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

Core Insights - The FDA has accepted and granted Priority Review for Jazz Pharmaceuticals' zanidatamab, targeting HER2-positive biliary tract cancer, with a PDUFA date set for November 29, 2024 [1][2] - Zanidatamab is positioned to be the first HER2-targeted treatment specifically indicated for patients with HER2+ locally advanced or metastatic biliary tract cancer [1][2] Company Overview - Jazz Pharmaceuticals is a global biopharma company focused on developing life-changing medicines for serious diseases, including a growing portfolio of cancer treatments [8] - The company is headquartered in Dublin, Ireland, and is committed to innovative research and development across oncology and neuroscience [8] Clinical Development - The BLA submission for zanidatamab is based on Phase 2b HERIZON-BTC-01 trial results, which showed a confirmed objective response rate of 41.3% in previously treated patients [2][4] - Ongoing Phase 3 HERIZON-BTC-302 trial aims to evaluate zanidatamab's efficacy and safety in combination with standard-of-care therapy for first-line advanced or metastatic HER2-positive biliary tract cancer [3] Regulatory Designations - Zanidatamab has received Breakthrough Therapy designation from the FDA for previously treated HER2 gene-amplified biliary tract cancers, along with two Fast Track designations [5] - The drug has also been granted Orphan Drug designations by both the FDA and the European Medicines Agency for biliary tract cancer and gastric cancer [5] Disease Context - Biliary tract cancer (BTC) accounts for less than 1% of all adult cancers globally and is associated with a poor prognosis, with approximately 12,000 new HER2+ BTC cases diagnosed annually in the U.S., Europe, and Japan [6]