NICE approves treatment for rare form of bile duct cancer

The National Institute for Health and Care Excellence (NICE) has granted conditional marketing authorisation for a treatment targeting a rare form of bile duct cancer.

Biopharmaceutical company Incyte’s Pemigatinib treatment was recommended by NICE for treating locally advanced or metastatic cholangiocarcinoma with a fibroblast growth factor receptor 2 (FGFR2) fusion or rearrangement.

The treatment has been designed as an option for patients whose cancer has progressed after chemotherapy. It’s estimated that around 50 people will be eligible for Pemigatinib which was approved only after Incyte provided more information to NICE about the therapy’s cost-effectiveness.

In the UK, over 2,000 people a year are diagnosed with cholangiocarcinoma, an aggressive form of cancer which can bring with it a five-year survival rate of less than 10%, depending when it’s diagnosed.

The decision by NICE means that pemigatinib is the first treatment for this type of cancer to be approved in the UK and Europe. It’s also the first therapy for patients with locally advanced or metastatic cholangiocarcinoma who have the FGFR2 fusion.

Meindert Boysen, deputy chief executive and director of the Centre for Health Technology Evaluation at NICE said: “I am pleased we are able to recommend this life-extending treatment for people with this extremely rare form of bile duct cancer. Treatment options for this form of cancer have not improved in a long time and we recognise there is an urgent unmet clinical need for people who have this disease.

“Today’s decision comes after the company was able to work with us in addressing the concerns highlighted by the committee in the previous draft guidance. This recommendation is a great example of how NICE and companies, by working closely and collaboratively, can really help make a difference to patients.”

The UK’s only cholangiocarcinoma charity AMMF has welcomed the news.

Helen Morement, CEO of AMMF, said: This historic decision by NICE is warmly welcomed by AMMF and the patients and clinical communities we support. It gives hope to those eligible cholangiocarcinoma patients who will be able to access the first ever targeted treatment for this most challenging of cancers."