The U.S. Food and Drug Administration has accepted a New Drug Application for zipalertinib to treat patients with locally advanced or metastatic non-small cell lung cancer with EGFR exon 20 insertion mutations whose disease has progressed on or after platinum-based chemotherapy, with or without amivantamab.

Taiho Oncology Inc., Taiho Pharmaceutical Co. Ltd., and Cullinan Therapeutics Inc. (NASDAQ: CGEM) announced the acceptance. The Prescription Drug User Fee Act target action date is February 27, 2027.

The application is supported by data from the Phase 2b REZILIENT1 clinical trial of zipalertinib monotherapy in patients with non-small cell lung cancer harboring EGFR exon 20 insertion mutations who have received prior therapy. The study met its primary endpoint of objective response rate.

In the primary efficacy population of 176 patients, including 51 who had received prior amivantamab, zipalertinib demonstrated a confirmed objective response rate of 35% with a median duration of response of 8.8 months. In patients treated after prior platinum-based chemotherapy only, the objective response rate was 40% with a median duration of response of 8.8 months.

Zipalertinib received Breakthrough Therapy Designation in 2021 for the treatment of patients with locally advanced or metastatic non-small cell lung cancer harboring EGFR exon 20 insertion mutations who have previously received platinum-based systemic chemotherapy.

Non-small cell lung cancer with EGFR exon 20 insertion mutations represents up to 4% of all non-small cell lung cancer cases globally, making them the third most common EGFR mutation subtype. In the United States, approximately 16% of patients with non-small cell lung cancer harbor EGFR mutations, with insertions at exon 20 accounting for up to 12% of these mutations.