Immunotherapy Approvals for Nasopharyngeal Carcinoma
The FDA has approved penpulimab-kcqx for certain patients with nasopharyngeal carcinoma.
The U.S. Food and Drug Administration (FDA) has approved penpulimab-kcqx in combination with chemotherapy for the first-line treatment of patients with nonkeratinizing nasopharyngeal carcinoma that is metastatic or has recurred.
Additionally, the FDA approved penpulimab-kcqx as a single agent for patients with metastatic nonkeratinizing nasopharyngeal carcinoma that has progressed on or after platinum-based chemotherapy and at least one other line of treatment.
Penpulimab-kcqx is a type of immunotherapy called an immune checkpoint inhibitor. It works by blocking suppressive signals to immune cells to enhance their cancer-fighting capabilities. This is the first FDA approval for penpulimab-kcqx.
The approval of the penpulimab-kcqx and chemotherapy combination is based on results from the randomized, multicenter, double-blind phase III Study AK105-304 trial. In the study, 291 patients with recurrent or metastatic nasopharyngeal carcinoma who had not received prior systemic chemotherapy for recurrent or metastatic disease were randomly assigned (1:1) to receive penpulimab-kcqx and chemotherapy followed by penpulimab-kcqx, or placebo and chemotherapy followed by placebo.
At a median follow-up of 19.1 months, penpulimab-kcqx reduced the risk of disease progression by 55%, with half of patients in the penpulimab-kcqx arm experiencing at least 9.6 months before tumor progression, compared with seven months in the placebo arm. At one-year follow-up, 31% of patients treated with penpulimab-kcqx and chemotherapy had not experienced disease progression, as compared with 11% of patients treated with placebo and chemotherapy. These results were presented at the AACR Annual Meeting 2025.
The approval of single-agent penpulimab-kcqx is based on results from the open-label, multicenter, single-arm phase II Study AK105-202 trial. In the trial, penpulimab-kcqx was used to treat 125 patients with unresectable or metastatic nonkeratinizing nasopharyngeal carcinoma whose disease had progressed after platinum-based chemotherapy and at least one other prior treatment. Twenty-eight percent (35/125) of patients responded to the immunotherapy.
The recommended dose for penpulimab-kcqx is 200 mg every three weeks until disease progression or unacceptable toxicity, for a maximum of two years.
Nasopharyngeal carcinoma is a type of head and neck cancer that affects the upper part of the throat behind the nose. Most forms of nasopharyngeal carcinoma are nonkeratinizing, and are linked to infection with the Epstein-Barr virus. According to federal statistics, it was estimated that 59,660 individuals would be diagnosed with head and neck cancer and approximately 12,770 patients would die of the disease in the United States in 2025.
The FDA rendered its decision on April 23, 2025. Check this resource for updated information on all therapeutics regulated by the FDA.