Futibatinib Receives Accelerated Approval for Cholangiocarcinoma

Cholangiocarcinoma micrograph.

The FDA has granted accelerated approval to futibatinib (Lytgobi®, Taiho Oncology, Inc.) for treatment of previously treated, unresectable, locally advanced, or metastatic intrahepatic cholangiocarcinoma (iCCA) with fibroblast growth factor receptor 2 (FGFR2) gene fusions or rearrangements.

“Survival outcomes are historically poor in patients with advanced/metastatic iCCA, with median overall survival times of approximately one year with first-line gemcitabine plus cisplatin and approximately six months with second-line chemotherapy,” wrote Lipika Goyal, MD, Assistant Professor of Medicine at Harvard Medicine School, and colleagues, in their publication of the FOENIX-CCA2 clinical trial (NCT02052778), on which the approval was based. “Futibatinib, a highly selective, irreversible FGFR1-4 inhibitor, demonstrated efficacy with durable responses in patients with iCCA harboring FGFR2 fusion/rearrangements in the pivotal FOENIX-CCA2 phase 2 study.”

Safety and efficacy were measured in the open-label, single-arm, multicenter trial, which enrolled 103 patients with previously treated cholangiocarcinoma with FGFR2 gene fusions or rearrangements to receive 20 mg of futibatinib orally once a day on a 21-day cycle until disease progression or unacceptable toxicity. The primary end points measured were overall response rate and duration of response.

At a median follow-up of 25 months, an independent review committee measured efficacy of futibatinib according to RECIST v1.1. The overall response rate was 41.7%, with a median duration of response of 9.7 months.

The most common adverse events experienced by 20% or more of patients being treated with futibatinib 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 erythrodysesthesia syndrome, and vomiting.

“Findings from the final analysis of FOENIX-CCA2 confirm the results of the primary analysis and reinforce the durable efficacy and continued tolerability of futibatinib in previously treated patients with advanced/metastatic iCCA harboring FGFR2 fusion/rearrangements,” concluded Dr. Goyal and colleagues in their abstract presented at the 2022 American Society of Clinical Oncology (ASCO) Annual Meeting.

The recommended dosage for patients being treated with futibatinib is 20 mg orally once a day, until disease progression or unacceptable toxicity occurs.

For More Information

Goyal L, Meric-Bernstam F, Hollebecque A, et al (2022). Updated results of the FOENIX-CCA2 trial: Efficacy and safety of futibatinib in intrahepatic cholangiocarcinoma (iCCA) harboring FGFR2 fusions/rearrangements. J Clin Oncol (ASCO Annual Meeting Abstracts), 40(suppl_16). Abstract 4009. DOI:10.1200/JCO.2022.40.16_suppl.4009

Clinicaltrials.gov (2022). A study of TAS-120 in patients with advanced solid tumors. NLM identifier: NCT02052778.

Lytgobi® (futibatinib) prescribing information (2022). Taiho Oncology, Inc. Available at: https://www.accessdata.fda.gov/drugsatfda_docs/label/2022/214801s000lbl.pdf

US Food and Drug Administration (2022). FDA grants accelerated approval to futibatinib for cholangiocarcinoma. Available at: https://www.fda.gov/drugs/resources-information-approved-drugs/fda-grants-accelerated-approval-futibatinib-cholangiocarcinoma?utm_medium=email&utm_source=govdelivery

Image credit: Nephron. Licensed under CC BY-SA 3.0

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