# Pemigatinib in the Real-World Management of Cholangiocarcinoma Through a Canadian Patient Support Program

## TL;DR

This study shows that pemigatinib is effective and well-tolerated in real-world Canadian patients with advanced cholangiocarcinoma linked to FGFR2 genetic changes.

## Contribution

The study provides real-world evidence of pemigatinib's effectiveness and safety in Canadian patients with FGFR2-positive cholangiocarcinoma.

## Key findings

- Over half of patients showed a measurable response to pemigatinib.
- Nearly 90% of patients had disease control, with an average delay in disease progression of one year.
- No patients discontinued treatment due to side effects.

## Abstract

Cholangiocarcinoma is a rare and aggressive cancer of the bile ducts. For some patients, this cancer is linked to a genetic change in the FGFR2 protein. In 2021, Health Canada approved pemigatinib as a targeted therapy for patients with previously treated, unresectable, locally advanced or metastatic cholangiocarcinoma with an FGFR2 fusion or rearrangement. However, there is little real-world data on the use of pemigatinib in these patients in the Canadian setting. This study included 18 patients across six provinces who received pemigatinib through a patient support program. Most had advanced disease, and many had already received several lines of chemotherapy. After starting pemigatinib, over half showed a measurable response in their cancer, and nearly 90% had some level of disease control. On average, pemigatinib delayed disease progression for approximately one year. Importantly, none of the patients stopped treatment because of side effects. These results are comparable to those from earlier clinical trials, suggesting pemigatinib is effective and well tolerated in real-world settings. These findings reinforce the clinical value of pemigatinib for Canadian patients with cholangiocarcinoma and underscore the need for timely access to both targeted therapies and comprehensive genetic testing to ensure patients receive the most effective, personalized care.

Background: In September 2021, pemigatinib received Health Canada approval for previously treated locally advanced/metastatic cholangiocarcinoma (CCA) with FGFR2 rearrangements/fusions. This retrospective study aimed to characterize the real-world management and outcomes of patients with CCA receiving pemigatinib through a Canadian patient support program (PSP). Methods: We evaluated a multi-centre case series of Canadian patients who were prescribed pemigatinib between September 2021 and January 2023 for eligible CCA diagnoses and enrolled in the PSP. The retrospective study data included demographic and disease-, treatment-, and outcome-related information, and these were collected using a survey of prescribing physicians. Results: Of the 26 patients who initiated pemigatinib in the PSP, we received survey responses for 18 (69%). Their median age was 57 years, 67% were female, 61% had stage IV disease, and 83% had intrahepatic CCA. Prior to pemigatinib, a partial hepatectomy was performed in 44% of the patients, and 66% of the patients received 2–4 prior lines of systemic therapy. All patients were treated with platinum-based regimens as the first-line treatment for unresectable/metastatic disease. The median follow-up time on pemigatinib was 12.6 (range: 2.3–28.4) months, and their median real-world progression-free survival (rwPFS) was 12.1 months (95% CI 7.2-NR). The physician-assessed objective response and disease control rates were 56% and 89%, respectively. For the nine patients who discontinued pemigatinib, the median treatment duration was 10.6 months (range: 0.8–21.7). Disease progression was the most common reason for discontinuation (89%). None discontinued due to adverse events. Conclusions: Objective response rates, disease control rates, and a PFS comparable to that in the phase 2 FIGHT-202 trial was reported with pemigatinib use in this Canadian PSP cohort.