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FDA Approves Futibatinib for Intrahepatic Cholangiocarcinoma With FGFR2 Gene Fusions or Rearrangements
The Food and Drug Administration (FDA) approved fibroblast growth factor receptor (FGFR) inhibitor futibatinib (TAS-120) for adult patients with unresectable, locally advanced or metastatic intrahepatic cholangiocarcinoma with FGFR2 gene fusions or other rearrangements that have been previously treated.
This accelerated approval was based on efficacy results from the open-label, single-arm phase 1/2 TAS-120-101 trial. The trial enrolled 103 patients with unresectable, locally advanced or metastatic intrahepatic cholangiocarcinoma with a FGFR2 gene fusion or other rearrangement who had previously been treated with at least 1 systemic gemcitabine and platinum-based chemotherapy. Patients were treated with 20 mg futibatinib orally, once daily, until either disease progression or unacceptable toxicity. The main efficacy outcomes were objective response rate and duration or response.
The objective response rate was 42% (95% confidence interval [CI], 32 to 52), with all responses being partial. The median duration of response was 9.7 months (95% CI, 7.6 to 17.1).
The most common adverse events, occurring in ≥20% of patients, were nail toxicity, musculoskeletal pain, constipation, diarrhea, fatigue, dry mouth, alopecia, stomatitis, abdominal pain, dry skin, arthralgia, dysgeusia, dry eye, nausea, decreased appetite, urinary tract infection, palmar-plantar erythodysesthesia syndrome, and vomiting.
Source:
FDA grants accelerated approval to futibatinib for cholangiocarcinoma. United States Food and Drug Administration. September 30, 2022. Accessed October 3, 2022. https://www.fda.gov/drugs/resources-information-approved-drugs/fda-grants-accelerated-approval-futibatinib-cholangiocarcinoma
