的总统研讨会上公布，并同时发表在 《新英格兰医学杂志》上。

In 2024, over 20,000 people in the US were treated for MIBC.1 Bladder cancer is considered muscle-invasive when there is evidence of the tumour invading the muscle wall of the bladder but no distant metastases.2 This represents a curative-intent setting, where the current standard of care is neoadjuvant chemotherapy and radical cystectomy.3 However, even after surgery, patients experience high rates of disease recurrence and have a poor prognosis.

2024 年，美国有超过 20,000 人接受了肌层浸润性膀胱癌治疗。1当有证据表明肿瘤侵入膀胱肌壁但没有远处转移时，膀胱癌被认为是肌层浸润性膀胱癌。2这代表 了一种治愈性治疗，目前的标准治疗是新辅助化疗和根治性膀胱切除术。3然而，即使手术后，患者的疾病复发率也很高，预后较差。

Matthew ND. Galsky, Lillian and Howard Stratton Professor of Medicine, Director of Genitourinary Medical Oncology, The Tisch Cancer Institute at the Icahn School of Medicine at Mount Sinai, New York, and NIAGARA investigator and steering committee member, said: “This approval for the durvalumab-based perioperative regimen is a major breakthrough for people with muscle-invasive bladder cancer, nearly half of whom see their cancer return despite chemotherapy and surgery with curative-intent. This durvalumab regimen significantly extended patients’ lives in the NIAGARA trial and has the potential to transform care.”

纽约西奈山伊坎医学院 Tisch 癌症研究所泌尿生殖医学肿瘤学主任、莉莲和霍华德·斯特拉顿医学教授、NIAGARA 研究员兼指导委员会成员 Matthew ND. Galsky 表示：“此次批准基于 durvalumab 的围手术期治疗方案对肌层浸润性膀胱癌患者来说是一项重大突破，这些患者中近一半的人尽管接受了化疗和手术，但癌症还是复发了。在 NIAGARA 试验中，这种 durvalumab 治疗方案显著延长了患者的生命，并有可能改变治疗方案。”

Dave Fredrickson, Executive Vice President, Oncology Haematology Business Unit, AstraZeneca, said: “Today’s approval for Imfinzi represents a paradigm shift, bringing the first perioperative immunotherapy to patients in the US with muscle-invasive bladder cancer and addressing a significant need for better treatment options. The NIAGARA trial showed more than 80 per cent of patients were still alive at two years, underscoring the potential of this innovative perioperative regimen to become a new standard of care in this setting.”

阿斯利康肿瘤血液学业务部执行副总裁 Dave Fredrickson 表示：“今天Imfinzi获批代表着一种模式转变，为美国肌层浸润性膀胱癌患者带来了首个围手术期免疫疗法，满足了对更好治疗方案的迫切需求。NIAGARA 试验表明，超过 80% 的患者在两年后仍然存活，这凸显了这种创新的围手术期治疗方案有可能成为这种情况下新的治疗标准。”

Meri-Margaret Deoudes, CEO of the Bladder Cancer Advocacy Network, said: “More than 20,000 people in the US were treated for muscle-invasive bladder cancer last year and there is a significant need for new treatment options that improve patient outcomes. The approval of the durvalumab perioperative regimen is welcome news, transforming how clinicians will tackle this disease in future and offering new hope to patients and their loved ones.”

膀胱癌宣传网络首席执行官 Meri-Margaret Deoudes 表示：“去年，美国有超过 20,000 人因肌层浸润性膀胱癌接受治疗，迫切需要新的治疗方案来改善患者的治疗效果。durvalumab 围手术期治疗方案的获批是一个好消息，它改变了临床医生未来治疗这种疾病的方式，并为患者及其亲人带来了新的希望。”

In the trial, patients were treated with four cycles of Imfinzi in combination with neoadjuvant chemotherapy before radical cystectomy followed by eight cycles of Imfinzi monotherapy, or neoadjuvant chemotherapy before radical cystectomy. In a planned interim analysis, the Imfinzi-based perioperative regimen demonstrated a 32% reduction in the risk of disease progression, recurrence, not undergoing surgery, or death versus the comparator arm (based on event-free survival [EFS] hazard ratio [HR] of 0.68; 95% confidence interval [CI] 0.56-0.82; p<0.0001). Estimated median EFS was not yet reached for the Imfinzi arm versus 46.1 months for the comparator arm. An estimated 67.8% of patients treated with the regimen were event free at two years compared to 59.8% in the comparator arm.

在试验中，患者在根治性膀胱切除术前接受 4 个周期的 Imfinzi联合新辅助化疗治疗，随后接受 8 个周期的 Imfinzi 单药治疗，或接受根治性膀胱切除术前的新辅助化疗。在计划的中期分析中，与对照组相比，以Imfinzi为基础的 围手术期方案显示疾病进展、复发、未接受手术或死亡的风险降低了 32%（基于无事件生存期 [EFS] 风险比 [HR] 为 0.68；95% 置信区间 [CI] 0.56-0.82；p<0.0001）。Imfinzi 组的估计中位 EFS 尚未达到， 而 对照组为 46.1 个月。据估计，接受该方案治疗的患者中有 67.8% 在两年内无事件发生，而对照组这一比例为 59.8%。

Results from the key secondary endpoint of overall survival (OS) showed that the Imfinzi-based perioperative regimen reduced the risk of death by 25% versus neoadjuvant chemotherapy with radical cystectomy (based on OS HR of 0.75; 95% CI 0.59-0.93; p=0.0106). Median survival was not yet reached for either arm. An estimated 82.2% of patients treated with the regimen were alive at two years compared to 75.2% in the comparator arm.

关键次要终点总生存期 (OS) 结果显示， 与根治性膀胱切除术的新辅助化疗相比，以Imfinzi为基础的围手术期方案可将死亡风险降低 25%（基于 OS HR 为 0.75；95% CI 0.59-0.93；p=0.0106）。两组的中位生存期均未达到。据估计，接受该方案治疗的患者两年内存活率为 82.2%，而对照组为 75.2%。

Imfinzi was generally well tolerated, and no new safety signals were observed in the neoadjuvant and adjuvant settings. Further, adding Imfinzi to neoadjuvant chemotherapy was consistent with the known profile for this combination and did not compromise patients’ ability to complete surgery compared to neoadjuvant chemotherapy alone. Immune-mediated adverse events (imAEs) were consistent with the known profile of Imfinzi, manageable and mostly low-grade.

Imfinzi总体耐受性良好，在新辅助和辅助治疗中未观察到新的安全信号。此外，将Imfinzi添加到新辅助化疗中符合这种组合的已知特性，并且与单独进行新辅助化疗相比，不会损害患者完成手术的能力。免疫介导不良事件 (imAE) 与Imfinzi的已知特性一致，可控且大多为低级别。

In February 2025, perioperative treatment with durvalumab (Imfinzi), neoadjuvant cisplatin-based chemotherapy and cystectomy was added to the NCCN Clinical Practical Guidelines in Oncology (NCCN Guidelines®) as a NCCN Category 1 Recommended regimen for patients with MIBC based on the data from NIAGARA.

2025 年 2 月，基于 NIAGARA 的数据，使用度伐单抗 ( Imfinzi ) 进行围手术期治疗、以顺铂为基础的新辅助化疗和膀胱切除术被添加到《NCCN 肿瘤学临床实用指南》（NCCN 指南®）中，作为 MIBC 患者的 NCCN 1 类推荐方案。

Imfinzi is also approved in Brazil in this setting based on the NIAGARA results. Regulatory applications are currently under review in the EU, Japan and several other countries.

根据 NIAGARA 试验结果， Imfinzi也已在巴西获得批准。欧盟、日本和其他几个国家目前正在审查其监管申请。