KalVista Pharmaceuticals has announced that the UK Medicines and Healthcare products Regulatory Agency (MHRA) has granted marketing authorisation for EKTERLY® (sebetralstat).

卡尔维斯塔制药公司宣布，英国药品和健康产品管理局（MHRA）已授予EKTERLY®（塞贝曲斯坦）上市许可。

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.

遗传性血管性水肿是一种罕见的遗传性疾病，由C1酯酶抑制蛋白缺乏或功能异常引起。这会导致激肽释放酶-激肽系统的失控激活，从而引发身体不同部位反复出现严重肿胀。这些发作可能伴有疼痛、导致行动不便，并有可能危及生命。

Clinical guidelines recommend treating attacks early to reduce their impact and duration, regardless of where they occur or how severe they are..

临床指南建议尽早治疗发作，以减少其影响和持续时间，无论发作发生在何处或严重程度如何。

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..

EKTERLY 是一种新型的口服血浆激肽释放酶抑制剂，用于按需治疗遗传性血管性水肿发作。它已获批用于12岁及以上人群，并正在研究用于更年幼的儿童。随着全球范围内多项监管审查的进行，EKTERLY 有望成为全球 HAE 管理中的核心选择。

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.

这标志着英国首次且唯一批准用于治疗 12 岁及以上青少年和成人遗传性血管性水肿 (HAE) 发作的口服按需治疗药物。

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.

EKTERLY 是由 KalVista 开发的一种血浆激肽释放酶抑制剂，旨在为遗传性血管性水肿（HAE）患者提供更易获取且便捷的急性发作管理选择。该药物在英国的获批紧随其于 2025 年 7 月获得美国食品药品监督管理局（FDA）的批准，体现了该公司扩大 Sebetralstat 全球可及性的更广泛计划。

The treatment was discovered in KalVista’s laboratories in Salisbury, United Kingdom..

该治疗方法是在英国索尔兹伯里 KalVista 的实验室中发现的。

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.

英国药品和健康产品管理局的决定基于第三阶段KONFIDENT研究的发现，这是有史以来在遗传性血管性水肿（HAE）领域进行的最大规模临床试验。该试验在全球20个国家的66个地点招募了136名患者。研究结果于2024年5月发表在《新英格兰医学杂志》上，结果显示，与安慰剂相比，EKTERLY能够更快缓解症状、减轻发作严重程度并加速恢复。

The safety profile of EKTERLY was also comparable to placebo, indicating it was well tolerated..

EKTERLY 的安全性与安慰剂相当，表明其耐受性良好。

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.

EKTERLY 在符合孤儿药资格标准后，已被列入 MHRA 的孤儿药注册名单。因此，该产品将在英国享有最长 10 年的市场独占期。

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..

今年早些时候，英国药品和健康产品管理局 (MHRA) 还在“药品早期获取计划”(EAMS) 下对 EKTERLY 发表了积极的科学意见。这使得临床医生能够在正式上市批准前为具有显著未满足医疗需求的患者开具该治疗药物。EKTERLY 将继续在 EAMS 框架下提供，直到英国国家卫生与临床优化研究所 (NICE) 做出报销决定，预计该决定将在 2026 年上半年作出。

KalVista has also submitted marketing applications in the European Union, Japan, and other global regions.

卡尔维斯塔还在欧盟、日本和其他全球地区提交了上市申请。