Remibrutinib

iStock, TBE The first oral BTK blocker for chronic spontaneous urticaria, Rhapsido offers a more convenient treatment option for patients who still show symptoms after antihistamine treatment. The FDA has approved Novartis’ remibrutinib for the treatment of chronic spontaneous urticaria. The drug will be marketed under the brand name Rhapsido . In particular, Rhapsido is indicated for adult patients who continue to suffer from the symptoms of hives even after antihistamine treatment. In the U.S., that means a market of more than half of the 1.7 million people with the condition, according to a company press release on Wednesday. Rhapsido will have a wholesale acquisition cost of $4,521 per 30-day supply, and will be available “in the coming days,” a Novartis spokesperson told BioSpace in an email. Rhapsido is the first oral and targeted BTK inhibitor for chronic spontaneous urticaria (CSU). Before its approval, patients who do not respond completely to antihistamines were given injectable therapies, such as Roche’s Xolair. In April this year, the FDA expanded treatment options for CSU when it approved Sanofi and Regeneron’s Dupixent for the indication. Unlike Xolair and Dupixent, which are monoclonal antibodies, Rhapsido is an orally available blocker of the BTK enzyme. This mechanism disrupts a signaling cascade and prevents the secretion of histamine, which causes the itching and swelling typical of CSU. Phase III data from the REMIX program, published in March in the New England Journal of Medicine , showed treatment with Rhapsido resulted in improvements in urticaria activity scores versus placebo, a benefit that was sustained through 24 weeks of follow-up. In a prepared statement alongside Novartis’ press announcement on Tuesday, Giselle Mosnaim, University of Chicago immunologist and investigator on the REMIX trial, called Rhapsido’s approval “significant,” noting that it provides an alternative “beyond existing injectable treatments and gives patients an oral option that can easily be incorporated into their daily lives.” Besides CSU, Novartis is also testing remibrutinib—Rhapsido’s active ingredient—in other immune indications. In March last year, the pharma reported findings from a small Phase IIb study, demonstrating that a 25-mg dose of the drug achieved a 50% reduction in abscesses and inflammatory nodules in 73% of treated patients with hidradenitis suppurativa, compared with 34.7% of placebo comparators. At the time of that mid-stage readout, Novartis said it had plans to approval in this indication before the end of 2024, but it is unclear if the company pushed through with those plans. In its latest earnings presentation , remibrutinib’s hidradenitis suppurativa program was listed as a late-stage project. Beyond immunology, Novartis is also testing remibrutinib as a therapy for multiple sclerosis, for which it is running two Phase III studies. Primary completion for both trials is expected in 2026 .

诺华（Novartis）今日宣布，Rhapsido（remibrutinib）已获得美国FDA批准，用于治疗H1抗组胺药不能充分控制症状的慢性自发性荨麻疹（CSU）成人患者。Rhapsido是一种每日口服两次的片剂，患者无需接受注射或实验室监测。根据新闻稿，它是FDA批准用于CSU治疗的首个布鲁顿酪氨酸激酶抑制剂（BTKi）。诺华已向包括欧盟、日本和中国在内的全球监管机构递交监管申请，并在中国获得优先审评资格。 慢性自发性荨麻疹是一种难以预测的严重皮肤疾病，影响0.5-1%的全球人群。其特征为出现瘙痒、疼痛性荨麻疹、和/或肿胀，持续至少6周，且发生原因未知。由于CSU的严重程度和不可预测性，对患者的生活质量造成严重影响。H1抗组胺药是CSU的一线治疗药物，然而约60%患者的症状通过单用抗组胺药的控制结果不佳，在用药后持续存在CSU症状。BTK是组胺释放的核心酶，当自发激活时，在CSU相关症状中起关键作用。 FDA此次批准主要是基于REMIX-1和REMIX-2临床3期试验的结果，入组的CSU患者在使用第二代H1抗组胺药后仍存有症状。分析显示，Rhapsido在第12周时相较安慰剂在瘙痒严重程度评分（ISS7）、荨麻疹严重程度评分（HSS7）和每周荨麻疹活动评分（UAS7）的基线变化方面具有显著优势。与安慰剂组相较，Rhapsido组显著更多患者在第2周和第12周实现疾病良好控制（UAS7≤6），且约三分之一患者在第12周时完全无瘙痒和荨麻疹。Rhapsido的安全性已得到验证，患者无需持续进行实验室监测。最常见的不良事件（发生率≥3%）为鼻塞、咽喉痛和流涕（鼻咽炎）、出血、头痛、恶心和腹痛。 值得一提的是，REMIX-1和REMIX-2的结果也在今年3月发布于《新英格兰医学杂志》当中。详细数据显示，Rhapsido起效快，治疗第1周即可减轻患者瘙痒和荨麻疹症状，治疗第24周时，半数患者症状得到控制，约30%患者瘙痒和荨麻疹完全消失。 ▲REMIX-1（图A）和REMIX-2试验（图B）Rhapsido组（蓝绿色）和安慰剂组（灰色）UAS7评分降幅对比（图片来源：参考文献[2]） Rhapsido是一种高选择性、口服BTK抑制剂，可阻断BTK级联反应，并阻止组胺释放引起的瘙痒、荨麻疹和肿胀。它在几种免疫介导的疾病中显示出良好的安全性特征，包括多发性硬化、化脓性汗腺炎和食物过敏。 参考资料： [1] Novartis receives FDA approval for Rhapsido® (remibrutinib), the only oral, targeted BTKi treatment for chronic spontaneous urticaria (CSU). Retrieved September 30, 2025 from https://www.novartis.com/news/media-releases/novartis-receives-fda-approval-rhapsido-remibrutinib-only-oral-targeted-btki-treatment-chronic-spontaneous-urticaria-csu [2] Martin Metz,  Ana Giménez-Arnau,  Michihiro Hide,  et al. Remibrutinib in Chronic Spontaneous Urticaria. Published March 5, 2025. N Engl J Med 2025;392:984-994. DOI: 10.1056/NEJMoa2408792 免责声明：本文仅作信息交流之目的，文中观点不代表药明康德立场，亦不代表药明康德支持或反对文中观点。本文也不是治疗方案推荐。如需获得治疗方案指导，请前往正规医院就诊。 版权说明：欢迎个人转发至朋友圈，谢绝媒体或机构未经授权以任何形式转载至其他平台。转载授权请在「药明康德」微信公众号回复“转载”，获取转载须知。 分享，点赞，在看，聚焦全球生物医药健康创新