The Cholangiocarcinoma Foundation (CCF) celebrates the November 30, 2020 FDA breakthrough therapy designation for Zanidatamab in patients with previously-treated HER2 gene-amplified biliary tract cancer. A HER-2 or Human Epidermal Growth Factor Receptor 2 mutation occurs when cancer causes the HER-2 cell to produce too much protein, resulting in cells dividing and growing too fast. HER-2 is one of the many identified cancer mutations that can now be treated with targeted therapies.

“This Breakthrough Therapy designation from the FDA, based on data generated in BTC patients treated in the initial Phase 1 trial, is recognition of the potential of Zanidatamab to provide a new approach to cancer treatment,” said Diana Hausman, M.D., Chief Medical Officer at Zymeworks.

HER2 is a well-described target for anti-cancer therapy. Tumor cells that produce a higher than normal level of HER2 tend to grow more quickly and spread to other parts of the body. About 0.9 - 8.5% of intrahepatic cholangiocarcinoma and 11-20% of extrahepatic cholangiocarcinoma patients have genetic mutations that express HER2. Zanidatamab offers hope to this significant subset of patients with this disease.

“Cholangiocarcinoma patients typically face a difficult diagnosis and poor prognosis,” said Stacie Lindsey, CEO and Founder of CCF. “Receiving this designation from the FDA will bring hope to our patients as Zanidatamab has the potential to be the first HER2-targeting therapy approved for metastatic BTC patients.”

“The recent FDA breakthrough therapy represents a milestone for cholangiocarcinoma in hopes of providing new treatment option for patients whose tumors harbor HER2 gene fusions and in doing so, establishing the role for tumor mutation profiling in all cholangiocarcinoma patients and the feasibility of clinical trials in molecularly-defined subsets.” said Kate Kelley, MD, Co-Chair of the Scientific and Medical Advisory Board of the Cholangiocarcinoma Foundation.

This Breakthrough Therapy designation was based on an ongoing clinical trial of Zanidatamab in patients with locally advanced (unresectable) and/or metastatic HER2-expressing tumors including BTC.

Updated clinical data for single agent Zanidatamab patients with BTC has been accepted for presentation at the upcoming ASCO GI- January 15-17, 2021.

“The recent FDA breakthrough therapy represents a milestone for cholangiocarcinoma in hopes of providing new treatment option for patients whose tumors harbor HER2 gene fusions and in doing so, establishing the role for tumor mutation profiling in all cholangiocarcinoma patients and the feasibility of clinical trials in molecularly-defined subsets.”

Kate Kelley, MD, Co-Chair of the Scientific and Medical Advisory Board of the Cholangiocarcinoma Foundation.