Pemigatinib for Previously Treated Advanced Cholangiocarcinoma

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In the phase II FIGHT-202 study reported in The Lancet Oncology, Ghassan K. Abou-Alfa, MD, and colleagues found that the fibroblast growth factor receptor 1 (FGFR1), FGFR2, and FGFR3 inhibitor pemigatinib was active in patients with previously treated locally advanced or metastatic cholangiocarcinoma with FGFR2 fusions or rearrangements.

Ghassan K. Abou-Alfa, MD

Ghassan K. Abou-Alfa, MD

As noted by the investigators, FGFR2 gene alterations are known to be involved in the pathogenesis of cholangiocarcinoma.

Study Details

In the study, 146 patients from sites in the United States, Europe, the Middle East, and Asia were enrolled between January 2017 and March 2019 into three cohorts: Those with FGFR2 fusions or rearrangements (n = 107); those with other FGF/FGFR alterations (n = 20); and those with no FGF/FGFR alterations (n = 18). One additional patient had an undetermined FGF/FGFR alteration.

Patients received a starting dose of oral pemigatinib at 13.5 mg once daily in a 21-day cycle with 2 weeks on and 1 week off until disease progression or unacceptable toxicity. The primary endpoint was the proportion of patients with FGFR2 fusions or rearrangements who achieved centrally assessed objective response.


Median follow-up was 17.8 months. An objective response was achieved in 38 (35.5%) of 107 patients with FGFR2 fusions or rearrangements, including a complete response in 3 patients. No responses were observed among the 20 patients with other FGF/FGFR alterations, the 18 with no FGF/FGFR alterations, or the 1 patient with an undetermined FGF/FGFR alteration.


  • Objective response was observed in 35.5% of patients with FGFR2 fusions or rearrangements.
  • No responses were observed among patients with other FGF/FGFR alterations or those with no FGF/FGFR alterations.

Median duration of response was 7.5 months among responders in the cohort with FGFR2 fusions or rearrangements, with 37% of responders maintaining response at 12 months. Progression-free survival was 6.9 months in this cohort, with a 12-month rate of 29%.

Adverse Events

Among all 146 patients, hyperphosphatemia was the most common any-grade adverse event (60%). Grade ≥ 3 adverse events occurred in 64% of patients, with the most common being hypophosphatemia (12%), arthralgia (6%), stomatitis (5%), hyponatremia (5%), abdominal pain (5%), and fatigue (5%). Serious adverse events occurred in 45% of patients, with the most common being abdominal pain (5%), pyrexia (5%), cholangitis (3%), and pleural effusion (3%).

Overall, 49% of patients died during the study; most deaths were related to disease progression (42%). No deaths were considered treatment-related.

The investigators concluded, “These data support the therapeutic potential of pemigatinib in previously treated patients with cholangiocarcinoma who have FGFR2 fusions or rearrangements.”

Dr. Abou-Alfa, of the Department of Medicine, Memorial Sloan Kettering Cancer Center, is the corresponding author for The Lancet Oncology article.

Disclosure: The study was funded by Incyte Corporation. For full disclosures of the study authors, visit