Summary

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• Pemigatinib (Pemazyre) is used for the treatment of adults with previously treated, unresectable locally advanced or metastatic cholangiocarcinoma with a fibroblast growth factor receptor 2 (FGFR2) fusion or other rearrangement as detected by an FDA-approved test. Additionally, it is utilized for adults with relapsed or refractory myeloid/lymphoid neoplasms (MLNs) with FGFR1 rearrangement.
• Two randomized controlled studies were analyzed to gather information about this drug. The first study examined its efficacy and safety in patients with urothelial carcinoma, while the second study focused on patients with cholangiocarcinoma.
• The studies included adult populations who had previously received treatment for either unresectable locally advanced or metastatic cancer, highlighting Pemigatinib's role in later stages of treatment. The first study addressed urothelial carcinoma, and the second evaluated cholangiocarcinoma, demonstrating broad potential applicability across different cancers.
• For effectiveness, the objective response rate varied between continuous dosing and intermittent dosing among patients with urothelial carcinoma, whereas it showed relatively high effectiveness among patients with cholangiocarcinoma, specifically those having FGFR2 fusions/rearrangements.
• The safety profile revealed common adverse events, including diarrhea, alopecia, stomatitis, and hyperphosphatemia among patients with urothelial carcinoma; hyperphosphatemia was the most common all-grade adverse event among patients with cholangiocarcinoma, along with frequent grade 3+ side effects requiring careful monitoring and management.
• Direct comparisons to other medications are challenging without specific comparative data, but Pemigatinib represents a critical option, fulfilling unmet needs, especially within subgroups demonstrating certain FGFR alterations, hinting at its role as a targeted therapy option.