. The Ministry of Health, Labour and Welfare (MHLW) in Japan has granted orphan drug designation to riliprubart, a monoclonal antibody that selectively inhibits activated C1s in the classical complement pathway for people with chronic inflammatory demyelinating polyneuropathy (CIDP). Despite available therapies, many CIDP patients are left with residual symptoms, including weakness, numbness, and fatigue that can lead to long-term morbidity and diminished quality of life.

日本厚生劳动省 (MHLW) 已授予riliprubart孤儿药资格，这是一种选择性抑制经典补体途径中活化C1s的单克隆抗体，用于治疗慢性炎症性脱髓鞘性多发性神经病（CIDP）患者。尽管已有治疗方法，但许多CIDP患者仍存在残留症状，包括可能导致长期发病和生活质量下降的虚弱、麻木和疲劳。

Approximately 30% of people with CIDP do not respond to standard therapies. The MHLW grants orphan drug designation to medicines that address rare medical diseases or conditions with unmet medical needs. There are currently approximately 4,000 people diagnosed with CIDP in Japan..

大约30%的CIDP患者对标准疗法没有反应。日本厚生劳动省（MHLW）授予孤儿药资格给那些针对存在未满足医疗需求的罕见疾病或病症的药物。目前，日本约有4,000人被诊断出患有CIDP。

Erik Wallstroem, MD, PhD

埃里克·瓦尔斯特罗姆，医学博士，哲学博士

Global Head of Neurology Development, Sanofi

赛诺菲全球神经学开发主管

“The orphan drug designation of riliprubart for people living with CIDP in Japan underscores our commitment to applying our deep understanding of the immune system to address rare neurological disorders with significant unmet medical needs. While CIDP therapies exist, many individuals continue to experience debilitating symptoms, including pain, fatigue and weakness.

“在日本，riliprubart获得用于治疗CIDP患者的孤儿药资格，这体现了我们致力于运用对免疫系统的深刻理解，来应对存在重大未满足医疗需求的罕见神经系统疾病。尽管已有CIDP疗法，但许多患者仍然经历着包括疼痛、疲劳和虚弱在内的衰弱症状。”

Our ongoing development of riliprubart reflects our dedication to challenging the status quo in neurology with the goal of improving people’s lives.”.

我们对riliprubart的持续开发反映了我们致力于挑战神经病学的现状，目标是改善人们的生活。”

Long-term, 76-week sustained efficacy and safety data from riliprubart’s phase 2 study were presented at the

在第2阶段研究中，riliprubart的76周长期持续有效性和安全性数据被展示。

Peripheral Nerve Society

周围神经学会

meeting in Edinburgh, UK on May 17-20, 2025. Findings suggest a potential sustained long-term benefit provided by riliprubart across a broad spectrum of participants with CIDP. Riliprubart is currently being tested in two separate phase 3 studies: MOBILIZE in patients refractory to standard of care (clinical study identifier: .

2025年5月17日至20日在英国爱丁堡召开的会议。研究结果表明，riliprubart可能为广泛的CIDP患者带来潜在的长期持续益处。Riliprubart目前正在进行两项独立的3期临床试验：针对标准治疗无效患者的MOBILIZE研究（临床试验标识符：）。

NCT06290128

NCT06290128

) and VITALIZE in IVIg-treated patients (clinical study identifier:

）和接受IVIg治疗的患者中的VITALIZE（临床研究标识符：

NCT06290141

NCT06290141

About riliprubart

关于riliprubart

SAR445088 (riliprubart) is an IgG4 humanized monoclonal antibody that selectively inhibits activated C1s in the classical complement pathway of the innate immune system. By blocking C1s, riliprubart has the potential to inhibit key inflammatory mechanisms that drive demyelination and axonal damage in CIDP.

SAR445088（利立普鲁巴）是一种IgG4人源化单克隆抗体，可选择性抑制先天免疫系统经典补体途径中的活化C1s。通过阻断C1s，利立普鲁巴有可能抑制导致CIDP中脱髓鞘和轴突损伤的关键炎症机制。

Riliprubart is currently under clinical investigation, and its safety and efficacy have not been evaluated by any regulatory authority. For more information on riliprubart clinical studies, please visit .

Riliprubart 目前正在临床研究中，其安全性和有效性尚未得到任何监管机构的评估。欲了解有关 riliprubart 临床研究的更多信息，请访问 。

About CIDP

关于CIDP

CIDP is a rare neurological condition that causes progressive weakness and sensory impairment in the arms and legs. CIDP occurs when the body’s immune system attacks the myelin sheaths around nerve cells in the peripheral nervous system. Timely diagnosis of CIDP is important because it allows for appropriate treatment, which is essential to preventing long-term disability.

CIDP是一种罕见的神经系统疾病，会导致四肢进行性无力和感觉障碍。CIDP发生的原因是人体免疫系统攻击周围神经系统中神经细胞周围的髓鞘。及时诊断CIDP非常重要，因为它能够确保适当的治疗，这对防止长期残疾至关重要。

However, despite available therapies, many individuals are left with residual symptoms, including weakness, numbness, and fatigue that can lead to long-term morbidity and diminished quality-of-life. Approximately 30% of people with CIDP do not respond to standard therapies. In people with CIDP who do respond, about 70% of the response is considered incomplete.

然而，尽管有可用的治疗方法，许多患者仍留有包括乏力、麻木和疲劳等残留症状，这些症状可能导致长期病痛和生活质量下降。大约30%的CIDP患者对标准治疗无响应。在对治疗有响应的CIDP患者中，约70%的治疗效果被认为是不完全的。

Less than one-third of people with CIDP remain in remission without continued therapy..

不到三分之一的CIDP患者在没有持续治疗的情况下仍能保持缓解状态。

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..

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和保护，同时拥有一条创新的研发管线，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影响。