Longer gaps between HAE treatment injections may be possible, study finds
Small study of Andembry found improved disease control and quality of life
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People with hereditary angioedema (HAE) showed better disease control and quality of life during treatment with Andembry (garadacimab-gxii), a small real-world study in Germany found. Many were also able to go longer between injections under medical supervision.
After about six months, nearly all patients who were assessed had well-controlled angioedema, and most showed meaningful improvements in quality of life. Patients continuing treatment had a median of six weeks between injections, compared with the approved monthly schedule. Some reached eight weeks. These longer intervals were used off-label, meaning they were outside the approved dosing schedule.
Patients reported no side effects during follow-up and described the injection pen and fine needle as comfortable to use.
Longer injection intervals may reduce HAE treatment burden
“[Andembry] showed high efficacy and safety and the gradual extension of injection intervals presented underline its potential for longer dosing intervals that may further reduce treatment burden,” the researchers wrote.
The study, “Experiences From the First Year of Using Garadacimab to Treat Hereditary Angioedema and Acquired Angioedema,” was published in Clinical and Translational Allergy.
HAE is a rare inherited condition that causes repeated swelling episodes affecting the skin, abdomen and airways. In the two most common forms, called HAE types 1 and 2, genetic mutations cause the body to make too little of a protein called C1 inhibitor (C1-INH) or produce a form that does not work properly. C1-INH helps control the production of bradykinin, a substance involved in swelling. Without enough working C1-INH, too much bradykinin is produced, causing fluid to leak from blood vessels into nearby tissues and leading to swelling.
Treatment of HAE includes medications taken as needed to treat swelling episodes and long-term treatments to help prevent future attacks.
Andembry was approved in 2025 in the U.S., Canada, European Union, and other regions to prevent swelling attacks in people with HAE ages 12 and older. It is given as an injection under the skin once a month. Andembry works by blocking activated factor XII, a protein that helps trigger the production of bradykinin.
The approvals were supported by results from the Phase 3 VANGUARD trial (NCT04656418), which included 64 adolescents and adults with HAE types 1 or 2. Results showed that patients receiving Andembry had nearly 90% fewer swelling attacks per month than those receiving a placebo. Most patients taking Andembry had no attacks during the six-month study, compared with none in the placebo group.
For the new real-world study, researchers in Germany examined the experiences of 29 patients who received Andembry at the Angioedema Center of Reference and Excellence (ACARE) in Berlin between March and December 2025. The group included people with hereditary and acquired angioedema.
Study includes patients with hereditary and acquired angioedema
Most participants — 27 (93%) — had HAE, while two (7%) had acquired angioedema. This form of angioedema can also result from C1-INH deficiency, which leads to excessive bradykinin production.
Before starting Andembry in routine care, 16 patients (55%) had used another preventive treatment, six (21%) had used only on-demand medications, and seven (24%) continued Andembry after participating in VANGUARD and its open-label extension (NCT04739059).
Patients often switched preventive treatments because they hoped for greater convenience or better disease control. Convenience was especially important for those previously taking Takhzyro (lanadelumab), another approved HAE preventive treatment generally given by injection under the skin every two weeks.
Patients were offered the option to gradually increase the time between Andembry injections beyond the approved monthly schedule. Those continuing treatment from VANGUARD and its extension could begin extending their intervals immediately if they were symptom-free. Patients newly starting Andembry in routine care could begin after two months, provided they had no symptoms and were tolerating treatment well.
Under medical supervision, patients gradually added three days, then four days, to the time between injections, repeating this pattern. If early warning signs or swelling episodes appeared, they stopped extending the interval and returned to a shorter schedule that had previously kept them symptom-free.
Three patients (10%) stopped Andembry after the initial loading dose or second dose because they felt the treatment was not working well enough. This left 26 patients for the main analysis.
After about six months of treatment, 24 of 25 patients (96%) with available Angioedema Control Test (AECT) results had well-controlled angioedema. Ten patients (40%) had complete control. The AECT measures disease control over the previous four weeks, with scores ranging from zero to 16. A score of 10 or higher indicates well-controlled disease, while 16 indicates complete control.
Among 20 patients with AECT scores available both before and during treatment, the average score increased significantly from 9.2 to 14.4 points. Fourteen patients (70%) had an improvement of at least three points, considered clinically meaningful.
Quality of life improves during treatment with Andembry
Quality of life also improved. Among 20 patients assessed before and during treatment, the average score on the Angioedema Quality of Life questionnaire (AE-QoL) fell significantly from 35.5 to 14.9 points. The questionnaire measures how angioedema affects daily life, with lower scores indicating less impact. Seventeen of the 20 patients (85%) had an improvement considered clinically meaningful.
Improvements in both disease control and quality of life were generally greater among patients who had previously used only on-demand medications than among those switching from another preventive treatment. Patients already receiving preventive treatment tended to have better disease control before starting Andembry, leaving less room for improvement.
At the latest assessment, the 26 patients who continued treatment had a median of 42 days, or six weeks, between injections, compared with the approved monthly interval of 31 days. Some had reached 56 days, or eight weeks. Three patients stayed on monthly injections from the start. Another 10 stopped extending their intervals after developing warning signs or swelling episodes and settled on fixed schedules — five returned to monthly dosing, while five maintained longer intervals. The remaining 13 patients were still gradually increasing the time between injections.
Patients reported no side effects during follow-up and described the injection pen and fine needle as pleasant to use.
The researchers concluded that “this early real-life observation confirmed the efficacy and safety of garadacimab as a new treatment option for [long-term preventive treatment] in HAE, while also demonstrating the potential to individualize the dosing interval according to the specific dosing needs of our HAE and AAE patients.”
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