FDA Approves Pembrolizumab for Resectable Locally Advanced Head Neck Cancer

The U.S. Food and Drug Administration (FDA) has approved pembrolizumab (Keytruda), an immune checkpoint inhibitor, for the treatment of patients with resectable locally advanced head and neck squamous cell carcinoma (HNSCC) whose tumors express PD-L1 with a combined positive score (CPS) of ≥1. This approval is based on results from the pivotal phase 3 KEYNOTE-689 trial, which showed that patients receiving pembrolizumab before, during, and after surgery had significantly longer event-free survival and greater tumor shrinkage compared to standard care. This marks the first approval of a checkpoint inhibitor in the curative, perioperative setting for HNSCC and is considered a major paradigm shift, offering a new neoadjuvant and adjuvant treatment option. Merck (MSD) highlighted that this perioperative regimen has the potential to change long-standing standards of care and is currently under review in other global markets. Additionally, a phase II trial comparing concurrent versus sequential pembrolizumab with chemoradiotherapy suggested that sequential administration may offer better progression-free survival and locoregional control, warranting further study. Experts emphasize that this development represents the first significant advance in head and neck cancer treatment in over two decades.