FDA approves Pemigatinib for CCA

The US pharmaceutical company, Incyte, has gained accelerated approval from the FDA (the US Food and Drug Administration), for their targeted therapy Pemazyre (pemigatinib) for the treatment of cholangiocarcinoma, (bile duct cancer)1.

This follows the outcome of the clinical trial, FIGHT-202, in which adult patients with previously treated, unresectable locally advanced or metastatic cholangiocarcinoma with an FGFR2 fusion or rearrangement received pemigatinib.

Whilst this is an important milestone as pemigatinib is the first targeted treatment for cholangiocarcinoma to receive FDA approval, AMMF understands from its advisors that this treatment is not yet available to UK or European CCA patients for the following reasons:

  • In the UK we are still bound by EMA2 (European Medicines Agency) approval; we are currently awaiting an update from Incyte re EU submission. Until that approval is received, it will not be licensed in the UK.
  • After EMA approval, NICE3 approval for the drug will be required; this would be linked to NICE approval for molecular testing – again something that is not regularly available for CCA patients in the UK outside of clinical trials.

However, it is important to bear in mind that even where this is currently approved for use (USA), it is for treatment following failure of chemotherapy. So newly diagnosed patients should not delay starting standard-of-care treatment.

The response rates to this treatment that have been seen so far appear to be consistent whenever it is given – it is not currently believed to be worse to receive FGFR treatment later, than if it had been given earlier.

AMMF’s advisors strongly advise newly diagnosed CCA patients to take part in the first-line clinical studies – for example, FIGHT-3024.  For more details on this and other clinical trials for CCA, see:  https://ammf.org.uk/clinical-trials/

1 To read the press release from the FDA, go to: www.fda.gov/news-events/press-announcements/fda-approves-first-targeted-treatment-patients-cholangiocarcinoma-cancer-bile-ducts

2EMA: the European Medicines Agency

3NICE: The UK’s National Institute for Health and Care Excellence

4FIGHT-302: Phase 3 Study of First-line Pemigatinib Versus Gemcitabine Plus Cisplatin for Cholangiocarcinoma With FGFR2 Fusions or Rearrangements.



22 April 2020