FDA Approves Akeso's Penpulimab-kcqx for Advanced Nasopharyngeal Carcinoma

Akeso, Inc., a prominent biopharmaceutical enterprise headquartered in Hong Kong, has achieved a significant breakthrough with the approval of its PD-1 monoclonal antibody, penpulimab-kcqx, by the U.S. Food and Drug Administration (FDA). This antibody, developed to treat non-keratinizing nasopharyngeal carcinoma (NPC), offers new therapeutic options for adults with recurrent or metastatic forms of this cancer. The approval allows penpulimab-kcqx to be utilized in combination with cisplatin or carboplatin and gemcitabine for first-line treatment. It also provides an option as a single agent for individuals with metastatic NPC who have experienced disease progression following platinum-based chemotherapy and at least one other previous therapy.

The journey to FDA approval marks penpulimab-kcqx as Akeso's inaugural internally developed biologic to receive such recognition in the U.S., highlighting the company's innovation in pharmaceutical development and its commitment to high international quality standards. This milestone underscores Akeso's strategic approach to international drug development and positions it for further involvement in global therapeutics markets.

Penpulimab-kcqx has already been approved in China for advanced NPC treatment, both as a first-line therapy and for subsequent lines. The recent U.S. approval expands treatment opportunities for American patients, reflecting the outcomes of international Phase III trials AK105-304 and AK105-202, which supported the Biologics License Applications for penpulimab-kcqx. These studies demonstrated significant clinical benefits and a favorable safety profile for the drug across two treatment stages for metastatic NPC. Specifically, trial AK105-304, a randomized, double-blind international study, included NPC patients from various ethnic backgrounds and is set to be featured at the 2025 American Association for Cancer Research Annual Meeting.

Boosting penpulimab-kcqx's profile, the FDA had previously granted it Breakthrough Therapy Designation, Orphan Drug Designation, and Fast Track Designation for NPC treatment, underscoring the urgent need for effective therapies in this area. According to the World Health Organization's 2020 Global Cancer Statistics, over 133,000 new NPC cases are diagnosed yearly worldwide, with the majority of these cases presenting as locally advanced. The prognosis for recurrent or metastatic NPC remains grim, with limited survival rates, highlighting the significance of this FDA approval in broadening the therapeutic options available to clinicians and patients.

Prof. Chaosu Hu from Fudan University Shanghai Cancer Center emphasized that this development enhances global treatment guidelines for advanced NPC and signifies China's growing influence in international drug innovation. Prof. Xiaozhong Chen from Zhejiang Cancer Hospital also noted the FDA's approval as a testament to the drug's efficacy and safety, aligning Chinese pharmaceutical advancements with global standards.

Dr. Yu Xia, the Founder, Chairwoman, President, and CEO of Akeso, expressed immense satisfaction with the FDA's approval, viewing it as a pivotal achievement not only for the company but also for providing a crucial immunotherapy option to NPC patients in the U.S. The success of penpulimab-kcqx highlights Akeso's dedication to addressing difficult-to-treat cancers and its ambition to continue advancing pioneering therapies. This includes their exploration of bispecific antibodies and CD47 inhibitors, aiming to challenge existing global care standards and maximize the potential of their treatment pipeline for cancer patients globally.