The FDA has approved penpulimab-kcqx (Akeso Biopharma Co, Ltd) with cisplatin or carboplatin and gemcitabine for the first-line treatment of adults with recurrent or metastatic non-keratinizing nasopharyngeal carcinoma (NPC) and as a single agent for adults with metastatic non-keratinizing NPC with disease progression on or after platinum-based chemotherapy and at least 1 other previous line of therapy.1
Penpulimab-kcqx with cisplatin or carboplatin and gemcitabine was evaluated in Study AK105-304 (NCT04974398), a double-blind, multicenter trial of 291 patients with recurrent or metastatic NPC who had not received previous systemic chemotherapy for recurrent or metastatic disease.
Patients were randomly assigned (1:1) to receive either penpulimab-kcqx with cisplatin or carboplatin and gemcitabine, followed by penpulimab-kcqx, or placebo with cisplatin or carboplatin and gemcitabine, followed by placebo. Chemotherapy regimens are described in the full prescribing information.
The median progression-free survival was 9.6 months (95% confidence interval [CI], 7.1-12.5) in the penpulimab-kcqx arm and 7 months (95% CI, 6.9-7.3) in the placebo arm (hazard ratio, 0.45 [95% CI, 0.33-0.62]; 2-sided P<.0001), with 31% and 11% of patients alive and progression-free after 12 months of follow-up in the penpulimab-kcqx and placebo arms, respectively. While overall survival results were immature, with 70% of prespecified deaths for the final analysis reported, no detrimental trend was observed.
Efficacy of single-agent penpulimab-kcqx was evaluated in Study AK105-202 (NCT03866967), an open-label, multicenter, single-arm trial. The overall response rate in that trial was 28% (95% CI, 20-37) and median duration of response was not reached (95% CI, 9.2-not estimable).
Immune-mediated adverse reactions occurred with penpulimab-kcqx including pneumonitis, colitis, hepatitis, endocrinopathies, nephritis with renal dysfunction, and skin adverse reactions.
The most common adverse reactions (≥20%) for penpulimab-kcqx with cisplatin or carboplatin and gemcitabine were nausea, vomiting, hypothyroidism, constipation, decreased appetite, decreased weight, cough, COVID-19 infection, fatigue, rash, and pyrexia. The most common adverse reactions (≥20%) for single-agent penpulimab-kcqx were hypothyroidism and musculoskeletal pain. Fatal adverse reactions occurred in 1% of patients, including 1 case each of pneumonitis, septic shock, colitis, and hepatitis.
Reference
- FDA. FDA approves penpulimab-kcqx for non-keratinizing nasopharyngeal carcinoma [press release]. April 23, 2025. Accessed April 30, 2025. www.fda.gov/drugs/resources-information-approved-drugs/fda-approves-penpulimab-kcqx-non-keratinizing-nasopharyngeal-carcinoma