FDA Approves Zanidatamab for Patients With Locally Advanced or Metastatic HER2-Positive Biliary Tract Cancer
On November 20th, 2024, the US Food and Drug Administration (FDA) approved zanidatamab for patients with unresectable locally advanced or metastatic human epidermal growth factor receptor 2 (HER2)-positive biliary tract cancer who experience disease progression on or after gemcitabine-based therapy. This accelerated approval was based on results from cohort 1 of the phase 2b HERIZON-BTC-01 trial.
In this multicenter, single-arm study, 40 patients with centrally confirmed and measurable HER2-amplified disease with a performance status of 0 or 1 and a HER2 immunohistochemistry score of 2+, 3+, or HER2-positive received 20 mg/kg of zanidatamab intravenously every 2 weeks. The primary end point was confirmed objective response rate (ORR) by independent central review. A key secondary end point was safety.
At a median follow-up of 12.4 months, the confirmed ORR was 41.3%. The most common grade 3 treatment-related adverse events included diarrhea (5%) and decreased ejection fraction (3%). No grade 4 treatment-related adverse events or deaths were reported.
Study investigator James Harding, MD, Memorial Sloan Kettering Cancer Center, New York, New York, noted "the significant unmet need for effective therapies for patients with [biliary tract and liver cancers].” He went on to state “Zanidatamab has demonstrated antitumor activity and is now a new option for patients with HER2-positive biliary tract cancer.”
Source:
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). Accessed on November 21, 2024. https://investor.jazzpharma.com/news-releases/news-release-details/jazz-pharmaceuticals-announces-us-fda-approval-ziiherar
Harding J, Fan J, Oh D-Y, et al. Zanidatamab for HER2-amplified, unresectable, locally advanced or metastatic biliary tract cancer (HERIZON-BTC-01): a multicentre, single-arm, phase 2b study. Lancet Oncol. 2023; 24(7):772-782. doi: 10.1016/S1470-2045(23)00242-5
