Summary

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• Pemazyre (pemigatinib) is indicated 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 and for the treatment of adults with relapsed or refractory myeloid/lymphoid neoplasms (MLNs) with FGFR1 rearrangement.
• This summary is based on the review of two randomized controlled trial(s). ^[1-2]
• In the FIGHT-201 study for urothelial carcinoma, the Objective Response Rate (ORR) was 17.8% (95% CI: 10.9% to 26.7%) for continuous dosing and 23.3% (95% CI: 15.5% to 32.7%) for intermittent dosing, with a ~24% ORR observed in patients with FGFR3 mutation S249C.
• In the FIGHT-202 study for cholangiocarcinoma with FGFR2 fusions or rearrangements, the ORR was 35.5% (95% CI: 26.5% to 45.4%), including 3 complete responses and 35 partial responses.
• Progression-Free Survival (PFS) and Overall Survival (OS) in the FIGHT-201 study for continuous dosing were 4.0 months (95% CI: 3.5-4.2) and 6.8 months (95% CI: 5.3-9.1), respectively, while intermittent dosing showed PFS of 4.3 months (95% CI: 3.9-6.1) and OS of 8.9 months (95% CI: 7.5-15.2).
• In the FIGHT-201 study, common treatment-emergent adverse events in patients with FGFR3 mutations included diarrhea (44.6%), alopecia (42.7%), stomatitis (42.7%), and hyperphosphatemia (42.7%). Acquired FGFR3 secondary resistance mutations were also detected at progression in some patients.
• In the FIGHT-202 study, the most frequent all-grade adverse event was hyperphosphatemia (60%). Grade 3 or worse adverse events included hypophosphatemia (12%) and various others (5%-6% each). Serious adverse events such as abdominal pain and pyrexia (5% each) were reported, with a 49% mortality rate during the study, primarily due to disease progression and no treatment-related deaths.
• The population types included patients aged ≥18 years with FGFR3 mutations or fusions/rearrangements in urothelial carcinoma and patients with locally advanced or metastatic cholangiocarcinoma. Subgroups in urothelial carcinoma revealed limited clinical activity in patients with other FGF/FGFR alterations, while in cholangiocarcinoma, a higher response rate was observed in patients with FGFR2 fusions or rearrangements compared to other subgroups.