FDA Officials Grant Accelerated Approval to Zanidatamab-hrii for Previously Treated, Unresectable, Metastatic HER2-Positive Biliary Tract Cancer

On November 20, 2024, officials with the FDA granted accelerated approval to zanidatamab-hrii (Ziihera; Jazz Pharmaceuticals, Inc), a bispecific HER2-directed antibody, for previously treated, unresectable or metastatic HER2-positive (IHC 3+) biliary tract cancer (BTC), as detected by an FDA-approved test.¹

On the same approval day, the FDA granted Ventana Pathway anti-HER-2/neu (4B5) Rabbit Monoclonal Primary Antibody (Ventana Medical Systems, Inc/Roche Diagnostics) as a companion diagnostic device to aid in identifying patients with BTC who may be eligible for this treatment.

As reported in HERIZON-BTC-01 (NCT04466891), an open-label, multicenter, single-arm trial in 62 patients with unresectable or metastatic HER2-positive (IHC 3+) BTC, the objective response rate was 52% (95% confidence interval [CI], 39-65) and median duration of response was 14.9 months (95% CI, 7.4-not estimable).

The prescribing information contains a Boxed Warning for embryo-fetal toxicity. The most common adverse reactions reported in at least 20% of patients who received zanidatamab were diarrhea, infusion-related reactions, abdominal pain, and fatigue.

FDA officials used the Assessment Aid for their review, which is a voluntary submission from the applicant to facilitate the FDA’s assessment.

Reference

1. Jazz Pharmaceuticals announces US FDA approval of Ziihera (zanidatamab-hrii) for the treatment of adults with previously treated, unresectable or metastatic HER2-positive (IHC 3+) biliary tract cancer (BTC). Press release. PR Newswire. Published November 20, 2024. Accessed December 5, 2024. www.prnewswire.com/news-releases/jazz-pharmaceuticals-announces-us-fda-approval-of-ziihera-zanidatamab-hrii-for-the-treatment-of-adults-with-previously-treated-unresectable-or-metastatic-her2-positive-ihc-3-biliary-tract-cancer-btc-302312216.html