FDA Approves Pembrolizumab as First-Line Treatment for PD-L1–Positive Non–Small Cell Lung Cancer
On October 24, 2016, the U.S. Food and Drug Administration (FDA) approved a checkpoint inhibitor, pembrolizumab (Keytruda), for the first-line treatment of patients with metastatic non–small cell lung cancer (NSCLC) whose tumors express programmed cell death ligand 1 (PD-L1). This approval also expands the indication in second-line treatment of NSCLC to include all patients with PD-L1–expressing NSCLC.
Approval was based on the results of two randomized, controlled trials—KEYNOTE-024 and KEYNOTE-010—which demonstrated statistically significant improvements in progression-free and overall survival for patients randomized to receive pembrolizumab compared with chemotherapy.
In the first-line KEYNOTE-024 trial, median progression-free survival in those who received pembrolizimab was 10.3 months (vs 6 months with chemotherapy); overall survival was also significantly improved with pembrolizumab (hazard ratio = 0.60; P < .005).
In the second-line KEYNOTE-010 study, two doses of pembrolizumab (2 mg/kg or 10 mg/kg every 3 weeks) were compared with docetaxel. Median survival with the 2-mg dose was 10.4 months and with the 10-mg dose was 12.7 months, compared with 8.5 months with docetaxel.