The U.S. Food and Drug Administration (FDA) has approved infigratinib (brand name: Trusetiq for the treatment of previously treated locally advanced or metastatic cholangiocarcinoma (CCA) harboring an FGFR2 gene fusion or rearrangement.

CCA is a rare 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).  FGFR2 fusions or rearrangements occur in about 10-16% of patients with iCCA. Fibroblast growth factor receptors (FGFRs) are believed to play an important role in tumor cell proliferation and survival, migration and angiogenesis. Infigratinib is an orally administered, ATP-competitive, tyrosine kinase inhibitor of FGFRs.

The approval of infigratinib is largely based on a phase 2 clinical study in which 108 patients who had undergone at least one prior treatment for advanced CCA received 125 mg of infigratinib daily for 21 days of 28-day cycles. The study’s primary endpoint demonstrated a confirmed objective response rate (ORR) of 23% (95% CI; 16–32%). The study also showed a median duration of response (DOR) of 5 months (95% CI; 3.7–9.3 months). Common adverse reactions include increased creatinine, increased phosphate, decreased phosphate, nail toxicity, stomatitis, increased alkaline phosphatase, decreased hemoglobin, increased alanine aminotransferase, dry eye, fatigue, increased lipase, decreased lymphocytes, increased calcium, decreased sodium, alopecia, increased triglycerides, increased aspartate aminotransferase, decreased platelets, increased urate, palmar-plantar erythrodysesthesia syndrome, arthralgia, and dysgeusia.

To learn more about CCA and other rare cancers, visit checkrare.com/diseases/cancers/