Medical Information

Futibatinib, as a highly selective FGFR inhibitor, has shown significant efficacy in solid tumors such as cholangiocarcinoma carrying FGFR2 gene mutations. Clinical studies have confirmed that its objective response rate and disease control rate are superior to traditional chemotherapy regimens, and it has manageable safety characteristics.

1. Core therapeutic effect

(1) Objective response rate: For patients with FGFR2 fusion/rearrangement cholangiocarcinoma, a phase II study showed an independently evaluated ORR of 41.7% and a median response duration of 9.7 months.

(2) Disease control: The overall disease control rate (DCR) reached 82.5%, and the median progression free survival (mPFS) was 9.0 months, significantly better than the 3-5 months of second-line chemotherapy.

2. Advantages of Mechanism of Action

(1) Targeting: Irreversible binding to the FGFR2 kinase domain, inhibiting FGFR1-4 activity at the nanomolar level, with selectivity more than 100 times higher than other kinases.

(2) Overcoming drug resistance: It can effectively inhibit gate region mutations such as FGFR2 V565F, solving the problem of acquired resistance to first generation FGFR inhibitors.

Performance of 3 special groups

(1) Previous treatment failures: Patients who have progressed with the gemcitabine regimen still show an ORR of 41.2%, and the efficacy is not affected by frontline treatment.

(2) Liver metastasis patients: There is no significant correlation between the incidence of liver dysfunction and baseline levels, and there is no need for routine dose adjustment.

4. Persistent characteristics of therapeutic efficacy

(1) Continuous response: The 12-month sustained remission rate remained at 35.8%, with the longest response duration exceeding 24 months.

(2) Survival benefit: The median overall survival (mOS) reached 21.7 months, nearly doubling compared to historical controls.