FDA Approves Akeso’s PD-1 Monoclonal Antibody for Recurrent or Metastatic Non-Keratinizing Nasopharyngeal Carcinoma

Image Credit: Adobe Stock Images/SKT Studio

The FDA has approved Akeso’s penpulimab-kcqx, a PD-1 monoclonal antibody, for two indications treating nasopharyngeal carcinoma (NPC). The drug is now indicated in combination with chemotherapy as a first-line treatment for adult patients with recurrent or metastatic non-keratinizing NPC and as a monotherapy for patients with metastatic NPC who have progressed following platinum-based chemotherapy and at least one additional line of therapy.¹

"We are very excited by the approval of penpulimab-kcqx's approval in the US FDA for first line and later line NPC,” said Yu Xia, founder, chairwoman, president, CEO, Akeso, in the press release “Beyond reaching our first international regulatory milestone, this approval also provides an important immunotherapy treatment option for patients with NPC in the United States. The FDA approval of penpulimab-kcqx not only highlights the quality of our innovation but also underscores Akeso's focus on delivering treatments for difficult to treat cancers for patients around the world. We are deeply grateful to all the researchers, participants, and patients who have contributed to this success. Akeso will continue to advance first and best in class therapies, including bispecific antibodies and CD47 inhibitors, challenge global standards of care and unlocking the full potential of our pipeline for cancer patients everywhere."

The approval was based on results from the randomized, double-blind, international Phase III AK105-304 trial and the pivotal AK105-202 study, both of which supported the Biologics License Applications for the treatment. Full data are expected to be presented at the 2025 American Association for Cancer Research Annual Meeting. The FDA had previously granted penpulimab-kcqx with Breakthrough Therapy Designation, Orphan Drug Designation, and Fast Track Designation.¹

"This milestone enhances international treatment guidelines for advanced NPC and extends the benefits of China's innovations to global patients, ultimately reshaping the treatment landscape for metastatic NPC worldwide,” said Chaosu Hu, principal investigator of penpulimab-kcqx, professor, Fudan University Shanghai Cancer Center, in the press release.

Earlier this week, Akeso also announced that its combination of ivonescimab plus chemotherapy demonstrated statistically significant and clinically meaningful improvements in progression-free survival. The results, from the Phase III HARMONi-6 trial, showed superiority over tislelizumab plus chemotherapy as a first-line treatment for advanced squamous non-small cell lung cancer.²

According to the American Cancer Society, there is fewer than one case of NPC per 100,000 people in most parts of the world, including the United States. However, the disease is significantly more common in regions such as South Asia, the Middle East, and North Africa. In certain areas of China, incidence rates reach 25 to 30 cases per 100,000 men and 15 to 20 cases per 100,000 women.

While the risk of developing NPC increases with age, cases can occur in childhood. In high-risk regions, the disease is most common among individuals aged 45 to 59 years. In lower-risk areas, it appears more frequently among those aged 15 to 24 years, then declines before peaking again between 65 and 79 years of age. Notably, men are two to three times more likely to be diagnosed with NPC than women.³

"The FDA approval of penpulimab-kcqx confirms its high efficacy and low toxicity, positioning China's innovative drug development in alignment with international standards,” said Xiaozhong Chen, investigator of penpulimab-kcq, in the press release.

References

1. Akeso Announces FDA Approval for Penpulimab-kcqx in Two BLA Indications for Comprehensive Treatment of Advanced Nasopharyngeal Carcinoma. PR Newswire. April 24, 2025. Accessed April 25, 2025. https://prnmedia.prnewswire.com/news-releases/akeso-announces-fda-approval-for-penpulimab-kcqx-in-two-bla-indications-for-comprehensive-treatment-of-advanced-nasopharyngeal-carcinoma-302437965.html

2. Akeso’s PD-1/VEGF Bispecific Antibody Demonstrates Meaningful Progression-Free Survival Benefits in Advanced Squamous NSCLC. PharmExec. April 23, 2025. Accessed April 25, 2025. https://www.pharmexec.com/view/akeso-pd-1-vegf-bispecific-antibody-demonstrates-meaningful-progression-free-survival-benefits-advanced-squamous-nsclc

3. Key Statistics for Nasopharyngeal Cancer. American Cancer Society. Accessed April 25, 2025. https://www.cancer.org/cancer/types/nasopharyngeal-cancer/about/key-statistics.html