HLB Faces U.S. Commercialization Test After Clearing FDA Hurdle With 'LYRFIGTU'

[Financial News] HLB is now facing a test of its ability to commercialize in the U.S. after securing approval from the U.S. Food and Drug Administration (FDA) for LYRFIGTU (active ingredient: lirafugratinib), a new treatment for cholangiocarcinoma. Having led the drug approval process through its U.S. subsidiary and targeting a local launch in the fourth quarter of this year, HLB will need to demonstrate its ability to turn the regulatory achievement into actual prescriptions and sales.
According to sources in the financial investment industry on the 27th, Elevar Therapeutics, HLB's U.S. subsidiary, received FDA approval on the 23rd (local time) for LYRFIGTU as a second-line treatment for patients with cholangiocarcinoma featuring FGFR2 fusions or rearrangements. Specifically, the approval covers adults with previously treated, unresectable locally advanced or metastatic cholangiocarcinoma.
According to HLB, this is the first case in which a South Korean pharmaceutical or biotech company directly submitted a new drug application (NDA) for a global oncology drug to the FDA and obtained approval. The actual application and approval process was led by Elevar, HLB's U.S. subsidiary.
The achievement is significant because HLB successfully connected the global rights it secured for the new drug to U.S. marketing approval. Since obtaining the global rights to LYRFIGTU in 2024, Elevar has led the necessary approval procedures by comprehensively analyzing and organizing existing clinical data and establishing a regulatory strategy. This has laid the groundwork for directly pursuing the approval and commercialization of an oncology drug in the U.S. market.
For patients, a new targeted therapy option has been added for cholangiocarcinoma with FGFR2 fusions or rearrangements, a disease in which treatment choices have been limited. LYRFIGTU is an irreversible targeted anticancer agent with high selectivity for FGFR2. It was designed to bind to FGFR2 and provide sustained inhibition while minimizing unnecessary inhibition of FGFR1, FGFR3 and FGFR4.
In the global Phase 1/2 ReFocus clinical trial, which served as the basis for the approval, LYRFIGTU recorded an objective response rate (ORR) of 45.7% and a median duration of response (mDOR) of 11.8 months in the approved patient population. The median progression-free survival (mPFS) was 11.3 months, while the 12-month progression-free survival rate was 49.2%. The company explained, "The safety profile confirmed in the clinical trial was predictable and manageable through dose adjustments."
Allison Schram, a gynecologic oncologist at Memorial Sloan Kettering Cancer Center (MSKCC), said, "LYRFIGTU demonstrated durable treatment responses and manageable safety, offering a new treatment possibility for patients with FGFR2 fusion-positive cholangiocarcinoma. It is important to conduct molecular testing from the initial diagnosis so that patients can receive appropriate treatment opportunities."
The key challenge is establishing the approved treatment in U.S. clinical practice. Because eligibility for treatment depends on the presence of FGFR2 fusions or rearrangements, identifying patients through molecular testing and ensuring access to the drug are crucial. How steadily Elevar can maintain supply and expand prescriptions after the launch is expected to serve as a benchmark for evaluating HLB's commercialization capabilities.
Elevar plans to launch LYRFIGTU in the U.S. market and supply it to patients during the fourth quarter of this year. If approval marked the crossing of the regulatory hurdle for sales, the post-launch phase will involve securing actual treatment demand from healthcare providers and patients.
Kim Dong-geon, CEO of Elevar, said, "The FDA approval of LYRFIGTU is meaningful because it provides patients with cholangiocarcinoma and their families with an important second-line treatment option. We will do our utmost to make LYRFIGTU available to healthcare providers and patients as quickly as possible and continue improving the treatment environment for patients who have had limited treatment options."
Meanwhile, Elevar submitted a marketing authorization application (MAA) to the European Medicines Agency (EMA) on the 14th for LYRFIGTU as a treatment for cholangiocarcinoma, launching the European approval process. A tumor-agnostic ReFocus202 trial is also underway for patients with various solid tumors featuring FGFR2 fusions or rearrangements.

[email protected] Choi Doo-sun Reporter