HUTCHMED's fanregratinib demonstrated a 42.5% objective response rate in pretreated patients with FGFR2 fusion-positive intrahepatic cholangiocarcinoma, according to pivotal Phase II data presented at the ESMO Gastrointestinal Cancers Congress 2026 in Munich — findings that underpin a New Drug Application already accepted for priority review by China's National Medical Products Administration.
The registration-enabling single-arm study (NCT04353375) enrolled 235 patients with advanced ICC across 53 sites in China, all of whom had received at least one prior line of systemic therapy; all had prior chemotherapy exposure and 72% had received prior immunotherapy. The Independent Review Committee-assessed ORR of 42.5% (95% CI: 30.0%–53.6%) met the primary endpoint. Median time to response was 1.4 months, median duration of response was 6.9 months (95% CI: 5.6–8.5), and disease control rate reached 83.9% (95% CI: 74.5%–90.9%). Median PFS was 6.9 months (95% CI: 4.1–8.2) and median OS was 16.6 months (95% CI: 12.4–16.6). Grade 3 or higher drug-related adverse events were reported in 48.3% of patients, most commonly liver enzyme elevations and palmar-plantar erythrodysesthesia syndrome; treatment discontinuation due to drug-related adverse events occurred in 2.2% of patients, and no treatment-related deaths were recorded.
The open-label, single-arm Phase II design — conducted exclusively in China — limits direct generalizability to broader global populations and precludes a randomized efficacy comparison. The absence of a control arm means OS and PFS findings reflect the natural history of a heavily pretreated cohort alongside drug effect, without formal separation of the two.