AstraZeneca’s Imfinzi Gains CHMP Backing as First Perioperative Immunotherapy for Muscle-Invasive Bladder Cancer in the EU
AstraZeneca has announced a significant regulatory milestone for its cancer immunotherapy, Imfinzi (durvalumab), with the drug receiving a positive recommendation from the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA). The CHMP has endorsed Imfinzi for approval in the European Union (EU) as a perioperative treatment for adult patients with resectable muscle-invasive bladder cancer (MIBC). If authorized, Imfinzi would become the first and only immunotherapy approved in the EU for use both before and after surgery in this patient population.
This recommendation covers the use of Imfinzi in combination with standard chemotherapy agents—gemcitabine and cisplatin—as neoadjuvant (pre-surgical) therapy, followed by Imfinzi monotherapy as adjuvant (post-surgical) treatment after radical cystectomy, a procedure involving complete bladder removal. The CHMP’s opinion is based on robust data from the pivotal Phase III NIAGARA trial, which demonstrated significant improvements in both event-free survival and overall survival among patients treated with this novel perioperative regimen.
Landmark Data from the NIAGARA Phase III Trial
The NIAGARA study was designed to evaluate the efficacy and safety of Imfinzi in the perioperative setting for patients with resectable MIBC. The results were unveiled 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 prespecified interim analysis, Imfinzi in combination with gemcitabine and cisplatin, followed by monotherapy with Imfinzi, demonstrated a 32% reduction in the risk of disease progression, recurrence, lack of surgery, or death compared to neoadjuvant chemotherapy followed by surgery alone. This was measured through the trial’s primary endpoint, event-free survival (EFS), yielding a hazard ratio (HR) of 0.68 (95% confidence interval [CI]: 0.56–0.82; p<0.0001). Notably, the median EFS for the Imfinzi-treated group had not yet been reached at the time of analysis, compared to 46.1 months in the control group. At the two-year mark, 67.8% of patients in the Imfinzi arm remained event-free, versus 59.8% in the control arm.
The trial also met its key secondary endpoint of overall survival (OS). The Imfinzi regimen led to a 25% reduction in the risk of death compared to neoadjuvant chemotherapy and surgery alone (OS HR: 0.75; 95% CI: 0.59–0.93; p=0.0106). Median OS had not been reached in either treatment group at the time of analysis. At two years, 82.2% of patients receiving the Imfinzi regimen were still alive, in contrast to 75.2% of patients in the comparator arm.
A Transformative Shift in MIBC Treatment
Dr. Michiel Van der Heijden, a medical oncologist and Group Leader at the Netherlands Cancer Institute and one of the NIAGARA trial investigators, emphasized the clinical importance of these findings.
“Approximately half of patients with muscle-invasive bladder cancer will experience disease recurrence despite curative-intent treatment with neoadjuvant chemotherapy and radical cystectomy,” he noted. “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.”
Bladder cancer becomes classified as muscle-invasive when the tumor infiltrates the muscular wall of the bladder but has not yet spread to distant organs. This form of the disease is aggressive, and while surgery and chemotherapy are often employed with curative intent, the risk of recurrence remains high. According to AstraZeneca, in 2024 alone, more than 35,000 patients across five major European markets were treated for MIBC, underscoring the urgent need for more effective treatment strategies.
AstraZeneca’s Commitment to Early-Stage Cancer Treatment
Susan Galbraith, Executive Vice President of Oncology R&D at AstraZeneca, reinforced the strategic significance of this development. “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,” she said. “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.”
Imfinzi’s potential approval in Europe would mark another milestone in AstraZeneca’s efforts to reshape cancer care through earlier intervention. The company has long maintained that treating cancer at earlier stages increases the likelihood of cure, and this CHMP recommendation further strengthens its position in the rapidly evolving field of immuno-oncology.
Safety Profile and Tolerability
Safety was a key component of the NIAGARA study, and the data show that Imfinzi was generally well tolerated in both the neoadjuvant and adjuvant settings. Importantly, the addition of Imfinzi to chemotherapy did not hinder patients’ ability to undergo bladder surgery—a critical consideration in the management of MIBC.
There were no new safety signals identified, and immune-mediated adverse events associated with Imfinzi were consistent with its established safety profile. These events were mostly low-grade and manageable, reinforcing the feasibility of incorporating the drug into routine clinical practice for perioperative use.
Regulatory Path Forward
Imfinzi has already gained approval in the United States and other countries for use in this setting, based on the NIAGARA trial results. Regulatory submissions are under active review in Japan and multiple additional countries worldwide, as AstraZeneca pursues a global strategy to establish Imfinzi as the first perioperative immunotherapy standard in MIBC.
This latest CHMP recommendation is a crucial step toward Imfinzi’s potential formal approval by the European Commission, which typically follows the CHMP’s advice within a few months. If granted, this authorization will expand Imfinzi’s indications in the EU and could offer new hope to thousands of patients facing this high-risk form of bladder cancer.
Imfinzi (durvalumab) is a human monoclonal antibody that binds to PD-L1, blocking its interaction with PD-1 and CD80, thereby enhancing the immune system’s ability to detect and destroy cancer cells. Imfinzi is part of AstraZeneca’s leading immunotherapy portfolio and has been approved in various indications, including non-small cell lung cancer, biliary tract cancer, and small cell lung cancer.
By extending its use into the perioperative treatment of MIBC, AstraZeneca aims to further solidify Imfinzi’s role as a cornerstone of immuno-oncology treatment strategies—especially in cancers with historically limited options in early-stage settings.
If the European Commission grants final marketing authorization, Imfinzi could become a game-changing addition to the standard of care for MIBC in Europe. The introduction of an immunotherapy-based perioperative regimen has the potential to significantly improve survival outcomes, reduce recurrence, and offer a better quality of life for patients battling this aggressive disease.
The CHMP’s recommendation represents a validation of the NIAGARA trial’s groundbreaking data and underscores the growing trend toward immunotherapy integration in earlier lines of cancer treatment. As regulatory bodies around the world continue to assess the NIAGARA data, patients and clinicians alike may soon have access to an innovative, evidence-based approach that reshapes expectations in muscle-invasive bladder cancer care.
