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Pemigatinib Shows ‘Therapeutic Potential’ For FGFR2-Altered Cholangiocarcinoma

Pemigatinib shows second-line activity against locally advanced or metastatic cholangiocarcinoma with fibroblast growth factor receptor 2 gene alterations
24 Mar 2020
Anticancer Agents;  Clinical Research;  Hepatobiliary Cancers;  Personalised/Precision Medicine

Author: By Lynda Williams, Senior medwireNews Reporter 


medwireNews: FIGHT-202 study findings suggest that locally advanced or metastatic cholangiocarcinoma patients with fibroblast growth factor receptor 2 (FGFR2) fusions or rearrangements may benefit from a potent oral FGFR1, 2 and 3 inhibitor treatment. 

Ghassan Abou-Alfa, from Memorial Sloan Kettering Cancer Center in New York, USA, and co-investigators say that the phase II trial results “support the therapeutic potential of pemigatinib in previously treated patients”. 

They report that 35.5% of 107 patients with FGFR2 fusions or rearrangements achieved an objective response to a daily pemigatinib dose of 13.5 mg given for 2 of every 3 weeks and 82.0% achieved disease control. This included three complete and 35 partial responses over a median follow-up of 15.4 months and a median treatment duration of 7.2 months. 

This antitumour activity “compares favourably with that reported for other second-line chemotherapy and targeted therapies”, the team says, although they “advise caution” as the overall survival data for the pemigatinib-treated patients are yet to mature. 

None of the 20 patients with other types of FGF or FGFR alterations or the 18 patients without any alterations achieved an objective response to treatment, and stable disease was achieved in 40.0% and 22.2%, respectively. 

Median progression-free survival was 6.9 months for patients with FGFR2 alterations, 2.1 months for patients with other FGF/FGFR alterations and 1.7 months for those with no alterations in these genes. 

The researchers report that 64% of patients experienced grade 3 or more severe adverse events, including hypophosphataemia (12%), arthralgia (6%), stomatitis (5%), hyponatraemia (5%), abdominal pain (5%) and fatigue (5%). 

And serious adverse events occurred in 45%, including abdominal pain (5%), pyrexia (5%), cholangitis (3%) and pleural effusion (3%). In all, 9% of patients discontinued treatment because of side effects and 14% required a dose reduction, but there were no treatment-related deaths. 

“Based on the encouraging findings from this study, an international, phase 3, randomised, active-controlled trial is currently recruiting patients to compare pemigatinib with gemcitabine plus cisplatin chemotherapy as first-line therapy for unresectable or metastatic cholangiocarcinoma with FGFR2 rearrangements (FIGHT-302)”, write Ghassan Abou-Alfa et al in The Lancet Oncology.

The authors of an accompanying comment note that “[c]ompared with previous studies of FGFR inhibitors, this study included the largest number of patients with advanced or metastatic cholangiocarcinoma and the results were robust.” 

While acknowledging that the response to pemigatinib in patients with FGFR2 alterations was “durable […], indicating a low drug resistance rate”, Feng Shen and co-authors, from Second Military Medical University in Shanghai, China, highlight the risk of acquired drug resistance for targeted therapies and the “need to comprehensively define the mutations that develop in response to FGFR inhibition in order to investigate novel inhibitors.” 

Nevertheless, they conclude that pemigatinib is “likely to add to the range of available treatments for cholangiocarcinoma with FGFR2 fusions or rearrangements, especially because existing systemic therapies offer inadequate efficacy.” 


Abou-Alfa GK, Sahai V, Hollebecque A, et al. Pemigatinib for previously treated, locally advanced or metastatic cholangiocarcinoma: a multicentre, open-label, phase 2 study. Lancet Oncol; Advance online publication 20 March 2020. https://doi.org/10.1016/S1470-2045(20)30109-1

Yang T, Liang L, Wang M-D, Shen F. FGFR inhibitors for advanced cholangiocarcinoma. Lancet Oncol; Advance online publication 20 March 2020. https://doi.org/10.1016/S1470-2045(20)30152-2

medwireNews (www.medwireNews.com ) is an independent medical news service provided by Springer Healthcare. © 2020 Springer Healthcare part of the Springer Nature group

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