Jazz Pharmaceuticals announces FDA approval of Ziihera in BTC

Jazz Pharmaceuticals (JAZZ) “announced the U.S. Food and Drug Administration accelerated approval of Ziihera 50mg/mL for injection for intravenous use for the treatment of adults with previously treated, unresectable or metastatic HER2-positive (IHC 3+) biliary tract cancer, or BTC, as detected by an FDA-approved test.1 Ziihera was approved under accelerated approval based on a 52% objective response rate and a median duration of response of 14.9 months as determined by independent central review. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial.1 The Phase 3 HERIZON-BTC-302 confirmatory trial is ongoing to evaluate zanidatamab in combination with standard-of-care therapy versus standard-of-care therapy alone in the first-line setting for patients with HER2-positive BTC. The FDA approval of Ziihera is based on compelling data from the HERIZON-BTC-01 trial, which included the evaluation of zanidatamab as a single agent in previously treated HER2-positive BTC and is the largest Phase 2b clinical trial to date specifically for this patient population. The trial achieved its primary endpoint of confirmed objective response rate by independent central review and results were presented at the American Society of Clinical Oncology Annual Meeting 2023, published in The Lancet Oncology, and included in the 2023 Best of ASCO(R) program. Longer follow-up data showing improvement upon previously reported DOR were reported at the ASCO Annual Meeting 2024.”