27 SEP 2026 — The United States Food and Drug Administration approved Lyrfigtu (lirafugratinib) on 23 September for patients with a rare bile-duct cancer whose tumours carry a particular genetic change. The applicant was Elevar Therapeutics, the American subsidiary of South Korean group HLB.
HLB describes it as the first time a Korean drug company has filed an application for a global cancer drug directly with the FDA and won approval. The drug itself was not discovered in Korea.
What was approved
The FDA's notice covers adults with cholangiocarcinoma, cancer of the bile ducts, that has already been treated, cannot be removed surgically, and has an FGFR2 gene fusion or other rearrangement. It is a second-line treatment for a genetically defined subset of an uncommon cancer.
This is a regular approval, not an accelerated one. It carried priority review, breakthrough therapy and orphan drug designations, and went through the FDA's real-time oncology review. The dose is 70 mg by mouth once a day.
The evidence behind it
The approval rests on REFOCUS (NCT04526106), a multicentre, open-label trial in 116 patients with a single arm, meaning every patient received the drug and there was no comparison group. In the FDA's figures, 46 per cent of patients had their tumours shrink by the trial's measure, with a 95 per cent confidence interval of 36 to 55 per cent, and the median response lasted 11.8 months.
Elevar's announcement adds a median progression-free survival of 11.3 months (95 per cent CI 9.2 to 14.8) and a 12-month progression-free rate of 49.2 per cent. Those figures are not on the FDA's notice. Neither source reports overall survival.
What the trial cannot show
A single-arm trial can show that tumours respond. It cannot show that patients live longer than they would have on another treatment. That is the usual limit of approvals in rare cancers with few options.
What the company says makes it different
Earlier drugs in this class block several related FGFR proteins at once, which brings side effects such as high blood phosphate. Elevar's release says lirafugratinib binds irreversibly, selectively targets FGFR2 while minimising off-target toxicities, and inhibits FGFR2 including many resistance mutations.
The release gives no comparative figures for any of those claims. The FDA's notice still lists hyperphosphatemia among its warnings, alongside eye toxicity, soft tissue mineralisation and harm to a developing foetus. The release names the most common side effects as nail changes, a hand-and-foot skin reaction, mouth sores and hair loss, each affecting at least one patient in five.
Whose drug it is
Lirafugratinib was developed by Relay Therapeutics in the United States as RLY-4008. Korea Biomedical Review reports that Elevar took exclusive global rights under licence in December 2024, ran the application, and plans a United States launch in the fourth quarter of 2026. A European marketing application was filed in September, the outlet adds.
The Korean first, then, is a claim about who took the drug through the regulator, not where it was invented. It is HLB's own description, reported by Korea Biomedical Review, and we found no independent check of it. The wording is narrow on purpose: a Korean company, filing directly, for a global cancer drug.
For the region's drug industry, that distinction is the point. Licensing a late-stage asset and doing the regulatory work in-house is how a company builds the capability to file its own discoveries later.
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