The European Medicines Agency has granted orphan designation to rilzabrutinib, a reversible covalent Bruton’s tyrosine kinase (BTK) inhibitor, for IgG4-related disease (IgG4-RD). EMA grants orphan designation to investigational therapies addressing rare, life-threatening or debilitating medical diseases or conditions that affect no more than 5 in 10,000 persons in the EU..

欧洲药品管理局（EMA）已授予可逆共价布鲁顿酪氨酸激酶（BTK）抑制剂rilzabrutinib孤儿药资格，用于治疗IgG4相关疾病（IgG4-RD）。EMA会为针对罕见、危及生命或致残性医学疾病或病症的在研疗法授予孤儿药资格，这些疾病或病症在欧盟中的发病率不超过每10,000人中5例。

Rilzabrutinib for the treatment of IgG4-related disease was evaluated in a phase 2 study (clinical study identifier:

Rilzabrutinib 治疗 IgG4 相关疾病的疗效在一项 2 期研究中得到了评估（临床研究标识符：

NCT04520451

NCT04520451

) and results were presented at the European Alliance of Associations for Rheumatology (EULAR) 2025 Congress. In IgG4-RD patients, treatment with rilzabrutinib for 52 weeks led to reduction in disease flare, other disease markers, and glucocorticoid sparing. The safety profile of rilzabrutinib in the study was consistent with previous studies, with no new safety signals observed.

）并在2025年欧洲风湿病学协会联盟（EULAR）大会上展示了结果。在IgG4-RD患者中，使用利扎布替尼治疗52周后，疾病复发减少，其他疾病标志物和糖皮质激素的使用也有所降低。研究中利扎布替尼的安全性与以往研究一致，未观察到新的安全性信号。

In addition to IgG4-related disease, rilzabrutinib has received orphan designations for immune thrombocytopenia (ITP) in the US, the EU, and Japan; and for

除了IgG4相关疾病外，rilzabrutinib还获得了美国、欧盟和日本的免疫性血小板减少症（ITP）孤儿药资格认定；以及

warm autoimmune hemolytic anemia

温暖型自身免疫性溶血性贫血

IgG4-RD

IgG4相关疾病

and

和

sickle cell disease

镰状细胞病

in the US. Rilzabrutinib has also been granted fast track designation in the US in ITP and IgG4-RD.

在美国，Rilzabrutinib 在 ITP 和 IgG4-RD 适应症中也获得了快速通道资格。

Rilzabrutinib is currently under regulatory review in the US, the EU, and China for its potential use in ITP. The target action date for the US FDA regulatory decision for ITP, which was granted fast track designation, is August 29, 2025.

Rilzabrutinib目前在美国、欧盟和中国正接受监管审查，用于潜在治疗ITP。美国FDA针对ITP的监管决策目标行动日期为2025年8月29日，该药物已被授予快速通道资格。

Rilzabrutinib is an investigational agent, and its safety and efficacy have not been evaluated by any regulatory authority.

Rilzabrutinib 是一种研究性药物，其安全性和有效性尚未经过任何监管机构的评估。

About rilzabrutinib

关于Rilzabrutinib

Rilzabrutinib is a novel, advanced, oral, reversible covalent BTK inhibitor that has the potential to be an effective new medicine for several rare immune-mediated or inflammatory diseases by working to restore immune balance via multi-immune modulation. BTK, expressed in B cells, macrophages, and other innate immune cells, plays a critical role in multiple immune-mediated disease processes and inflammatory pathways.

Rilzabrutinib 是一种新型、先进、口服、可逆的共价 BTK 抑制剂，通过多免疫调节来帮助恢复免疫平衡，有潜力成为治疗多种罕见免疫介导或炎症性疾病的高效新药。BTK 在 B 细胞、巨噬细胞和其他先天免疫细胞中表达，在多种免疫介导的疾病过程和炎症通路中发挥关键作用。

With the application of the TAILORED COVALENCY.

随着定制共价性的应用。

technology, rilzabrutinib can selectively inhibit the BTK target while potentially reducing the risk of off-target side effects.

技术上，rilzabrutinib可以有选择地抑制BTK靶点，同时可能减少脱靶副作用的风险。

About IgG4-RD

关于IgG4-RD

IgG4-RD is a progressive, relapsing, chronic immune-mediated rare disease, which can manifest in almost every organ and can lead to organ damage and irreversible dysfunction with a sometimes-fatal outcome. People with IgG4-RD experience frequent flare-ups of the condition characterized by periods of exacerbated symptoms.

IgG4-RD 是一种进行性、复发性、慢性免疫介导的罕见疾病，几乎可以影响每个器官，导致器官损伤和不可逆的功能障碍，有时甚至可能致命。患有 IgG4-RD 的人会经历病情频繁发作，症状在发作期加剧。

It affects approximately eight out of 100,000 adult patients in the US each year. Due to its rarity and challenges with diagnosis, the global prevalence of IgG4-RD is unknown..

在美国，每年大约每10万名成人患者中有8人受到影响。由于其罕见性和诊断的困难，IgG4-RD的全球患病率尚不清楚。

About Sanofi

关于赛诺菲

Sanofi is an R&D driven, AI-powered biopharma company committed to improving people’s lives and delivering compelling growth. We apply our deep understanding of the immune system to invent medicines and vaccines that treat and protect millions of people around the world, with an innovative pipeline that could benefit millions more. Our team is guided by one purpose: we chase the miracles of science to improve people’s lives; this inspires us to drive progress and deliver positive impact for our people and the communities we serve, by addressing the most urgent healthcare, environmental, and societal challenges of our time..

赛诺菲是一家以研发为驱动、以人工智能为支撑的生物制药公司，致力于改善人们的生活并实现引人注目的增长。我们凭借对免疫系统的深刻理解，研发药物和疫苗，为全球数百万人提供治疗与保护，同时我们的创新研发管线有望惠及更多人群。我们的团队秉承一个使命：追寻科学奇迹以改善人类生活；这激励我们推动进步，为我们服务的员工和社区带来积极影响，应对当今最紧迫的医疗、环境和社会挑战。

Sanofi is listed on EURONEXT: SAN and NASDAQ: SNY

赛诺菲在欧洲证券交易所上市，代码为SAN，在纳斯达克上市，代码为SNY。