A new targeted treatment for a rare and aggressive bile duct cancer has been approved by NICE, offering patients the chance of longer survival and improved quality of life
The targeted drug is designed for patients with advanced cholangiocarcinoma, an aggressive bile duct cancer that is often diagnosed at a late stage and has limited treatment options. Clinical evidence suggests it can help some patients live significantly longer, while also maintaining quality of life.
Clinicians and patient groups have welcomed this decision, emphasising that it marks a significant step forward in the treatment of a disease with historically low survival rates.
New targeted drug offers hope for patients with advanced bile duct cancer
Zanidatamab, also known as Ziihera and made by Jazz Pharmaceuticals, has been recommended by NICE in a final draft guidance for adults with HER2-positive advanced biliary tract cancer. HER2-positive means that the cancer cells have higher-than-normal levels of the protein HER2 (human epidermal growth factor receptor 2), which can help the cancer grow.
Bile tract cancer is often diagnosed late, when it can no longer be removed by surgery. Fewer than one in three people in England survive for a year after diagnosis, and until now, treatment options for patients whose cancer had progressed were limited.
Around 65 people a year in England are expected to be eligible for zanidatamab, which is given by intravenous infusion once every two weeks. It binds to a protein called HER2 on the surface of cancer cells and slows or stops their growth.
Trial results show significant survival gains as experts welcome NHS access
According to clinical trial data, patients treated with zanidatamab lived for an average of 18 months after starting the drug—substantially longer than the six-month average for those on standard chemotherapy.
Unlike standard chemotherapy, which is administered via a surgically implanted device, zanidatamab does not require surgical implantation, offering a less invasive treatment option.
“I am pleased we have been able to recommend this effective new treatment option, the first HER2-targeted drug NICE has recommended for people with biliary tract cancer in England. Patients and clinical experts involved in the appraisal told us how this treatment would make a huge difference by extending people’s lives and improving their quality of life – something that was not always possible with chemotherapy.
We are determined to ensure NHS patients in England have access to the most promising new treatments while providing value for the taxpayer,” said Helen Knight, director of medicines evaluation at NICE.
“AMMF welcomes NICE’s positive recommendation of zanidatamab. Until now, people with HER2-positive cholangiocarcinoma (bile duct cancer), gallbladder cancer, or ampullary cancer whose disease had progressed on first-line therapy had no targeted treatment options. During NICE’s appraisal, we heard from patients and their families about the positive difference zanidatamab provided for them. We are therefore pleased that more people with advanced HER2-positive disease will be able to access this life-extending treatment through the NHS,” added Helen Morement, CEO of AMMF – The Cholangiocarcinoma Charity.
“Cancer diagnosis can turn a family’s world upside down, and people diagnosed with this rare and aggressive cancer have had so few options for so long. Thanks to these changes, more people will have precious extra time with the people they love. Through the National Cancer Plan, this government is committed to making sure NHS patients get access to the best new treatments, and today’s recommendation delivers exactly that,” concluded Sharon Hodgson MP, Minister for Public Health.
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