KalVista Pharmaceuticals, Inc. reported that the U.S. Food and Drug Administration (FDA) has approved the marketing of EKTERLY(R) (sebetralstat) to treat acute attacks of hereditary angioedema (HAE) in adults and adolescents aged ≥12 years, a landmark advancement. This represents the entry of the first oral, on-demand therapy for a rare condition that previously required injectable treatments for the management of symptoms.
HAE is a rare genetic condition characterized by spontaneous episodes of swelling, which can be painful and, in some cases, life-threatening, that can occur in various parts of the body, including the airway. Until now, patients were limited to intravenous or subcutaneous treatments to manage sudden attacks. EKTERLY, as an oral, on-demand treatment, offers a much-needed alternative.
“Today’s approval changes the landscape for people living with HAE,” said Ben Palleiko, CEO of KalVista. “EKTERLY allows patients to treat an attack immediately, no matter where they are, without needing needles or specialized administration. This milestone represents the commitment of the KalVista team to explore better options for the HAE community, and we are deeply thankful to each of the patients, physicians, and organizations who supported this effort.”
The approval was supported by robust data from KalVista’s KONFIDENT phase 3 trial. This trial was the largest study ever conducted in this disease area. Results published in The New England Journal of Medicine showed that patients treated with EKTERLY had a faster symptom improvement, with milder attacks and quicker resolution compared to placebo. The safety profile was comparable to placebo, with headache being the most reported side effect.
Dr. Marc A. Riedl, MD, a leading HAE specialist at the University of California, San Diego, and a principal investigator in the trial, emphasized the clinical value of the new therapy. “Having an oral option aligns with treatment guidelines that promote early treatment. And for many patients, it offers greater independence, eliminating the need to delay treatment due to the complexities of injection-based therapies.”
Anthony J. Castaldo, CEO, U.S. Hereditary Angioedema Association, shares similar significance. “This is a major breakthrough in the HAE community. It reduces the treatment burden and provides more flexibility for patients, enabling quicker and more effective control of attacks.”
KalVista confirmed that EKTERLY will be launched in the U.S. without delay. Healthcare providers can now prescribe the medications, and the company has launched KalVista CaresTM, a patient support program to assist with insurance navigation, access resources, and provide tailored treatment support.
EKTERLY’s recommended dosing is to take two tablets (600 mg) at the first sign of an attack, with one additional dose allowed if symptoms persist or recur after three hours. Patients are advised not to exceed four tablets (1,200 mg) within a 24-hour period.
KalVista is also actively pursuing regulatory approvals in other global markets and is conducting studies to evaluate sebetralstat in young pediatric populations, aiming to expand access to treatment worldwide. For individuals and families living with hereditary angioedema, EKTERLY represents a welcoming new option in handling sudden attacks that bring rapid relief and greater autonomy within each.
References: KalVista Pharmaceuticals. KalVista Pharmaceuticals announces FDA approval of EKTERLY® (sebetralstat), first and only oral on-demand treatment for hereditary angioedema. Business Wire. Published July 7, 2025. Accessed July 8, 2025. https://www.businesswire.com/news/home/20250702871458/en/KalVista-Pharmaceuticals-Announces-FDA-Approval-of-EKTERLY-sebetralstat-First-and-Only-Oral-On-demand-Treatment-for-Hereditary-Angioedema
