Virtually no effective therapies are available for the substantial proportion of patients with cholangiocarcinoma with disease progression after surgery and standard-of-care chemotherapy with gemcitabine plus cisplatin. Now, results of the single-arm phase II FIGHT-202 trial provide evidence of clinical benefit with the FGFR1–3 inhibitor pemigatinib in this setting.
FIGHT-202 involved patients with locally advanced or metastatic cholangiocarcinoma harbouring FGFR2 fusions or rearrangements (n = 107), other alterations in FGF or FGFR (n = 20) or no alterations in these genes (n = 18), with disease progression after at least one line of systemic therapy. At a median of 17.8 months, 38 patients with FGFR2 fusions or rearrangements (35.5%; 95% CI 26.5–45.4) had an objective response, including three complete responses. No responses were observed in the other two subgroups. The median progression-free survival durations were 6.9 months, 2.1 months and 1.7 months in patients with FGFR2 fusions or rearrangements, other FGF or FGFR alterations and no alterations, respectively. Although data were not mature at the time of reporting, the median overall survival durations were 21.1 months, 6.7 months and 4.0 months, respectively. The incidence of grade ≥3 and serious AEs was 64% and 45%, respectively; 6 patients had fatal AEs that were not deemed to be treatment-related.
The results of FIGHT-202 suggest that the 10–16% of patients with cholangiocarcinoma harbouring FGFR2 fusions might derive considerable benefit from pemigatinib. These results warrant further studies including an active comparator arm to further establish the clinical benefit from premigatinib in this population.
Abou-Alfa, G. K. et al. Pemigatinib for previously treated, locally advanced or metastatic cholangiocarcinoma: a multicentre, open-label, phase 2 study. Lancet Oncol. https://doi.org/10.1016/S1470-2045(20)30109-1 (2020)
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Romero, D. Benefit from pemigatinib in cholangiocarcinoma. Nat Rev Clin Oncol 17, 337 (2020). https://doi.org/10.1038/s41571-020-0369-z