Ziihera(zanidatamab)

Ziihera (zanidatamab) is indicated as monotherapy for the treatment of adults with unresectable locally advanced or metastatic HER2-positive (IHC3+) biliary tract cancer (BTC) who have previously received at least one line of systemic therapy. The marketing authorization application was submitted on May 23, 2024, and a CHMP positive opinion was issued on April 25, 2025. Orphan medicinal product designation was granted on July 19, 2021. The drug received approval in 2022, with final results for conditional marketing authorization due by September 2029.

In efficacy studies, the confirmed Overall Response Rate (ORR) in the total population was 37.9% (95% CI: 27.7, 49.0%). Specifically, in Cohort 1, the confirmed ORR was 41.3% (95% CI: 30.4, 52.8%), with 3.8% achieving a Complete Response (CR) and 37.5% a Partial Response (PR). The median Duration of Response (DOR) was 4.7 months, and the median Progression-Free Survival (PFS) was 2.4 months. The Disease Control Rate (DCR) was 52.4%.

Regarding safety, treatment-emergent adverse events occurred in 96.6% of patients. The mean duration of treatment was 7.51 months, with a median duration of 5.06 months. Discontinuation due to radiographic progression was observed in 102 patients in Part 2 of Study 101, while 18 patients discontinued due to clinical progression.