KalVista Pharma has finally received FDA approval for its oral kallikrein inhibitor sebetralstat, becoming the first treatment in the US that can provide oral, on-demand treatment of hereditary angioedema (HAE) attacks.

KalVista制药公司终于获得了FDA对其口服激肽释放酶抑制剂Sebetralstat的批准，成为美国首个可以提供口服按需治疗遗传性血管性水肿（HAE）发作的药物。

The drug has been approved under the Ekterly brand name for use in patients aged 12 and over with HAE, which affects about 1 in 10,000 to 1 in 50,000 people worldwide and leads to unpredictable bouts of debilitating and painful swelling in various parts of the body.

该药物已获准以Ekterly品牌名用于12岁及以上患有HAE的患者，HAE影响全球约万分之一到五万分之一的人口，并导致身体各部位出现不可预测的、使人衰弱且疼痛的肿胀发作。

The FDA had been due to deliver a verdict on

美国食品药品监督管理局（FDA）原本应该发布一份决定

sebetralstat

赛贝曲普坦

by 17th June, but the review was delayed and due to what the company suggested was disruption at the agency caused by sweeping job cuts.

截至6月17日，但审查因公司所称的机构因大规模裁员而造成的混乱而被推迟。

There have been unconfirmed

存在未经证实的

reports

报告

that FDA Commissioner Marty Makary had sought a complete response letter (CRL) for the application as a punishment for blaming the shake-up at the regulator for the delay, but was prevented from doing so on concerns it would make the agency look capricious.

FDA专员马蒂·马卡里曾要求对申请发出完整回应函（CRL），以此作为对将延迟归咎于监管机构改组的惩罚，但因担忧此举会让机构显得反复无常而未能如愿。

With that speculation in the past, Anglo-US KalVista can now plan for the launch of what it says will be the first new on-demand treatment for HAE attacks in a decade, with chief executive Ben Palleiko calling the approval 'a defining moment' for people living with the disease. 'Ekterly enables people to treat attacks the moment symptoms begin, wherever they are,' he said..

过去这种猜测一直存在，现在英美合资的KalVista公司可以计划推出其声称是十年来首个按需治疗HAE发作的新疗法。首席执行官本·帕莱科称此次获批是“对HAE患者来说具有决定性意义的时刻”。他说：“Ekterly使人们能够在症状刚开始时就进行治疗，无论他们身在何处。”

There is already an oral plasma kallikrein inhibitor on the market – BioCryst Pharma's Orladeyo (berotralstat). However, that is designed for daily dosing to prevent attacks, while KalVista's drug would be taken when a patient feels an attack coming on. If the swelling in an attack affects areas like the throat or airways it can have life-threatening consequences..

市场上已经有一种口服血浆激肽释放酶抑制剂——BioCryst制药公司的Orladeyo（贝罗司他）。然而，该药物设计为每日服用以预防发作，而KalVista的药物则是在患者感到即将发作时才服用。如果发作导致喉咙或气道等部位肿胀，可能会带来危及生命的后果。

Even with the use of long-term prophylaxis as a preventative therapy, most people living with HAE continue to have unpredictable attacks and require ready access to on-demand medication, according to KalVista.

据 KalVista 称，即使使用长期预防疗法，大多数遗传性血管性水肿患者仍会继续出现不可预测的发作，并需要随时获得按需药物。

Until now, drugs designed to treat acute episodes had to be delivered by subcutaneous injection or infusion, in some cases by a healthcare professional, which can delay treatment. Moreover, these are typically not able to prevent attacks entirely, and sometimes multiple doses are needed to get symptoms under control..

直到现在，用于治疗急性发作的药物都必须通过皮下注射或输注的方式给药，有时需要由医疗专业人员进行操作，这可能会延误治疗。此外，这些方法通常无法完全预防发作，有时需要多次给药才能控制症状。

KalVista said it will launch Ekterly immediately and will price it in the same ballpark as other on-demand therapies. Further details will be available after an investor presentation later today.

KalVista 表示将立即推出 Ekterly，并将其定价在与其他按需治疗相同的范围内。更多细节将在今天晚些时候的投资者报告后公布。

Other on-demand HAE drugs include Takeda's bradykinin B2 receptor antagonist Firazyr (icatibant) and plasma kallikrein inhibitor Kalbitor (ecallantide), and Pharming's Ruconest and CSL Behring's Berinert, both recombinant C1 esterase inhibitors.

其他按需使用的HAE药物包括武田的缓激肽B2受体拮抗剂Firazyr（艾卡肽）和血浆激肽释放酶抑制剂Kalbitor（依卡兰肽），以及Pharming公司的Ruconest和CSL Behring公司的Berinert，两者均为重组C1酯酶抑制剂。

Analysts at Jefferies have previously said that if approved, sebetralstat could make sales of around $500 million a year in the US and Europe.

Jefferies的分析师此前曾表示，如果获得批准，sebetralstat在美国和欧洲的年销售额可能达到5亿美元左右。