Pemigatinib (Pemazyre®) can provide symptomatic benefits to patients with cholangiocarcinoma (CCA) according to a small case series of its real-world clinical use published in Drugs in Context.

The patients in the case series experienced symptomatic relief, which improved their quality of life even when radiological evaluation indicated stable disease, the researchers noted. This benefit ranged between 6 and 14 months during the study, which was similar to the median duration of 9.13 months observed during the phase 2 FIGHT-202 clinical trial, they added.

Read more about CCA therapies

Continue Reading

In the first case report, a female patient experienced symptomatic benefit within 3 weeks of starting pemigatinib. These included improvements in nausea, early satiety, ascites, and performance status from 2 to 1. Treatment was continued for 10 cycles, each lasting 21 days, until clinical progression and worsening ascites were observed.

The second patient was started on pemigatinib after significant disease progression, and recurrent abdominal pain was observed. She had a rapid resolution of abdominal pain within 3 days of treatment initiation, which allowed her to resume daily activities. A partial response on CT scans was observed after 2 months of treatment. However, new neck and shoulder pain, along with disease progression on MRI, were observed after 5 months of treatment, leading to discontinuation.

The third clinical case involved a 67-year-old male who began treatment with pemigatinib. The treatment helped him achieve stable disease and led to a duration of benefit of 14 months before multifocal progression was observed and treatment was discontinued. The patient experienced several toxicity-related symptoms, including diarrhea, dry mouth, and painless nail changes during treatment, but most were grade 1 or 2 and did not interfere with routine daily activities.

“In conclusion, patients with extensive metastatic CCA with FGFR2 fusion or rearrangement can benefit from late-line pemigatinib therapy. In our practice, a significant and durable symptomatic benefit was obtained in patients receiving pemigatinib, even without a radiological response,” the authors wrote.

Pemigatinib is approved for the treatment of locally advanced or metastatic CCA with FGFR2 gene fusions or rearrangement. The authors noted that only around 50 patients have received treatment with it in the United Kingdom so far and that many oncologists have limited experience with its use. Due to this, they recommend routine monitoring of patients during the use of pemigatinib.


Franklin H. Clinical experience with pemigatinib for previously treated metastatic cholangiocarcinoma: practical considerations from clinical cases. Drugs in Context. Published August 14, 2023. Accessed August 30, 2023.