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News
A marketing authorization application has been submitted to the European Medicines Agency for lirafugratinib, a highly selective FGFR2 inhibitor, as a second-line treatment for metastatic cholangiocarcinoma. Backed by the ReFocus trial, the drug showed a 46.5% response rate and a median overall survival of nearly two years. It already has FDA priority review status in the US.
A marketing authorization application (MAA) has been submitted to the European Medicines Agency (EMA) for lirafugratinib, an oral, selective FGFR2 inhibitor, as a second-line treatment for patients with advanced or metastatic cholangiocarcinoma (bile duct cancer) harboring FGFR2 fusions or rearrangements. The submission is supported by data from the phase 1/2 ReFocus trial.
What sets lirafugratinib apart from existing FGFR inhibitors is its precision — it selectively targets FGFR2 rather than the entire FGFR family, reducing off-target side effects like hyperphosphatemia and diarrhea. It also works as an irreversible inhibitor, forming a lasting bond with its target, which may help overcome resistance mutations seen with reversible drugs.
Why it matters: Cholangiocarcinoma is a rare and aggressive cancer with limited second-line options. Lirafugratinib's strong efficacy data and cleaner safety profile — compared to pan-FGFR inhibitors — could offer a meaningful new option for patients in Europe. The drug already holds FDA priority review status in the US, signaling momentum on both sides of the Atlantic.
