FDA Approves Adjuvant Nivolumab for the Treatment of Patients With High-Risk Urothelial Carcinoma

The FDA has approved nivolumab for patients with high-risk urothelial carcinoma regardless of previous treatment with neoadjuvant chemotherapy, nodal involvement, or PD-L1 status.

The use of adjuvant nivolumab (Opdivo) in patients with urothelial carcinoma who are at a high risk of recurrence following radical resection was approved by the FDA regardless of prior treatment with neoadjuvant chemotherapy, nodal involvement, or PD-L1 status, according to a press release from Bristol Myers Squibb.

The approval is based on the results of the phase 3 CheckMate 274 trial (NCT02632409), in which a 240 mg dose of nivolumab (n = 353) was compared with placebo (n = 356). Findings from the study indicated that patients who were treated with nivolumab experienced a median disease-free survival (DFS) of 20.8 months (95% CI, 16.5-27.6) compared with 10.8 months (95% CI, 8.3-13.9) in the placebo cohort. Nivolumab decreased the risk of disease recurrence of death by 30% vs placebo (HR, 0.70; 95% CI, 0.57-0.86; P = .0008).

“This approval is a major milestone for patients who have undergone major surgery to remove the bladder or parts of the urinary tract and are in need of additional treatment approaches that can help reduce the risk of their UC returning,” primary investigator Matthew D. Galsky, MD, professor of medicine, director of Genitourinary Medical Oncology, co-director of the Center of Excellence for Bladder Cancer, and associate director for Translational Research at The Tisch Cancer Institute and the Icahn School of Medicine at Mount Sinai, said in a press release. “Nivolumab provides a new FDA-approved treatment shown to reduce the risk of disease recurrence or death based on the safety and efficacy findings from CheckMate 274, and has the potential to become a new standard of care option in this setting.”

Other findings indicated that among patients who had a PD-L1 expression status of 1% or more (n = 140), the median DFS was not reached (95% CI, 21.2–not evaluable) compared with 8.4 months (95% CI, 5.6-21.2) among those who received placebo (n = 142). In this population nivolumab reduced the risk of disease recurrence of death by 45% (HR, 0.55; 95% CI, 0.39-0.77; P = .0005).

Nivolumab may cause severe and fatal immune-mediated adverse effects such as pneumonitis, colitis, hepatitis, and hepatotoxicity, endocrinopathies, dermatologic AEs, and nephritis with renal dysfunction. The agent may also cause infusion reactions and complications with allogeneic hematopoietic stem cell transplant.

“At Bristol Myers Squibb, our leading research in immunotherapy has helped transform the way many cancers are treated, and we are continuing to bring these advancements to patients with earlier stages of disease, particularly in challenging cancers with significant unmet need,” Adam Lenkowsky, senior vice president and general manager of U.S. Cardiovascular, Immunology and Oncology at Bristol Myers Squibb, said in a press release. “[Urothelial carcinoma] is the third type of cancer where Opdivo has been the first approved PD-1 inhibitor in the adjuvant setting. Now with this advancement, we can offer new hope to the conversations between health care providers and their UC patients where historically no approved treatment options have existed to help prevent disease recurrence post-surgery.”

This approval follows a priority review for nivolumab in this indication that was granted in April 2021. This decision by the FDA comes ahead of the target action date of September 3, 2021.


U.S. Food and Drug Administration approves Opdivo® (nivolumab) for the adjuvant treatment of patients with high-risk urothelial carcinoma. News release. Bristol Myers Squibb. August 20, 2021. Accessed August 20, 2021. https://bit.ly/3y8Yfo5

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