Oral Ekterly gives relief to kids with HAE, trial data show
Treatment provides early, effective management for children aged 2-11
Ekterly (sebetralstat), given as a tablet that dissolves on the tongue, resulted in early, effective, and safe management of swelling attacks in children with hereditary angioedema (HAE) aged 2-11, according to interim data from a clinical trial.
The ongoing Phase 3 study, KONFIDENT-KID (NCT06467084), which has enrolled 36 children with HAE, also revealed that the attack frequency in that age group was higher than previously thought, developer Kalvista Pharmaceuticals said in a company press release.
“These interim data reveal just how profoundly the burden of hereditary angioedema has been underestimated in children,” Paul Audhya, MD, chief medical officer of Kalvista, said in an email to Angiodema News. “In KONFIDENT-KID, we observed an attack rate far higher than what’s been historically understood — not because the disease has changed, but perhaps because, for the first time, treatment options have.”
The data were presented at the American College of Allergy, Asthma & Immunology (ACAAI) 2025 annual scientific meeting, held Nov. 6-10 in Orlando, Florida.
“We’re finally seeing the true scope of this disease, and that understanding is essential to improving care for pediatric patients,” Audhya said.
Patients prefer oral treatment to injections
Meanwhile, new treatment satisfaction results from a questionnaire given during the ongoing extension study KONFIDENT-S (NCT05505916) showed that participants ages 12 and older who switched from injectable on-demand treatments to oral Ekterly were very or extremely satisfied.
HAE is marked by the overproduction of bradykinin, a signaling molecule that causes blood vessels to widen and become more permeable. As a result, fluid can leak into deeper layers of the skin or mucous membranes, triggering episodes of swelling.
Ekterly is designed to inhibit kallikrein, an enzyme that drives the production of bradykinin. By blocking kallikrein during an attack, the medication is expected to lower bradykinin levels and help control swelling.
Among approved treatments for HAE attacks, only Berinert (human C1 esterase inhibitor) is authorized in the U.S. for use in children younger than 12. It must be administered via intravenous (into-the-vein) infusion.
“The invasiveness of injectable therapies has long deterred parents and caregivers of pediatric patients from treating anything but the most severe attacks,” Audhya said. “With [Ekterly] — the first and only oral on-demand option, those barriers could be eliminated.”
Ekterly was recently approved for HAE patients aged 12 and older in regions including the U.S., the U.K., and the European Union and Switzerland. It’s also approved in Iceland, Liechtenstein, and Norway.
Its approvals were primarily supported by results from the Phase 3 KONFIDENT trial (NCT05259917), demonstrating that on-demand Ekterly outperformed a placebo in providing faster symptom relief and quicker resolution of attacks. Findings from KONFIDENT-S continue to show that the medicine can rapidly ease swelling, including in severe episodes and those involving the airways.
Interim data from KONFIDENT-KID were reported by Adil Adatia, MD, assistant professor at the University of Alberta, in an oral presentation titled, “Sebetralstat for On-demand Treatment of Hereditary Angioedema in Pediatric (2-11y) Patients: Interim Results from KONFIDENT-KID.”
KONFIDENT-KID recently expanded its enrollment to include 36 children, aged 2-11, across seven countries in North America, Europe, and Asia, in response to high demand. Based on body weight, participants were assigned one of three Ekterly doses (75 mg, 150 mg, or 300 mg) administered as a tablet that rapidly dissolves in the mouth without the need for water.
Among 26 children who received on-demand Ekterly treatment for 65 attacks, representing a mean of 0.8 attacks per month, more than half of the attacks were moderate (53.8%), while 32.3% were mild and 7.7% severe. Primary attack locations were the abdomen (43.1%), arms/hands (27.7%), head/face/neck (12.3%), and legs/feet (10.8%).
The median time to treatment, as reported by caregivers or patients, was 30 minutes, with most attacks (61.3%) being treated within one hour of onset. Overall, in nearly all attacks (78.9%), symptom relief was achieved within 12 hours. In participants who received the 150 mg dose, the median time to symptom relief onset was 1.5 hours.
Ekterly was well tolerated, with no reports of serious or treatment-related adverse events, and no signs of swallowing difficulties.
“These interim results from KONFIDENT-KID are highly promising as they demonstrate that [Ekterly] can … halt attack progression at an early stage and provide rapid relief,” Adatia said. “If approved, [Ekterly] for children would be a true breakthrough in HAE care.”
Patient satisfaction data from KONFIDENT-S were described by Maeve E. O’Connor, MD, an allergist and immunologist with Integrative Allergy & Immunology Care in Charlotte, North Carolina, in a poster titled, “Satisfaction with Sebetralstat for HAE Attacks in Patients Switching from Parenteral On-demand Treatments in KONFIDENT-S.”
In KONFIDENT-S, 1,089 attacks were treated with Ekterly among participants who had previously received injectable on-demand therapies. Based on a 7-point scale ranging from -3 (extremely dissatisfied) to 3 (extremely satisfied), the median treatment satisfaction score with Ekterly was 2, or very satisfied. Participants were satisfied, very satisfied, or extremely satisfied with Ekterly in treating nearly all attacks (84%).
“The high satisfaction scores reported by patients who have successfully transitioned from injectable products to [Ekterly] are extremely encouraging and speak directly to the profound impact of having a simple, effective, and reliable oral option readily available,” O’Connor said.
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