[비즈한국] Having faced three failures in its attempts to secure approval for a liver cancer treatment, HLB has finally crossed the threshold for U.S. Food and Drug Administration (FDA) new drug approval with a treatment for bile duct cancer. It was not rivoceranib, which HLB had been striving to get approved in the U.S. for a long time, but lirafugratinib, introduced from an external source in late 2024, that secured the FDA's green light first.
With this approval, HLB has secured its first anti-cancer drug available for sale in the U.S. Furthermore, since its U.S. subsidiary, Elevar Therapeutics (Elevar), holds the global development and commercialization rights directly, it is significant that a Korean company now holds the reins for global commercialization.
On the 23rd (local time), the FDA approved Elevar's lirafugratinib (U.S. product name: Lyfiktua) as a treatment for adult patients with unresectable, locally advanced, or metastatic bile duct cancer who possess FGFR2 gene fusions or other rearrangements and have received prior treatment.
Lirafugratinib is an oral targeted anti-cancer drug that selectively inhibits FGFR2. It is a substance previously developed by the U.S. biotech company Relay Therapeutics under the name RLY-4008. In December 2024, Elevar signed an exclusive licensing agreement with Relay to secure global development and commercialization rights. Elevar plans to launch lirafugratinib in the U.S. market within the fourth quarter of this year.
FDA Approval Before Rivoceranib… HLB Holds Global Rights to Lirafugratinib
It is noteworthy that lirafugratinib, rather than the long-pursued rivoceranib, received FDA approval.
Lirafugratinib secured FDA clearance only about two years after being added to HLB’s pipeline.
In the global Phase 1/2 clinical trials that served as the basis for FDA approval, lirafugratinib recorded an Objective Response Rate (ORR) of 46% and a median Duration of Response (mDOR) of 11.8 months. Previously disclosed analyses showed an ORR of 46.5%, a median Progression-Free Survival (PFS) of 11.3 months, and a median Overall Survival (OS) of 22.8 months.
Another reason this approval is drawing attention in the Korean pharmaceutical and bio-industry is the structure of the global licensing rights.
Leclaza, the non-small cell lung cancer treatment from Yuhan Corporation that received FDA approval before lirafugratinib, has its global rights (excluding Korea) held by the global pharmaceutical company Janssen. This means the lead in overseas market development and sales belongs to Janssen.
In contrast, while the original developer of lirafugratinib is the U.S.-based Relay Therapeutics, HLB’s U.S. subsidiary, Elevar, secured the global development and commercialization rights.
Under the contract, Elevar will pay up to $500 million (683.4 billion KRW), including upfront payments and regulatory/sales milestones, along with tiered royalties based on global sales. While not all revenue will flow directly to the HLB Group, the difference is clear in that the entity for global commercialization, including the U.S. market, is Elevar.
17 Years for a Global Anti-Cancer Drug… Jin Yang-gon's Persistence Bears First Fruit with Lirafugratinib
This approval marks a significant turning point in the global anti-cancer drug business that Jin Yang-gon, Chairman of the HLB Group, has pursued for 17 years.
Chairman Jin began his full-scale entry into anti-cancer drug development in 2009, triggered by rivoceranib. Starting with the decision to invest in the U.S. firm LSK BioPartners, which was developing rivoceranib at the time, HLB has since expanded its anti-cancer business with the goal of global clinical trials and FDA approval.
The process was far from easy. After exploring various cancer types, rivoceranib was positioned as a first-line treatment for liver cancer, and positive results were secured in global Phase 3 clinical trials in combination with the immune checkpoint inhibitor camrelizumab from China's Hengrui Pharma. Based on this, HLB submitted a New Drug Application (NDA) to the FDA in 2023.
However, approval was blocked at the threshold three times. Following the first Complete Response Letter (CRL) in 2024, a second one followed in 2025, and a third this year. As problems with manufacturing facilities and regulatory procedures were repeated, independent of the clinical results, skepticism grew in the market regarding the FDA approval prospects that HLB had emphasized. Each time the company's expectations diverged from actual review results, investor criticism and fatigue accumulated.
Nevertheless, Chairman Jin did not back away from the global anti-cancer business. While continuing to push for rivoceranib approval, he expanded the company's pipeline with the U.S. subsidiary Elevar at the center. In late 2024, he secured the global development and commercialization rights for lirafugratinib from Relay Therapeutics.
Ultimately, it was not rivoceranib, which he had devoted 17 years to, but the late-joining lirafugratinib that brought HLB its first FDA-approved anti-cancer drug.
The question now facing HLB is how much it can generate in actual prescriptions and revenue in the U.S. market. As the first hurdle of regulatory approval has been cleared with the approval of lirafugratinib, it is expected to serve as the next testing ground for Chairman Jin’s global anti-cancer drug business.