FDA Approves Nivolumab for Resected Esophageal or GEJ Cancer

On May 20, 2021, the Food and Drug Administration approved nivolumab (Opdivo, Bristol-Myers Squibb Company) for patients with completely resected esophageal or gastroesophageal junction (GEJ) cancer with residual pathologic disease who have received neoadjuvant chemoradiotherapy.

Efficacy was evaluated in CHECKMATE-577, a randomized, multicenter, double-blind trial in 794 patients with completely resected (negative margins) esophageal or GEJ cancers who had residual pathologic disease following concurrent chemoradiotherapy. Patients were randomized (2:1) to receive either nivolumab 240 mg or placebo every 2 weeks for 16 weeks followed by 480 mg of nivolumab or placebo every 4 weeks beginning at week 17 for up to one year of treatment.

The main efficacy outcome measure was disease-free survival (DFS), defined as the time between randomization date and the first recurrence (local, regional, or distant from the primary resected site) date, or death, from any cause, as assessed by the investigator prior to subsequent anti-cancer therapy.

CHECKMATE-577 demonstrated a statistically significant improvement in DFS for patients receiving nivolumab as compared to those on the placebo arm. The median DFS was 22.4 months (95% CI: 16.6, 34.0) versus 11 months (95% CI: 8.3, 14.3), respectively (HR 0.69; 95% CI: 0.56, 0.85; p=0.0003). The DFS benefit was observed regardless of tumor PD-L1 expression and histology.

Nivolumab received orphan drug designation for this indication. A description of FDA expedited programs is in the Guidance for Industry: Expedited Programs for Serious Conditions-Drugs and Biologics.