Regulatory approval

Published by the Food and Drug Administration. Accelerated

The U.S. Food and Drug Administration (FDA) granted accelerated approval to zanitdatamab for the treatment of adult patients with previously treated, unresectable or metastatic HER2-positive (IHC 3+) biliary tract cancer (BTC), as detected by an FDA-approved test. The package insert states that this indication is approved under accelerated approval based on overall response rate and duration of response and that continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial(s).

This is written in the approval document as:

ZIHERA is a bispecific HER2-directed antibody indicated for the treatment of adults with previously treated, unresectable or metastatic HER2-positive (IHC 3+) biliary tract cancer (BTC), as detected by an FDA-approved test. This indication is approved under accelerated approval based on overall response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial(s).

Citation

Astellas Pharma US, Inc. Ziihera (zanidatamab-hrii) [package insert]. U.S. Food and Drug Administration website. https://www.accessdata.fda.gov/drugsatfda_docs/nda/2024/761416Orig1s000Lbl.pdf. Revised November 2024. Accessed January 10, 2025.

Therapeutic response

Precision oncology relationships for therapeutic response derived from this regulatory approval.

Organizations	Biomarkers	Cancer types	Therapies
FDA (2)	HER2-positive	Intraductal Papillary Neoplasm of the Bile Duct	Zanidatamab
FDA (2)	HER2-positive	Intracholecystic Papillary Neoplasm	Zanidatamab
FDA (2)	HER2-positive	Cholangiocarcinoma	Zanidatamab

Contributions

Date	Contributor	Description
2025-01-10	Van Allen lab	Additional FDA approvals for initial version
2024-11-20	Food and Drug Administration	Indication received accelerated approval.