First FDA Approval of a New Drug Developed by a Korean Company: HLB’s Cholangiocarcinoma Treatment

The U.S. Food and Drug Administration (FDA) has approved lirfugratinib (ingredient name: lirafugratinib), a treatment for cholangiocarcinoma developed by Elevar Therapeutics, an HLB Group affiliate. It is the first time the FDA has approved a new drug developed by a Korean company.

According to Fierce Pharma on the 25th (local time), the FDA authorized lirfugratinib for patients with previously treated cholangiocarcinoma harboring FGFR2 gene fusions or other rearrangements.

HLB Group Chairman Jin Yang-gon said in a YouTube announcement, “The greatest gift from this new drug approval is the confidence that has spread throughout the company, along with the achievement of becoming a company with a global oncology drug.” He added, “Our goal was to complete new drug development independently, without relying on technology exports.”

Unlike existing FGFR inhibitors, including Incyte’s Pemazyre and Taiho’s Lytgobi, lirfugratinib was designed to increase selectivity for FGFR2 while sparing other proteins in the FGFR family, reducing toxicity.

In 2024, Elevar licensed the asset from Relay Therapeutics for an upfront payment, $75 million in regulatory milestones and up to $425 million in future commercial milestones.

In the single-arm REFOCUS clinical trial, lirfugratinib achieved an objective response rate of 46% and a median duration of response of 11.8 months among 116 patients with cholangiocarcinoma who had not received an FGFR inhibitor but had received prior chemotherapy or chemoimmunotherapy. Median progression-free survival was 11.3 months.

Rupika Goyal, MD, of the Stanford Cancer Center, lead author of the REFOCUS trial, said in an announcement on the 23rd, “Lirfugratinib’s unique irreversible covalent mechanism of action potently and durably inhibits FGFR2, including resistance mutations that can emerge with existing FGFR inhibitors.” She added, “This approval provides an important next-generation precision medicine option for patients with advanced cholangiocarcinoma.”

Elevar plans to make lirfugratinib available to patients in the U.S. this year. Chairman Jin said the company also submitted a marketing authorization application to the European Medicines Agency (EMA) on the 15th.

Chairman Jin also discussed the drug’s potential in other cancer types, including in tumor-agnostic trials, and opportunities for combination regimens with large pharmaceutical companies. He said he expects clinical trial enrollment to accelerate following the approval.

Meanwhile, the first U.S. approval for Elevar and its parent company, HLB, had been expected to be a first-line combination treatment for liver cancer developed in collaboration with China’s Hengrui Pharmaceuticals. However, beginning with two complete response letters (CRLs) in May 2024, the application was rejected three times because of manufacturing deficiencies at Hengrui’s production facilities.

According to Chairman Jin, Hengrui resolved the issues after the most recent rejection in July, and the companies are collecting updated safety data for resubmission.