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NICE approves treatment for rare bile duct cancer

Published on 22/07/21 at 09:03am

NICE has approved Pemigatinib (Pemazyre) as first treatment for people with a rare form of bile duct cancer (cholangiocarcinoma).

Pemigatinib, made by the Incyte Corporation, is recommended for treating locally advanced or metastatic cholangiocarcinoma with a fibroblast growth factor receptor 2 (FGFR2) fusion or rearrangement.

The treatment is an option for adults whose cancer has progressed after chemotherapy, around 50 people will be eligible for the treatment.

As the number of people in the clinical trial was so small, robust data collecting was difficult. However, clinical evidence suggests that pemigatinib works better than current treatments at this stage of cancer.

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after the company was able to work with us in addressing the concerns highlighted by the committee in the previous draft guidance.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.”

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.

"Over the past 20 years we have tirelessly campaigned for more research and awareness of this neglected cancer on behalf of patients and their loved ones. This NICE decision will not only give eligible patients in England and Wales access to an alternative to chemotherapy, but it opens the door for cholangiocarcinoma patients to have molecular testing.

“Molecular testing signals a new dawn in our understanding of cholangiocarcinoma, bringing us one important step closer to finding further new treatment options for this devastating cancer."

Patients with cholangiocarcinoma − often referred to as one of the ‘Cinderella' cancers because of its neglected status in regard to awareness and available treatments − are frequently diagnosed too late to benefit from surgery, currently the only potentially curative treatment. 

In the UK around 2,430 people are diagnosed with cholangiocarcinoma every year. Around 70% of these are diagnosed with unresectable, locally advanced, or metastatic disease, and these patients have an estimated five-year survival rate of less than 10%.

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