HLB Submits European Marketing Authorization Application for New Cholangiocarcinoma Drug

HLB announced that its U.S. subsidiary, Elevate Therapeutics, has submitted a Marketing Authorization Application (MAA) for 'Rylapugratinib', an FGFR2-targeted anticancer drug, to the European Medicines Agency (EMA).

According to HLB on September 15, this application is based on the results of the global phase 1/2 'ReFocus' clinical study. In patients with FGFR2 fusion-positive advanced or metastatic cholangiocarcinoma (CCA) who had previously received one or more systemic therapies, Rylapugratinib recorded an objective response rate (ORR) of 46.5% as the primary endpoint, based on evaluation by an Independent Review Committee (IRC). The median duration of response (mDOR), a key secondary endpoint, was 11.8 months. The median progression-free survival (mPFS) was 11.3 months, and the median overall survival (mOS) was 22.8 months.

Rylapugratinib is an irreversible inhibitor that selectively targets FGFR2 and is differentiated from existing non-selective FGFR inhibitors, which broadly inhibit FGFR1, FGFR3, and FGFR4. Due to this high selectivity, the incidence rates of hyperphosphatemia and diarrhea—commonly seen with FGFR inhibitors—were reported as 20.7% and 21.6%, respectively.

Rylapugratinib received Orphan Drug Designation (ODD) from the U.S. Food and Drug Administration (FDA) in 2022 and Breakthrough Therapy Designation (BTD) in 2023. In January of this year, Elevate submitted a New Drug Application (NDA) for Rylapugratinib to the FDA. The FDA designated it for Priority Review and is currently reviewing the application. The Prescription Drug User Fee Act (PDUFA) target date for review is September 25. In addition, Elevate is conducting the global phase 2 tumor-agnostic 'ReFocus202' trial of Rylapugratinib in various solid tumors with FGFR2 fusions and rearrangements.

Donggun Kim, CEO of Elevate Therapeutics, said, "Rylapugratinib is a new drug candidate that has demonstrated clinically meaningful efficacy and differentiated safety data in line with its high FGFR2 selectivity and sustained inhibition mechanism. We will ensure smooth progress in the approval process in both the U.S. and European Union, while also expanding Rylapugratinib’s therapeutic potential to a variety of solid tumors through tumor-agnostic clinical trials."

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