KalVista Pharmaceuticals Announces Inclusion of EKTERLY® (sebetralstat) as a First-Line Therapy for Adolescents 12 and Older in International Pediatric HAE Guideline
Guideline recommends ensuring on-demand treatment is available anytime, anywhere and prioritizes early intervention and rapid self-administration, reinforcing the unique value of EKTERLY
KalVista Pharmaceuticals, Inc. (NASDAQ:KALV) today announced that the newly published International Guideline on the Diagnosis and Management of Pediatric Patients With Hereditary Angioedema recommends EKTERLY® (sebetralstat) as a first-line therapy for the acute treatment of hereditary angioedema (HAE) attacks in adolescents aged 12 years and older.1
Based on robust, high-quality evidence, including clinical trial data demonstrating consistent efficacy, rapid symptom relief and a favorable safety profile, the guideline committee issued a strong recommendation for the first-line use of sebetralstat in adolescents 12 and older with HAE. The recommendation comes just months after the launch of EKTERLY in the US and Germany and follows seven global regulatory approvals received in 2025. EKTERLY is the first and only oral on-demand treatment for people 12 and older with HAE.
"We are encouraged to see this international guideline reflect the progress the HAE community has made toward more patient-centered care, particularly for adolescents," said Paul Audhya, MD, MBA, Chief Medical Officer of KalVista. "The first-line recommendation for EKTERLY so soon after becoming commercially available underscores the strength of our clinical data and reinforces the importance of ensuring patients have immediate access to effective on-demand therapy. As the first and only oral on-demand treatment for HAE, EKTERLY uniquely enables guideline-aligned care by supporting early intervention and simplifying self-administration, empowering adolescents 12 and older to treat at the earliest recognition of an attack."
The guideline emphasizes the importance of early intervention, noting that early treatment of attacks is associated with better outcomes and results in shorter, less severe episodes. However, real-world data show that adolescents with HAE have historically faced substantial delays administering treatment, waiting nearly eight hours on average to treat attacks due to the anxiety and challenges associated with injectable therapies.2
To support early intervention, the guideline recommends ensuring pediatric patients have on-demand therapy available anytime, anywhere, consider treatment of all attacks, and have sufficient supply to treat at least two attacks, even when receiving long-term prophylaxis. In addition, it recommends having on-demand treatment available during potentially triggering events such as medical, dental and surgical procedures, while emphasizing that the activities and lifestyle of patients should be kept as normal as possible, provided they have rapid access to on-demand medication.
"While general protocols have existed for many years, these new recommendations recognize that children require care tailored to their unique needs to manage this rare and potentially life-threatening genetic condition," said Mauro Cancian, MD, PhD, Head of the Allergy Division at the University of Padua in Padova, Italy. "HAE presents distinct physiological and psychological hurdles, particularly in adolescents, from puberty-driven flare-ups to the trauma of frequent injections. By moving beyond a 'one-size-fits-all' approach, the guidelines provide a clear framework for care that supports not only rapid life-saving treatment, but also a child's long-term health and emotional well-being."
EKTERLY is currently approved in the United States, European Union, United Kingdom, Switzerland, Australia, Singapore and Japan for the treatment of acute attacks of HAE in people 12 years of age and older. It is marketed in the US and Germany, with additional global launches anticipated in 2026 and beyond. KalVista continues to advance the KONFIDENT-KID trial—the fastest-enrolling pediatric HAE trial conducted to date—and is on track to submit a new drug application for sebetralstat in patients aged 2 to 11 in the third quarter of 2026.
Developed by an International Steering Committee, Taskforce and the Hereditary Angioedema Working (HAWK) Group, the guideline was published in Allergy, the official journal of the European Academy of Allergy and Clinical Immunology (EAACI).
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 KONFIDENT-KID
KONFIDENT-KID is an open label clinical trial of sebetralstat for on-demand treatment of HAE attacks in pediatric patients. Originally designed to enroll 24 pediatric patients, the trial was met with high demand and will ultimately enroll approximately 36 children aged two to 11 years across seven countries in North America, Europe and Asia. KONFIDENT-KID will collect safety, pharmacokinetic and efficacy data for each patient for up to one year and features a proprietary pediatric oral disintegrating tablet (ODT) formulation of sebetralstat. If approved, sebetralstat would be the first oral on-demand therapy for this age group, and only the second FDA-approved on-demand therapy of any type for this population.
About KalVista Pharmaceuticals, Inc.
KalVista is a global pharmaceutical company dedicated to delivering life-changing oral therapies for individuals affected by rare diseases with significant unmet needs. The KalVista team discovered and developed EKTERLY®—the first and only oral on-demand treatment for hereditary angioedema (HAE)—and continues to work closely with the global HAE community to improve treatment and care for this disease around the world. For more information about KalVista, please visit www.kalvista.com and follow us on LinkedIn, X, Facebook and Instagram.
INDICATION AND IMPORTANT SAFETY INFORMATION
What is EKTERLY® (sebetralstat)?
EKTERLY is a prescription medicine used to treat sudden (acute) attacks of hereditary angioedema (HAE) in adults and children aged 12 years of age and older. It is not known if EKTERLY is safe and effective in children under 12 years of age.
IMPORTANT SAFETY INFORMATION
Before taking EKTERLY, tell your healthcare provider about all of your medical conditions, including if you:
- Are pregnant or planning to become pregnant. It is not known if EKTERLY can harm your unborn baby.
- Are breastfeeding or plan to breastfeed. It is not known if EKTERLY passes into your breast milk. Talk to your healthcare provider about the best way to feed your baby while taking EKTERLY.
- Have liver problems.
Tell your healthcare provider about all of the medicines you take, including prescription and over-the-counter medicines, vitamins, and herbal supplements. Taking EKTERLY with certain other medicines can cause side effects or affect how well EKTERLY or the other medicines work. Especially tell your healthcare provider if you take any of the following, as their use with EKTERLY is not recommended: itraconazole, phenytoin, efavirenz.
Know the medicines you take. Keep a list of them to show your healthcare provider or pharmacist when you get a new medicine.
What are the possible side effects of EKTERLY?
The most common side effects of EKTERLY include headache. For more information, ask your healthcare provider or pharmacist. Talk to your doctor for medical advice about side effects.
You are encouraged to report side effects related to KalVista products by calling 1-855-258-4782. If you prefer, you may contact the U.S. Food and Drug Administration (FDA) directly. Visit www.fda.gov/medwatch or call 1-800-FDA-1088.
Please click here for full Prescribing Information, including Patient Information.
Forward-Looking Statements
This press release contains "forward-looking" statements within the meaning of the safe harbor provisions of the U.S. Private Securities Litigation Reform Act of 1995. Forward-looking statements can be identified by words such as: "anticipate," "intend," "plan," "goal," "seek," "believe," "project," "estimate," "expect," "position," "strategy," "future," "likely," "may," "should," "will" and similar references to future periods. These statements are subject to numerous risks and uncertainties that could cause actual results to differ materially from what we expect. Examples of forward-looking statements include, among others, information relating to our business and business plans, the success of our efforts to commercialize EKTERLY® (sebetralstat), our ability to successfully obtain foreign regulatory approvals for sebetralstat, our expectations about the safety and efficacy of sebetralstat, the timing of clinical trials and their results, our ability to commence clinical studies or complete ongoing clinical studies, including our KONFIDENT-S and KONFIDENT-KID trials, and the ability of EKTERLY to treat HAE. Further information on potential risk factors that could affect our business and financial results are detailed in our filings with the Securities and Exchange Commission, including in our annual report on Form 10-K for the year ended April 30, 2025, our quarterly reports on Form 10-Q, and our other reports that we may make from time to time with the Securities and Exchange Commission. We undertake no obligation to publicly update any forward-looking statement, whether written or oral, that may be made from time to time, whether as a result of new information, future developments or otherwise.
1Farkas H., Martinez-Saguer I., Bork K., et al. International guideline on the diagnosis and management of pediatric patients with hereditary angioedema. Allergy. 2026;1–30. https://doi.org/10.1111/all.70207
2Christiansen S, et al. Ann Allergy Asthma Immunol. 2025; doi:10.1016/j.anai.2024.12.012
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Ryan Baker
Head, Investor Relations
(617) 771-5001
ryan.baker@kalvista.com
Molly Cameron
Director, Corporate Communications
(978) 339-3378
molly.cameron@kalvista.com