Zanidatamab Biliary Cancer Therapy: Advancing Targeted Treatment for HER2-Positive Patients

Zanidatamab biliary cancer therapy has been recommended by NICE for routine use in the NHS to treat adults with unresectable locally advanced or metastatic HER2-positive biliary tract cancer who have received at least one prior line of systemic therapy.

Targeted Option for Rare Aggressive Cancer

The committee concluded that zanidatamab biliary cancer therapy is likely to extend progression-free survival and overall survival compared with standard care. Given the poor prognosis of this aggressive disease, the most likely cost-effectiveness estimates fall within the range NICE considers an acceptable use of NHS resources. The treatment will be available in England within 90 days of final guidance publication.

In the pivotal HERIZON-BTC-01 trial, patients with IHC3-positive tumours achieved median progression-free survival of 7.2 months and median overall survival of 18.1 months. These results compare favourably against historical data for FOLFOX chemotherapy plus active symptom control. Real-world evidence from England and France further supported these findings, reinforcing the value of zanidatamab biliary cancer therapy in a population of approximately 50 eligible patients per year.

Addressing Longstanding Unmet Need

Biliary tract cancer is typically diagnosed late, with fewer than one in three patients in England surviving one year. Until now, second-line options after progression on standard immuno-chemotherapy have offered only modest survival gains and significant treatment burden. Zanidatamab biliary cancer therapy targets the 5–10% of cholangiocarcinomas and up to 20% of gallbladder cancers that overexpress HER2, filling a clear gap in care for this molecular subset.

Balanced Decision Despite Evidence Challenges

The appraisal accepted a naïve indirect comparison between the single-arm zanidatamab trial and the ABC-06 study. After rigorous review of survival modelling, utility values and uncertainty, the incremental cost-effectiveness ratio was judged to lie within an acceptable range when applying the severity weight. The very small patient population, confidential discount, and additional benefits such as reduced carer burden and lower hospital resource use helped support the positive recommendation.

(Source: National Institute for Health and Care Excellence)