On November 10, 2016, the U.S. Food and Drug Administration (FDA) approved nivolumab (Opdivo) for the treatment of patients with recurrent or metastatic squamous cell carcinoma of the head and neck with disease progression on or after a platinum-based therapy.
Approval was based on data from an international, multicenter, open-label, randomized trial (CheckMate 141) comparing nivolumab with investigator’s choice of chemotherapy (either cetuximab [Erbitux], methotrexate, or docetaxel) in patients with recurrent or metastatic squamous cell carcinoma of the head and neck with disease progression on or within 6 months of receiving platinum-based chemotherapy.
The trial enrolled 361 patients randomized (2:1) to nivolumab at 3 mg/kg every 2 weeks intravenously (n = 240) or investigator’s choice (n = 121) of either intravenous cetuximab at 400 mg/m2 once, then 250 mg/m2 weekly (n = 15), intravenous methotrexate at 40 mg/m2 weekly (n = 52), or intravenous docetaxel at 30 mg/m2 weekly (n = 54) until disease progression or unacceptable toxicity.
The trial demonstrated a statistically significant and clinically meaningful improvement in overall survival associated with the nivolumab arm (hazard ratio = 0.7, 95% confidence interval [CI] = 0.52–0.92, P = .0101, stratified log rank test). Estimated median overall survival was 7.5 months (95% CI = 5.5–9.1) in the nivolumab arm and 5.1 months (95% CI = 4.0–6.0) for investigator’s choice.
Serious adverse reactions occurred in 49% of patients receiving nivolumab. The most frequent serious adverse reactions reported in at least 2% of patients receiving nivolumab were pneumonia, dyspnea, respiratory failure, respiratory tract infection, and sepsis. The most common adverse reactions occurring in more than 10% of nivolumab-treated patients and at a higher incidence than investigator’s choice were cough and dyspnea. ■