Imfinzi approved in the US as the first and only immunotherapy combination for patients with BCG naïve, high risk non muscle invasive bladder cancer

The approval is based on POTOMAC Phase III trial results which showed a 32% reduction in the risk of high risk disease recurrence, progression, or death after one year of Imfinzi added to BCG, compared with BCG alone.

AstraZeneca’s Imfinzi (durvalumab) in combination with Bacillus Calmette-Guérin (BCG) induction and maintenance therapy has secured US approval for the treatment of adult patients with BCG naïve, high risk non muscle invasive bladder cancer (NMIBC). The Food and Drug Administration (FDA) approval is based on positive findings from the POTOMAC Phase III trial, which were presented at the European Society for Medical Oncology (ESMO) Congress 2025 and published simultaneously in The Lancet.

In 2024, more than 31,000 people in the US received treatment for high risk NMIBC, a curative intent setting in which the current standard of care is tumour resection followed by BCG instilled directly into the bladder. Approximately half of patients with NMIBC are classified as high risk for disease recurrence or progression based on cancer characteristics such as tumour grade, stage, and specific tumour features. As many as 80% of high risk patients experience disease recurrence within five years of treatment.

Neal Shore, MD, FACS, Director of START Carolinas / Head of the Carolina Urologic Research Center and co-principal investigator in the trial, said: “The durvalumab plus BCG regimen is the first new therapy approved in over 30 years for patients with BCG-naïve, high-risk non-muscle-invasive bladder cancer. Unfortunately, many of these patients experience disease recurrence requiring repeated surgical procedures, as well as disease progression resulting in surgical removal of their bladder. The POTOMAC trial demonstrates that the durvalumab with BCG induction and maintenance regimen reduces the risk of disease recurrence, progression or death for patients by almost a third compared to BCG alone, heralding a marked advancement for patients with high-risk non-muscle-invasive bladder cancer.”

Dave Fredrickson, Executive Vice President, Oncology Haematology Business Unit, AstraZeneca, said: “Today’s approval for Imfinzi brings the first immunotherapy combination regimen to patients in the US with BCG-naïve, high-risk non-muscle-invasive bladder cancer, an early setting that builds on the positive impact Imfinzi is already having in muscle-invasive disease. The early and sustained disease-free survival benefit demonstrated by Imfinzi plus BCG in the POTOMAC trial is an important advance for patients at risk of early disease recurrence and signals a shift in the standard of care.”

Meri-Margaret Deoudes, CEO of the Bladder Cancer Advocacy Network, said: “It is devastating for patients with high-risk non-muscle-invasive bladder cancer to face the common, early and repeated disease recurrences that are the hallmark of this disease, let alone the prospect of progressing to more advanced disease and life-changing surgeries. New and effective treatment options that address their significant burden are always good news and are urgently needed, so today’s approval could offer meaningful hope for patients and their families.”

The POTOMAC findings showed that adding one year of Imfinzi to BCG induction and maintenance therapy produced a 32% reduction in the risk of high risk disease recurrence, progression, or death in patients with BCG naïve, high risk NMIBC compared with BCG alone (disease free survival hazard ratio 0.68; 95% confidence interval 0.50 to 0.93; p=0.0154). With a median follow up of more than five years (60.7 months), the Imfinzi regimen provided an early and sustained disease free survival benefit, beginning less than four months after the start of treatment. Estimated median disease free survival had not yet been reached in either arm.

The safety and tolerability of Imfinzi plus BCG induction and maintenance therapy was consistent with the known safety profiles of each individual medicine, and no new safety signals emerged after a median follow up of more than five years for disease free survival. The addition of Imfinzi did not affect patients’ ability to complete BCG induction and maintenance therapy, and had no meaningful impact on patient reported quality of life.

Regulatory submissions based on the POTOMAC findings are currently under review in the European Union, Japan, and several other countries.

Earlier in the week, positive high level results were announced from the VOLGA Phase III trial, which showed that perioperative treatment with Imfinzi combined with neoadjuvant enfortumab vedotin (EV) produced statistically significant and clinically meaningful improvements in event free survival (EFS) and overall survival (OS) in patients with muscle invasive bladder cancer (MIBC) who were ineligible for, or had declined, cisplatin based chemotherapy. Perioperative Imfinzi plus Imjudo (tremelimumab) in combination with neoadjuvant EV also delivered a statistically significant and clinically meaningful improvement in event free survival and a favourable trend in overall survival; however, the overall survival data were not statistically significant at this planned interim analysis and will be formally reassessed at a later analysis.

Imfinzi is also approved in several countries for patients with cisplatin eligible MIBC, based on the NIAGARA Phase III trial, and is currently being studied in locally advanced or metastatic disease in the NILE Phase III trial.

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Related Topics and Keywords

AstraZeneca, BCG naïve, bladder cancer, Imfinzi, immunotherapy, non–muscle-invasive bladder cancer