Incyte announced that Health Canada has granted a Notice of Compliance with conditions for Pemazyre® (pemigatinib), a selective fibroblast growth factor receptor (FGFR) inhibitor, for the treatment of adults with previously treated, unresectable locally advanced or metastatic cholangiocarcinoma with a fibroblast growth factor receptor 2 (FGFR2) fusion or other rearrangement. The conditional approval is based on data from the FIGHT-202 study evaluating pemigatinib as a treatment for patients with previously treated, locally advanced or metastatic cholangiocarcinoma. Study results demonstrated that in patients harbouring FGFR2 fusions or rearrangements (Cohort A), pemigatinib monotherapy resulted in an overall response rate (ORR) of 35.5% (primary endpoint) and median duration of response (DOR) of 9.1 months (secondary endpoint) with a median follow-up of 15.4 months. Adverse events observed included serous retinal detachment (SRD) and hyperphosphatemia. The most common adverse reactions (incidence =15%) are hyperphosphatemia, alopecia, diarrhea, fatigue, nail toxicity, dysgeusia, nausea, constipation, stomatitis, dry mouth, decreased appetite, vomiting, dry eye, arthralgia, abdominal pain, hypophosphatemia, dry skin, edema peripheral, weight decreased, headache, urinary tract infection, dehydration, hypercalcemia, and palmar-plantar erythrodysaesthesia syndrome. Cholangiocarcinoma is a rare cancer that forms in the bile duct. It is classified based on its anatomical origin: intrahepatic cholangiocarcinoma (iCCA) occurs in the bile duct inside the liver, and extrahepatic cholangiocarcinoma occurs in the bile duct outside the liver. Patients with cholangiocarcinoma are often diagnosed at a late or advanced stage when the prognosis is poor.1,2 The incidence of cholangiocarcinoma varies regionally and ranges between 0.3-3.4 per 100,000 in North America and Europe.1 FGFR2 fusions or rearrangements occur almost exclusively in iCCA, where they are observed in 10-16% of patients.3,4,5,6 FGFRs play an important role in tumour cell proliferation and survival, migration, and angiogenesis (the formation of new blood vessels). Activating fusions, rearrangements, translocations, and gene amplifications in FGFRs are closely correlated with the development of various cancers. About FIGHT-202: The FIGHT-202 is a multi-center, open-label, single-arm, Phase 2 study (NCT02924376) that evaluated the safety and efficacy of pemigatinib – a selective fibroblast growth factor receptor (FGFR) inhibitor – in adult (age =18 years) patients with previously treated, locally advanced or metastatic cholangiocarcinoma with documented FGFR2 fusion or rearrangement. Patients were enrolled into one of three cohorts – Cohort A (FGFR2 fusions or rearrangements), Cohort B (other FGF/FGFR genomic alterations) or Cohort C (no FGF/FGFR genomic alterations). All patients received 13.5 mg pemigatinib orally once daily (QD) on a 21-day cycle (two weeks on/one week off) until radiological disease progression or unacceptable toxicity. The primary endpoint of FIGHT-202 was overall response rate (ORR) in Cohort A, assessed by independent review per RECIST v1.1. Secondary endpoints include ORR in Cohorts B, A plus B, and C; and duration of response (DOR).