On September 30, 2022, the U.S. Food and Drug Administration (FDA) granted accelerated approval to futibatinib (Lytgobi®) for adult patients with previously treated, unresectable, locally advanced or metastatic intrahepatic cholangiocarcinoma harboring fibroblast growth factor receptor 2 (FGFR2) gene fusions or other rearrangements.

FDA Grants Accelerated Approval to Futibatinib for Cholangiocarcinoma

Efficacy was evaluated in TAS-120-101 (NCT02052778), a multicenter, open-label, single-arm trial that enrolled 103 patients with previously treated, unresectable, locally advanced or metastatic intrahepatic cholangiocarcinoma harboring a FGFR2 gene fusion or other rearrangement. The presence of FGFR2 fusions or other rearrangements was determined using next-generation sequencing testing. Patients received 20 mg of futibatinib orally once daily until they experienced disease progression or unacceptable toxicity.

The major efficacy outcome measures were overall response rate (ORR) and duration of response (DoR) as determined by an independent review committee according to RECIST v1.1. ORR was 42% (95% CI = 32, 52); all 43 responders achieved partial responses. The median DoR was 9.7 months (95% CI = 7.6, 17.1).

The most common adverse reactions reported in 20% or more 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 erythrodysesthesia syndrome, and vomiting.

The recommended futibatinib dose is 20 mg orally once daily until patients experience disease progression or unacceptable toxicity.

View the full prescribing information for futibatinib.

The review used the Real-Time Oncology Review pilot program, which streamlined data submission prior to the filing of the entire clinical application, and the Assessment Aid, a voluntary submission from the applicant to facilitate FDA’s assessment.

The application was granted priority review and breakthrough designation. A description of FDA expedited programs is in the Guidance for Industry: Expedited Programs for Serious Conditions-Drugs and Biologics. The application also was granted orphan drug designation.

Healthcare professionals should report all serious adverse events suspected to be associated with the use of any medicine and device to FDA’s MedWatch Reporting System or by calling 800-FDA-1088.

For assistance with single-patient investigational new drug applications, contact OCE’s Project Facilitate at 240-402-0004 or email OncProjectFacilitate@fda.hhs.gov.