. The US Food and Drug Administration (FDA) has granted orphan drug designation to rilzabrutinib, an investigational, novel, advanced, oral, reversible Bruton’s tyrosine kinase (BTK) inhibitor, for two rare diseases, warm autoimmune hemolytic anemia (wAIHA) and IgG4-related disease (IgG4-RD). There is still a significant unmet medical need for these two rare diseases, and neither have any currently approved medicine.

美国食品药品监督管理局（FDA）已授予rilzabrutinib孤儿药资格，这是一种研究性、新型、先进、口服、可逆的Bruton酪氨酸激酶（BTK）抑制剂，用于治疗两种罕见疾病：温抗体型自身免疫性溶血性贫血（wAIHA）和IgG4相关疾病（IgG4-RD）。这两种罕见疾病目前仍存在显著的未满足医疗需求，并且尚无获批的治疗药物。

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

FDA授予用于治疗美国境内患者少于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

全球开发主管，罕见病部门

“Orphan drug designation for these two rare, immune-mediated conditions validates our ongoing commitment to pursuing potential first- and best-in-class medicines for diseases that affect small populations but persist with unmet medical need. Our continued exploration of rilzabrutinib across multiple indications speaks to our belief in its potential for multi-immune modulation, as well as our belief in supporting treatment options, no matter how rare a condition.”.

“这两种罕见的免疫介导疾病获得孤儿药资格认定，证明了我们持续致力于为影响小众但存在未满足医疗需求的疾病开发潜在的首创和最佳药物。我们在多个适应症中继续探索rilzabrutinib，体现了我们对其在多免疫调节方面潜力的信心，同时也表明我们支持无论多么罕见病症的治疗选择。”

Rilzabrutinib is currently under regulatory review in the US, the EU, and China for its potential use in immune thrombocytopenia (ITP). The target action date for the FDA regulatory decision for ITP, which was granted fast track designation, is August 29, 2025. Rilzabrutinib also received orphan drug designation for ITP in the US, EU, and Japan..

Rilzabrutinib目前在美国、欧盟和中国正处于监管审查中，用于潜在治疗免疫性血小板减少症（ITP）。FDA对该药物用于ITP的监管决定目标行动日期为2025年8月29日，该药物已被授予快速通道资格。Rilzabrutinib还获得了美国、欧盟和日本针对ITP的孤儿药资格认定。

wAIHA and IgG4-RD supporting data

wAIHA 和 IgG4-RD 支持数据

Results from a phase 2b study on wAIHA

关于wAIHA的2b期研究结果

presented at ASH 2024

在2024年ASH会议上发表

(clinical study identifier: NCT05002777) demonstrated that treatment with rilzabrutinib showed clinically meaningful outcomes on response rate and disease markers.

（临床研究标识符：NCT05002777）表明，使用rilzabrutinib治疗在反应率和疾病标志物方面显示出具有临床意义的结果。

In IgG4-RD patients, results from a phase 2a study (clinical study identifier: NCT04520451) showed treatment with rilzabrutinib for 52 weeks led to reduction in disease flare, other disease markers, and glucocorticoid sparing. More detailed results will be shared at a forthcoming medical meeting.

在IgG4-RD患者中，一项2a期研究（临床研究标识符：NCT04520451）的结果显示，使用rilzabrutinib治疗52周可减少疾病复发、其他疾病标志物以及糖皮质激素的用量。更详细的结果将在即将举行的医学会议上分享。

The safety profile of rilzabrutinib in both studies was consistent with previous studies.

两项研究中，利扎布替尼的安全性特征与之前的研究一致。

About rilzabrutinib

关于利扎布替尼

Rilzabrutinib is an investigational, oral, reversible, BTK inhibitor that has the potential to be a first- and best-in-class treatment of several immune-mediated diseases. BTK, expressed in B cells, macrophages, and other innate immune cells, plays a critical role in inflammatory pathways and multiple immune-mediated disease processes.

Rilzabrutinib 是一种研究性、口服、可逆的 BTK 抑制剂，有潜力成为多种免疫介导疾病的首选用药和最佳治疗选择。BTK 在 B 细胞、巨噬细胞和其他先天免疫细胞中表达，于炎症通路和多种免疫介导的疾病过程中发挥关键作用。

With the application of Sanofi’s TAILORED COVALENCY.

随着赛诺菲的TAILORABLE COVALENCY技术的应用。

technology, rilzabrutinib can selectively inhibit the BTK target while potentially reducing the risk of off-target side effects. Based on its ability to drive multi-immune modulation, rilzabrutinib holds great promise in the treatment of multiple clinical indications.

技术上，利扎布替尼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。基于其驱动多免疫调节的能力，利扎布替尼在治疗多种临床指征方面具有很大的潜力。

About wAIHA

关于wAIHA

Affecting one to three people out of 100,000 in the US each year, wAIHA is a rare, potentially life-threatening, autoimmune disorder where autoantibodies lead to the premature destruction of the body’s own red blood cells (hemolysis). People living with wAIHA may experience debilitating fatigue, thromboembolism, dizziness, palpitations, and shortness of breath as the rate of production of new red blood cells in their bone marrow cannot compensate quickly enough for premature destruction of red blood cells..

每年在美国，每 100,000 人中就有 1 到 3 人受到影响，温抗体型自身免疫性溶血性贫血 (wAIHA) 是一种罕见的、可能危及生命的自身免疫性疾病，自身抗体导致体内红细胞过早破坏（溶血）。wAIHA 患者可能会出现令人虚弱的疲劳、血栓栓塞、头晕、心悸和呼吸急促，因为他们的骨髓中新生红细胞的生成速度无法足够快地补偿红细胞的过早破坏。

About IgG4-RD

关于IgG4-RD

IgG4-RD affects approximatively eight out of 100,000 adult patients in the US each year and is a rare, progressive, relapsing, chronic fibro-inflammatory condition which can manifest in almost every organ and can lead to organ damage and irreversible dysfunction with a sometimes-fatal outcome.

IgG4-RD 每年在美国大约影响八万分之一的成年患者，是一种罕见的、进行性的、反复发作的慢性纤维炎症性疾病，可累及几乎所有的器官，导致器官损伤和不可逆的功能障碍，有时甚至可能致命。

About ITP

关于ITP

ITP is a rare, complex autoimmune disorder characterized by low platelet counts (less than 100,000/μL) resulting from both increased platelet destruction and decreased platelet production. Beyond bruising and bleeding, which can include potentially life-threatening episodes like intracranial hemorrhage, people living with ITP may experience arterial or venous thrombosis, which can result from the ITP itself, from other medical comorbidities, or may also be associated with the use of certain other ITP treatments.

ITP是一种罕见的、复杂的自身免疫性疾病，其特征是血小板计数低（少于100,000/μL），这是由于血小板破坏增加和血小板生成减少所致。除了瘀伤和出血（可能包括危及生命的事件，如颅内出血）之外，ITP患者可能会经历动脉或静脉血栓形成，这可能是由ITP本身、其他医疗合并症引起的，也可能与某些其他ITP治疗的使用有关。

Additionally, people living with ITP often experience easily overlooked symptoms that can significantly impair their quality of life, such as unexplained fatigue, anxiety or depression, and cognitive impairment. .

此外，ITP患者通常会出现容易被忽视的症状，这些症状会严重影响他们的生活质量，例如无法解释的疲劳、焦虑或抑郁以及认知障碍。

About Sanofi

关于赛诺菲

We are an innovative global healthcare company, driven by one purpose: we chase the miracles of science to improve people’s lives. Our team, across the world, is dedicated to transforming the practice of medicine by working to turn the impossible into the possible. We provide potentially life-changing treatment options and life-saving vaccine protection to millions of people globally, while putting sustainability and social responsibility at the center of our ambitions..

我们是一家创新的全球医疗保健公司，由一个目标驱动：我们追逐科学的奇迹以改善人们的生活。我们的团队遍布全球，致力于通过努力将不可能变为可能来改变医学实践。我们为全球数百万人提供可能改变生命的治疗选择和挽救生命的疫苗保护，同时将可持续发展和社会责任置于我们抱负的核心。