Cancer Res Treat.  2024 Jul;56(3):847-855. 10.4143/crt.2023.1197.

Global Expanded Access Program for Pemigatinib in Patients with Previously Treated Locally Advanced or Metastatic Cholangiocarcinoma and Fibroblast Growth Factor Receptor Gene Alterations

• Lindley A
• Prager G
• Bitzer M
• Burn TC
• Lihou CF
• Croft E

• KMID: 2557671
• DOI: http://doi.org/10.4143/crt.2023.1197

Abstract

Purpose
Pemigatinib is a fibroblast growth factor receptor-2 (FGFR2) inhibitor approved for use in patients with previously treated cholangiocarcinoma (CCA) and FGFR2 fusions or rearrangements. This ongoing global Expanded Access Program (EAP) allows physicians in regions where pemigatinib is not commercially available to request pemigatinib for patients with locally advanced or metastatic CCA who, in the physician’s opinion, could benefit from pemigatinib treatment.
Materials and Methods
Eighty-nine patients from Europe, North America, and Israel were treated from January 2020 through September 2021.
Results
Patients had FGFR gene fusions (68.5%), rearrangements (12.4%), translocations (5.6%), amplifications (2.2%), and other alterations (11.2%). Median duration of treatment in the EAP was 4.0 months (range, 0.1 to 13.6 months). The most frequently reported adverse event (AE) was hyperphosphatemia (22.5%); the most common serious AE was cholangitis (3.4%). Treatment discontinuation was associated with reports of AEs for seven patients (7.9%). AEs associated with pemigatinib were consistent with those observed in clinical trials.
Conclusion
Efficacy was not assessed in this EAP. However, some patients remained on treatment for up to a year, suggesting that they observed a benefit from treatment. Patients with CCA should undergo molecular testing to identify those who could benefit from targeted treatments such as pemigatinib.

Keyword

Pemigatinib; Cholangiocarcinoma; fusions; rearrangements; Expanded Access Program

Figure

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  Fig. 1. Distribution of patients and FGFR2 tests in Europe (A), North America (B), and Israel (C). a)One patient had parallel testing with Illumina and Archer, b)One patient had parallel testing with Illumina and ThermoFisher Oncomine; therefore, percentages add up to > 100%.

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  Fig. 2. Duration of therapy. Gray arrow indicates the patient was still on treatment at data cutoff (September 30, 2021). *Indicates that the patient was reported to be discontinuing the Expanded Access Program due to the program being discontinued or being transitioned to commercially available pemigatinib. Patients for whom the specific type of FGFR2 or FGFR3 alteration was unreported were categorized generally as ‘FGFR2 alteration’ or ‘FGFR3 alteration.’

Reference

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