Drug Trials Snapshots: EKTERLY
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 EKTERLY Prescribing Information for all of 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).
EKTERLY (sebetralstat)
(ek-TURR-lee)
KalVista Pharmaceuticals Limited
Approval date: July 03, 2025
DRUG TRIALS SNAPSHOT SUMMARY:
What is the drug for?
EKTERLY is a plasma kallikrein inhibitor that is used for the treatment of acute attacks of hereditary angioedema (HAE) in adult and pediatric patients aged 12 years and older.
How is this drug used?
EKTERLY is an oral tablet. Two tablets are taken at the earliest recognition of an HAE attack.
Who participated in the clinical trials?
The FDA approved EKTERLY based on evidence from a clinical trial (KONFIDENT) that enrolled 110 patients with HAE type I or II. The trial was conducted at 66 sites in 20 countries, including Australia, Bulgaria, Canada, France, Germany, Greece, Hungary, Israel, Italy, Japan, Netherlands, New Zealand, North Macedonia, Poland, Portugal, Romania, Slovakia, Spain, the United Kingdom, and the United States.
How were the trials designed?
Researchers tested EKTERLY's benefits and side effects in a clinical study called KONFIDENT. In this study, participants tried three different treatments in random order: EKTERLY 600 mg, EKTERLY 300 mg, and a placebo (inactive pill that looks like the real medicine). The main goal of the study was to measure how quickly EKTERLY started working. Researchers tracked how long it took for people to feel at least "a little better" twice in a row within 12 hours of taking their first dose.
How were the trials designed?
To participate in the study, patients needed to have at least two HAE attacks in the three months before joining. People who were already in a similar study could also join. Most participants (92%) had HAE type I while 8% had HAE type II. Before the study began, most participants (78%) were taking medicine only when they had an HAE attack, while 22% were taking chronic medicine to prevent HAE attacks. Once the study started, participants took the study medicine as soon as they felt an attack beginning. They could use the medicine to treat attacks anywhere on their body, except for severe throat attacks.
DEMOGRAPHICS SNAPSHOT
The FDA primary efficacy population for this application included 110 participants with HAE type I or II. Figure 1 summarizes how many male and female patients were enrolled into the efficacy population in the clinical trial used to evaluate the safety and efficacy of EKTRELY.
Figure 1. Baseline Demographics by Sex, Efficacy Population
Source: Adapted from FDA Review
Figure 2 summarizes the percentage of patients by race in the efficacy population that enrolled into the clinical trial used to evaluate the safety and efficacy of EKTERLY.
Figure 2. Baseline Demographics by Race, Efficacy Population
Source: Adapted from FDA Review
Figure 3 summarizes the percentage of patients by age in the efficacy population that enrolled into the clinical trial used to evaluate the safety and efficacy of EKTERLY.
Figure 3. Baseline Demographics by Age, Efficacy Population
Source: Adapted from FDA Review
Figure 4 summarizes the percentage of patients by ethnicity in the efficacy population that enrolled into the clinical trial used to evaluate the safety and efficacy of EKTERLY.
Figure 4. Baseline Demographics by Ethnicity, Efficacy Population
Source: Adapted from FDA Review
Who participated in the trials?
Table 1. Baseline Demographics by Age, Race, Sex, and Ethnicity
| Demographic | EKTERLY N=110 |
|---|---|
| Sex, n (%) | |
| Female | 66 (60) |
| Male | 44 (40) |
| Age group, years, n (%) | |
| 12 to <18 | 13 (12) |
| ≥18 | 97 (88) |
| Mean age, years, (SD) | 38 (15) |
| Race, n (%) | |
| Asian | 10 (9) |
| Black or African American | 1 (1) |
| White | 92 (84) |
| Other | 1 (1) |
| Not reported | 6 (5) |
| Ethnicity, n (%) | |
| Hispanic or Latino | 7 (7) |
| Not Hispanic or Latino | 95 (86) |
| Unknown | 8 (7) |
| HAE subtype, n (%) | |
| HAE Type I | 101 (92) |
| HAE Type II | 9 (8) |
| Background HAE prophylaxis, n (%) | 24 (22) |
Source: Adapted from FDA Review
Abbreviations: HAE, hereditary angioedema
What are the benefits of this drug?
In the KONFIDENT trial, EKTERLY 600 mg worked faster than the placebo (inactive pill) to start relieving symptoms of an HAE attack. When participants took EKTERLY 600 mg, 76% of them (71 out of 93 people) felt at least "a little better" within 12 hours. When participants took the placebo, only 49% of them (41 out of 84 people) felt this same level of improvement within 12 hours.
What are the benefits of this drug (results of trials used to assess efficacy)?
Figure 5 demonstrates the efficacy results from KONFIDENT that compared EKTERLY to placebo. More people who took EKTERLY felt better after 12 hours compared to people who took a placebo.
Figure 5. Time to Beginning of Symptom Relief Within 12 Hours of First Dose Administration With EKTERLY in KONFIDENT, Efficacy Population
Source: EKTERLY Prescribing Information
Figure 6. Subgroup Analysis on Median Time and 95% Confidence Interval (Hours) for Beginning of Symptom Relief Within 12 Hours of First Dose Administration With EKTERLY in KONFIDENT, Efficacy Population
Source: Adapted from FDA Review
Abbreviations: IMP, investigational medical product
Were there any differences in how well the drug worked in clinical trials among sex, race, and age?
- Sex: EKTERLY worked similarly in males and females.
- Race: EKTERLY worked similarly in White and Asian participants. The number of participants of other races was limited; therefore, differences in response for other races could not be determined.
- Age: EKTERLY worked similarly in adolescents and adults, including participants older than 65 years of age.
What are the possible side effects?
The most common side effect of EKTERLY was headache.
What are the possible side effects (results of trials used to assess safety)?
Table 2 summarizes the safety results from KONFIDENT.
Table 2. Safety Results, Safety Population
| Adverse Reaction | EKTERLY 600 mg N=93 n (%) |
Placebo N=83 n (%) |
|---|---|---|
| Headache | 3 (3.2) | 1 (1.2) |
Source: Adapted from FDA Review
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 White and Asian patients. The number of patients of other races was small; therefore, differences in how the drug worked in other races could not be determined.
- Age: The occurrence of side effects was similar in adolescent and adults, including participants 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.
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