Immunotherapy Approved in Combination With BCG for High-risk Bladder Cancer
The FDA approved durvalumab with Bacillus-Calmette Guérin for patients with high-risk, BCG-naïve non-muscle invasive bladder cancer.
The U.S. Food and Drug Administration (FDA) has approved durvalumab (Imfinzi) in combination with Bacillus Calmette-Guérin (BCG) for the treatment of adults with high-risk non-muscle invasive bladder cancer (NMIBC) who have not received prior treatment with BCG.
As a type of immunotherapy known as an immune checkpoint inhibitor, durvalumab, a monoclonal antibody, binds to the human programmed death ligand-1 (PD-L1), which both tumor and normal cells can express in inflammatory environments to regulate immune activity. Blocking PD-L1 from interacting with the PD-1 and B7.1 proteins on T cells prevents the suppression of those T cells, essentially releasing the brakes on the immune system. BCG is a strain of bacteria that has long been used in the treatment of bladder cancer. BCG can stimulate immune responses that counter the tumor, but its precise anticancer mechanisms of action remain unknown.
Prior to this approval, patients with NMIBC were eligible for immune checkpoint inhibitor therapy only after their disease had progressed on or after BCG treatment. This approval makes immune checkpoint inhibitor therapy available earlier in the course of treatment, before disease progression.
The approval was based on results from POTOMAC, a randomized, open-label, multicenter phase III clinical trial that enrolled 1,018 patients with high-risk NMIBC who had not been treated with BCG in the past three years and who had undergone surgical tumoral resection. Patients were randomly assigned on a 1:1:1 basis to receive one of three treatments: durvalumab plus BCG induction and maintenance (i.e., an initial round of BCG treatments followed by subsequent rounds over several months or years); BCG induction and maintenance alone; or durvalumab with BCG induction but no BCG maintenance (i.e., an initial round of BCG only). The approval was determined by the performance of the durvalumab with BCG induction and maintenance arm vs. the BCG-only with induction and maintenance arm.
After a median follow-up of 60.7 months, patients who received durvalumab and BCG induction and maintenance were 32% less likely than their counterparts in the BCG induction and maintenance arm to have experienced high-risk NMIBC recurrence, persistent carcinoma in situ, muscle-invasive bladder cancer, metastasis, or death. In both arms, at the time of follow-up, fewer than half of the patients in both arms had experienced any of the aforementioned disease progression events.
The recommended dose of durvalumab is based on body weight. For patients weighing less than 30 kg, 20 mg/kg should be administered; for patients weighing 30 kg or more, 1,500 mg should be administered. Regardless of body weight, intravenous infusions of durvalumab should be given every four weeks for 13 four-week cycles with BCG induction and maintenance. Treatment should continue until either the 13 cycles conclude or recurrence of high-risk disease, disease progression, or unacceptable toxicity occurs.
Bladder cancer originates in the lining of the bladder, and NMIBC refers to bladder cancer that has not yet spread to the muscles immediately surrounding the bladder lining. According to federal statistics, it was estimated that 84,530 individuals would be diagnosed with bladder cancer and 17,870 patients would die of the disease in the United States in 2026.
The FDA rendered its decision on May 28, 2026. Check this resource for updated information on all therapeutics regulated by the FDA