HLB applies for European approval of FGFR2-targeted anticancer drug 'Lirafugratinib'

[Financial News] HLB is expanding its entry into the global market by initiating the approval process for its FGFR2-targeted anticancer drug 'Lirafugratinib' in Europe, following the US.
HLB announced on the 15th that its U.S. subsidiary, Eleva Therapeutics, submitted a Marketing Authorization Application (MAA) for lirafugratinib to the European Medicines Agency (EMA) on the 14th (local time) as a second-line treatment for patients with advanced or metastatic cholangiocarcinoma with FGFR2 fusion and rearrangement.
This application is based on the results of the global Phase 1/2 'ReFocus' clinical trial. In patients with FGFR2 fusion-positive advanced or metastatic cholangiocarcinoma who had received at least one previous systemic treatment, the objective response rate (ORR) was 46.5%. This is the evaluation standard of the Independent Review Committee (IRC).
The median duration of response (mDOR) was 11.8 months, the median progression-free survival (mPFS) was 11.3 months, and the median overall survival (mOS) was 22.8 months.
Lirafugratinib is an irreversible inhibitor that selectively targets FGFR2. Unlike existing non-selective FGFR inhibitors that broadly inhibit FGFR1, 2, 3, and 4, it is characterized by enhanced selectivity for FGFR2. The incidence rates of hyperphosphatemia and diarrhea were found to be 20.7% and 21.6%, respectively.
The approval review is currently underway in the United States. Lirafugratinib received Orphan Drug Designation (ODD) from the U.S. Food and Drug Administration (FDA) in 2022 and Breakthrough Drug Designation (BTD) in 2023. Eleva submitted a New Drug Application (NDA) to the FDA in January of this year and was designated for priority review. The deadline for the review under the Prescription Drug User Fee Act (PDUFA) is the coming 25th.
HLB is also conducting clinical trials to expand its therapeutic scope beyond cholangiocarcinoma to various solid tumors with FGFR2 gene mutations. Eleva is conducting the tumor-agnostic global Phase 2 trial 'ReFocus202' targeting solid tumors with FGFR2 fusion and rearrangement.
Patient enrollment and medication are underway in Korea, the United States, the United Kingdom, and the European Union (EU), with a target of an interim analysis in 2027. However, the schedule is subject to change depending on patient enrollment status and FDA requirements.
"Lirapugratinib is a novel drug candidate that has confirmed clinically significant therapeutic effects and differentiated safety data consistent with high FGFR2 selectivity and a sustained inhibition mechanism," said Kim Dong-gun, CEO of Eleva Therapeutics. "We will proceed with the approval process in the United States and the European Union without setbacks, while expanding the potential for treatment to various solid tumors through clinical trials regardless of cancer type."

[email protected] Jeong Sang-hee Reporter