FDA approves penpulimab-kcqx for non-keratinizing nasopharyngeal carcinoma

Regulatory approval: On April 23, 2025, the FDA approved penpulimab-kcqx for adults with recurrent or metastatic non-keratinizing nasopharyngeal carcinoma (NPC). Approvals include first-line use with cisplatin or carboplatin plus gemcitabine, and single-agent use for metastatic non-keratinizing NPC after progression on or after platinum-based chemotherapy and at least one additional prior line. In AK105-304, penpulimab combination improved PFS vs placebo; OS was immature with no detrimental trend reported.

FDA approval (April 23, 2025)

The FDA approved penpulimab-kcqx (Akeso Biopharma Co., Ltd.) for adults with recurrent or metastatic non-keratinizing nasopharyngeal carcinoma (NPC):

  • First-line: penpulimab-kcqx with cisplatin or carboplatin plus gemcitabine.
  • Previously treated: single-agent penpulimab-kcqx for metastatic non-keratinizing NPC with progression on or after platinum-based chemotherapy and at least one other prior line.

Full prescribing information is expected on Drugs@FDA.

Supporting evidence

AK105-304 (NCT04974398): randomized, double-blind, multicenter (n=291) in recurrent/metastatic NPC without prior systemic chemotherapy for recurrent/metastatic disease. Primary endpoint PFS (BIRC, RECIST v1.1): median 9.6 vs 7.0 months (HR 0.45, 95% CI 0.33–0.62; p<0.0001) for penpulimab combo vs placebo combo. OS was immature (70% of prespecified deaths reported), with no detrimental trend observed.

AK105-202 (NCT03866967): open-label, single-arm (n=125; single country) in unresectable/metastatic non-keratinizing NPC after platinum and ≥1 additional line. ORR 28% (95% CI 20–37); median DOR not reached (95% CI 9.2–not estimable).

Safety and dosing (from FDA communication)

Immune-mediated adverse reactions included pneumonitis, colitis, hepatitis, endocrinopathies, nephritis, and skin reactions. Fatal adverse reactions occurred in 1%.

Recommended dose: 200 mg q3w with chemotherapy (max 24 months); 200 mg q2w as monotherapy (max 24 months).

Regulatory designations

Application had fast track, breakthrough, and orphan drug designations; FDA review used an Assessment Aid.

Sources