Framingham, Massachusetts & Salisbury, England — November 6, 2025 : KalVista Pharmaceuticals, Inc. (Nasdaq: KALV) presented new data at the American College of Allergy, Asthma & Immunology (ACAAI) 2025 Annual Scientific Meeting, showcasing both high patient satisfaction and promising pediatric outcomes for EKTERLY® (sebetralstat), the first and only oral on-demand treatment for hereditary angioedema (HAE). Findings from the KONFIDENT-S and KONFIDENT-KID clinical studies underscore the therapy’s effectiveness, safety, and transformative potential for both adult and pediatric populations living with this rare, potentially life-threatening condition.
Science Significance
In the KONFIDENT-S study, adults with HAE who transitioned from injectable on-demand therapies (icatibant, pdC1INH, rhC1INH) to oral sebetralstat reported 84% satisfaction, with most being very or extremely satisfied. Over 1,000 attacks were treated effectively, with rapid relief and high treatment adherence. Meanwhile, interim data from KONFIDENT-KID demonstrated that children aged 2–11 receiving a weight-based, oral disintegrating tablet (ODT) formulation achieved a median symptom relief within 1.5 hours and had no serious adverse events. These results establish sebetralstat as a safe, effective, and non-invasive alternative to injections—representing a major scientific advancement in pediatric HAE management.
Regulatory Significance
EKTERLY® (sebetralstat) has already been approved in the U.S., EU, UK, and Switzerland for treating acute HAE attacks in patients 12 years and older. The new pediatric trial results bolster the company’s ongoing regulatory submissions for extending approval to children aged 2–11 years. By meeting key safety and efficacy endpoints, sebetralstat strengthens KalVista’s position with regulators as the first company to pioneer a universal, age-inclusive, oral on-demand treatment for hereditary angioedema.
Business Significance
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The success of EKTERLY® reflects KalVista’s innovation-driven business model targeting rare diseases with high unmet needs. These data reinforce the company’s commercial trajectory, building on the product’s early global launch success in adults and adolescents. As awareness of oral treatment benefits grows—combined with 89% of patients expressing a preference for oral therapy—KalVista’s market expansion potential is strong. With additional data emerging from global KONFIDENT trials, EKTERLY® is poised to become a foundational therapy for HAE management worldwide, driving both clinical adoption and shareholder value.
Patients’ Significance
For patients, the introduction of an oral, non-injectable on-demand option transforms the daily reality of managing HAE attacks. Current injectable treatments pose emotional and physical challenges, especially for children who face anxiety, pain, and logistical barriers. EKTERLY® offers ease of use, faster administration (within 30 minutes of attack onset), and quicker symptom relief, enabling patients and caregivers to treat early and prevent progression. This evolution in HAE care could significantly improve quality of life, reduce trauma, and empower self-management across age groups.
Policy Significance
The availability of EKTERLY® aligns with public health goals to enhance rare disease care and ensure treatment accessibility. Findings from a Delphi consensus study involving 19 HAE experts emphasized the need for early, easy-to-administer treatments within 60 minutes of symptom onset—a target EKTERLY® meets effectively. As regulators and health systems focus on improving equitable access to advanced therapies, EKTERLY®’s oral format supports policy shifts toward patient-centric, at-home care models, reducing healthcare resource burden and enhancing long-term outcomes.
With compelling data from adult and pediatric clinical programs, KalVista Pharmaceuticals has redefined the standard of care for hereditary angioedema through its first-in-class oral plasma kallikrein inhibitor, EKTERLY®. As regulatory expansion and global availability progress, the therapy’s strong patient satisfaction and safety profile position it to become the cornerstone of on-demand HAE management. EKTERLY® not only meets clinical and commercial milestones but also represents a significant step toward accessible, injection-free treatment for rare disease patients worldwide.
Source: KalVista Pharmaceuticals, Inc. press release
