"These data highlight the significant unmet needs that exist for children living with HAE and the families who care for them," said Ben Palleiko, Chief Executive Officer of KalVista. "Currently, the only FDA-approved on-demand treatment for children ages 2–11 requires intravenous administration, which can place a considerable burden on both children and caregivers during already stressful attacks. Across real-world treatment patterns, healthcare resource utilization, and caregiver experiences, these findings reinforce the need for new treatment options that are easy to use and support early, consistent treatment of attacks. As the first and only oral on-demand treatment for HAE, EKTERLY® has the potential to meaningfully improve the treatment experience for this pediatric population and serve as a family-wide solution across generations."
On-Demand Treatments for Hereditary Angioedema and Healthcare Resource Utilization in Pediatric (2–11 Years) Patients: A US Claims Database Analysis was presented at ISPOR by Alice Wang, Senior Director, Global Health Economics and Value Demonstration, KalVista Pharmaceuticals.
- Real-world claims data showed that while about two thirds of children aged 2–11 were prescribed IV pdC1INH—the only FDA-approved on-demand HAE treatment for this population—the majority of treatments utilized were off-label (primarily icatibant), suggesting families and providers may be seeking less invasive treatment options.
- Although the mean on-demand dosing rate was 0.43 per patient per month, the rate for pdC1INH was far less (0.28) than that of icatibant (0.72).
- Healthcare resource utilization was substantial with 47% of patients requiring emergency room care and 17% utilizing home-health services.
Burden of Injectable On-Demand Treatment for Hereditary Angioedema Attacks in Children will be presented at EAC by Timothy Craig, DO, Professor, Departments of Medicine and Pediatrics, Division of Pulmonary, Allergy and Critical Care Medicine, Penn State Health.
- A caregiver survey found that nearly half of children did not receive on-demand treatment for their most recent HAE attack.
- Among children who did treat their last attack, 93.7% experienced anxiety about using injectable on-demand treatment, with 68.8% reporting extreme anxiety.
- Among the most common reasons for not treating or feeling anxious about treatment were fear of needles and anticipated burning, stinging or pain from the injection.
- Seventy-five percent experienced a side effect from treatment, most commonly pain from the needle and burning, stinging or pain while injecting treatment.
Caregiver Burden Associated with Injectable On-Demand Treatment of Hereditary Angioedema Attacks in Children will be presented at EAC by H. Henry Li, MD, Institute for Asthma and Allergy.
- The same caregiver survey found that during their child's last HAE attack, 43% occurred outside the home and 37% occurred while they were not with their child.
- 63% of caregivers had difficulty administering treatment and 31% took their child to a hospital or emergency center for treatment.
- Caregiver anxiety was high, with 90% of caregivers reporting anxiety about deciding to treat, including 60% who felt extremely anxious.
- Mean time to treatment was 2.8 hours, with only 31% of caregivers treating in less than one hour.
- The majority of caregivers reported that the most important factor that could make on-demand treatment better for their child would be an oral therapy.
"Across real-world datasets, we consistently see a substantial treatment burden for young children with HAE and their caregivers, often resulting in treatment avoidance or long delays in on-demand treatment. We also observe high rates of anxiety among children and caregivers related to injectable treatments. We believe these challenges drive higher-than-expected healthcare resource utilization, including emergency room visits and home healthcare support for treatment administration," said Paul Audhya, MD, MBA, Chief Medical Officer of KalVista. "It is therefore not surprising that caregivers identify oral therapy as the most important factor that could improve on-demand treatment for their child. Ultimately, these presentations reinforce the value of an oral on-demand therapy that can be taken anytime, anywhere, without the challenges associated with injectables, and may help enable guideline-aligned care for the first time."
KalVista continues to execute the KONFIDENT-KID trial—the fastest-enrolling pediatric HAE trial conducted to date—and intends to submit a new drug application for sebetralstat in children aged 2–11 in the third quarter of 2026.