Jazz Pharmaceuticals has announced that the U.S. Food and Drug Administration (FDA) has granted accelerated approval for Ziihera (zanidatamab-hrii), a treatment for adults with previously treated, unresectable, or metastatic HER2-positive biliary tract cancer (BTC). This approval is based on data from the HERIZON-BTC-01 trial, which demonstrated a 52% objective response rate and a median duration of response of 14.9 months. The drug, developed in collaboration with Beigene, represents a significant advancement as it is the first dual HER2-targeted bispecific antibody approved for this indication in the U.S.

The FDA's decision was supported by the results of a mid-stage study involving 87 patients, where Ziihera showed promising efficacy. However, serious adverse reactions were reported in 53% of patients, including diarrhea, infusion-related reactions, and abdominal pain. Despite these challenges, the approval offers hope for patients with limited treatment options and a poor prognosis, as BTC has a five-year survival rate of less than 5% in the metastatic setting.

Jazz Pharmaceuticals, headquartered in Dublin, Ireland, is committed to further research on zanidatamab in BTC and other HER2-expressing solid tumors. The company plans to conduct a Phase 3 confirmatory trial to evaluate the drug in combination with standard-of-care therapy. This trial is crucial for converting the accelerated approval into a standard approval, contingent upon demonstrating clinical benefit.

The approval of Ziihera is a milestone in the treatment of BTC, providing a new option for patients and potentially improving their quality of life. Jazz Pharmaceuticals continues to innovate in the field of oncology, aiming to transform the lives of patients with serious diseases.