WILMINGTON, Del. — AstraZeneca said the U.S. Food and Drug Administration has approved Imfinzi in combination with Bacillus Calmette-Guérin induction and maintenance therapy for adult patients with BCG-naïve, high-risk non-muscle-invasive bladder cancer.
The company said the approval makes the Imfinzi combination the first immunotherapy regimen approved in the U.S. for this patient group. The decision was based on results from the Phase 3 POTOMAC trial, which showed that adding one year of Imfinzi to BCG induction and maintenance therapy reduced the risk of high-risk disease recurrence, progression or death by 32% compared with BCG alone.
Non-muscle-invasive bladder cancer is often treated with tumor resection followed by BCG therapy delivered directly into the bladder. AstraZeneca said more than 31,000 people in the U.S. were treated for high-risk non-muscle-invasive bladder cancer in 2024. About half of patients with non-muscle-invasive bladder cancer are considered at high risk for recurrence or progression, depending on tumor grade, stage and other tumor features.
The company said up to 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 trial showed a disease-free survival hazard ratio of 0.68, with a 95% confidence interval of 0.50 to 0.93 and a p-value of 0.0154. AstraZeneca said the benefit appeared early and was sustained, beginning less than four months after treatment started. With median follow-up of more than five years, estimated median disease-free survival had not yet been reached in either treatment arm.
The safety and tolerability of Imfinzi plus BCG were consistent with the known safety profiles of the individual medicines, the company said. No new safety signals were identified, and the addition of Imfinzi did not compromise patients’ ability to complete BCG induction and maintenance therapy or have a meaningful impact on patient-reported quality of life.
AstraZeneca said regulatory submissions based on the POTOMAC results are under review in the European Union, Japan and several other countries.
Imfinzi is also approved in several countries for patients with cisplatin-eligible muscle-invasive bladder cancer based on the Phase 3 NIAGARA trial. The drug is also being studied in locally advanced or metastatic disease in the Phase 3 NILE trial.
