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 drug.
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 ANDEMBRY 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).
ANDEMBRY (garadacimab)
an-DEM-bree
CSL Behring
Original Approval date: June 17, 2025
DRUG TRIALS SNAPSHOT SUMMARY:
What is the drug for?
ANDEMBRY is a medication used to prevent attacks of hereditary angioedema (HAE) in patients 12 years of age and older.
HAE is a rare disease that runs in families. People with HAE have repeated episodes (attacks) of swelling without hives or itching. Swelling is most often in the skin, throat, airway, and stomach area.
How is this drug used?
ANDEMBRY is given once a month by injection under the skin.
Who participated in the clinical trials?
The FDA approved ANDEMBRY based on evidence from a clinical trial of 64 patients with HAE. The trial was conducted at 28 centers in seven countries including Canada, Germany, Hungary, Israel, Japan, Netherlands, and the United States. Among the 64 patients in the trial, 33% were from the United States.
How were the trials designed?
ANDEMBRY was tested in a clinical trial of 64 patients with HAE. In the trial, researchers wanted to see if ANDEMBRY was better than placebo in reducing HAE attacks. Patients were treated with ANDEMBRY or placebo once a month. The primary endpoint was the number of HAE attacks per month over a six-month treatment period.
How were the trials designed?
The safety and efficacy of ANDEMBRY were evaluated in a six-month, randomized, double-blind, placebo-controlled, parallel-group, phase 3 trial in patients 12 years of age and older with HAE. The trial included a two-month run-in period during which patients had to demonstrate two or more acute HAE attacks. Eligible patients were randomized in a 1:1 ratio to receive ANDEMBRY 200 mg or placebo every month over a six-month treatment period. The primary endpoint was the time-normalized number of investigator-confirmed HAE attacks per month during the six-month treatment period.
DEMOGRAPHICS SNAPSHOT
Figure 1 shows how many male and female patients were enrolled in the clinical trial used to evaluate the efficacy of ANDEMBRY.
Figure 1. Baseline Demographics by Sex, Efficacy Population
Source: Adapted from FDA Review
Figure 2 shows how many patients by race were enrolled in the clinical trial used to evaluate the efficacy of ANDEMBRY.
Figure 2. Baseline Demographics by Race, Efficacy Population
Source: Adapted from FDA Review
Figure 3 shows how many patients by age were enrolled in the clinical trial used to evaluate the efficacy of ANDEMBRY.
Figure 3. Baseline Demographics by Age, Efficacy Population
Source: Adapted from FDA Review
Figure 4 shows how many patients by ethnicity were enrolled in the clinical trial used to evaluate the efficacy of ANDEMBRY.
Figure 4. Baseline Demographics by Ethnicity, Efficacy Population
Source: Adapted from FDA Review
Who participated in the trials?
Table 1. Baseline Demographics of Efficacy Trial
| Characteristic | ANDEMBRY N=39 n (%) |
Placebo N=25 n (%) |
|---|---|---|
| Sex | ||
| Female | 24 (62) | 14 (56) |
| Male | 15 (38) | 11 (44) |
| Age group, years | ||
| <18 | 4 (10) | 2 (8) |
| 18 to 65 | 31 (80) | 23 (92) |
| >65 | 4 (10) | 0 |
| Race | ||
| White | 33 (84) | 22 (88) |
| Asian | 4 (10) | 2 (8) |
| Black or African American | 0 | 1 (4) |
| Hawaiian or Pacific Islander | 1 (3) | 0 |
| Other | 1 (3) | 0 |
| Ethnicity | ||
| Hispanic or Latino | 1 (3) | 2 (8) |
| Not Hispanic or Latino | 37 (94) | 23 (92) |
| Unknown | 1 (3) | 0 |
| Geographic region | ||
| United States | 12 (31) | 9 (36) |
| Rest of the world | 27 (69) | 16 (64) |
Source: Adapted from FDA Review
What are the benefits of this drug?
In patients 12 years of age and older with HAE, ANDEMBRY helped reduce the number of HAE attacks per month over a six-month period.
What are the benefits of this drug (results of trials used to assess efficacy)?
The trial demonstrated that treatment with ANDEMBRY was superior to placebo in patients 12 years of age and older with HAE. This was measured by the primary endpoint of the time-normalized number of investigator-confirmed HAE attacks per month during a six-month treatment period. ANDEMBRY showed a reduction in the time-normalized number of investigator-confirmed HAE attacks per month compared to placebo.
Table 2. Time-Normalized Number of Investigator-Confirmed HAE Attacks per Month, Efficacy Population
| Parameter | ANDEMBRY N=39 |
Placebo N=25* |
|---|---|---|
| LS mean (95% CI) | 0.2 (0.1, 0.5) | 2.1 (1.5, 2.9) |
| LS mean difference (95% CI) | -1.9 (-2.6, -1.0) |
|
| LS mean percent reduction (95% CI) | 89.2 (75.6, 95.2) |
|
Source: Adapted from FDA Review
* One patient in placebo group removed from analysis due to receiving less than 30 days of study treatment
Abbreviations: CI, confidence interval; HAE, hereditary angioedema; LS, least squares; N, number of patients in treatment group
Were there any differences in how well the drug worked in clinical trials among sex, race, age, and ethnicity?
- Sex: ANDEMBRY worked similarly in males and females.
- Race: The number of patients of races other than White or Asian was small; therefore, differences in how ANDEMBRY worked among races could not be determined.
- Age: ANDEMBRY worked similarly in patients between 12 to 18 years of age, between 18 to 65 years of age, and older than 65 years of age.
- Ethnicity: The study did not include a sufficient number of Hispanic or Latino patients; therefore, differences in how ANDEMBRY worked among ethnicities could not be determined.
Were there any differences in how well the drug worked in clinical trials among sex, race, and age groups?
Subgroup analyses of the primary endpoint based on sex, race, age, and ethnicity were performed. In general, there were no significant differences in the efficacy of ANDEMBRY based on the demographic subgroups, but some subgroups were small.
Table 3. Efficacy Results by Subgroup, Efficacy Population
| Subgroup | ANDEMBRY | Placebo | LS Mean Difference (95% CI) |
||
|---|---|---|---|---|---|
| n | LS Mean | n* | LS Mean | ||
| Sex | |||||
| Female | 24 | 0.4 | 14 | 1.9 | -1.5 (-2.3, -0.8) |
| Male | 15 | 0.1 | 10 | 2.2 | -2.1 (-3.1, -1.0) |
| Race | |||||
| Asian | 4 | 1.0 | 2 | 3.2 | -2.2 (-4.8, 0.4) |
| White | 33 | 0.2 | 21 | 2.0 | -1.8 (-2.4, -1.2) |
| Other1 | 2 | 0.1 | 1 | 0.2 | -0.1 (X, X) |
| Age group, years | |||||
| 12 to <18 | 4 | 0.4 | 2 | 0.7 | -0.3 (-1.9, 1.3) |
| 18 to 65 | 31 | 0.3 | 22 | 2.1 | -1.8 (-2.5, -1.2) |
| >65 | 4 | 0.0 | 0 | X | X (X, X) |
| Ethnicity2 | |||||
| Hispanic or Latino | 1 | 0.2 | 2 | 0.7 | -0.5 (X, X) |
| Not Hispanic or Latino | 37 | 0.3 | 22 | 2.1 | -1.9 (-2.8, -1.6) |
Source: Adapted from FDA Review
* One patient in placebo group removed from analysis due to receiving less than 30 days of study treatment
1 Includes Black or African American, Native Hawaiian or other Pacific Islander, and other races
2 Ethnicity unknown for one patient in ANDEMBRY group
Abbreviations: CI, confidence interval; LS mean, least squares mean; n, number of patients; X, value cannot be calculated due to low number of patients
What are the possible side effects?
The most common side effects of ANDEMBRY included abdominal pain and nasopharyngitis (which includes symptoms of runny nose, nasal congestion, sore throat, or upper respiratory infection).
What are the possible side effects (results of trials used to assess safety)?
The safety profile of ANDEMBRY was based on the safety population from the trial. The most common adverse reactions are shown in Table 4.
Additionally, more patients treated with ANDEMBRY compared to placebo had prolongations of prothrombin time (PT) and activated partial thromboplastin time (aPTT). The PT and aPTT prolongations were not associated with bleeding events.
Table 4. Safety Results, Safety Population
| Adverse Reaction | ANDEMBRY N=39 n (%) |
Placebo N=25 n (%) |
|---|---|---|
| Nasopharyngitisa | 8 (21) | 3 (12) |
| Abdominal pain | 3 (8) | 0 |
Source: Adapted from FDA Review
a Nasopharyngitis is composed of several similar terms including nasopharyngitis, rhinitis, and upper respiratory infections.
Abbreviations: N, number of patients in treatment group; n, number of patients with adverse reaction
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 number of patients of races other than White or Asian was small; therefore, differences in the occurrence of side effects among races could not be determined.
- Age: The occurrence of side effects was similar in patients between 12 to 18 years of age, between 18 to 65 years of age, and older than 65 years of age.
- Ethnicity: The study did not include a sufficient number of Hispanic or Latino patients; therefore, differences in the occurrence of side effects among ethnicities could not be determined.
Were there any differences in side effects of the clinical trials among sex, region, and age groups?
A safety analysis by key demographic subgroups including age, gender at birth, and region (United States versus outside the United States) was performed. There were no significant differences noted between subgroups.
Table 5. Side Effects by Sex, Age, and Region, Safety Population
| Parameter | ANDEMBRY N=39 n/Ns (%) |
Placebo N=25 n/Ns (%) |
|---|---|---|
| Sex | ||
| Female | 16/24 (67) | 11/14 (79) |
| Male | 9/15 (60) | 4/11 (36) |
| Age group, years | ||
| ≤17 | 2/4 (50) | 2/2 (100) |
| >17 | 23/35 (66) | 13/23 (57) |
| Region or country | ||
| United States | 7/12 (58) | 6/9 (67) |
| Outside the United States | 18/27 (67) | 9/16 (56) |
Source: Adapted from FDA Review
Abbreviations: N, number of patients in the treatment group; n, number of patients in the subgroup with adverse event; Ns, total number of patients for each specific subgroup and were assigned to that specific arm
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.
