Jazz Pharmaceuticals plc (JAZZ) has announced that the U.S. Food and Drug Administration (FDA) has granted accelerated approval for Ziihera (zanidatamab-hrii) 50mg/mL for injection, intended for intravenous administration. This approval is specifically for treating adults with HER2-positive (IHC 3+), previously treated, unresectable, or metastatic biliary tract cancer.
The FDA's accelerated approval was informed by data demonstrating a 52% objective response rate and a median response duration of 14.9 months, as assessed by independent central review (ICR) from the HERIZON-BTC-01 clinical trial.
The ongoing approval for this use may require validation and detailed evidence of clinical benefits through a confirmatory trial. The Phase 3 HERIZON-BTC-302 confirmatory trial is currently underway, aiming to assess zanidatamab combined with standard-of-care therapy, compared to standard-of-care therapy alone, for first-line treatment in patients with HER2-positive biliary tract cancer (BTC).
Jazz Pharmaceuticals has highlighted the potential risk of embryo-fetal harm if Ziihera is administered during pregnancy, emphasizing the importance for patients to be mindful of this risk and to use effective contraception.
Zanidatamab is also under investigation for efficacy in several other cancer types, including Phase 3 trials for gastroesophageal adenocarcinomas (GEAs) and metastatic breast cancer (mBC). The HERIZON-GEA-01 trial is evaluating zanidatamab in combination with chemotherapy, with or without tislelizumab, as a first-line treatment for patients with advanced or metastatic HER2-positive GEAs. Meanwhile, the EmpowHER-303 trial examines zanidatamab combined with a physician's choice of chemotherapy for treating HER2-positive mBC in patients who have either progressed on or cannot tolerate a previous trastuzumab deruxtecan regimen.
