South Korean pharmaceutical and biotech company HLB has successfully secured new drug marketing approval from the US Food and Drug Administration (FDA) for the first time in 19 years. However, rather than its long-pursued liver cancer treatment—which HLB has been developing since 2007—the approved drug is Liptivo, a cholangiocarcinoma (bile duct cancer) treatment acquired from a US biotech firm in 2024. According to the company, this marks the first instance where a South Korean company has directly submitted a New Drug Application (NDA) for an anticancer drug and gained approval from the FDA.
HLB announced on September 27 that its US subsidiary, Elevar Therapeutics, received marketing authorization from the FDA for Liptivo (generic name: lirafugratinib), a fibroblast growth factor receptor 2 (FGFR2) targeted therapy.
Liptivo was approved as a second-line treatment for patients with advanced cholangiocarcinoma harboring FGFR2 gene alterations who have received prior therapies. Cholangiocarcinoma is a rare cancer occurring in the bile ducts, which are tubes carrying bile generated by the liver. Clinical data showed that 46% of patients experienced complete disappearance or reduction of tumors, with a median duration of response lasting 11.8 months.
Previously, Yuhan Corporation’s lung cancer treatment Leclaza (US brand name: Lazcluze) won FDA approval in 2024, but the regulatory process was handled by Janssen, which secured global development and commercialization rights excluding South Korea.
Milestone Achievement: Direct FDA Application by a Korean Company
Dr. Lipika Goyal of the Stanford Cancer Center, who led the Liptivo clinical trials, stated, “Liptivo can powerfully and persistently inhibit FGFR2, including resistance mutations that may arise with existing FGFR inhibitors. It will serve as an important next-generation precision medicine treatment option for advanced cholangiocarcinoma patients.”
Liptivo was originally a candidate substance developed under the name ‘RLY-4008’ by the US-based Relay Therapeutics. In December 2024, Elevar secured global development and commercialization rights through a deal worth up to $500 million (approx. 680 billion KRW), subsequently taking charge of clinical data analysis, regulatory strategies, NDA submissions, and FDA communications. Unlike the traditional model of out-licensing internally developed drugs overseas, this case involved bringing in external clinical assets to directly drive approval and commercialization.
Meanwhile, HLB’s combination therapy of ‘rivoceraninib’ and Hengrui Medicine’s ‘camrelizumab’ for liver cancer, which the company has long pushed for FDA approval, has yet to cross the finish line. Since 2024, it has received three Complete Response Letters (CRLs), and in July, issues arose at Hengrui Medicine’s active pharmaceutical ingredient manufacturing facility. HLB subsequently announced that the facility was classified with a ‘Voluntary Action Indicated’ (VAI) status following an FDA inspection, resolving major issues, and the company plans to pursue a resubmission for FDA approval.
Jin Yang-gon, Chairman of HLB Group, stated, “Our goal was to independently complete new drug development without resorting to technology export. The experience and capabilities accumulated during the drug approval process have become a tremendous asset.”
Commercialization and Future Outlook
The next agenda for HLB is commercialization and market expansion. Elevar plans to launch Liptivo in the US market in the fourth quarter of this year. The company also intends to pursue label expansion into other FGFR2-altered solid tumors. Fierce Pharma noted that Liptivo will compete with existing FGFR inhibitors such as Incyte’s ‘Pemazyre’ and Taiho Oncology’s ‘Lytgobi,’ explaining that Liptivo was designed to selectively target FGFR2 to minimize impacts on other similar proteins.
Eun-kyung Choi (choi.eunkyung@joongang.co.kr)
