Priority Review for Zanidatamab in HER2-Positive Metastatic Biliary Tract Cancer

Dublin, May 29, 2024 — Jazz Pharmaceuticals plc announced that the U.S. Food and Drug Administration (FDA) has accepted its Biologics License Application (BLA) for zanidatamab. This human epidermal growth factor receptor 2 (HER2)-targeted bispecific antibody is proposed for the treatment of patients with previously treated, unresectable, locally advanced, or metastatic HER2-positive biliary tract cancer (BTC). The FDA has granted Priority Review status, setting the target action date under the Prescription Drug User Fee Act (PDUFA) for November 29, 2024. 

If approved, zanidatamab will be the first HER2-targeted therapy specifically for patients suffering from HER2+ locally advanced or metastatic BTC. Rob Iannone, M.D., M.S.C.E., Executive Vice President and Global Head of Research and Development at Jazz Pharmaceuticals, emphasized the importance of this designation, noting the critical need for new therapeutic options for these patients, who face a poor prognosis.

The BLA for zanidatamab is supported by data from Cohort 1 of the Phase 2b HERIZON-BTC-01 clinical trial (NCT05152147), which involved patients with unresectable, locally advanced, or metastatic HER2-positive BTC. The trial reported a confirmed objective response rate (cORR) of 41.3% by independent central review. These results were presented at the 2023 American Society of Clinical Oncology (ASCO) Annual Meeting and published in The Lancet Oncology. Further data on overall survival, updated duration of response, and additional long-term follow-up will be presented at the ASCO Annual Meeting in 2024.

In addition to the Phase 2b trial, the HERIZON-BTC-302 Phase 3 trial (NCT06282575) is currently underway. This global, open-label, randomized study is evaluating the efficacy and safety of zanidatamab in combination with standard-of-care therapy, compared to standard-of-care therapy alone, in first-line advanced or metastatic HER2-positive BTC. This trial is anticipated to confirm the efficacy of zanidatamab in BTC.

About Zanidatamab

Zanidatamab is an investigational bispecific antibody that targets HER2 by binding to two non-overlapping epitopes through biparatopic binding. This design facilitates multiple mechanisms of action, including dual HER2 signal blockade, removal of HER2 from the cell surface, and immune-mediated cytotoxicity, resulting in significant antitumor activity. Zanidatamab is being developed for various HER2-expressing solid tumors in collaboration with BeiGene, Ltd., under license agreements from Zymeworks.

The FDA has granted several designations to zanidatamab, including Breakthrough Therapy status for previously treated HER2 gene-amplified BTC, and two Fast Track designations: one as a single agent for refractory BTC and another in combination with chemotherapy for first-line gastroesophageal adenocarcinoma (GEA). Moreover, zanidatamab has received Orphan Drug Designations for BTC and GEA from the FDA, as well as the European Medicines Agency for BTC and gastric cancer. It has also been granted Breakthrough Therapy designation by China’s Center for Drug Evaluation (CDE).

About Biliary Tract Cancer (BTC)

Biliary tract cancer, which includes gallbladder cancer and intrahepatic and extrahepatic cholangiocarcinoma, accounts for less than 1% of all adult cancers worldwide and is associated with a poor prognosis. HER2 is a validated target for antitumor therapy in various cancers. Approximately 12,000 individuals are diagnosed annually with HER2+ BTC across the U.S., Europe, and Japan.

About Jazz Pharmaceuticals

Jazz Pharmaceuticals plc is a global biopharmaceutical company focused on developing life-changing medicines for patients with serious diseases. Their portfolio includes leading therapies for sleep disorders and epilepsy, as well as a growing selection of cancer treatments. Headquartered in Dublin, Ireland, Jazz Pharmaceuticals operates with research and development labs, manufacturing facilities, and employees worldwide, committed to serving patients.

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