The US Food and Drug Administration (FDA) has approved Lyrfigtu (lirafugratinib) for the treatment of cholangiocarcinoma with FGFR2 fusion or other rearrangement.
Cholangiocarcinoma is a rare, aggressive cancer that forms in the bile duct. Patients with cholangiocarcinoma are often diagnosed at a later stage when the prognosis is poor. The disease can be classified based on whether the cancer is in the bile duct within the liver (intrahepatic cholangiocarcinoma [iCCA]) or outside the liver (extrahepatic cholangiocarcinoma).
Lirafugratinib is a potent, selective and oral small molecule inhibitor of FGFR2, a receptor tyrosine kinase that is frequently altered in certain cancers.
The approval is based on results from the phase 1/2 ReFocus clinical trial (NCT04526106). Lirafugratinib demonstrated a confirmed objective response rate of 46% and a median duration of response of 11.8 months. Median progression-free survival was 11.3 months, with a 12-month rate of 49.2%.
Additionally, the safety profile was predictable and manageable through dose adjustments.
To learn more about cholangiocarcinoma and other rare cancers, visit https://checkrare.com/diseases/cancers/
Tags: cancer, gastro, regulations, treatment