On March 4, 2022, the FDA accelerated the approval of the PD-1 inhibitor nivolumab (Opdivo; Bristol Myers Squibb) in combination with platinum-doublet chemotherapy for the neoadjuvant treatment of adults with resectable non–small-cell lung cancer (NSCLC). This represents the first FDA approval for neoadjuvant therapy for early-stage NSCLC.
The efficacy was evaluated in the CheckMate-816 study, a randomized, open-label clinical trial of patients with resectable, stage IB (≥4 cm), stage II, or stage IIIA NSCLC. Patients were enrolled regardless of the tumor PD-1 or PD-L1 status. A total of 358 patients were randomized to nivolumab plus platinum-doublet chemotherapy or to platinum chemotherapy alone, administered every 3 weeks for up to 3 cycles in each arm.
The main efficacy outcomes were event-free survival (EFS) and pathologic complete response (pCR). The median EFS was 31.6 months in the nivolumab plus chemotherapy arm and 20.8 months in the chemotherapy-alone arm. The hazard ratio was 0.63 (97.38% confidence interval, 0.43-0.91; P = .0052). The pCR rate was 24% with nivolumab plus chemotherapy versus 2.2% with chemotherapy alone.
The most common (≥20%) adverse reactions were nausea, constipation, fatigue, decreased appetite, and rash.
