Background: Patients with advanced cholangiocarcinoma (CCA) have limited treatment options. Fibroblast growth factor receptor (FGFR) fusions are found in 10-16% of patients with CCA. Infigratinib is a selective FGFR2 inhibitor. The aim of this study was to evaluate the efficacy and safety of infigratinib for the treatment of advanced CCA.

Methods: PubMed, Embase®, and Cochrane via Ovid platform were searched on 11^th October 2021 for studies reporting safety and efficacy of infigratinib in patients with advanced CCA. No restriction regarding year of publication was applied. Two reviewers independently searched for articles and extracted data, with differences resolved through consensus.

Results: Of 166 identified studies, only two studies satisfied our inclusion criteria. Both were Phase II, single-arm studies with 125 mg of oral infigratinib once daily for 21 days of 28-day cycle. In Javle et al. 2021, treatment with infigratinib resulted in an overall response rate of 23.1%, a complete response rate of 1%, and a partial response rate of 22%. The progression-free survival (PFS) rate at 6 and 12 months was 56.49% and 16.88%, respectively. The median overall survival (OS) with infigratinib was 12.2 months, while OS rates at 6, 12, 24 months were 87.4%, 50.5%, 27.1% respectively. Grade 3 and grade 4 adverse events were reported in 56.0% and 8.0% of patients respectively. No treatment-related deaths were observed. Goyal et al. 2019, reported a mean PFS of 8.4 months.

Conclusion: Infigratinib demonstrated good therapeutic activity and a fair safety profile in locally advanced or metastatic CCA. Further studies are needed to ascertain the therapeutic benefit of infigratinib in CCA. PROOF 301, a phase III multicenter, open label, randomized trial of infigratinib versus standard of care in advanced/metastatic CCA is ongoing.