BACKGROUND: Fibroblast growth factor receptor (FGFR) 2 gene alterations are 
involved in the pathogenesis of cholangiocarcinoma. Pemigatinib is a selective, 
potent, oral inhibitor of FGFR1, 2, and 3. This study evaluated the safety and 
antitumour activity of pemigatinib in patients with previously treated, locally 
advanced or metastatic cholangiocarcinoma with and without FGFR2 fusions or 
rearrangements.
METHODS: In this multicentre, open-label, single-arm, multicohort, phase 2 study 
(FIGHT-202), patients aged 18 years or older with disease progression following 
at least one previous treatment and an Eastern Cooperative Oncology Group (ECOG) 
performance status of 0-2 recruited from 146 academic or community-based sites 
in the USA, Europe, the Middle East, and Asia were assigned to one of three 
cohorts: patients with FGFR2 fusions or rearrangements, patients with other 
FGF/FGFR alterations, or patients with no FGF/FGFR alterations. All enrolled 
patients received a starting dose of 13·5 mg oral pemigatinib once daily (21-day 
cycle; 2 weeks on, 1 week off) until disease progression, unacceptable toxicity, 
withdrawal of consent, or physician decision. The primary endpoint was the 
proportion of patients who achieved an objective response among those with FGFR2 
fusions or rearrangements, assessed centrally in all patients who received at 
least one dose of pemigatinib. This study is registered with ClinicalTrials.gov, 
NCT02924376, and enrolment is completed.
FINDINGS: Between Jan 17, 2017, and March 22, 2019, 146 patients were enrolled: 
107 with FGFR2 fusions or rearrangements, 20 with other FGF/FGFR alterations, 18 
with no FGF/FGFR alterations, and one with an undetermined FGF/FGFR alteration. 
The median follow-up was 17·8 months (IQR 11·6-21·3). 38 (35·5% [95% CI 
26·5-45·4]) patients with FGFR2 fusions or rearrangements achieved an objective 
response (three complete responses and 35 partial responses). Overall, 
hyperphosphataemia was the most common all-grade adverse event irrespective of 
cause (88 [60%] of 146 patients). 93 (64%) patients had a grade 3 or worse 
adverse event (irrespective of cause); the most frequent were hypophosphataemia 
(18 [12%]), arthralgia (nine [6%]), stomatitis (eight [5%]), hyponatraemia 
(eight [5%]), abdominal pain (seven [5%]), and fatigue (seven [5%]). 65 (45%) 
patients had serious adverse events; the most frequent were abdominal pain 
(seven [5%]), pyrexia (seven [5%]), cholangitis (five [3%]), and pleural 
effusion (five [3%]). Overall, 71 (49%) patients died during the study, most 
frequently because of disease progression (61 [42%]); no deaths were deemed to 
be treatment related.
INTERPRETATION: These data support the therapeutic potential of pemigatinib in 
previously treated patients with cholangiocarcinoma who have FGFR2 fusions or 
rearrangements.
FUNDING: Incyte Corporation.