FDA Approves AstraZeneca’s Imfinzi for Muscle-Invasive Bladder Cancer

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The FDA has approved AstraZeneca’s Imfinzi (durvalumab) as the first and only perioperative immunotherapy for muscle-invasive bladder cancer (MIBC). The approval, granted under Priority Review status, was based on data from the NIAGARA Phase III trial presented at the 2024 European Society for Medical Oncology Congress and published in The New England Journal of Medicine.¹

“This approval for the durvalumab-based perioperative regimen is a major breakthrough for people with muscle-invasive bladder cancer, nearly half of whom see their cancer return despite chemotherapy and surgery with curative-intent,” NIAGARA investigator and steering committee member Matthew Galsky, MD, Lillian and Howard Stratton professor of medicine, director, genitourinary medical oncology, the Tisch Cancer Institute at the Icahn School of Medicine at Mount Sinai, New York, said in a press release. “This durvalumab regimen significantly extended patients’ lives in the NIAGARA trial and has the potential to transform care.”

The randomized, open-label, multi-center NIAGARA trial evaluated perioperative Imfinzi in 1,063 patients before and after radical cystectomy. Patients were randomly assigned to receive either Imfinzi plus neoadjuvant chemotherapy before cystectomy, followed by Imfinzi monotherapy, or neoadjuvant chemotherapy alone with no post-surgery treatment. The study’s dual primary endpoints were event-free survival (EFS) and pathologic complete response at the time of cystectomy, while key secondary endpoints included overall survival (OS) and safety.

Results showed that the Imfinzi regimen reduced the risk of disease progression, recurrence, failure to undergo surgery, or death by 32% (HR 0.68; p<0.0001). At two years, 67.8% of patients in the Imfinzi arm were event-free compared to 59.8% in the comparator arm. Median EFS was not yet reached in the Imfinzi arm but was 46.1 months in the comparator arm. OS data showed a 25% reduction in the risk of death (HR 0.75; p=0.0106), with 82.2% of patients in the Imfinzi arm alive at two years versus 75.2% in the comparator arm.

The Imfinzi regimen was well tolerated, with no new safety signals observed. Immune-related adverse events were consistent with its known safety profile.¹

“Today’s approval for Imfinzi represents a paradigm shift, bringing the first perioperative immunotherapy to patients in the United States with muscle-invasive bladder cancer and addressing a significant need for better treatment options,” said Dave Fredrickson, EVP, oncology hematology business unit, AstraZeneca, in the press release. “The NIAGARA trial showed more than 80% of patients were still alive at two years, underscoring the potential of this innovative perioperative regimen to become a new standard of care in this setting.”

Bladder cancer is the ninth most common cancer worldwide, with approximately 614,000 new cases diagnosed annually. In the United States, an estimated 84,870 new cases are expected by the end of 2025. Among patients with MIBC who undergo bladder removal surgery, 50% experience disease recurrence.1,2 Since 2013, bladder cancer incidence has declined by about 1% per year, a trend the American Cancer Society attributes to earlier detection, improved treatments, and increased screening.²

“More than 20,000 people in the US were treated for muscle-invasive bladder cancer last year and there is a significant need for new treatment options that improve patient outcomes,” said Meri-Margaret Deoudes, CEO, Bladder Cancer Advocacy Network, in the press release. “The approval of the durvalumab perioperative regimen is welcome news, transforming how clinicians will tackle this disease in future and offering new hope to patients and their loved ones.”

References

1. Imfinzi approved in the US as first and only perioperative immunotherapy for patients with muscle-invasive bladder cancer. AstraZeneca. March 31, 2025. Accessed March 31, 2025. https://www.astrazeneca.com/media-centre/press-releases/2025/imfinzi-approved-in-the-us-for-bladder-cancer.html

2. Key Statistics for Bladder Cancer. American Cancer Society. Accessed March 31, 2025. https://www.cancer.org/cancer/types/bladder-cancer/about/key-statistics.html