Dive Brief:

简报：

Pharvaris said Wednesday its pill for the rare swelling disorder hereditary angioedema succeeded in a Phase 3 trial,

法瓦里斯周三表示，其用于治疗罕见的遗传性血管性水肿的药物在第三阶段试验中取得了成功，

helping to begin relieving symptoms

帮助开始缓解症状

of an attack within 1.28 hours, significantly faster than a placebo.

在1.28小时内发生攻击，明显快于安慰剂。

The Netherlands-based biotechnology company said it plans on asking the Food and Drug Administration in the first half of 2026 to approve deucrictibant. An extended release formulation of the medication is also in a Phase 3 trial to test whether it can prevent HAE attacks, with data expected next year..

总部位于荷兰的生物技术公司表示，计划在2026年上半年请求食品和药物管理局批准“deucrictibant”。该药物的缓释配方也正在进行第三阶段试验，以测试其是否能够预防HAE发作，预计明年将获得数据。

If approved, deucrictibant would be entering an increasingly competitive market. The first oral HAE drug to relieve attacks, Kalvista’s Ekterly,

如果获得批准，deucrictibant 将进入一个竞争日益激烈的市场。首款缓解 HAE 发作的口服药物，Kalvista 的 Ekterly，

got an FDA nod

获得了FDA的批准

this year, while two injectable drugs, including one to relieve attacks, also

今年，同时有两种注射药物，其中一种用于缓解发作，也

recently

最近

launched

已启动

.

。

Dive Insight:

深入洞察：

Treatment of HAE began with plasma products designed to replace a protein called C1 that regulates blood vessel dilation, but many of these early products could only be administered intravenously. That began to change in the early 2010s with Firazyr, now sold by Takeda, as the first self-administered injection for HAE attacks..

HAE的治疗始于设计用来替代调节血管扩张的C1蛋白的血浆产品，但许多早期产品只能通过静脉注射给药。这种情况在2010年代初期开始改变，当时Takeda推出的Firazyr成为首个用于HAE发作的自我注射药物。

More medicines designed to relieve symptoms of attacks or reduce their frequency have been introduced in the years since. However, 2025 broke new ground with the approval of three drugs: Ekterly, and the preventive shots Andembry from CSL and Dawnzera from Ionis Pharmaceuticals.

近年来，已有更多旨在缓解发作症状或减少其频率的药物问世。然而，2025年因三种药物的批准而开创了新天地：Ekterly，以及CSL的预防性注射药物Andembry和Ionis Pharmaceuticals的Dawnzera。

Ekterly’s launch pointed to strong demand for an oral drug. Sales of $13.7 million for the

Ekterly的上市表明了对口服药物的强劲需求。销售额达到1370万美元。

third quarter of 2025

2025年第三季度

well exceeded Leerink Partners’ forecast of $4 million. In assessing the deucrictibant data, Leerink analyst Joseph Schwartz wrote “the market is very receptive to new options in the HAE space.”

远超Leerink Partners预测的400万美元。在评估deucrictibant数据时，Leerink分析师Joseph Schwartz写道：“市场对HAE领域的新选择非常欢迎。”

”We continue to believe that the HAE market is large enough for multiple players, especially as it continues to expand with the entrance of new options,” Schwartz wrote.

“我们仍然认为HAE市场足够大，可以容纳多个参与者，尤其是随着新选择的出现，市场还在继续扩大，”施瓦茨写道。

On its primary endpoint, time to onset of symptom response, deucrictibant’s median 1.28 hours is numerically better than Ekterly’s 1.79 hours for its FDA-approved first dose, Schwartz added, although the two drugs haven’t been tested head-to-head. More impressive, he wrote, was the 11.95 hours median time to complete resolution of symptoms for deucrictibant, a measure on which Ekterly took more than 24 hours..

施瓦茨补充道，在其主要终点——症状缓解的起效时间上，deucrictibant的中位时间为1.28小时，数值上优于Ekterly首个FDA批准剂量的1.79小时，尽管这两种药物尚未进行过头对头试验。他写道，更令人印象深刻的是，deucrictibant的症状完全缓解的中位时间为11.95小时，而Ekterly在这一指标上耗时超过24小时。

Stifel analyst Paul Matteis, who covers Kalvista, said the differences in effectiveness might not necessarily drive patients to switch once they have begun taking Ekterly, however.

Stifel 分析师保罗·马蒂斯（Paul Matteis）在谈到Kalvista时说，然而，疗效上的差异不一定就会驱使患者在开始服用Ekterly后转而更换药物。

“If you look historically at the on demand market, subtle differences in efficacy haven't really been the determinant of majority share,” Matteis wrote. Within a few months, Kalvista penetrated the market by about 10% and “will have the opportunity to entrench itself further” before deucrictibant might gain approval..

“如果你从历史上看按需市场，疗效上的细微差异并不是决定多数份额的关键因素，”马特伊斯写道。在几个月内，Kalvista的市场渗透率达到了约10%，并且“将有机会进一步巩固自身地位”，然后deucrictibant可能会获得批准。