Novartis has claimed a first-in-class regulatory approval in the US for oral BTK inhibitor remibrutinib as a second-line treatment for chronic spontaneous urticaria (CSU), also known as chronic hives.

诺华公司声称，其口服BTK抑制剂remibrutinib已在美国获得首个同类监管批准，作为慢性自发性荨麻疹（CSU），也称为慢性荨麻疹的二线治疗药物。

The FDA has cleared

FDA已经批准

remibrutinib

瑞米布鲁替尼

under the Rhapsido trade name as the first oral alternative to current injectable treatments, namely Novartis' anti-IgE antibody Xolair (omalizumab), which has been a mainstay of refractory treatment for

在Rhapsido的商标下，作为当前可注射治疗的首个口服替代品，即诺华公司的抗IgE抗体Xolair（奥马珠单抗），这一直是难治性治疗的中流砥柱。

CSU

加州州立大学

for many years, and Sanofi/Regeneron's recently approved IL-4 and IL-13 inhibitor

多年来，赛诺菲/再生元最近批准的IL-4和IL-13抑制剂

Dupixent

杜匹鲁单抗

(dupilumab).

（度普利尤单抗）。

CSU affects around 1% of the global population and, in severe cases, causes itching and swelling that can last for weeks at a time and resist even very high doses of antihistamines, which are the first-line therapy of choice but don't work in around 60% of cases.

慢性自发性荨麻疹（CSU）影响全球约1%的人口，在严重情况下，会导致持续数周的瘙痒和肿胀，甚至抵抗极高的抗组胺药剂量。抗组胺药是首选的一线治疗药物，但在大约60%的病例中不起作用。

Novartis estimates that there are around 1.7 million people with uncontrolled CSU in the US alone, with more than half not responding adequately to antihistamines alone, but fewer than 20% of eligible patients are currently receiving the injectable drugs second-line.

诺华估计，仅在美国，就有大约170万慢性自发性荨麻疹（CSU）得不到控制的患者，其中超过一半的人单独使用抗组胺药效果不佳，但目前只有不到20%的符合条件的患者正在接受二线注射药物治疗。

There is no question that CSU is enormously debilitating, with almost two-thirds of patients experiencing mental health disorders, including depression and anxiety, so the availability of an additional therapeutic option for those most severely affected by symptoms is a major step forward, according to the Allergy & Asthma Network patient organisation..

根据过敏与哮喘网络患者组织的说法，毫无疑问，CSU（慢性自发性荨麻疹）造成了极大的衰弱影响，近三分之二的患者出现心理健康问题，包括抑郁和焦虑，因此为那些症状最严重的患者提供额外的治疗选择是一个重大进步。

'Many CSU patients feel misunderstood and settle for treatments that don't fully meet their needs,' said Lynda Mitchell, the group's chief executive. 'This convenient new oral therapy offers a promising new way to manage CSU and potentially improve daily life for those living with this challenging condition.'.

“许多CSU患者感到被误解，并接受不能完全满足他们需求的治疗，”该组织首席执行官琳达·米切尔表示。“这种方便的新口服疗法提供了一种管理CSU的有前景的新方法，并有可能改善那些面对这一挑战性病症的患者的日常生活。”

The FDA approval comes on the back of a pair of phase 3 trials –

FDA 的批准基于两项 3 期试验的结果——

REMIX-1 and REMIX-2

REMIX-1 和 REMIX-2

– which showed that a twice-daily dose of Rhapsido achieved significant improvements in urticaria activity, itch severity, and hive severity scores compared to placebo.

— 结果显示，与安慰剂相比，每日两次服用Rhapsido可显著改善荨麻疹活动、瘙痒严重程度和风团严重程度评分。

Almost half of the patients treated with Novartis' drug were completely free of itch and hives at the end of the 52-week follow-up period.

几乎一半的患者在为期 52 周的随访期结束后，使用诺华药物治疗后完全摆脱了瘙痒和荨麻疹。

Novartis said that Rhapsido will cost $4,521 for a 30-day supply at list prices and will be available to patients within the next few days. Meanwhile, applications have been filed in the EU, China, and Japan.

诺华表示，Rhapsido 的定价为 4,521 美元/30 天疗程，并将在未来几天内提供给患者。同时，该公司已向欧盟、中国和日本提交了上市申请。

Analysts have suggested that approval in CSU alone could make the drug a blockbuster, with sales of more than $1 billion a year, and Novartis is running phase 3 trials for chronic inducible urticaria (CINDU) and multiple sclerosis (MS), and phase 2 studies in hidradenitis suppurativa (HS) and food allergy indications..

分析师表示，仅获得 CSU 批准就可能使该药成为重磅药物，年销售额超过 10 亿美元。诺华正在开展针对慢性诱导性荨麻疹 (CINDU) 和多发性硬化症 (MS) 的 3 期试验，以及针对化脓性汗腺炎 (HS) 和食物过敏适应症的 2 期研究。

Sanofi is also developing an oral BTK inhibitor called

赛诺菲公司也在开发一种名为 BTK 抑制剂的口服药物

rilzabrutinib

利扎布替尼

for CSU, which has had phase 2 results in hand since 2024 but has not yet advanced into phase 3 for this indication. It is also being tested for asthma, IgG4-related disease, and other inflammatory diseases and was recently approved by the FDA as

对于CSU，该药物自2024年以来已取得第二阶段试验结果，但尚未针对这一适应症进入第三阶段。同时，它也正在被测试用于哮喘、IgG4相关疾病及其他炎症性疾病，并且最近刚获得FDA批准作为。

Wayrilz

瓦伊利兹

for immune thrombocytopenia (ITP).

免疫性血小板减少症（ITP）。