Seeking better treatments for patients with intrahepatic cholangiocarcinoma (iCCA) and FGFR2 gene rearrangement (futibatinib)

Narrative

Cholangiocarcinoma is an aggressive cancer originating in the bile ducts. Approximately 20% patients will have the intrahepatic form of the disease (iCCA), where cancer forms in the bile ducts inside the liver. Fibroblast growth factor receptor 2 (FGFR2) is a gene which makes a protein involved in cell division. Around 10-16% of patients with iCCA will typically have an FGFR2 gene rearrangement, including fusions which promote tumour proliferation (growth).

Futibatinib (known as Lytgobi) is an example of a precision medicine/targeted therapy. It binds to FGFR2 and inhibits the signalling pathway.

In September 2022, based on our research as part of the international FOENIX-CCA2 trial, FDA approved futibatinib (known as Lytgobi) to treat patients with iCCA harbouring fibroblast growth factor receptor 2 (FGFR2).

National Comprehensive Cancer Network (NCCN) guidance was updated in October 2022 to recommend futibatinib in CCA with FGFR2 fusions or rearrangements.

In July 2023, the European Commission gave Lytgobi conditional marketing approval throughout EU.

In September 2024, NICE recommended futibatinib as a treatment option for these patients citing FOENIX-CCA2 as the source of the clinical evidence.

Impact date	2022
Category of impact	Health and wellbeing
Impact level	Adoption