Futibatinib for FGFR2+ Advanced Cholangiocarcinoma Hits the Market

Treatment with futibatinib is now available for patients with previously treated, unresectable, locally advanced or metastatic intrahepatic FGFR2-muatant cholangiocarcinoma.

Futibatinib (Lytgobi) is now available for the treatment of adult patients with previously treated, unresectable, locally advanced or metastatic intrahepatic cholangiocarcinoma harboring fibroblast growth factor receptor 2 (FGFR2) gene fusions or other rearrangements.1

Previously in September 2022, the FDA granted an accelerated approval to the kinase inhibitor futibatinib in this patient population. However, the company noted that the continued approval for this indication is subject to change based on verification and description of clinical benefit in a confirmatory trial.

The FDA approval for futibatinib was supported by findings from the phase 1/2 TAS-120-101 (NCT02052778). In the study, investigators assessed the use of futibatinib in 132 patients with previously treated, unresectable, locally advanced, or metastatic intrahepatic cholangiocarcinoma harboring a FGFR2 gene fusion or other rearrangement. Data showed futibatinib to reach an objective response rate (ORR) of 42% (95% CI, 32-52), with a 9.7-month (95% CI: 7.6, 17.1) median duration of response (DOR).

Regarding adverse events because of futibatinib, the most common included 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.

TAS-120-101 was an open-label, nonrandomized, phase 1/2 study evaluating the FGFR inhibitor futibatinib. The goals of the study included to evaluate the safety, tolerability, pharmacokinetics, pharmacodynamic, and anti-tumor activity of futibatinib in patients with advanced solid tumors who have genomic FGF/FGFR abnormalities as well as those who do not.

The study was conducted in 3 parts, including a dose-escalation portion to determine the maximum tolerated dose or recommended phase 2 dose (RP2D) of futibatinib, a phase 1 expansion portion to evaluate the safety and efficacy of futibatinib even further in patients with tumors harboring FGF/FGFR aberrations, and a phase 2 portion which will confirm ORR of futibatinib.2

Patients aged 18 years and older with histologically or cytologically confirmed, locally advanced, or metastatic cancer, an ECOG performance status of 0 or 1, and adequate organ function were enrolled in the study.

In phase 1, patients who had any type of solid tumor and disease progression following standard therapies or patients who were intolerant to prior standard therapies were enrolled.

The dose-expansion portion enrolled patients with 1 FGF/FGFR aberration who had either primary central nervous system tumors, advanced urothelial carcinoma with FGFR3 fusions or FGFR3 activating mutation, breast cancer, gastric cancer, or other solid tumor types harboring FGFR gene fusions or activating mutation. In this portion, patients must have had disease progression after standard therapies or bentolerant to prior standard therapies, and have measurable disease.

Then in the last portion of the study, patients with iCCA and FGFR2 gene rearrangements or fusions were enrolled who had been treated with at least 1 prior systemic gemcitabine and platinum-based chemotherapy, had radiographic progression of disease, no prior treatment with an FGFR inhibitor, and measurable disease.

"Onco360 is honored to partner with Taiho Oncology and become a specialty pharmacy provider for Lytgobi patients," said Benito Fernandez, chief commercial officer of Onco360, in a press release. "We are committed to supporting the highly specialized needs of patients battling treatment-experienced, FGFR2-mutant, advanced cholangiocarcinoma patients across the United States."

Lytgobi® (futibatinib) now available from Onco360 for the treatment of adult patients with previously treated, unresectable, locally advanced or metastatic intrahepatic cholangiocarcinoma harboring FGFR2 gene fusions or other rearrangements. News release. Onco360. February 13, 2023. Accessed February 14, 2023. https://yhoo.it/3lBoHq3
A study of TAS-120 in patients with advanced solid tumors. ClinicalTrials.gov. Updated January 10, 2022. Accessed February 14, 2023. https://clinicaltrials.gov/ct2/show/NCT02052778
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