FDA Approves Opdivo for Select Patients With Urothelial Carcinoma

The FDA, under accelerated approval, has approved intravenous Opdivo (nivolumab, Bristol-Myers Squibb) for patients with locally advanced or metastatic urothelial carcinoma whose disease has progressed during or following platinum-containing chemotherapy or whose disease progressed within 12 months of receiving neoadjuvant or adjuvant treatment with platinum-containing chemotherapy.

Approval was based on results of  CheckMate-275, an open-label, single-arm, multicenter trial in which 270 patients with metastatic or surgically unresectable locally advanced urothelial carcinoma with measurable disease received intravenous Opdivo 3 mg/kg every 2 weeks until disease progression or unacceptable toxicity. 

The study found that 53/270 (19.6%) responded to treatment, with complete response in 7/270 (2.6%) and partial response in 46/270 (17%) patients. The median duration of response was 10.3 months (range, 1.9+ - 12.0+ months), and the median time to respond was 1.9 months.

Serious adverse events occurred in 54% of patients, including urinary tract infections, sepsis, diarrhea, small intestine obstruction, and deterioration in physical health. Fatigue, musculoskeletal pain, nausea, and decreased appetite were the most common adverse events.

Among the 270 patients, 17% discontinued treatment and 46% had a dose delay due to adverse events. Overall, four patients died of treatment-related pneumonitis or cardiovascular failure.

Continued approval of Opdivo for this indication may be contingent on findings from confirmatory trials. —Mary Beth Nierengarten