FDA Approval Summary: Nivolumab for adjuvant treatment of adults with completely resected esophageal/gastroesophageal junction cancer and residual pathologic disease.

The Food and Drug Administration (FDA) approved nivolumab on May 20, 2021, for the adjuvant treatment of completely resected (negative margins) esophageal or gastroesophageal junction cancer in patients who had residual pathologic disease following chemoradiotherapy (CRT). The approval was based on data from the double-blind CheckMate 577 trial which randomly allocated patients to receive nivolumab or placebo. Disease-free survival (DFS) was the primary endpoint. At the time of the final DFS analysis and the pre-specified interim overall survival (OS) analysis, the estimated median DFS was 22.4 months (95% confidence interval [CI]: 16.6, 34.0) in the nivolumab arm vs. 11.0 months (95% CI: 8.3, 14.3) in the placebo arm, with a hazard ratio (HR) of 0.69 (95% CI: 0.56, 0.85; two-sided p-value= 0.0003). An unblinded review of OS did not indicate a detrimental effect on survival. Adverse reactions occurring in ≥20% of patients receiving nivolumab were fatigue/asthenia, diarrhea, nausea, rash, musculoskeletal pain, and cough. Approval of nivolumab is likely to change the treatment paradigm for the adjuvant treatment of patients with completely resected (negative margins) esophageal or gastroesophageal junction cancer who have residual pathologic disease following CRT based on the study results and favorable risk: benefit of nivolumab administration.