AstraZeneca has secured US approval for a major new immunotherapy advance.

 

The pharma leader's drug Imfinzi (durvalumab) combined with BCG therapy has been cleared for adults with BCG-naïve, high-risk non-muscle-invasive bladder cancer (NMIBC). This marks the first and only immunotherapy combination in this setting.

 

The decision by the Food and Drug Administration follows results from the POTOMAC Phase III trial, which showed the regimen cut the risk of high-risk disease recurrence, progression, or death by 32% compared with BCG alone after one year of treatment. The benefit was measured using disease-free survival, with a hazard ratio of 0.68.

 

The study tracked patients for more than five years (median 60.7 months), with protection against disease returning emerging in under four months and continuing long term.

 

Neal Shore, 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.”

 

AstraZeneca said the approval marks the first immunotherapy combination to reach this early-stage bladder cancer setting, an area where recurrence rates remain high and treatment options have been limited for decades.

 

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.”

 

Patient advocates also welcomed the development.

 

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, involving 1,018 patients across more than 120 centres in 12 countries, compared Imfinzi plus BCG against standard BCG therapy. 

 

The combination showed an early and sustained disease-free survival advantage, while maintaining a safety profile consistent with known effects of each treatment and without reducing patients’ ability to complete therapy.

 

In context, NMIBC accounts for more than 70% of bladder cancer cases, and up to 80% of high-risk patients experience recurrence within five years — underscoring the commercial and clinical significance of the result.