What's Happening?
KalVista Pharmaceuticals has presented new data at the American College of Allergy, Asthma & Immunology (ACAAI) 2025 Annual Scientific Meeting regarding EKTERLY® (sebetralstat), an oral treatment for hereditary
angioedema (HAE). The data highlights patient satisfaction and the potential of EKTERLY® in treating children aged 2-11. The KONFIDENT-S study showed high satisfaction among patients who switched from injectable treatments to sebetralstat, with 84% of attacks treated rated as satisfied or very satisfied. The KONFIDENT-KID study demonstrated the effectiveness of sebetralstat in pediatric patients, showing rapid relief from HAE attacks with no serious adverse events. The findings underscore the unmet need for non-injectable treatments, as current options for children involve intravenous or subcutaneous administration, which can be burdensome.
Why It's Important?
The introduction of EKTERLY® as an oral treatment for HAE represents a significant advancement in patient care, particularly for children who face challenges with injectable treatments. The high satisfaction rates and effective relief provided by sebetralstat could lead to improved adherence to treatment guidelines and better management of HAE attacks. This development is crucial for families affected by HAE, offering a more convenient and less invasive option. The potential approval of EKTERLY® for pediatric use could transform HAE treatment, reducing the burden on patients and caregivers and addressing the previously hidden disease burden due to the limitations of injectable therapies.
What's Next?
KalVista Pharmaceuticals is continuing its efforts to expand the use of EKTERLY® globally, with ongoing studies and regulatory applications in key markets. The company aims to establish EKTERLY® as a foundational therapy for HAE management worldwide. If approved for pediatric use, EKTERLY® could become a breakthrough in HAE care, providing early and consistent treatment for attacks. The company is also focusing on addressing the barriers to on-demand treatment identified in consensus studies, aiming to improve early intervention and adherence to guidelines.
Beyond the Headlines
The shift towards oral treatments for HAE reflects broader trends in patient-centered care, emphasizing convenience and reducing treatment burdens. The success of EKTERLY® could influence future pharmaceutical developments, encouraging more research into oral therapies for other rare diseases. Additionally, the findings highlight the importance of addressing patient preferences and unmet needs in drug development, potentially leading to more personalized and effective treatment options.
