HUTCHMED (00013) has announced that its New Drug Application for fanregratinib (HMPL-453) has received conditional approval from China's National Medical Products Administration (NMPA). The approval covers the treatment of adult patients with advanced, metastatic, or unresectable intrahepatic cholangiocarcinoma who have FGFR2 fusions or rearrangements and have previously undergone systemic therapy. The therapy will be marketed in China under the brand name ATLED® (Chinese: 爱领达®).
Intrahepatic cholangiocarcinoma is a highly aggressive malignancy originating from the bile duct epithelium within the liver. It accounts for approximately 8.2% to 15.0% of primary liver cancers, making it the second most common type after hepatocellular carcinoma. In recent years, the incidence of intrahepatic cholangiocarcinoma has shown a steady upward trend, with a five-year overall survival rate of approximately 9%. Among global patients with intrahepatic cholangiocarcinoma, around 10% to 15% harbor FGFR2 fusions or rearrangements.
The approval is supported by data from the Phase II registration cohort of a single-arm, multicenter, open-label Phase II/IIIb pivotal clinical trial conducted in China (NCT04353375). The results were recently presented at the 2026 European Society for Medical Oncology (ESMO) Gastrointestinal Cancers Congress. The study met its primary endpoint, demonstrating robust and clinically meaningful responses in previously treated patients with advanced intrahepatic cholangiocarcinoma harboring FGFR2 fusions or rearrangements. The objective response rate (ORR) assessed by an Independent Review Committee (IRC) was 42.5% (95% CI: 30.0%–53.6%). Key secondary endpoints also showed consistent clinical activity, with rapid onset of action and a median time to response of 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) reached 83.9% (95% CI: 74.5%–90.9%). Additionally, 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).
Mr. Zheng Zefeng, Acting Chief Executive Officer and Chief Financial Officer of HUTCHMED, stated: "Intrahepatic cholangiocarcinoma is a highly prevalent and devastating subtype of primary liver cancer, carrying a heavy disease burden, and previous targeted treatment options have been extremely limited. We are delighted to see ATLED® receive NMPA approval, which directly addresses a significant therapeutic gap in this clinical field in China. This approval brings an important new treatment option to a large number of patients with previously treated advanced intrahepatic cholangiocarcinoma. We are fully prepared to leverage our established commercial team and infrastructure to deliver this precision therapy to patients as quickly as possible."
The Phase IIIb portion of the clinical trial will serve as a confirmatory study to further validate the clinical benefit and safety profile of ATLED® in this indication. Patient enrollment for this confirmatory cohort was initiated in January 2026. ATLED® (fanregratinib, HMPL-453) is a novel, highly selective, and potent inhibitor of FGFR 1, 2, and 3. Aberrant FGFR signaling has been identified as a driver of tumor growth, promotion of angiogenesis, and resistance to anti-tumor therapies. Abnormal FGFR genetic alterations are considered driving factors for tumor cell proliferation in a variety of solid tumors. HUTCHMED currently holds all worldwide rights to fanregratinib.
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