The FDA has approved Akeso’s differentiated PD-1 monoclonal antibody, penpulimab-kcqx, for two therapeutic indications. Specifically, this injection has been approved as a first-line treatment for adult patients with recurrent or metastatic non-keratinizing nasopharyngeal carcinoma (NPC) when combined with carboplatin or cisplatin and gemcitabine. Additionally, penpulimab-kcqx has also been approved as a monotherapy for adult patients with metastatic non-keratinizing NPC whose disease has progressed following platinum-based chemotherapy and at least one other prior therapy. In the U.S., patients with advanced NPC now have a new immunotherapy option of penpulimab-kcqx.
The WHO 2020 Global Cancer Statistics report that more than 133,000 new cases of NPC are diagnosed globally each year and that over 70% of patients present with a locally advanced condition at admission. The recurrent or metastatic NPC is showing a poor prognosis and short survival time. The FDA approval of penpulimab-kcqx will broaden treatment options for NPC patients and offer potential benefits to a larger number of individuals.
This approval is based on the results from the Phase III clinical trial AK105-304 and the pivotal AK 105-202 clinical study. These studies provided the necessary data to support the two Biologics License Applications (BLA) for penpulimab-kcqx. These two clinical studies showed the clinical efficacy and safety of the drug throughout two treatment stages for metastatic NPC.
AK105-304 (NCT04974398) is a randomized, double-blind, multicenter clinical study that randomly assigned patients to receive either Penpulimab (AK105) with chemotherapy or a placebo with chemotherapy as first-line treatment for metastatic or recurrent NPC. A total of 296 patients were enrolled in this study. The primary outcome was progression-free survival (PFS), and secondary outcomes were overall survival (OS), objective response rate (ORR), duration of response (DoR), disease control rate (DCR), and adverse events (AEs). All the outcomes were measured with a follow-up period of up to 2 years.
The FDA has granted penpulimab-kcqx Orphan Drug Designation, Fast Track Designation, and Breakthrough Therapy Designation to the NPC treatment at the 2025 AACR Annual meeting. These designations highlight the significant unmet need that this therapy addresses.
Penpulimab-kcqx is available as an injection with a concentration of 100 mg/10 mL (10 mg/mL) solution in a single-dose vial. The combination medicine could lead to side reactions such as constipation, vomiting, nausea, weight loss, coughing, thyroid problems, a lack of appetite, weakness, rash, and COVID-19 infection. When used as a single agent, the common AEs (≥20%) were hypothyroidism and anemia.
According to Prof. Chaosu Hu and Prof. Xiaozhong Chen, the FDA has approved penpulimab-kcqx, a Chinese medicine, which broadens globally treatment recommendations for advanced nasopharyngeal carcinoma, strengthens medical advances, and supports its remarkable efficacy and safety profile.
Dr. Yu Xia, Founder, Chairwoman, President, and CEO of Akeso, expressed excitement about this approval, which offers a crucial immunotherapy option for US NPC patients. Akeso remains committed to advancing therapies and improving global cancer care with innovative treatments.
References:
- Akesobio. Akeso Announces FDA Approval for Penpulimab-kcqx in Two BLA Indications for Comprehensive Treatment of Advanced Nasopharyngeal Carcinoma. Published April 25, 2025. Accessed April 28, 2025. Akeso, Inc. | Akeso Announces FDA Approval for Penpulimab-kcqx in Two BLA Indications for Comprehensive Treatment of Advanced Nasopharyngeal Carcinoma
- U.S. Food and Drug Administration (FDA). Label PENPULIMAB-KCQX. 2025. Label PENPULIMAB-KCQX
- ClinicalTrials.gov. A Study of Penpulimab (AK105) in the First-line Treatment of Recurrent or Metastatic Nasopharyngeal Carcinoma. NCT04974398. A Study of Penpulimab (AK105) in the First-line Treatment of Recurrent or Metastatic Nasopharyngeal Carcinoma
