AstraZeneca gets USFDA approval for perioperative Imfinzi for bladder cancer

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.

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

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.”

Dave Fredrickson, Executive Vice President, Oncology Haematology Business Unit, AstraZeneca, said, “The 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.”

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.”

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.

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.

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.

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.

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.