Sebetralstat Shows Promise for On-Demand HAE Treatment in Children, With Adil Adatia, MD

In a late-breaking presentation at the 2025 American College of Allergy, Asthma & Immunology (ACAAI) Annual Scientific Meeting, Adil Adatia, MD, FRCPC, from the University of Alberta, shared interim results from the KONFIDENT-KID trial assessing sebetralstat for on-demand treatment of hereditary angioedema (HAE) in children aged 2 to 11 years.

“It’s very reassuring to see that this tablet was used quickly after the onset of symptoms,” Adatia told HCPLive during an on-site interview. “So with very young children, it is challenging to do clinical trials because they may not report specific symptoms attributable to a hereditary angioedema attack.”

In the United States, the only currently approved on-demand treatment option for children with HAE is intravenous plasma-derived C1 inhibitor therapy. Because IV administration can be difficult to perform at home, many pediatric attacks remain untreated or are managed in emergency settings, delaying care.

Sebetralstat, an oral plasma kallikrein inhibitor already approved for patients aged ≥ 12 years, was evaluated for safety, pharmacokinetics, and efficacy in younger children. The trial included 3 dosing cohorts based on age.

Interim results indicated no treatment-emergent adverse events, suggesting a favorable safety profile consistent with data seen in adults. Pharmacokinetic findings showed plasma concentrations aligned with prior studies, and efficacy analyses demonstrated a median time to first improvement of four hours.

Participants initiated treatment at a median of 30 minutes after symptom onset, highlighting the potential of oral therapy to enable faster, at-home intervention. The study used an oral disintegrating tablet formulation designed to dissolve quickly on the tongue or in a small amount of liquid, facilitating administration in young children.

“…the time to event analysis showed that patients were better with a median of 4 hours, and so patients were getting rapid improvement,” Adatia said.

According to the findings, early treatment was associated with reduced attack duration and improved symptom control. The interim data support the continued evaluation of oral on-demand options for pediatric HAE, which may improve timely access to therapy and overall quality of care.

“I think in the future oral on-demand treatment will become the standard, and most patients will use oral on-demand treatments because they're much easier to use and they can be administered much more rapidly,” Adatia said. “When you administer treatment early compared to the onset of symptoms, you can get a much faster resolution, so I think that is hopefully the direction that [HAE care] will take.”

Adatia has no relevant reported disclosures.

References

Adatia A. Sebetralstat for On-demand Treatment of Hereditary Angioedema in Pediatric (2-11y) Patients: Interim Results from KONFIDENT-KID. Late-breaker presented at ACAAI 2025 in Orlando, Florida, on November 8, 2025.