FDA Approves KalVista’s EKTERLY® as First Oral On-Demand Treatment for Hereditary Angioedema
KalVista Pharmaceuticals, Inc. has announced a landmark development in the treatment of hereditary angioedema (HAE): the U.S. Food and Drug Administration (FDA) has officially approved EKTERLY® (sebetralstat), the first and only orally administered on-demand therapy for treating acute HAE attacks in adults and adolescents aged 12 years and older.
This approval represents a pivotal shift in how HAE can be managed, offering patients a more convenient, rapid, and accessible solution during the onset of life-disrupting—and sometimes life-threatening—attacks.
A New Era for HAE Management
Hereditary angioedema is a rare and potentially serious genetic disorder characterized by unpredictable and recurring episodes of severe swelling in various parts of the body, including the face, extremities, gastrointestinal tract, and airway. These attacks can be painful, disfiguring, and even fatal when they affect the throat.
Until now, patients living with HAE have had to rely on treatments that require either intravenous (IV) or subcutaneous (SC) injections—methods that can be difficult to administer, especially during emergencies or when access to medical facilities is limited.
EKTERLY (sebetralstat), a novel oral plasma kallikrein inhibitor, provides an entirely new approach. Its oral formulation allows patients to respond immediately to symptoms as they arise—at home, at work, or on the go—without the need for injections or complex medical procedures.
A Defining Moment for the HAE Community
Ben Palleiko, CEO of KalVista, emphasized the importance of this milestone for patients and the broader HAE community.
“The FDA approval of EKTERLY is a defining moment for people living with HAE,” said Palleiko. “EKTERLY enables people to treat attacks the moment symptoms begin, wherever they are. This approval affirms the strength of our science and deep commitment to the HAE community.”
Palleiko further expressed gratitude to the teams and individuals involved in achieving this success, from KalVista’s scientists and staff to the patients and healthcare professionals who participated in clinical trials. He also acknowledged the support of advocacy organizations such as the Hereditary Angioedema Association (HAEA) and HAEi, which have played vital roles in supporting awareness and research.
“EKTERLY has the potential to become the foundational treatment for HAE,” he added. “Our focus now is on delivering it to the people who need it.”
Expert Support from the HAE Medical Community
The approval of EKTERLY has been warmly welcomed by experts in the field, including those involved in its development and clinical evaluation.
Anthony J. Castaldo, CEO of the U.S. Hereditary Angioedema Association, underscored the significance of the treatment’s oral delivery format:
“As the first orally administered on-demand therapy for HAE attacks, EKTERLY provides patients and physicians with an important and welcome advance in HAE treatment options.”
Dr. Marc A. Riedl, Professor of Medicine at the University of California, San Diego, and Clinical Director at the U.S. HAEA Center, also highlighted the importance of timely treatment:
“Until now, on-demand treatment relied on injectable subcutaneous or intravenous administration, often resulting in delayed intervention. Having an oral option empowers patients to treat attacks early, which aligns with treatment guidelines and advances our goal as physicians to reduce the overall burden of disease.”
According to Dr. Riedl, having an oral medication allows for earlier response during attacks, reducing severity and duration, which can significantly improve quality of life and independence for patients.
Groundbreaking Clinical Trial Results
The FDA’s approval of EKTERLY was based on robust data from KalVista’s Phase 3 KONFIDENT trial, the largest HAE clinical trial program ever conducted. The study included 136 patients across 66 clinical sites in 20 countries, providing a globally representative and statistically rigorous data set.
The results, published in The New England Journal of Medicine in May 2024, showed that EKTERLY:
- Provided significantly faster symptom relief compared to placebo
- Reduced attack severity and duration
- Was well tolerated with a safety profile similar to placebo
In parallel, KalVista conducted the KONFIDENT-S open-label extension study, which simulated real-world use of EKTERLY. As of September 2024, the study showed that patients were able to treat their attacks a median of just 10 minutes after onset. Symptom relief began in a median of 1.3 hours, even in challenging cases such as laryngeal attacks, abdominal attacks, and breakthrough episodes despite prophylactic treatments.
Importantly, the safety profile in over 1,700 treated attacks in KONFIDENT-S remained consistent with the initial Phase 3 trial results, confirming both the reliability and tolerability of EKTERLY in broader usage.
Immediate U.S. Launch and Patient Support
KalVista has moved quickly to make EKTERLY available to those who need it. The company confirmed that U.S. physicians can begin prescribing EKTERLY immediately, marking the start of a new standard of care for acute HAE treatment.
To support patients and ensure equitable access, KalVista has also launched KalVista Cares™, a dedicated patient assistance and support program. This initiative offers a comprehensive range of services, including:
- Help with insurance coverage and reimbursement
- Personalized access support
- Ongoing assistance and educational resources throughout the treatment journey
KalVista Cares is designed to simplify the process of starting and continuing therapy, particularly for patients new to managing HAE or transitioning from injectable treatments.
Looking Ahead
With the FDA approval of EKTERLY, KalVista Pharmaceuticals has achieved a major milestone—not just in terms of business success, but in transforming how a rare and burdensome disease is treated.
This breakthrough reflects years of scientific innovation, collaboration, and patient advocacy, and it marks a new chapter in the ongoing effort to provide people living with HAE the tools they need to live more freely and confidently.
As KalVista continues to expand access to EKTERLY and explore further applications of sebetralstat, the HAE community now has greater hope for faster, easier, and more effective control over this complex condition.
For more information, visit EKTERLY.com.
About Hereditary Angioedema
Hereditary angioedema (HAE) is a rare genetic disease resulting in deficiency or dysfunction in the C1 esterase inhibitor (C1INH) protein and subsequent uncontrolled activation of the kallikrein-kinin system. People living with HAE experience painful and debilitating attacks of tissue swelling in various locations of the body that can be life-threatening depending on the area affected. Treatment guidelines recommend treating attacks as early as possible to prevent progression of swelling and shorten the time to attack resolution, and to consider treatment for all attacks, regardless of anatomic location or severity.
About EKTERLY® (sebetralstat)
EKTERLY (sebetralstat), a novel plasma kallikrein inhibitor, is the first and only oral on-demand therapy approved by the U.S. FDA for the treatment of acute attacks of hereditary angioedema (HAE) in people 12 years of age and older. With ongoing studies exploring its use in children aged two to 11 and multiple regulatory applications under review in key global markets, EKTERLY has the potential to become the foundational therapy for HAE management worldwide.
