HLB’s experimental drug gains FDA Fast-track review status
Korean biotechnology firm HLB said its experimental bile duct cancer treatment received FDA priority review, accelerating the regulatory process and setting up a potential decision by late September.
HLB said the U.S. Food and Drug Administration granted priority review to lirafugratinib, shortening the review period and paving the way for potential approval by Sept. 27.
Key facts
- The FDA accepted the new drug application for lirafugratinib (RLY-4008).
- The review period was shortened to six months from the standard 10 months.
- A regulatory decision is expected by Sept. 27.
- The treatment is aimed at bile duct cancer patients with FGFR2 gene alterations who have already received prior treatment.
Korean biotechnology firm HLB said its experimental bile duct cancer treatment has been flagged for priority review from the U.S. Food and Drug Administration, speeding up the regulatory process and paving the way for potential approval by late September.
The company said its U.S. subsidiary, Elevar Therapeutics, was notified Thursday that the FDA had accepted its new drug application for lirafugratinib (RLY-4008), a targeted therapy for bile duct cancer patients with fibroblast growth factor receptor 2 (FGFR2) gene alterations who have received prior treatment.
The designation shortens the review period to six months from the standard 10 months, with a decision expected by Sept. 27, the company said.
Lirafugratinib as a second-line bile duct cancer treatment
Lirafugratinib is being evaluated as a second-line treatment for patients with cholangiocarcinoma, a rare and often aggressive cancer of the bile ducts. HLB said the drug demonstrated an objective response rate of 47 percent in clinical trials, compared to 36 percent for pemigatinib and 42 percent for futibatinib, both approved FGFR inhibitors.
The company also said the treatment showed a manageable safety profile, with side effects that were predictable and controllable through dose adjustments. That safety profile, together with what HLB described as competitive clinical data, supports the company’s push toward regulatory approval and commercialization.
HLB plans regulatory push and commercialization
HLB said it plans to devote significant resources to securing regulatory approval and preparing for commercialization, citing what it described as competitive clinical data. The company presented the FDA review milestone as an important regulatory step for Elevar Therapeutics and for its broader oncology strategy.
Kim Dong-gun, chief executive of Elevar Therapeutics, said the FDA’s decision to grant priority review and begin a full evaluation marked a pivotal moment for the company. “This is a highly encouraging signal for our corporate value,” Kim said.
He added that the company would focus all capabilities on major milestones, including potential approval and commercialization of a liver cancer treatment by July and this bile duct cancer therapy by September.
Broader development across multiple cancer types
The company added that it is also accelerating broader clinical trials of lirafugratinib across multiple cancer types. That expansion suggests HLB sees the drug as a broader precision oncology candidate beyond its current bile duct cancer application, especially in tumors tied to FGFR2-related pathways.
The latest FDA priority review status therefore represents more than a faster regulatory timetable. For HLB and Elevar Therapeutics, it is also a meaningful validation point for lirafugratinib, its clinical performance, its safety profile and its potential market positioning in the treatment of cholangiocarcinoma and other cancers.
