Molecular Profiling, Targeted Therapies and Immunotherapy – updates

Update 22 July 2021: Pemigatinib: NICE has now approved pemigatinib (Pemazyre®) for those with cholangiocarcinoma who have an FGFR2 fusion.  Pemigatinib is the first ever targeted therapy approved for CCA.  This approval will not only give those eligible patients in England and Wales access to an alternative to chemotherapy, but importantly this new NICE guidance now means molecular profiling will be mandated as standard of care and so opens the door for all those diagnosed with cholangiocarcinoma to have molecular testing carried out.  

For AMMF’s report on the NICE approval for pemigatinib, click here.
For the statement from NICE, click here

Update 07 February 2022: The Scottish Medicines Consortium (SMC) committee has now approved pemigatinib (Pemazyre®) for use by NHS Scotland for the treatment of adults with locally advanced or metastatic cholangiocarcinoma with an FGFR2fusion that has progressed after at least one prior line of systemic therapy.

The relevant Detailed Advice Document can be found here and a link to the Decision Explained public information summary can be found here.

Update 31 January 2024: Ivosidenib: NICE has now approved the targeted therapy ivosidenib (TIBSOVO®) for those with cholangiocarcinoma who have an IDH1 mutation, and who have already undergone one other treatment.  To see the full guidance from NICE, click here 

Immunotherapy

Update 10 January 2024: Durvlumab: NICE now officially recommend the immunotherapy durvalumab plus the gemcitabine and cisplatin combination as a first line treatment for those with locally advanced, unresectable, or metastatic cholangiocarcinoma (CCA) or gallbladder cancer in adults. All patients diagnosed with CCA or gallbladder cancer should discuss this option with their oncologist.  To download the full guidance from NICE click here