Dr Goyal on Efficacy Data With Lirafugratinib in FGFR2-Altered Cholangiocarcinoma

“The ORR was 46.5%. [Study investigators] also looked at some secondary end points, such as [median] DOR, [which] was 11.8 months.”

Lipika Goyal, MD, an associate professor in the University Medical Line in the Division of Oncology of the Department of Medicine at Stanford University, as well as a member of the Stanford Cancer Institute, discussed data from the phase 1/2 ReFocus trial (NCT04526106) evaluating lirafugratinib (RLY-4008) in FGFR2-altered cholangiocarcinoma (CCA).

Overall, study investigators evaluated the main key efficacy parameters that are examined across all the FGFR inhibitor trials in this setting, Goyal stated. She noted that the study’s primary end point was overall response rate (ORR) per independent review committee (IRC).

Findings from the pivotal cohort (n = 116) presented at the 2026 Gastrointestinal Cancers Symposium showed that the IRC-assessed ORR for patients with FGFR inhibitor–naive, chemotherapy-exposed CCA (n = 114) was 46.5% (95% CI, 37.1%-56.1%) with lirafugratinib. Best overall responses included a complete response rate of 2.6%, a partial response rate of 43.9%, and a stable disease rate of 50.0%.

Additionally, the disease control rate (DCR) was 96.5% (95% CI, 91.3%-99.0%), with a median duration of response (DOR) of 11.8 months (95% CI, 7.5-13.0). The median progression-free survival (PFS) was 11.3 months (95% CI, 9.2-14.8) and the median overall survival (OS) was 22.8 months (95% CI, 18.1-27.2).

Of note, these data supported a new drug application for lirafugratinib as a second-line treatment option for patients with pretreated CCA harboring FGFR2 fusions or rearrangements, which was granted priority review by the FDA in March 2026. The regulatory agency has assigned a Prescription Drug User Fee Act target action date of December 3, 2026.