. The US Food and Drug Administration (FDA) has granted orphan drug designation to rilzabrutinib, a novel, advanced, oral, reversible Bruton's tyrosine kinase (BTK) inhibitor that works via multi-immune modulation, to target a reduction in vaso-occlusive crises, which may occur via inflammation, in sickle cell disease.

美国食品药品监督管理局（FDA）已授予rilzabrutinib孤儿药资格，这是一种新型、先进、口服、可逆的布鲁顿酪氨酸激酶（BTK）抑制剂，通过多途径免疫调节发挥作用，旨在减少镰状细胞病中可能由炎症引发的血管阻塞性危象。

The FDA grants orphan drug designation to investigational therapies addressing rare medical diseases or conditions that affect fewer than 200,000 people in the US..

美国食品药品监督管理局（FDA）授予用于治疗美国境内患病人数少于20万人的罕见疾病或病症的在研疗法孤儿药资格。

Karin Knobe, MD, PhD

卡琳·克诺贝，医学博士，哲学博士

Global Head of Development, Rare Diseases

全球研发主管，罕见病部门

“Receiving our fourth orphan drug designation for rilzabrutinib reinforces our continued dedication to developing medicines to address the unmet medical needs of people living with rare diseases. People with sickle cell disease often live with severe episodes of pain from vaso-occlusive crises and other complications that can significantly impact both quality of life and life expectancy.

“Rilzabrutinib获得我们的第四项孤儿药资格认定，再次证明了我们持续致力于开发药物以满足罕见病患者的未竟医疗需求。镰状细胞病患者常常因血管阻塞危象及其他并发症而经历剧烈的疼痛发作，这些症状可能严重影响生活质量和预期寿命。”

There remains a need for novel treatment approaches to address these experiences by modulating the immune system responses that can contribute to sickle cell disease pathogenesis.”.

仍然需要通过调节可能导致镰状细胞病发生的免疫系统反应，来开发新的治疗方法以应对这些症状。

In addition to sickle cell disease, rilzabrutinib has received orphan drug designation for immune thrombocytopenia (ITP) in the US, the EU, and Japan, for

除了镰状细胞病外，利扎布替尼还获得了美国、欧盟和日本的免疫性血小板减少症（ITP）孤儿药资格认定，用于

warm autoimmune hemolytic anemia

温暖型自身免疫性溶血性贫血

(wAIHA) in the US and the EU, and for

（wAIHA）在美国和欧盟，以及

IgG4-related disease

IgG4相关疾病

(IgG4-RD) in the US.

美国的 IgG4-RD。

The safety and efficacy of rilzabrutinib have not been determined by any regulatory authority. Rilzabrutinib is currently under regulatory review in the US, the EU, and in China for its potential use in ITP. The target action date for the FDA regulatory decision for ITP, which was granted fast track designation, is August 29, 2025..

任何监管机构都尚未确定利扎布替尼的安全性和有效性。利扎布替尼目前在美国、欧盟和中国正接受监管审查，用于其在ITP中的潜在应用。FDA针对ITP的监管决定的目标行动日期为2025年8月29日，该药物已被授予快速通道资格。

Sickle cell disease supporting data

镰状细胞病支持数据

Preclinical data on sickle cell disease was

镰状细胞病的临床前数据

presented at ASH 2024

在2024年ASH会议上展示

showing that rilzabrutinib helped reduce vaso-occlusion –blockage of blood vessels – and inflammation in transgenic mice with sickle cell disease.

显示rilzabrutinib有助于减少患有镰状细胞病的转基因小鼠的血管阻塞和炎症。

About rilzabrutinib

关于rilzabrutinib

Rilzabrutinib is a novel, advanced, oral, reversible Bruton’s tyrosine kinase (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、可逆的布鲁顿酪氨酸激酶 (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 Sanofi’s TAILORED COVALENCY.

随着赛诺菲的TAILORED COVALENCY技术的应用。

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

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

About sickle cell disease

关于镰状细胞病

Sickle cell disease is a group of rare, genetic blood disorders in which red blood cells are misshapen, typically in a sickle or crescent shape, causing them to get stuck in small blood vessels, blocking blood flow. This can lead to episodes of severe pain as well as other health complications including infections, stroke, lung, eye, and kidney disease.

镰状细胞病是一组罕见的遗传性血液疾病，患者的红细胞呈镰刀形或新月形，导致它们卡在小血管中，阻碍血流。这会引发剧烈疼痛，并导致包括感染、中风、肺部、眼部和肾脏疾病在内的其他健康问题。

Sickle cell disease affects more than 100,000 people in the US, approximately 90% of whom are African American and 3-9% of whom are Hispanic or Latino. Approximately 1 in 365 babies of African American descent born in the US have sickle cell disease, while an estimated 1 in 13 are carriers of the disease.

镰状细胞病影响着美国超过10万人，其中约90%是非裔美国人，3-9%是西班牙裔或拉丁裔。在美国出生的非裔婴儿中，大约每365名就有1名患有镰状细胞病，而据估计每13人中有1人是该病的携带者。

People in the US with sickle cell disease have an estimated life expectancy that is 20 years shorter than average..

美国镰状细胞病患者的预期寿命比平均寿命短20年。

About Sanofi

关于赛诺菲

Sanofi is an R&D driven, AI-powered biopharma company committed to improving people’s lives and creating 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..

赛诺菲是一家以研发为驱动、以人工智能为助力的生物制药公司，致力于改善人们的生活并创造引人注目的增长。我们运用对免疫系统的深刻理解，研发能够治疗和保护全球数百万人的药物和疫苗，同时拥有一个创新的研发管线，有望使数百万人进一步受益。

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.

我们的团队以一个目标为指导：我们追逐科学的奇迹以改善人们的生活；这激励我们推动进步，通过应对当今最紧迫的医疗、环境和社会挑战，为我们服务的人们和社区带来积极的影响。

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。