AstraZeneca Plc (LON:AZN) Imfinzi (durvalumab) has been recommended for approval in the European Union (EU) for the treatment of adult patients with resectable muscle-invasive bladder cancer (MIBC) in combination with gemcitabine and cisplatin as neoadjuvant treatment, followed by Imfinzi as monotherapy adjuvant treatment after radical cystectomy (surgery to remove the bladder).
Recommendation based on NIAGARA Phase III trial results which showed a 32% reduction in the risk of recurrence and a 25% reduction in the risk of death for the Imfinzi regimen vs. neoadjuvant chemotherapy alone
The Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency based its positive opinion 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 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 alone (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.
Dr Michiel Van der Heijden, medical oncologist and Group Leader at the Netherlands Cancer Institute, and investigator in the NIAGARA trial, said: “Approximately half of patients with muscle-invasive bladder cancer will experience disease recurrence despite curative-intent treatment with neoadjuvant chemotherapy and surgery to remove the bladder. This recommendation for the durvalumab-based perioperative regimen brings us closer to providing an important new treatment option that has demonstrated a significant survival benefit, with the potential to transform the treatment approach for patients across Europe.”
Susan Galbraith, Executive Vice President, Oncology Haematology R&D, AstraZeneca, said: “The Imfinzi-based perioperative regimen in the NIAGARA Phase III trial enabled survival of more than 80 per cent of patients at two years after treatment. This supports our strategy of moving our innovative medicines into the earlier stages of disease where the opportunity for treatment with curative intent is greatest. If approved, this novel approach will become a much-needed new treatment option for patients in Europe and could become the new standard of care in this setting.”
In 2024, over 35,000 people in the five major European countries 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 Despite this representing a curative-intent setting, where the current standard-of-care is neoadjuvant chemotherapy and radical cystectomy, many patients experience disease recurrence after surgery and have a poor prognosis.3
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 were consistent with the known profile of Imfinzi, manageable and mostly low-grade.
Imfinzi is approved in the US and other countries in this setting based on the NIAGARA results. Regulatory applications are currently under review in Japan and several other countries.
References
- AstraZeneca PLC. Investor Relations Epidemiology Spreadsheet. Available at: https://www.astrazeneca.com/investor-relations.html. Accessed May 2025
- American Cancer Society. What Is Bladder Cancer? Available at: https://www.cancer.org/cancer/bladder-cancer/about/what-is-bladder-cancer.html. Accessed May 2025.
- Witjes JA, et al. EAU Guidelines on muscle-invasive and metastatic bladder cancer. Eur Urol. 2021;1-94.
