Javle M, Roychowdhury S, Kelley RK, et al. - Infigratinib represents a potential new therapeutic option for previously treated patients with locally advanced or metastatic cholangiocarcinoma harboring FGFR2 gene fusions or rearrangements, as it showed promising clinical activity and a manageable adverse event profile in this setting.

• In this multicenter, open-label, single-arm, phase 2 study, patients were administered 125 mg of oral infigratinib once daily for 21 days of 28-day cycles until disease progression, intolerance, withdrawal of consent, or death.

• The blinded independent central review-assessed objective response rate of 23·1% was recorded after a median follow-up of 10·6 months, with one confirmed complete response in a patient who only had non-target lesions identified at baseline and 24 partial responses.

• Hyperphosphatemia, stomatitis, fatigue, and alopecia were the most common treatment-emergent adverse events.

• Dry eyes were the most common ocular toxicity observed.

• No treatment-related deaths were recorded.