FDA Okays Perioperative Durvalumab for Bladder Cancer

The US Food and Drug Administration (FDA) has approved a perioperative durvalumab (Imfinzi, AstraZeneca) regimen for adults with muscle-invasive bladder cancer.

Specifically, the indication is for neoadjuvant durvalumab in combination with gemcitabine and cisplatin chemotherapy followed by adjuvant single agent durvalumab after radical cystectomy.

Approval followed Priority Review and was based on efficacy demonstrated in the randomized, open-label, phase 3 NIAGARA trial that showed significant improvements in survival outcomes with the durvalumab-containing regimen vs chemotherapy and surgery alone, according to the FDA approval notice.

In 1063 patients randomized 1:1 to the durvalumab or control arm, median event-free survival (EFS), the major efficacy outcome, was not reached in the durvalumab arm (hazard ratio [HR], 0.68) at the time of a prespecified interim analysis. In the control arm, the EFS was 46.1 months. Median overall survival was not reached in either arm but was estimated at 82.2% vs 75.2% at 2 years in the treatment and control arms, respectively (HR, 0.75).

Adverse reactions were consistent with those known to be associated with durvalumab and platinum-based chemotherapy, and no new safety signals were observed.

According to the prescribing information, durvalumab dosing is weight-based: Patients with a body weight ≥ 30 kg should be given a dose of 1500 mg every 3 weeks with chemotherapy and 1500 mg as a single agent every 4 weeks as adjuvant therapy. Those weighing < 30 kg should receive a 20 mg/kg dose with chemotherapy every 3 weeks and 20 mg/kg every 4 weeks as adjuvant therapy. Treatment should continue until disease progression that precludes definitive surgery, recurrence, and unacceptable toxicity or until a maximum of eight cycles after surgery.

“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 Matthew Galsky, MD, of the Mount Sinai Hospital, New York, stated in a press release. “This durvalumab regimen significantly extended patients’ lives in the NIAGARA trial and has the potential to transform care.” 

Meri-Margaret Deoudes, chief executive officer of the Bladder Cancer Advocacy Network, noted in the press release that “[m]ore 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.

“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,” she said. 

Sharon Worcester, MA, is an award-winning medical journalist based in Birmingham, Alabama, writing for Medscape Medical News, MDedge, and other affiliate sites. She currently covers oncology, but she has also written on a variety of other medical specialties and healthcare topics. She can be reached at [email protected] or on X @SW_MedReporter.