KalVista Pharmaceuticals' EKTERLY® Gains FDA Approval as First Oral On-Demand Hereditary Angioedema Treatment

KalVista Pharmaceuticals, Inc., a prominent biopharmaceutical company, has announced a significant milestone in the treatment of hereditary angioedema (HAE) with the approval of EKTERLY® (sebetralstat) by the U.S. Food and Drug Administration (FDA). This approval marks the introduction of the first oral on-demand treatment for acute HAE attacks, available to both adults and children aged 12 and older. This development is expected to revolutionize the management of HAE, a rare and potentially life-threatening genetic condition characterized by unpredictable episodes of severe swelling in various parts of the body.

EKTERLY's approval is a pivotal moment for those living with HAE, as it provides a more convenient treatment option compared to the existing therapies that require intravenous or subcutaneous administration. This advancement allows patients to manage their condition more independently and efficiently, offering rapid relief from symptoms and quicker resolution of attacks, regardless of the severity or location of the attack, patient age, or concurrent use of long-term prophylaxis. The new oral medication is particularly beneficial since it aligns with treatment guidelines that emphasize early intervention to prevent the progression of swelling and reduce the overall burden of the disease.

KalVista CEO Ben Palleiko expressed gratitude towards the team, patients, healthcare providers, and associated organizations like HAEA and HAEi, for their roles in achieving this milestone. The company is committed to making EKTERLY readily available to patients in need, highlighting its potential to become a cornerstone therapy for HAE management. In support of this goal, KalVista has initiated the KalVista Cares™ program, which aims to offer personalized assistance to patients, including help with insurance coverage, access support, and ongoing treatment guidance.

The efficacy and safety of EKTERLY were demonstrated in the KONFIDENT phase 3 clinical trial, the largest of its kind for HAE. Published in the New England Journal of Medicine, the trial results revealed that EKTERLY provides significantly faster symptom relief, reduces the severity of attacks, and ensures quicker resolution compared to a placebo. The trial involved 136 patients from 66 clinical sites across 20 countries, establishing EKTERLY's effectiveness and safety profile, which was similar to that of a placebo.

Following its approval, EKTERLY is set to be launched immediately in the U.S., and physicians can begin prescribing it. The real-world KONFIDENT-S open-label extension trial reinforced the clinical trial findings, showing that relief from symptoms could begin in as little as 1.3 hours, particularly in laryngeal and abdominal attacks, as well as breakthrough attacks among those on long-term prophylaxis. With over 1,700 attacks analyzed, the safety profile remained consistent with earlier studies.

As part of their strategy to expand EKTERLY's availability, KalVista is conducting further studies to explore its use in younger children aged two to 11, and multiple regulatory applications are under review in key global markets. The company maintains its focus on addressing the significant unmet needs of individuals with rare diseases through the development of transformative oral therapies.

KalVista will host a conference call and webcast to discuss the approval of EKTERLY, providing an opportunity for stakeholders to gain further insight into this groundbreaking advancement in HAE treatment. This event reflects KalVista's ongoing dedication to improving the lives of those affected by rare diseases through innovative medical solutions.