The UK’s National Institute for Health and Care Excellence (NICE) has issued a positive recommendation for Incyte Biosciences UK’s Pemazyre for the treatment of the rare bile duct cancer cholangiocarcinoma (CCA).

NICE’s guidance recommends Pemazyre (pemigatinib) for the treatment of CCA with fibroblast growth factor receptor 2 (FGFR2) fusion or rearrangement that has progressed after at least one prior line of systemic therapy.

CCA is a rare type of liver cancer that forms in the bile duct, and is classified based on its origin – intrahepatic CCA occurs in the bile duct inside the liver while extrahepatic CCA occurs in the bile duct outside the liver.

In the UK, approximately 2,430 CCA patients are diagnosed each year with around 70% of these being diagnosed with unresectable, locally advanced or metastatic disease.

The NICE decision is based on data from the FIGHT-202 study, which evaluated the FGFR isoform 1,2 and 3 inhibitor in adult patients with previously treated, locally advanced or metastatic CCA with documented FGF/FGFR status.

In this study, among patients with FGFR2 fusion or rearrangements, Pemazyre monotherapy resulted in an overall response rate (ORR) of 37% and a median duration of response of eight months.

“Incyte welcomes this positive decision from NICE today which enables timely access to this innovative medicine for all eligible patients for whom rapid access to treatment is essential,”  said Mark Tanner, general manager, Incyte Biosciences UK.

“We are delighted NICE has acknowledged the value Pemazyre will bring to this fortunately rare but important group of patients, who until now have had extremely limited treatment options. We look forward to working in partnership with all those providing care for CCA patients for many years to come,” he added.