The FDA came to Elevar Therapeutics bearing gift ahead of the mid-autumn festival.
The U.S. agency has approved the company’s Lyrfigtu (lirafugratinib) for patients with previously treated bile duct cancer harboring an FGFR2 gene fusion or other rearrangement.
As Jin Yang-gon, chairman of HLB Group, Elevar’s parent company, noted in a YouTube announcement, Lyrfigtu marks the first novel cancer drug from a Korean company to secure FDA approval.
“The greatest gift brought by this new drug approval is the newfound confidence spreading throughout the company, bolstered by the achievement of becoming a firm with a global anti-cancer drug,” Jin said during the auto English-dubbed announcement. “Our goal was to complete new drug development independently, rather than relying on technology exports, which require waiting for others to develop and produce results.”
Compared with existing FGFR inhibitors such as Incyte’s Pemazyre and Taiho’s Lytgobi, Lyrfigtu was designed with increased selectivity for FGFR2, while sparing other proteins of the FGFR family, to minimize toxicities. Elevar obtained the asset from Relay Therapeutics in 2024 for $75 million in upfront and regulatory milestone payments and up to $425 million in potential commercial milestones.
In a single-arm trial called Refocus, Lyrfigtu registered an objective response rate of 46% and median duration of response reaching 11.8 months among 116 patients with cholangiocarcinoma new to FGFR inhibitor treatment but who had received prior chemotherapy or chemoimmunotherapy. The median progression-free survival was 11.3 months.
“Lyrfigtu’s unique, irreversible, covalent-binding mechanism enables potent and sustained inhibition of FGFR2, including many resistance mutations that can emerge with earlier FGFR inhibitors,” Lipika Goyal, M.D., from the Stanford Cancer Center and lead author of the Refocus trial, said in a Sept. 23 announcement. “This FDA approval brings an important next-generation precision medicine option directly to patients with advanced bile duct cancer.”
Elevar expects to make Lyrfigtu to patients in the U.S. this year. The company also filed an application with the European Medicines Agency on Sept. 15, Jin said.
Jin touted Lyrfigtu’s potential in other cancer types, including ongoing clinical trials in a tumor-agnostic setting, as well as opportunities to be combined with other anticancer drugs by Big Pharma. Now that the drug has been approved, the company expects patient enrollment in clinical trials to accelerate, Jin said.
The milestone of an FDA approval came years late for Elevar and its parent HLB. The company’s first U.S. approval was supposed to be the combination of camrelizumab and rivoceranib, partnered with China’s Hengrui Pharma, for the first-line treatment of liver cancer. However, beginning with a pair of complete response letters in May 2024, the two drugs have been rejected three times, all because of manufacturing shortfalls at Hengrui’s facilities.
Following the most recent snub in July, Hengrui has solved the issues, and the partners are now gathering updated safety data for a resubmission, according to Jin.