FDA Approves Lyrfigtu for Advanced Cholangiocarcinoma

The U.S. Food and Drug Administration approved Lyrfigtu (lirafugratinib) for adults with previously treated, unresectable, locally advanced or metastatic cholangiocarcinoma with FGFR2 gene fusions or rearrangements on September 23, 2026.

This development introduces a new treatment option for a rare form of bile duct cancer, with Elevar Therapeutics, a subsidiary of HLB, announcing the approval as a second-line therapy for eligible patients.

The FDA’s decision was based on data from the Phase 1/2 ReFocus trial, which was conducted globally and involved multiple centers. The trial used an open-label, single-arm design and assessed the drug’s effectiveness in patients who had previously received treatment for cholangiocarcinoma.

Results from the ReFocus study showed an objective response rate of about 46 percent and a median duration of response of nearly 12 months. These findings supported the agency’s approval for patients whose cancer is not amenable to surgical removal and has specific genetic alterations.

What the numbers show

• FDA approval date: September 23, 2026.
• Objective response rate in trial: approximately 46%.
• Median duration of response: about 11.8 months.
• Estimated 8,000 new cholangiocarcinoma cases per year in the U.S.

Cholangiocarcinoma is an uncommon cancer, with roughly 8,000 new diagnoses each year in the United States. The disease affects the bile ducts and often presents treatment challenges, especially in advanced stages or when surgery is not possible.

HLB acquired the global development and commercialization rights for lirafugratinib in 2024. Following this acquisition, Elevar Therapeutics led the regulatory submission process that resulted in the FDA’s approval.

The approval of Lyrfigtu marks the first time a South Korean pharmaceutical company has directly obtained U.S. approval for a novel oncology drug, according to media reports. HLB’s direct submission of the New Drug Application and subsequent approval represent a milestone for the company and the country’s pharmaceutical sector.

Elevar Therapeutics stated that it plans to launch Lyrfigtu in the U.S. market during the fourth quarter of 2026. The company announced its intention to make the drug available as a second-line treatment for patients with FGFR2-altered cholangiocarcinoma.

* This article is based on publicly available information at the time of writing.