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  5. Drug Trials Snapshots: ZIIHERA
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Drug Trials Snapshots: ZIIHERA

HOW TO USE THIS SNAPSHOT 
The information provided in Snapshots highlights who participated in the key clinical trials that supported the original FDA approval of this drug, and whether there were differences among sex, race, age, and ethnic groups. The “MORE INFO” bar shows more detailed, technical content for each section. The Snapshot is intended as one tool for consumers to use when discussing the risks and benefits of the drugs.

LIMITATIONS OF THIS SNAPSHOT 
Do not rely on Snapshots to make decisions regarding medical care. Always speak to your healthcare provider about the benefits and risks of a drug.

Some of the information in this Snapshot is for presentation purposes and does not represent the approved conditions of use of this drug. Refer to the ZIIHERA Prescribing Information for all the approved conditions of use of this drug (e.g., indication(s), population(s), dosing regimen(s), safety information).

Snapshots are limited to the information available at the time of the original approval of the drug and do not provide information on who participated in clinical trials that supported later approvals for additional uses of the drug (if applicable).

ZIIHERA (zanidatamab-hrii) 
(zye-HAYR-rah) 
Jazz Pharmaceuticals Ireland Limited 
Approval date: November 20, 2024


DRUG TRIALS SNAPSHOT SUMMARY:

What is the drug for?

ZIIHERA is a bispecific HER2-directed antibody that is used to treat adults with previously treated, unresectable or metastatic HER2-positive (immunohistochemistry [IHC 3+]) biliary tract cancer (BTC), as detected by an FDA-approved test.

How is this drug used?

ZIIHERA is an injection. It is given by a healthcare professional directly into the vein (intravenous infusion) once every two weeks.

Who participated in the clinical trials?

FDA granted accelerated approval to ZIIHERA based predominantly on evidence from Cohort 1 of HERIZON-BTC-01 (NCT04466891) trial, which included 80 patients with previously treated unresectable or metastatic HER2-positive (IHC 2+ or 3+) BTC (cholangiocarcinoma or gall bladder cancer). The trial was conducted at 32 sites in 9 countries including Canada, Chile, China, France, Italy, the Republic of Korea, Spain, the United Kingdom, and the United States. This trial includes 17 patients treated in the United States.

The efficacy findings (N=62) represent the indicated population who had HER2 IHC 3+ expression and the safety findings (N=80) represents all study participants in Cohort 1 of HERIZON-BTC-01.

How were the trials designed?

ZIIHERA was evaluated in the HERIZON-BTC-01 study, which enrolled 87 patients with previously treated unresectable BTC in which there were extra copies of the gene that makes the HER2 protein. These patients were divided into two groups, Cohort 1 and Cohort 2. The main group (Cohort 1) had HER2 protein levels scored as intermediate (a score of 2+) or high (a score of 3+). Within this group of 80, there were 62 patients with a high score (known as IHC 3+). The second group included 7 patients who had low or no HER2 protein. All patients received ZIIHERA; there was no comparison group in the study. The trial measured how many patients had tumors that decreased in size after starting treatment, the length of time that the patient’s tumor responded to treatment, the amount of time that patients survived without disease progression, and how long patients lived. The analyses focused on the 80 patients in Cohort 1, and in particular on the 62 patients with high HER2 scores (IHC 3+).


DEMOGRAPHICS SNAPSHOT

Figure 1 summarizes how many male and female patients were enrolled in the clinical trial used to evaluate the efficacy of ZIIHERA.

Figure 1. Baseline Demographics by Sex, Efficacy Population

Pie chart summarizing how many male and female patients were in the clinical trial. In total, 28 (45%) male patients and 34 (55%) female patients participated in the clinical trial.

 

Source: Adapted from FDA Review

Figure 2 summarizes how many patients by race were enrolled in the clinical trial used to evaluate the efficacy of ZIIHERA.

Figure 2. Baseline Demographics by Race, Efficacy Population

Pie chart summarizing how many White, Asian, American Indian or Alaska Native, and not reported or unknown patients were in the clinical trial. In total, 19 (31%) White patients, 38 (61%) Asian patients, 1 (2%) American Indian or Alaska Native patient, and 4 (6%) not reported or unknown patients participated in the clinical trial.

Source: Adapted from FDA Review

Figure 3 summarizes how many patients by age were enrolled in the clinical trial used to evaluate the efficacy of ZIIHERA.

Figure 3. Baseline Demographics by Age, Efficacy Population

Pie chart summarizing how many patients by age were in the clinical trial. In total, 33 (63%) patients younger than 65 years of age and 29 (47%) patients 65 years of age and older participated in the clinical trial.

Source: Adapted from FDA Review

Figure 4 summarizes how many patients by ethnicity were enrolled in the clinical trial used to evaluate the efficacy of ZIIHERA.

Figure 4. Baseline Demographics by Ethnicity, Efficacy Population

Pie chart summarizing how many Hispanic, not Hispanic, and not reported patients were in the clinical trial. In total, 5 (8%) Hispanic or Latino patients, 55 (89%) not Hispanic or Latino patients, and 2 (3%) not reported patients participated in the clinical trial.

Source: Adapted from FDA Review

What are the benefits of this drug?

In the HERIZON-BTC-01 study 52% of patients with HER2-positive IHC3+ BTC had their tumors respond to treatment. The median duration of response was 14.9 months.

Were there any differences in how well the drug worked in clinical trials among sex, race, and age?

  • Sex: ZIIHERA worked similarly males and females.
  • Race: ZIIHERA worked similarly in all races.
  • Age: ZIIHERA worked similarly in patients younger and older than 65 years of age.

What are the possible side effects?

ZIIHERA may cause serious side effects including diarrhea, decrease in heart function, reactions during infusion, and harm to unborn baby.

The most commonly observed side effects (reported by greater than 20% of patients) include: diarrhea, reactions during infusion, abdominal pain, and fatigue.

Permanent discontinuation due to a side effect occurred in 2.5% of patients who received ZIIHERA.

Were there any differences in side effects among sex, race and age?

  • Sex: The occurrence of side effects was similar in males and females.
  • Race: The occurrence of side effects was similar in Asian and White patients.
  • Age: The occurrence of side effects was similar in patients younger and older than 65 years of age.

GLOSSARY

CLINICAL TRIAL: Voluntary research studies conducted in people and designed to answer specific questions about the safety or effectiveness of drugs, vaccines, other therapies, or new ways of using existing treatments. 
COMPARATOR: A previously available treatment or placebo used in clinical trials that is compared to the actual drug being tested. 
EFFICACY: How well the drug achieves the desired response when it is taken as described in a controlled clinical setting, such as during a clinical trial. 
PLACEBO: An inactive substance or “sugar pill” that looks the same as, and is given the same way as, an active drug or treatment being tested. The effects of the active drug or treatment are compared to the effects of the placebo. 
SUBGROUP: A subset of the population studied in a clinical trial. Demographic subsets include sex, race, and age groups.

PRESCRIBING INFORMATION

 

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