FRAMINGHAM, Mass. — KalVista Pharmaceuticals Inc. said new data presented at ISPOR 2026 and the Eastern Allergy Conference highlight the burden of injectable on-demand treatment for children ages 2 to 11 with hereditary angioedema.
The Framingham-based company said the presentations showed substantial treatment burden, caregiver anxiety, delayed or untreated attacks and high health care resource use among young children living with hereditary angioedema, or HAE.
“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.”
KalVista said one presentation at ISPOR examined on-demand treatments for HAE and health care resource use among pediatric patients ages 2 to 11 using a U.S. claims database. The analysis found that about two-thirds of children in that age group were prescribed intravenous plasma-derived C1 inhibitor, or IV pdC1INH, the only FDA-approved on-demand HAE treatment for the population.
The company said most treatments used in the claims analysis were off-label, primarily icatibant, suggesting families and providers may be seeking less invasive treatment options. The mean on-demand dosing rate was 0.43 per patient per month, while the rate for pdC1INH was 0.28 compared with 0.72 for icatibant.
KalVista said health care resource use was substantial, with 47% of patients requiring emergency room care and 17% using home-health services.
A separate caregiver survey being presented at the Eastern Allergy Conference found that nearly half of children did not receive on-demand treatment for their most recent HAE attack. Among children who did receive treatment for their last attack, 93.7% experienced anxiety about using injectable on-demand treatment, including 68.8% who reported extreme anxiety.
The company said common reasons for not treating or feeling anxious about treatment included fear of needles and anticipated burning, stinging or pain from injections. Seventy-five percent of children experienced a side effect from treatment, most commonly needle pain and burning, stinging or pain during injection.
Another EAC presentation focused on caregiver burden associated with injectable on-demand treatment. KalVista said the same survey found that 43% of children’s most recent HAE attacks occurred outside the home and 37% occurred while the caregiver was not with the child.
The survey also found that 63% of caregivers had difficulty administering treatment and 31% took their child to a hospital or emergency center for treatment. Ninety percent of caregivers reported anxiety about deciding to treat, including 60% who said they felt extremely anxious.
KalVista said the mean time to treatment was 2.8 hours, with only 31% of caregivers treating in less than one hour. Most caregivers said the most important factor that could improve on-demand treatment 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 said it continues to conduct the KONFIDENT-KID trial, which it described as the fastest-enrolling pediatric HAE trial conducted to date. The company said it plans to submit a new drug application for sebetralstat in children ages 2 to 11 in the third quarter of 2026.
