Durvalumab Granted Priority Review by FDA for Muscle-Invasive Bladder Cancer Treatment

AstraZeneca’s durvalumab (IMFINZI) has been granted Priority Review by the US Food and Drug Administration (FDA) for the treatment of patients with muscle-invasive bladder cancer (MIBC). This decision is based on data from the phase 3 NIAGARA trial, which demonstrated a statistically significant and clinically meaningful improvement in event-free survival (EFS) and overall survival (OS) for patients receiving perioperative durvalumab in combination with standard therapies. FDA approval would make durvalumab the only perioperative immunotherapy regimen available in the curative-intent setting.

Susan Galbraith, Executive Vice President of Oncology R&D at AstraZeneca, emphasized the importance of this potential new option: “Nearly half of patients with muscle-invasive bladder cancer will see their cancer return or progress despite curative-intent treatment…. Today’s Priority Review designation recognizes the urgent need for new options for these patients and the potential of IMFINZI to transform the standard of care as the first and only perioperative immunotherapy regimen to delay recurrence and extend survival in this setting.”

The Priority Review designation is based on findings from the NIAGARA phase 3 trial, presented at the 2024 European Society for Medical Oncology (ESMO) Congress and published in The New England Journal of Medicine. The trial evaluated a perioperative regimen of durvalumab combined with neoadjuvant chemotherapy before surgery and durvalumab as adjuvant monotherapy post-surgery, compared to the standard approach of neoadjuvant chemotherapy followed by surgery.

The trial results showed:

• A 32% reduction in the risk of disease progression, recurrence, failure to undergo surgery, or death for the durvalumab regimen (EFS hazard ratio [HR], 0.68; 95% confidence interval [CI]: 0.56-0.82; P<.0001). At two years, 67.8% of patients receiving durvalumab were event-free compared to 59.8% in the comparator arm.
• A 25% reduction in the risk of death (OS HR, 0.75; 95% CI: 0.59-0.93; P=.0106). Two-year overall survival rates were 82.2% for the durvalumab group compared to 75.2% for the comparator group.

Median EFS and OS were not yet reached in the durvalumab arm at the time of analysis, signaling robust durability of benefit.

Durvalumab was well tolerated, with no new safety concerns reported in either the neoadjuvant or adjuvant settings. The combination of durvalumab and chemotherapy did not interfere with patients’ ability to complete all cycles of chemotherapy or undergo surgery. The safety profile of durvalumab was consistent with previous studies, and adverse events were manageable.

Regulatory applications for durvalumab in this indication are also under review in the European Union, Japan, and other countries. The FDA’s Priority Review status indicates that the agency will aim to make a decision by the second quarter of 2025, expediting the availability of this potential treatment option for MIBC patients in the US.