Phase 2 Trial Produces Promising Results for Pemigatinib in Cholangiocarcinoma

If approved, pemigatinib would be the first targeted therapy for patients with cholangiocarcinoma, a rare cancer that impacts the bile ducts. The disease is often diagnosed at a late or advanced stage.

Findings from a phase 2 study assessing pemigatinib in patients with previously treated, advanced cholangiocarcinoma are supporting Incyte’s planned submission for a New Drug Application for the selective fibroblast growth factor receptor (FGFR) inhibitor by the end of the year.

If approved, pemigatinib would be the first approved targeted therapy for patients with cholangiocarcinoma, a rare cancer that impacts the bile ducts. The disease is often diagnosed at a late or advanced stage.

“Patients with cholangiocarcinoma face a significant challenge as they cope with a life-threatening condition that is often diagnosed once it has progressed into late stages,” Arndt Vogel, MD, senior consultant and professor at Hannover Medical School, said in a statement. “As a physician, I am encouraged to see the data from the FIGHT-202 study, which demonstrate the potential that pemigatinib has to become an important and much needed target option for this patient population.”

Findings from the FIGHT-202 study, presented at the European Society of Medical Oncology 2019 Congress in Barcelona, Spain, showed that pemigatinib resulted in an overall response rate of 36% and median progression-free survival of 6.9 months following a median of 15 months of follow-up in patients with FGFR2 fusions or rearrangements.

The study included 3 cohorts: those with FGFR2 fusions or rearrangements, those with other FGF/FGFR genetic alterations, and those with no FGF/FGFR genetic alterations. All patients received 13.5 mg pemigatinib orally once daily on a 21-day cycle until disease progression or until unacceptable toxicity.

Three of the 107 patients with FGFR2 fusions or rearrangements achieved a complete response rate while 35 achieved a partial response. Among these patients, there was a disease control rate of 82% and the median duration of response was 7.5 months. Preliminary data on overall survival showed a median overall survival of 21.1 months.

“We are excited to share updated data for pemigatinib, which may provide a promising and targeted treatment approach for patients with cholangiocarcinoma harboring FGFR2 fusions or rearrangements,” said Peter Langmuir, MD, group vice president, targeted therapies, Incyte, in a statement. “Patients with advanced cholangiocarcinoma face a poor prognosis, and currently there is no standard of care beyond first-line chemotherapy.”

The safety analysis of 146 patients indicated that pemigatinib was generally well tolerated, with the most common treatment-emergent adverse event being grade 1 or 2 hyperphosphatemia. This adverse event was managed with a low phosphate diet, phosphate binders and diuretics, or dose reduction or interruption.

The most common grade 3 or higher treatment-emergent adverse event was hypophosphatemia (12%); however, none of the cases were considered clinically significant or serious and none resulted in a dose reduction or discontinuation.

In June, Incyte announced that the first patient had been treated in a phase 3 trial comparing pemigatinib with gemcitabine with cisplatin chemotherapy as first-line therapy for the diesase.

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