Pemigatinib: A Review in Advanced Cholangiocarcinoma
1Springer Nature, Private Bag 65901, Mairangi Bay, Auckland, 0754, New Zealand. demail@springer.com.
Targeted Oncology
|January 11, 2024
Summary
Pemigatinib is an effective targeted therapy for advanced cholangiocarcinoma (CCA) with FGFR2 fusions. This fibroblast growth factor receptor inhibitor demonstrated significant objective responses and manageable safety in a phase 2 study.
Area of Science:
- Oncology
- Pharmacology
- Genetics
Background:
- Cholangiocarcinoma (CCA) is a rare and aggressive cancer.
- FGFR2 gene fusions or rearrangements are key drivers in a subset of CCA.
- Limited effective treatment options exist for previously treated advanced CCA.
Purpose of the Study:
- To evaluate the efficacy and safety of pemigatinib in patients with advanced cholangiocarcinoma (CCA) harboring FGFR2 gene fusions or rearrangements.
- To assess objective response rate (ORR), progression-free survival (PFS), and overall survival (OS).
Main Methods:
- Single-arm, phase 2 study (FIGHT-202).
- Adult patients with previously treated, unresectable locally-advanced or metastatic CCA with FGFR2 alterations received pemigatinib.
- Treatment involved pemigatinib 13.5 mg orally once daily (2 weeks on, 1 week off).
Main Results:
- Objective response in just over a third of patients (ORR).
- Median progression-free survival (PFS) of 7.0 months and overall survival (OS) of 17.5 months.
- Generally well-tolerated with a manageable safety profile; hyperphosphatemia was the most common adverse event, mostly grade 1-2.
Conclusions:
- Pemigatinib provides a valuable targeted therapy option for pretreated patients with advanced CCA and FGFR2 alterations.
- The drug demonstrated significant clinical activity and an acceptable safety profile.
- Further confirmation is ongoing in a phase 3 study (FIGHT-302).
