KalVista Pharmaceuticals Receives Approval for EKTERLY® (sebetralstat), the First Oral On-Demand Treatment for Hereditary Angioedema
Wednesday, July 16, 2025
KalVista Pharmaceuticals has announced that the UK Medicines and Healthcare products Regulatory Agency (MHRA) has granted marketing authorisation for EKTERLY® (sebetralstat).
Hereditary angioedema is a rare genetic condition caused by a deficiency or dysfunction of the C1 esterase inhibitor protein. This leads to uncontrolled activation of the kallikrein-kinin system and results in recurring attacks of severe swelling in various parts of the body. These attacks can be painful, disabling, and potentially life-threatening. Clinical guidelines recommend treating attacks early to reduce their impact and duration, regardless of where they occur or how severe they are.
EKTERLY is a novel oral plasma kallikrein inhibitor developed for the on-demand treatment of hereditary angioedema attacks. It is approved for use in individuals aged 12 years and older and is currently under investigation for use in younger children. With multiple regulatory reviews underway worldwide, EKTERLY has the potential to become a central option in the global management of HAE.
This marks the first and only oral on-demand treatment approved in the UK for hereditary angioedema (HAE) attacks in adults and adolescents aged 12 years and older.
EKTERLY is a plasma kallikrein inhibitor developed by KalVista to offer a more accessible and convenient option for HAE patients managing acute attacks. The approval in the UK follows its earlier authorisation by the US Food and Drug Administration (FDA) in July 2025 and reflects the company’s broader plan to expand access to sebetralstat globally. The treatment was discovered in KalVista’s laboratories in Salisbury, United Kingdom.
The MHRA's decision is based on findings from the phase 3 KONFIDENT study, the largest clinical trial ever conducted in HAE. The trial enrolled 136 patients across 66 sites in 20 countries. Published in The New England Journal of Medicine in May 2024, the results demonstrated that EKTERLY provided faster symptom relief, reduced the severity of attacks, and led to quicker resolution compared to placebo. The safety profile of EKTERLY was also comparable to placebo, indicating it was well tolerated.
EKTERLY has been included in the MHRA’s Orphan Register after meeting the criteria for Orphan Designation. As a result, the product will benefit from up to 10 years of market exclusivity in the UK.
Earlier this year, the MHRA also issued a positive scientific opinion for EKTERLY under the Early Access to Medicines Scheme (EAMS). This allowed clinicians to prescribe the treatment before formal marketing approval for patients with significant unmet medical needs. EKTERLY will continue to be available under the EAMS framework until a reimbursement decision is made by the National Institute for Health and Care Excellence (NICE), which is expected in the first half of 2026.
KalVista has also submitted marketing applications in the European Union, Japan, and other global regions.
Source: kalvista.com
