US approves targeted treatment for advanced bile duct cancer

The US Food and Drug Administration has approved a new oral treatment for some adults with advanced bile duct cancer, adding an option after earlier therapy.

The drug, lirafugratinib, is approved for previously treated adults whose cancer is locally advanced and cannot be removed surgically, or has spread to other parts of the body. Their tumours must also have specific changes in the FGFR2 gene, known as fusions or rearrangements. The medicine is designed to block the FGFR2 protein involved in cancer growth.

The approval was supported by a clinical trial involving 116 previously treated patients who had not received an FGFR-targeting medicine. Tumours shrank in 46% of patients, and responses lasted a median of 11.8 months among those who responded. These findings concern a specific patient group, rather than everyone with bile duct cancer.

Bile duct cancer is a rare cancer affecting the tubes that carry bile from the liver. About 8,000 people are diagnosed in the United States each year.

Elevar Therapeutics, the US subsidiary of South Korea’s HLB, plans to launch lirafugratinib in the United States in the fourth quarter of 2026. It has also submitted an application for European approval, although that process is not yet complete.