HLB gets FDA green light for Refiktu in bile duct cancer

By Elena MacLeod · September 28, 2026, 11:14 AM · 3 mins read

HLB’s US arm Elevar Therapeutics has landed FDA approval for Refiktu, an oral drug for FGFR2-altered cholangiocarcinoma. This first for South Korean pharma opens the door to global expansion.

Elevar Therapeutics, the US branch of HLB, now holds FDA approval for Refiktu as a second-line treatment for cholangiocarcinoma patients with FGFR2 fusion or rearrangement. This is a first. No South Korean pharmaceutical or biotech company has ever directly filed and won FDA approval for a new cancer drug before.

Elevar is moving fast. The company plans to launch Refiktu in the US in the fourth quarter. At the same time, it has already filed for European approval with the European Medicines Agency. The goal is bigger than the US. HLB wants Refiktu to compete worldwide in oncology.

The FDA approved lirafugratinib (marketed as Lyrfigtu) for adults with previously treated unresectable, locally advanced or metastatic cholangiocarcinoma harboring FGFR2 gene fusion or rearrangement on September 23, 2026.

U.S. FDA

Refiktu works by targeting FGFR2, a genetic change found in some bile duct cancers. It is taken by mouth. Elevar is also testing Refiktu in other solid tumors with FGFR2 fusion or rearrangement. The company wants to see if the drug can help more patients beyond cholangiocarcinoma.

The FDA approval notice spells out the details. In the US, the drug is called Lyrfigtu. Its generic name is lirafugratinib. It is approved for adults with previously treated, unresectable, locally advanced or metastatic cholangiocarcinoma with FGFR2 gene fusion or rearrangement. Elevar Therapeutics, part of HLB, received the approval. The decision is based on results from the phase 1/2 ReFocus trial, which tested the safety and effectiveness of this oral FGFR2 inhibitor in these patients.

HLB is using this win to push its pipeline forward. The company is getting ready to resubmit its application for Rivoceranib, a liver cancer drug that did not get FDA approval before. HLB says it has fixed all the FDA's concerns about its Chinese partner's compliance with US Good Manufacturing Practice rules. The company is gathering more safety data. It will resubmit once everything is ready.

The FDA decision for Lyrfigtu was directly linked to results from the phase 1/2 ReFocus study, which provided the clinical evidence supporting its use in previously treated cholangiocarcinoma with FGFR2 alterations.

CancerNetwork

HLB is not stopping with Refiktu. HLB Therapeutics is about to announce global Phase 3 results for its keratitis drug candidate. Verismo, a US subsidiary of HLB Innovation, is also close to releasing clinical trial data for its CAR-T therapy for blood cancers. CAR-T is a hot area. Recent reports have shown promise for CAR-T in hard-to-treat cancers.

Refiktu’s US launch is coming soon. HLB is running several clinical and regulatory projects at once. The company now faces a new test: can it turn this FDA approval into a lasting presence in oncology, or will it be a one-off? For now, HLB has broken a barrier that stood for years in South Korean pharma. The industry is watching. The next moves matter.

Topics:

Biotechnology Newsroom

Elena MacLeod

Elena MacLeod is Clinical Biotechnology Editor at GenoMethods, covering CAR-T, engineered cell therapies, gene therapy, clinical trials, cancer immunology and regulatory developments. Her evidence-first reporting focuses on trial design, patient populations, safety, efficacy, response durability and the limitations that determine how early clinical results should be interpreted.