HLB’s cholangiocarcinoma drug lirafugratinib is approaching its U.S. Food and Drug Administration (FDA) decision date, with the target action date set for September 25. The company said no additional review or manufacturing-facility issues have emerged so far.
According to HLB on September 22, its U.S. subsidiary Elevar Therapeutics submitted a New Drug Application (NDA) for lirafugratinib in January, and the FDA granted the application priority review. The target action date is September 25, 2026.
The drug’s proposed U.S. brand name, Lytgobi—referred to in the company’s materials as Lytgobi—has also received conditional acceptance from the FDA. The agency reviews proposed drug names before approval and may conditionally accept them. However, conditional acceptance of a product name is a separate process from drug approval and does not mean that the marketing authorization has been granted.
Lirafugratinib is being developed as a second-line treatment for patients with advanced or metastatic cholangiocarcinoma harboring FGFR2 fusions or rearrangements. FGFR proteins transmit signals involved in cell growth, and genetic abnormalities such as fusions or rearrangements can cause abnormal signaling that promotes tumor growth.
The oral small-molecule drug is designed to selectively inhibit FGFR2. Unlike some existing FGFR inhibitors that target multiple FGFR subtypes, lirafugratinib was developed with high selectivity for FGFR2. HLB has highlighted this selectivity as a potential way to reduce adverse effects associated with broader FGFR inhibition.
The global Phase 1/2 ReFocus study supporting the regulatory submission reported an objective response rate (ORR) of 46.5% among patients with previously treated, FGFR2 fusion-positive advanced or metastatic cholangiocarcinoma. The median duration of response was 11.8 months, median progression-free survival was 11.3 months, and median overall survival was 22.8 months.
The FDA review is now in its final stage. Elevar held a late-cycle meeting with the FDA in July to review key issues arising during the later stages of the application review. According to the company, no major disagreements or new review issues were identified at that time.
HLB said no additional review issues have emerged since the meeting and that no particular problems involving the manufacturing facilities have been raised so far.
An HLB representative said, “There have been no significant additional issues since the late-cycle meeting, and there are currently no particular issues concerning the manufacturing facilities.”
The company added that the final approval decision rests with the FDA and that it is awaiting the outcome. Details of the U.S. launch timing and commercialization strategy will be disclosed after the regulatory decision.
Elevar is also pursuing expansion into Europe and additional indications. On September 15, the company submitted an application to the European Medicines Agency (EMA) for marketing authorization of lirafugratinib. It is also conducting the global Phase 2 ReFocus202 trial in patients with various solid tumors carrying FGFR2 fusions or rearrangements.
