KalVista Pharmaceuticals Secures UK MHRA Approval for EKTERLY® (sebetralstat), Paving the Way for First Oral On-Demand Treatment for Hereditary Angioedema in the Region
KalVista Pharmaceuticals, Inc. (Nasdaq: KALV), a clinical-stage pharmaceutical company focused on developing innovative oral therapies for rare diseases, has reached a pivotal regulatory milestone with the United Kingdom’s Medicines and Healthcare products Regulatory Agency (MHRA) granting marketing authorization for EKTERLY® (sebetralstat). This newly approved plasma kallikrein inhibitor becomes the first and only oral on-demand therapy for the treatment of hereditary angioedema (HAE) attacks in patients aged 12 and older in the UK.
A Landmark Moment in HAE Treatment
Hereditary angioedema is a rare, potentially life-threatening genetic disorder marked by recurring episodes of swelling in various parts of the body, including the extremities, gastrointestinal tract, face, and airway. Until now, UK patients in need of immediate symptom relief during HAE attacks had access only to injectable therapies. The approval of EKTERLY represents a major step forward in patient care by offering a rapid, effective, and easy-to-administer oral treatment.
“This approval in the UK is especially meaningful for KalVista because EKTERLY was discovered in our Salisbury, UK laboratories,” said Ben Palleiko, CEO of KalVista Pharmaceuticals. “Bringing this innovation full circle—from discovery to approval in its country of origin—underscores the extraordinary capabilities of our team and our commitment to delivering transformative therapies to patients worldwide. With this milestone, we continue executing what we believe will be the fastest global launch of an HAE therapy ever.”
Backed by Robust Phase 3 Data
The MHRA’s decision is underpinned by compelling efficacy and safety data from the KONFIDENT Phase 3 clinical trial—the largest study of its kind ever conducted in HAE. The trial enrolled 136 patients from 66 clinical centers across 20 countries and was designed to evaluate the effectiveness of sebetralstat as an on-demand treatment during acute HAE attacks.
Findings from the KONFIDENT study were published in the New England Journal of Medicine in May 2024 and demonstrated that EKTERLY significantly outperformed placebo in multiple key measures:
- Faster symptom relief
- Reduced attack severity
- Rapid attack resolution
Importantly, EKTERLY was also shown to be well-tolerated, with a safety profile comparable to placebo, providing additional confidence for both physicians and patients in its use during unpredictable and distressing HAE episodes.
Clinical Perspective and Real-World Impact
For physicians working on the frontlines of HAE treatment, the MHRA’s decision marks a turning point. Dr. Sinisa Savic, Professor of Clinical Immunology at St James’s University Hospital in Leeds and an investigator in the KONFIDENT trial, emphasized the clinical significance of this development.
“The MHRA approval of EKTERLY brings us one step closer to transforming how we manage HAE attacks in the UK,” Dr. Savic noted. “For the first time, patients will have access to an oral on-demand treatment, which could improve both clinical outcomes and overall quality of life. I look forward to seeing its positive impact on the HAE community.”
Orphan Designation and Market Exclusivity
Further solidifying its regulatory status, EKTERLY has met the MHRA’s Orphan Designation criteria and will be added to the agency’s Orphan Register. This designation is granted to treatments developed for rare conditions that offer a significant benefit over existing options, and it allows EKTERLY to benefit from up to 10 years of market exclusivity in the UK. Such exclusivity is essential in incentivizing continued innovation in rare disease therapeutics.
Early Access Scheme and Path to NHS Availability
Even before full marketing authorization, EKTERLY was already reaching UK patients through the Early Access to Medicines Scheme (EAMS). In March 2025, the MHRA issued a positive scientific opinion under EAMS, enabling healthcare providers to prescribe EKTERLY to patients with urgent unmet needs prior to its formal approval.
This provisional access underscores the treatment’s critical value in urgent care scenarios, particularly when rapid relief is necessary and traditional injections may not be feasible or preferred. EKTERLY will continue to be available under EAMS until the National Institute for Health and Care Excellence (NICE) completes its review process, which is expected to result in a decision on National Health Service (NHS) coverage in the first half of 2026.
Global Expansion and Regulatory Momentum
The UK approval follows a similar landmark decision by the U.S. Food and Drug Administration (FDA), which granted marketing authorization for EKTERLY on July 3, 2025, for the treatment of HAE attacks in patients 12 years of age and older. KalVista is actively seeking regulatory approvals in other key global markets, including the European Union, Japan, and additional countries where HAE patient communities remain underserved.
This synchronized global strategy positions KalVista to rapidly expand access to EKTERLY, leveraging the strength of its clinical evidence and regulatory credibility across multiple regions.
KalVista’s successful navigation of the UK’s regulatory landscape, coupled with prior FDA approval and ongoing international submissions, reflects a cohesive global launch plan for EKTERLY. The company is firmly committed to ensuring widespread access to its therapy and furthering its leadership in HAE innovation.
This approval also illustrates the strategic importance of KalVista’s UK-based R&D operations, which have played a central role in discovering and developing sebetralstat. As the company builds out its commercial infrastructure and engages with healthcare systems worldwide, EKTERLY is poised to become a standard-of-care treatment for acute HAE attacks—redefining what’s possible for patients with this debilitating condition.
About KalVista Pharmaceuticals
KalVista Pharmaceuticals, Inc. is a clinical-stage pharmaceutical company dedicated to the discovery, development, and commercialization of small-molecule protease inhibitors for diseases with significant unmet need. The company’s focus includes treatments for hereditary angioedema and other kallikrein-mediated diseases. With a robust R&D pipeline and a strong foundation in protease biology, KalVista aims to transform treatment paradigms for patients worldwide.
Key Highlights:
- MHRA grants UK marketing authorization for EKTERLY® (sebetralstat)
- First and only oral on-demand HAE treatment approved in the UK
- Approval based on largest HAE clinical trial (KONFIDENT)
- Published in the New England Journal of Medicine (May 2024)
- Continues availability via EAMS while awaiting NICE NHS review (anticipated H1 2026)
- Already FDA-approved in the U.S. (July 2025)
- Regulatory submissions pending in EU, Japan, and other markets
With this latest approval, KalVista moves one step closer to fulfilling its mission: delivering patient-centered, breakthrough treatments for those living with hereditary angioedema, wherever they are in the world.
