Ziihera receives orphan drug designation in Korea

first HER2 bispecific antibody

Approved by the FDA in November last year… BeiGene owns domestic sales rights

Attention rises on biliary tract cancer treatment options...confirms efficacy through Phase IIb trial

The first HER2 bispecific antibody drug Ziihera has received an orphan drug designation in Korea.

 

The Ministry of Food and Drug Safety (MFDS) recently announced so through the first orphan drug designation in the new year.

 

Its specific indication is for the treatment of adult patients with previously treated unresectable locally advanced or metastatic HER2-positive (IHC3+) biliary tract cancer.

 

Ziihera (zanidatamab), a bispecific antibody that targets the HER2 gene, received accelerated approval from the U.S.

 

FDA in November last year after demonstrating efficacy in patients with biliary tract cancer.

 

This is the first time that a bispecific antibody targeting the HER2 gene has been used as a second-line treatment option for patients with biliary tract cancer who are identified as HER2-positive (IHC 3+).

 

Biliary tract cancer is a fatal disease with a poor prognosis and a low five-year survival rate of less than 5% for metastatic disease.

 

The company demonstrated Ziihera’s efficacy through the single-arm phase IIB HERIZON-BTC-01 study.

 

In the trial, the drug met the primary endpoint of confirmed objective response rate (cORR) by independent central review (ICR).

 

The objective response rate was 52%, and the median duration of response (DOR) was 14.9 months.

 

The safety profile of Ziihera was demonstrated in the HERIZON-BTC-01 trial in 80 patients.

 

During the study, 53% of patients treated with Ziihera experienced an adverse event.

 

The most common adverse events were diarrhea, infusion-related reactions, abdominal pain, and fatigue.

 

Serious adverse events occurring in 2% or more of patients were biliary obstruction, biliary infection, sepsis, pneumonia, diarrhea, gastric obstruction, and fatigue.

 

The trial results were presented at the 2023 American Society of Clinical Oncology (ASCO) Annual Meeting and published in The Lancet Oncology.

 

Long-term follow-up data showing improvement in the duration of response were reported at the 2024 ASCO Annual Meeting.

 

Meanwhile, BeiGene has the domestic rights to Ziihera.

 

The drug is currently being studied in multiple cancers, including Phase III trials in gastroesophageal adenocarcinoma (GEA) and metastatic breast cancer (mBC).

 

It is also currently being studied in the Phase III HERIZON-BTC-302 trial, which compares the combination of Ziihera and standard of care (Soc) to Soc alone in patients with HER2-positive biliary tract cancer.