Jazz Pharmaceuticals Completes Biologics License Application for Zanidatamab Targeting Previously Treated HER2-Positive Metastatic Biliary Tract Cancer
Wednesday, April 03, 2024
Jazz Pharmaceuticals plc (Nasdaq: JAZZ) has announced the completion of the rolling submission of a Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) for accelerated approval of zanidatamab, a HER2-targeted bispecific antibody, as a treatment for previously-treated, unresectable, locally advanced, or metastatic HER2-positive biliary tract cancer (BTC). This submission marks a significant step forward as zanidatamab could potentially become the first HER2-targeted therapy approved for BTC in the United States.
According to Rob Iannone, M.D., M.S.C.E., Executive Vice President and Global Head of Research and Development at Jazz Pharmaceuticals, the submission represents a critical milestone in bringing zanidatamab, a targeted therapeutic option, to patients suffering from HER2-positive BTC. He noted the particularly dismal prognosis associated with this type of cancer, with a five-year overall survival rate of less than 5%. Zanidatamab is a biparatopic HER2-targeted bispecific antibody designed to bind two distinct epitopes of HER2 simultaneously, resulting in multiple mechanisms of action. It is being evaluated as a potential treatment option for various HER2-expressing cancers, with ongoing Phase 3 trials in first-line (1L) BTC, 1L gastroesophageal adenocarcinoma (GEA), and previously treated breast cancer.
The BLA submission includes data from the Phase 2b HERIZON-BTC-01 trial, which assessed zanidatamab in previously treated HER2-positive BTC patients. In this trial, zanidatamab demonstrated a promising confirmed objective response rate (cORR) and a median duration of response (DOR). The safety profile of zanidatamab was manageable and tolerable, with a low incidence of adverse events leading to treatment discontinuation.
The Phase 3 HERIZON-BTC-302 trial has recently begun enrollment and will evaluate the efficacy and safety of zanidatamab in combination with standard-of-care therapy compared to standard-of-care therapy alone in 1L advanced or metastatic HER2-positive BTC.
Zanidatamab is an investigational bispecific antibody developed by Jazz Pharmaceuticals and BeiGene, Ltd., under license agreements from Zymeworks. It has received Breakthrough Therapy designation from the FDA for its development in patients with previously treated HER2 gene-amplified BTC, as well as Fast Track and Orphan Drug designations for various indications.
Biliary tract cancer (BTC), including gallbladder cancer and intrahepatic and extrahepatic cholangiocarcinoma, is a rare cancer type with a poor prognosis. Approximately 12,000 individuals are diagnosed with BTC annually across the U.S., Europe, and Japan, with most patients diagnosed with unresectable tumors. The human epidermal growth factor receptor 2 (HER2) is a validated target for antitumor therapy in other cancers, making it a promising target for the treatment of BTC.
Source: jazzpharma.com
