On May 29, the National Medical Products Administration (NMPA) approved the marketing of Zanidatamab, submitted by BeiGene, for the treatment of patients with unresectable locally advanced or metastatic biliary tract cancer (BTC) who have previously received systemic therapy and exhibit high HER2 expression (IHC 3+).
Zanidatamab is an investigational HER2-targeted bispecific antibody that can simultaneously bind two non-overlapping epitopes of the HER2 receptor, known as biparatopic binding. This unique design and increased binding results in multiple mechanisms of action, including dual HER2 signal blockade, removal of HER2 protein from the cell surface, and immune-mediated cytotoxicity leading to encouraging antitumor activity in patients.
Receptor tyrosine-protein kinase, commonly known as Human Epidermal Growth Factor Receptor 2 (HER2) or CD340, is located on the surfaces of cells. This protein is a member of the Epidermal Growth Factor Receptor (EGFR) family and is encoded by the Erythroblastic oncogene B (ERBB2) gene. The ERBB2 gene is vital for normal processes such as cell division, differentiation, and survival. Through various signaling pathways, HER2 receptors promote cell proliferation and inhibit apoptosis (programmed cell death). It is crucial to regulate these receptors to prevent uncontrolled cell growth. Cancers that overexpress or amplify the HER2 gene are referred to as HER2-positive malignancies. This gene serves as an important biomarker and is a key target for drug therapy.
However, when HER2 is amplified or overexpressed, unchecked cell proliferation and division accelerate the progression of cancer. This amplification or overexpression is usually found across various tumor types and could have important therapeutic significance for malignancies that are not usually expected to respond well to anti-HER2 treatments.
HER2-positivity is found in various types of malignancies like gastric, biliary tract, breast, esophageal, ovarian, and uterine cancers. The incidence, description, and treatment trends for individual HER2-positive tumors are present in Table . Recently, an association has been discovered with pancreatic and biliary tract cancers (BTC). Patients with HER2-positive cancers tend to have poorer prognosis compared to those with HER2-negative cancers, as these malignancies are more aggressive. However, new treatment strategies have shown promising results.
This approval is based on the results of the pivotal clinical study HERIZON-BTC-01 (NCT04466891). This was a global, multicenter, open-label, single-arm, Phase 2b study designed to evaluate the efficacy and safety of zanidatamab in patients with previously treated, unresectable, HER2-amplified advanced or metastatic BTC.
The study enrolled 87 BTC patients, including 62 with high HER2 expression. The results showed that Zanidatamab monotherapy demonstrated clinically meaningful efficacy in previously treated patients with HER2-high BTC. The independently reviewed confirmed objective response rate (ORR) was 51.6%, with a median duration of response (mDoR) of 14.9 months, a median progression-free survival (mPFS) of 7.2 months, and a median overall survival (mOS) of 18.1 months.
At present, there are still many clinical trials of new anti-cancer technologies in China seeking patients. Consultation on new drugs and technologies, you can contact Beijing South Region Oncology Hospital International Department.
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Post time: Jun-06-2025