AstraZeneca’s IMFINZI (durvalumab), in combination with Bacillus Calmette-Guérin (BCG) induction and maintenance therapy, has been approved in the US for the treatment of adult patients with BCG-naïve, high-risk Non-Muscle-Invasive Bladder Cancer (NMIBC).

The approval by the Food and Drug Administration (FDA) is based on positive results from the POTOMAC phase 3 trial which were presented at the European Society for Medical Oncology (ESMO) Congress 2025 and simultaneously published in The Lancet.

In 2024, more than 31,000 people in the US were treated for high-risk NMIBC, a curative-intent setting where the standard of care is tumor resection followed by BCG treatment directly into the bladder. About half of patients with NMIBC are at high-risk for disease recurrence or progression based on certain characteristics of their cancer, such as tumor grade, stage and specific tumor features. Up to 80 percent of high-risk patients experience disease recurrence within 5 years of treatment.

Neal Shore, MD, FACS, Director, 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.”

Results from the POTOMAC trial showed adding 1 year of treatment with IMFINZI to BCG induction and maintenance therapy demonstrated a 32 percent reduction in the risk of high-risk disease recurrence, progression or death in patients with BCG-naïve, high-risk NMIBC compared to BCG alone (based on a Disease-Free Survival (DFS) hazard ratio of 0.68; 95 percent confidence interval 0.50-0.93; P=0.0154). With a median follow-up of over 5 years (60.7 months), the IMFINZI regimen delivered an early and sustained DFS benefit starting less than 4 months after beginning treatment. Estimated median DFS was not yet reached for either arm.

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.”

The safety and tolerability of IMFINZI plus BCG induction and maintenance therapy was consistent with the known safety profiles of the individual medicines, with no new safety signals identified with a median follow-up of over 5 years for DFS. The addition of IMFINZI did not compromise 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 results are under review in the European Union (EU), Japan and several other countries.

Meri-Margaret Deoudes, CEO, 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.”

AstraZeneca recently announced positive high-level results from the VOLGA phase 3 trial, showing that perioperative treatment with IMFINZI in combination with neoadjuvant Enfortumab Vedotin (EV) demonstrated 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 tremelimumab-actl in combination with neoadjuvant EV demonstrated a statistically significant and clinically meaningful improvement in EFS and a favorable trend for OS; however, the OS data were not statistically significant at this planned interim analysis and will be formally reassessed at a subsequent analysis.

IMFINZI is also approved in several countries for patients with cisplatin-eligible MIBC, based on the NIAGARA phase 3 trial, and continues to be investigated in locally advanced or metastatic disease in the NILE phase 3 trial.