培美替尼治疗胆管癌的效果好吗？

The American company Incyte develops an FGFR1/2/3 inhibitor. Pemetinib (4.5, 9, 13.5 mg) was approved by the US FDA for accelerated approval on April 20, 2020, for adult patients with previously treated unresectable locally advanced or metastatic cholangiocarcinoma associated with FGFR2 gene fusion or other rearrangements. So, is pemetinib effective in treating cholangiocarcinoma?

The effect of pemetinib in the treatment of cholangiocarcinoma

A multicenter, open-label, single-arm trial, WARR-202 (NCT 02924376), evaluated pemsire in 107 patients with locally advanced, unresectable, or metastatic cholangiocarcinoma whose disease had progressed on or after at least 1 prior line of therapy and with FGFR2 gene fusion or nonfusion rearrangements, as determined by clinical trial assays performed at a central laboratory. Eligible in-frame fusions and other rearrangements are predicted to have a breakpoint in intron 17/exon 18 of the FGFR2 gene, leaving the FGFR2 kinase domain intact.

Patients received 21 days of treatment with PEMAZYRE, 13.5 mg orally once daily for 14 days, followed by 7 days off. PEMAZYRE should be administered until disease progression or unacceptable toxicity. The primary efficacy outcome measures were overall response rate (ORR) and duration of response (DoR) as determined by an independent review committee (IRC) according to RECIST v1.1.

The median age was 56 years (range: 26 to 77 years), 61% were female, 74% were white, and 95% had baseline Eastern Cooperative Oncology Group (ECOG) performance status of 0 (42%) or 1 (53%). 98% of patients had intrahepatic cholangiocarcinoma. In-frame FGFR2 gene fusions were present in 86% of patients, with the most common FGFR2 fusion being FGF r 2-BIC 1 (34%). 14% of patients developed additional FGFR2 rearrangements that could not be confidently predicted as in-frame fusions, including rearrangements without an identifiable partner gene. All patients had received at least 1 prior systemic treatment regimen, 27% had 2 prior regimens, and 12% had 3 or more prior regimens. 96% of patients had received platinum-based therapy, of which 76% had received gemcitabine/cisplatin.

Efficacy results are summarized in Table 1. The median time to response was 2.7 months (range, 0.7–6.9 months).

Table 1: Efficacy results of war-202

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