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Futabatinib for Patients with Intrahepatic Cholangiocarcinoma and FGFR2 Fusions or Rearrangements

Intrahepatic cholangiocarcinoma has a higher incidence in Asian than in Western countries and carries a poor prognosis
20 Nov 2020
Anticancer agents & Biologic therapy;  Gastrointestinal cancers;  Personalised medicine

Meaningful clinical benefit was provided by the FGFR1–4 inhibitor futibatinib in patients with refractory intrahepatic cholangiocarcinoma (iCCA) harbouring FGFR2 gene fusions or other rearrangements, including those enrolled in Asian countries. These findings were presented at the ESMO Asia Virtual Congress 2020, held from 20 to 22 November 2020.

Junji Furuse of the Department of Medical Oncology, Kyorin University Hospital in Tokyo Japan presented data from a preplanned interim analysis of the global phase II FOENIX-CCA2 study (NCT02052778), which evaluated the efficacy, safety, and quality of life (QoL) of futibatinib, a highly selective irreversible FGFR1–4 inhibitor, in patients with iCCA and FGFR2 fusions and/or rearrangements. He pointed out that futibatinib had already demonstrated safety and preliminary efficacy in similar patients.

FOENIX-CCA2 enrolled 103 patients with unresectable/metastatic iCCA and FGFR2 fusion/rearrangements who experienced disease progression after ≥1 line of systemic therapy that included gemcitabine–cisplatin, but not prior FGFR inhibitors.

All patients received oral futibatinib at 20 mg once daily until disease progression/intolerability.

The primary endpoint of the study was objective response rate (ORR) per independent central radiology review and RECIST v1.1, and secondary endpoints included disease control rate (DCR), duration of response (DoR), progression-free survival (PFS), safety, and patient-reported outcomes (PROs). Subgroup analysis of ORR (per baseline demographic, fusion partner, and the presence of other molecular alterations, such as TP53) was also performed.

Response was consistent between the general population and patient subgroups

This planned interim analysis reported data from 67 patients who had a minimum follow-up period of 6 months; of these, 54% of patients were white and 24% were Asian; 55% of patients had received ≥2 prior therapy lines. Eighty-two percent of patients had tumours with an FGFR2 fusion, and BICC1 was the fusion partner in 15 patients.

An ORR of 37.3% was reported in this interim analysis. The DCR was 82.1%, and median DoR was 8.3 months.


Best overall response in the FOENIX-CCA2 study.

© Junji Furuse.

Objective responses occurred in all subgroups of patients evaluated and were consistent across baseline characteristics that included age, FGFR2 fusion partner (BICC1; 33.3%), or the presence of another genetic mutation (TP53; 16.7%). Notably, an ORR of 57.1% was observed in the subgroup of patients aged ≥65 years.

The median PFS was 7.2 months with futibatinib treatment.

The most common any-grade treatment-related adverse events (TRAEs) included hyperphosphataemia (in 81% of patients), diarrhoea in 37%, and dry mouth in 33% of patients. Grade 3 hyperphosphatemia occurred in 27% of patients. No grade 4/5 TRAEs were reported. TRAEs were managed with dosing interruption in 55% of patients and with dose reduction in 51%; one patient discontinued treatment due to a TRAE.

PROs remained stable through 273 days (13 cycles) of treatment.

The US Food and Drug Administration Office of Orphan Drug Development granted futibatinib orphan drug status for the treatment of cholangiocarcinoma in May 2018.


According to the authors, futibatinib provided durable objective responses in patients with iCCA and FGFR2 fusions/rearrangements, including within patient subgroups. 

In addition, adverse events were manageable, and QoL as assessed by PROs was maintained throughout treatment.

The study was funded by Taiho Oncology, Inc., and Taiho Pharmaceutical Co., Ltd.


116MO – Furuse I, Goyal L, Meric-Bernstam F, et al. Efficacy, safety, and quality of life (QoL) with futibatinib in patients (pts) with intrahepatic cholangiocarcinoma (iCCA) harbouring FGFR2 fusions/rearrangements: FOENIX-CCA2. ESMO Asia Virtual Congress 2020 (20-22 November).

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