Infigratinib Approved for Cholangiocarcinoma

Area of Science:

• Oncology
• Pharmacology

Background:

• Cholangiocarcinoma is a rare and aggressive bile duct cancer.
• Treatment options for advanced or metastatic cholangiocarcinoma are limited.
• Fibroblast growth factor receptor 2 (FGFR2) fusions are key drivers in a subset of these cancers.

Purpose of the Study:

• To evaluate the efficacy and safety of infigratinib in patients with previously treated, advanced cholangiocarcinoma harboring FGFR2 alterations.

Main Methods:

• A phase II, single-arm, open-label study was conducted.
• Patients received oral infigratinib daily.
• Tumor response was assessed using RECIST criteria, and progression-free survival was a key endpoint.

Main Results:

• The overall response rate (ORR) was 23%.
• Median progression-free survival (PFS) was 7.3 months.
• Hyperphosphatemia occurred in 77% of patients, generally manageable.

Conclusions:

• Infigratinib demonstrates meaningful clinical activity in patients with advanced cholangiocarcinoma and FGFR2 alterations.
• The safety profile is consistent with previous studies, with hyperphosphatemia being the most frequent adverse event.
• Infigratinib represents a new targeted therapy option for this patient population.