Zanidatamab Granted Priority Review for HER2-Positive Metastatic Biliary Tract Cancer
Target Action (PDUFA) Date set for
If approved, zanidatamab will be the first HER2-targeted treatment specifically indicated for patients with HER2+ locally advanced or metastatic biliary tract cancer
"The priority review designation for zanidatamab underscores the critical need for new treatment options for patients with locally advanced or metastatic HER2-positive BTC, a devastating disease with a poor prognosis," said
Jazz's BLA submission is based on results from Cohort 1 of the Phase 2b HERIZON-BTC-01 clinical trial (NCT05152147) of zanidatamab in previously treated patients with unresectable, locally advanced, or metastatic HER2-positive BTC (defined as in situ hybridization [ISH] positive and immunohistochemistry [IHC] 2+ or 3+). The trial demonstrated a primary endpoint of 41.3% [95% confidence interval (CI): 30.4, 52.8] confirmed objective response rate (cORR) by independent central review (ICR) and results were presented at the
Additionally, the global, open-label, randomized HERIZON-BTC-302 Phase 3 trial (NCT06282575) to evaluate the efficacy and safety of zanidatamab in combination with standard-of-care therapy against standard-of-care therapy alone in first-line advanced or metastatic HER2-positive BTC is ongoing and is open for enrollment. HERIZON-BTC-302 is expected to serve as the confirmatory trial for zanidatamab in BTC.
About Zanidatamab
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. Zanidatamab is being developed in multiple clinical trials as a targeted treatment option for patients with solid tumors that express HER2. Zanidatamab is being developed by Jazz and BeiGene, Ltd. (BeiGene) under license agreements from Zymeworks, which first developed the molecule.
The U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy designation for zanidatamab development in patients with previously treated HER2 gene-amplified biliary tract cancers (BTC), and two Fast Track designations for zanidatamab: one as a single agent for refractory BTC and one in combination with standard of care chemotherapy for 1L gastroesophageal adenocarcinoma (GEA). Additionally, zanidatamab has received Orphan Drug designations from FDA for the treatment of BTC and GEA, as well as Orphan Drug designation from the European Medicines Agency for the treatment of BTC and gastric cancer. Zanidatamab was also granted Breakthrough Therapy designation from the Center for Drug Evaluation (CDE) in China.
About Biliary Tract Cancer
BTC, including gallbladder cancer and intrahepatic and extrahepatic cholangiocarcinoma, account for <1% of all adult cancers globally and are often associated with a poor prognosis1,2. The human epidermal growth factor receptor 2 (HER2) is a well-validated target for antitumor therapy in other cancers. Across the
About
Jazz Pharmaceuticals plc
Caution Concerning Forward-Looking Statements
This press release contains forward-looking statements, including, but not limited to, statements related to our opportunity to deliver this treatment option to the BTC community with the potential to be the first HER2-targeted treatment specifically indicated for HER2+ BTC and other statements that are not historical facts. These forward-looking statements are based on
Contacts:
Jazz Media Contact:
Head of Global Strategic Brand Engagement
CorporateAffairsMediaInfo@jazzpharma.com
Jazz Investor Contact:
Vice President, Head, Investor Relations
investorinfo@jazzpharma.com
References:
1 Valle JW, et al.
2 Siegel RL, et al. CA Cancer J Clin 2022; 72;7-33
3 BTC overall diagnosed patients as per SEER 22
4 Assumes anatomic subsites intrahepatic CCA, extrahepatic CCA, gallbladder cancer, and BTC unspecified
5 Assumes HER2 positivity rates per anatomical subsite from: Galdy, S., Lamarca, A., McNamara, M.G. et al. Cancer Metastasis Rev 36, 141–157 (2017),
6 Major markets: U.K,
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