Jazz Pharmaceuticals Gains Conditional EU Approval for Ziihera® to Treat Advanced HER2-Positive Biliary Tract Cancer
Wednesday, July 02, 2025
Jazz Pharmaceuticals plc has received conditional marketing authorisation from the European Commission (EC) for Ziihera® (zanidatamab) as a treatment for adults with unresectable locally advanced or metastatic HER2-positive biliary tract cancer (BTC) who have previously received at least one line of systemic therapy.
Biliary tract cancers, including gallbladder cancer and cholangiocarcinoma, are rare and aggressive, with most cases identified at a late stage when curative surgery is not an option. Globally, around 26% of BTC cases are HER2-positive, a status linked with poorer outcomes compared to HER2-negative disease.
Ziihera is the first HER2-targeted treatment to be conditionally authorised in the European Union for this patient group. Its continued approval will depend on the results of the ongoing Phase 3 HERIZON-BTC-302 trial, which is evaluating zanidatamab combined with standard therapy against standard therapy alone in newly diagnosed patients.
The European Commission's decision is based on findings from the Phase 2b HERIZON-BTC-01 trial, which assessed Ziihera in previously treated patients with advanced or metastatic HER2-positive BTC. This study included 87 participants, with 80 of them confirmed to have HER2-positive tumours. The trial met its main objective, showing a confirmed objective response rate (cORR) of 41.3%, as independently reviewed. Two patients had complete responses. The median duration of response was 14.9 months, and median overall survival was 15.5 months.
A subgroup analysis of patients with IHC 3+ tumours (n=62) showed even better outcomes, with a cORR of 51.6%, a median response duration of 14.9 months, and overall survival of 18.1 months.
Ziihera is administered as an intravenous infusion at a dose of 20 mg/kg every two weeks, continuing until disease progression or unacceptable side effects occur.
The safety of zanidatamab was evaluated in all 87 trial participants. The most common side effects included diarrhoea (46%), infusion-related reactions (33.3%), abdominal pain (26.4%), anaemia (25.3%) and fatigue (24.1%). Serious side effects were reported in 16.1% of patients, with the most frequent being diarrhoea, fatigue, and raised liver enzyme levels.
This conditional approval represents a key step for patients with limited treatment options in advanced HER2-positive BTC. It highlights the growing role of targeted therapies in addressing unmet medical needs, especially in rare cancers. The company is continuing to recruit patients for the Phase 3 study and is exploring the use of zanidatamab in other HER2-expressing cancers.
The EC's authorisation covers all European Union countries, as well as Iceland, Norway, and Liechtenstein.
More information, including full safety details and dosage guidelines, can be found in the Summary of Product Characteristics for Ziihera.
Source: prnewswire.com
