China's NMPA Approves Fanregratinib for Pretreated FGFR2+ Intrahepatic Cholangiocarcinoma

China’s National Medical Products Administration (NMPA) has granted conditional approval to fanregratinib (Atled) for the treatment of adult patients with advanced, metastatic, or unresectable intrahepatic cholangiocarcinoma harboring an FGFR2 fusion or rearrangement who have previously received systemic therapy.¹

The approval of fanregratinib, a selective, oral FGFR1/2/3 inhibitor, was supported by the phase 2 registration cohort of the single-arm, multicenter, open-label, pivotal phase 2/3b trial (NCT04353375), which was conducted across 53 sites in China.

Data presented at the 2026 ESMO Gastrointestinal Cancers Congress showed the study met its primary end point, with an independent review committee (IRC)–assessed objective response rate (ORR) of 42.5% (95% CI, 30.0%–53.6%) among pretreated patients with advanced FGFR2 fusion/rearrangement–positive intrahepatic cholangiocarcinoma.² The median time to response was 1.4 months, the median duration of response (DOR) was 6.9 months (95% CI, 5.6–8.5), and the disease control rate (DCR) was 83.9% (95% CI, 74.5%–90.9%).

The median progression-free survival (PFS) was 6.9 months (95% CI, 4.1–8.2), and the median overall survival (OS) was 16.6 months (95% CI, 12.4–16.6).²

“As a major and devastating subtype of primary liver cancer, intrahepatic cholangiocarcinoma carries an immense disease burden with historically limited targeted options. We are thrilled by the NMPA approval of fanregratinib, which directly addresses this critical therapeutic gap in China,” Johnny Cheng, acting chief executive officer and chief financial officer of HUTCHMED, stated in a news release.¹ “This approval unlocks an important new treatment alternative for a substantial population of pretreated [patients with] advanced intrahepatic cholangiocarcinoma. We are fully prepared to leverage our established commercial infrastructure to bring this precision medicine to patients as rapidly as possible.”

How was the fanregratinib registration trial designed?

The pivotal phase 2/3b trial was a single-arm, multicenter, open-label study evaluating the efficacy, safety, and pharmacokinetics of fanregratinib in patients with advanced intrahepatic cholangiocarcinoma harboring FGFR2 fusions or rearrangements.^2,3 The registration cohort enrolled adult patients with locally advanced, unresectable, or metastatic intrahepatic cholangiocarcinoma who had received at least 1 prior line of systemic therapy; all patients had received prior chemotherapy, and 72% had received prior immunotherapy.² Patients were required to have measurable disease per RECIST 1.1 criteria and an ECOG performance status of 0 or 1.³

Enrolled patients received fanregratinib at 300 mg orally once daily for 14 consecutive days followed by 7 days off, in 21-day cycles.³

The primary end point was ORR; key secondary end points included DCR, time to response, DOR, PFS, and OS.

What is the safety profile of fanregratinib?

Fanregratinib exhibited a manageable safety profile consistent with the known mechanism of selective FGFR inhibitors, according to HUTCHMED.² Grade 3 or higher treatment-related adverse effects (TRAEs) were reported in 48.3% of patients, with the most common being liver enzyme elevations and palmar-plantar erythrodysesthesia syndrome.

TRAEs led to treatment discontinuation in 2.2% of patients, and no treatment-related deaths were reported.

What are the next steps for fanregratinib in intrahepatic cholangiocarcinoma?

The NMPA granted the approval on a conditional basis, and the phase 3b portion of the trial will serve as the confirmatory study to further validate the clinical benefit and safety of fanregratinib in this setting, and enrollment in the confirmatory cohort was initiated in January 2026.¹

The NDA had previously been accepted for review and granted priority review by the NMPA in December 2025.²

The approval adds a targeted option to an advanced biliary tract cancer treatment landscape that has continued to generate activity, including recent news that first-line ivonescimab plus chemotherapy improved OS vs durvalumab (Imfinzi) plus chemotherapy in patients with advanced biliary tract cancer.⁴

References

1. HUTCHMED announces NMPA approval for Atled (fanregratinib) for the treatment of patients with FGFR2-fusion/rearrangement intrahepatic cholangiocarcinoma. News release. HUTCHMED. August 28, 2026. Accessed August 28, 2026. https://www.hutch-med.com/fanregratinib-icc-nmpa-approval/
2. HUTCHMED highlights pivotal phase II data for fanregratinib in intrahepatic cholangiocarcinoma presented at ESMO Gastrointestinal Cancers Congress 2026. News release. HUTCHMED. June 25, 2026. Accessed August 28, 2026. https://www.hutch-med.com/fanregratinib-icc-esmogi26/
3. An open-label, single-arm, multicenter phase 2/3b clinical study to evaluate the efficacy, safety, and pharmacokinetics of HMPL-453 tartrate in patients with advanced intrahepatic cholangiocarcinoma harbouring FGFR2 fusion/rearrangement. ClinicalTrials.gov. Updated December 31, 2025. Accessed August 28, 2026. https://clinicaltrials.gov/study/NCT04353375
4. First-line ivonescimab plus chemotherapy meets OS end point in advanced biliary tract cancer. OncLive. Published August 26, 2026. Accessed August 28, 2026. https://www.onclive.com/view/first-line-ivonescimab-plus-chemotherapy-meets-os-end-point-in-advanced-biliary-tract-cancer