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Is Futibatinib of Benefit in Patients With FGFR2-Rearranged Intrahepatic Cholangiocarcinoma?

By: Joshua Swore, PhD
Posted: Thursday, March 9, 2023

The FGFR1–4 inhibitor futibatinib may provide clinical benefits for patients with FGFR2-rearranged intrahepatic cholangiocarcinoma with the possibility to scale to other cancers that respond to FGFR2 inhibition, according to a study published in The New England Journal of Medicine. “Clinical and translational research has shown that FGFR2 fusion or rearrangement–positive intrahepatic cholangiocarcinoma is a treatable cancer,” said Lipika Goyal, MD, of Stanford University School of Medicine, Palo Alto, California, and colleagues. “Data from this the value of molecular profiling in identifying tumors that are likely to respond to FGFR2 inhibition.”

The phase II, open-label FOENIX-CCA2 trial enrolled 103 patients with unresectable or metastatic FGFR2 fusion or rearrangement–positive intrahepatic cholangiocarcinoma. All patients had disease progression after one or more previous lines of non–FGFR-inhibiting therapy. In the present study, patients received a 20-mg dose of futibatinib once daily in a continuous regimen, with a median follow-up of 17.1 months.

The authors reported that 42% of patients achieved a treatment-related response (95% confidence interval = 32%–52%), with a median duration of 9.7 months. The authors noted that responses were consistent across all patients with varying disease states, mutations, and prior treatments. Progression-free survival and overall survival of these patients were 9.0 months and 21.7 months, respectively.

In terms of toxicity, the researchers recorded grade 3 treatment-related hyperphosphatemia in 30% of patients. Other treatment-related adverse events included increased aspartate aminotransferase level, stomatitis, and fatigue. A total of 2% of patients discontinued futibatinib therapy because of treatment-related toxicity. Of note, no treatment-related deaths were reported, and quality of life was maintained throughout the term of the study.

Disclosure: For full disclosures of the study authors, visit

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