Targeting Fibroblast Growth Factor Receptor Alterations in Cholangiocarcinoma and Other Solid Tumours: A New Frontier in Personalised Medicine

Abstract

Cholangiocarcinoma (CCA) is a cancer of the bile duct with poor prognosis and increasing incidence. Filippo de Braud gave an overview of CCA and its classification and highlighted key unmet needs in both diagnosis and treatment. Juan Valle explained that intrahepatic CCA (iCCA) accounts for 11% of the entire cancer of unknown primary (CUP) population, as well as 33% of patients with liver-inclusive disease. It is, therefore, vital that CUP with a ‘liver-dominant lesion’ is reviewed by a specialist team familiar with iCCA management. Angela Lamarca emphasised that managing CAA requires a multidisciplinary approach to both diagnosis and treatment. Precision medicine is now a reality in biliary tract carcinoma (BTC), particularly iCCA, so molecular testing is essential to ensure targeted therapy options are considered. David Malka explored existing and emerging standard-of-care (SOC) treatments for advanced BTC, which represents a target-rich disease. Updated guidelines now recommend targeted treatment for second-line iCCA in patients with appropriate alterations, underscoring the importance of systematic and early molecular profiling. A wide range of malignancies harbour FGFR alterations at varying frequencies, and the FGFR inhibitor pemigatinib is already approved for the treatment of CCA with FGFR2 fusions or rearrangements. Antoine Hollebecque highlighted the final results from the Phase II FIGHT-202 study, in which pemigatinib achieved an overall response rate (ORR) of 37% in the target population with a manageable safety profile.