Hutchmed (China) (AIM:HCM) reported pivotal Phase II results for fanregratinib, its selective FGFR inhibitor, in patients with advanced intrahepatic cholangiocarcinoma carrying FGFR2 fusions or rearrangements, with data presented at the ESMO Gastrointestinal Cancers Congress in Munich on 4 July.
The registration-enabling trial, conducted across 53 sites in China, enrolled patients who had received at least one prior line of systemic therapy; all had received chemotherapy and 72% had prior immunotherapy exposure.
The study met its primary endpoint, with an independent review committee assessing an objective response rate of 42.5%, and a disease control rate of 83.9%.
Median time to response was 1.4 months, median duration of response was 6.9 months, and median overall survival reached 16.6 months.
Grade 3 or higher drug-related adverse events occurred in 48.3% of patients, most commonly liver enzyme elevations and hand-foot skin reaction; treatment discontinuation due to adverse events was 2.2%, and no treatment-related deaths were recorded.
The data underpin a New Drug Application that China's National Medical Products Administration accepted and granted priority review in December 2025.
"The results represent an important milestone in the targeted treatment landscape for FGFR2-altered ICC," said Professor Jianming Xu of the Chinese PLA General Hospital and lead principal investigator.
