Elevar Submits EMA Marketing Authorization Application for Lirafugratinib in FGFR2-Fusion Cholangiocarcinoma
核心洞察
Elevar Therapeutics (搜索) has filed a marketing authorization application with the European Medicines Agency (搜索) for lirafugratinib as second-line treatment of advanced or metastatic FGFR2 (搜索) fusion-positive cholangiocarcinoma (搜索).
The filing rests on Phase 1/2 ReFocus data showing a 46.5% objective response rate by independent review, with median duration of response of 11.8 months.
Median progression-free survival reached 11.3 months and median overall survival 22.8 months in patients who had received one prior line of systemic therapy.
Elevar Therapeutics (搜索), Inc., a majority-owned subsidiary of HLB Co., Ltd. (搜索), announced on Sept. 15, 2026 that it has submitted a marketing authorization application (MAA) to the European Medicines Agency (搜索) (EMA) for lirafugratinib as a second-line treatment for patients with advanced or metastatic cholangiocarcinoma (搜索) (CCA) harboring FGFR2 (搜索) fusions or rearrangements. The submission initiates the regulatory process in the European Union and follows the company's earlier U.S. filing, expanding its global market presence for the drug candidate.
ReFocus Data Underpin the Filing
The application is based on results from the Phase 1/2 ReFocus study, whose primary endpoint is objective response rate (ORR). In patients with advanced or metastatic FGFR2 (搜索) fusion-positive CCA who had received a prior line of systemic therapy, lirafugratinib achieved an ORR of 46.5% as assessed by an independent review committee.
Median duration of response, a key secondary endpoint, was 11.8 months. Median progression-free survival was 11.3 months, and median overall survival was 22.8 months.
"Lirafugratinib is a novel drug candidate that has demonstrated meaningful clinical activity and a differentiated safety profile consistent with its high selectivity for FGFR2 (搜索) and sustained inhibitory mechanism," said Dong-Gun Kim, chief executive officer of Elevar. "We will continue to advance the regulatory processes in the United States and European Union while broadening the therapeutic potential of lirafugratinib across multiple solid tumors through our tumor-agnostic clinical development program."
Selectivity Distinguishes the Agent From Pan-FGFR Inhibitors
Lirafugratinib (also known as RLY-4008) is a potent, selective, oral small molecule inhibitor of FGFR2 (搜索), a receptor tyrosine kinase frequently altered in certain cancers. FGFR2 is one of four members of the FGFR family, a set of closely related proteins with highly similar protein sequences and properties.
The agent is an irreversible inhibitor that selectively targets FGFR2 (搜索), differentiating it from existing pan-FGFR inhibitors that broadly inhibit FGFR1, FGFR3 and/or FGFR4 in addition to FGFR2. Consistent with this high selectivity, the rates of hyperphosphatemia and diarrhea — adverse events commonly associated with non-selective FGFR inhibitors — were 20.7% and 21.6%, respectively.
Elevar holds an exclusive license to lirafugratinib from Relay Therapeutics, Inc. for commercialization worldwide.
Parallel U.S. Review and Tumor-Agnostic Development
Lirafugratinib received Orphan Drug Designation from the U.S. Food and Drug Administration (搜索) (FDA) in 2022 and Breakthrough Therapy Designation in 2023. Elevar submitted a New Drug Application for lirafugratinib to the FDA in January 2026, and the application was granted Priority Review. The FDA is currently reviewing the application, with a Prescription Drug User Fee Act target action date of Sept. 25, 2026.
Beyond CCA, Elevar is conducting ReFocus202, a global Phase 2 study evaluating lirafugratinib in patients with various solid tumors harboring FGFR2 (搜索) fusions or rearrangements. Patient enrollment and dosing are underway at clinical sites globally, including in the EU, U.K., Korea and U.S. Through this program, the company seeks to expand the therapeutic potential of lirafugratinib beyond CCA to a broader range of solid tumors with an FGFR2 fusion or rearrangement.
The trial is currently enrolling additional patients with previously treated, advanced or metastatic solid tumors other than CCA harboring FGFR2 (搜索) fusion or rearrangement who have not been treated with prior FGFR inhibitors.
Elevar notes in its forward-looking statements that FDA or EMA approval does not guarantee commercial success or any particular level of market adoption, reimbursement, revenue generation, or clinical outcomes in broader patient populations.