FDA approves lirafugratinib for previously treated, unresectable, locally advanced or metastatic cholangiocarcinoma

On September 23, 2026, the Food and Drug Administration approved lirafugratinib (Lyrfigtu, Elevar Therapeutics, Inc.), a kinase inhibitor, for the treatment of adults with previously treated unresectable, locally advanced or metastatic cholangiocarcinoma harboring a fibroblast growth factor receptor 2 (FGFR2) gene fusion or other rearrangement.

Full prescribing information for Lyrfigtu will be posted on Drugs@FDA.

Efficacy and Safety

Efficacy was evaluated in REFOCUS (NCT04526106), a multicenter, open-label, single-arm trial in 116 patients with unresectable or metastatic cholangiocarcinoma who were naïve to FGFR inhibitor treatment and had received prior chemotherapy or chemoimmunotherapy. Patients received lirafugratinib 70 mg once daily until disease progression or unacceptable toxicity.

The major efficacy outcome measures were objective response rate (ORR) and duration of response (DOR) as determined by an independent review committee according to RECIST v1.1. ORR was 46% (95% CI: 36, 55) and median DOR was 11.8 months (95% CI: 7.5, 13.0).

The prescribing information includes warnings and precautions for ocular toxicity, hyperphosphatemia and soft tissue mineralization, and embryo-fetal toxicity.

Recommended Dosage

The recommended lirafugratinib dose is 70 mg orally once daily until disease progression or unacceptable toxicity.

This review used the Real-Time Oncology Review (RTOR) 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 the FDA’s assessment.

This application was granted priority review. Lirafugratinib received breakthrough therapy and orphan drug designations. A description of FDA expedited programs is in the Guidance for Industry: Expedited Programs for Serious Conditions-Drugs and Biologics.

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 1-800-FDA-1088.

For assistance with single-patient INDs for investigational oncology products, healthcare professionals may contact OCE’s Project Facilitate at 240-402-0004 or email  OncProjectFacilitate@fda.hhs.gov.