The US Food and Drug Administration (FDA) has granted orphan drug designation to SAR446523, an IgG1-based Antibody-Dependent Cellular Cytotoxicity-enhanced (ADCC) monoclonal antibody (mAb) targeting G-protein coupled receptor family C group 5 member D (GPRC5D) for the potential treatment of patients with relapsed or refractory multiple myeloma (R/R MM).

美国食品药品监督管理局（FDA）已授予SAR446523孤儿药资格，这是一种基于IgG1的增强抗体依赖性细胞毒作用（ADCC）单克隆抗体（mAb），靶向G蛋白偶联受体C家族第5组成员D（GPRC5D），用于潜在治疗复发或难治性多发性骨髓瘤（R/R MM）患者。

GPRC5D is highly expressed on plasma cells in MM patients, with low expression in healthy tissues. 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..

GPRC5D在MM患者的浆细胞上高表达，在健康组织中表达较低。FDA授予孤儿药资格给那些针对在美国影响少于20万人的罕见疾病或病症的研究性疗法。

The orphan drug designation is a significant milestone in our ongoing efforts to develop innovative treatments in multiple myeloma,” said

“孤儿药指定是我们持续努力在多发性骨髓瘤中开发创新疗法的重要里程碑，”

Alyssa Johnsen, MD, PhD

艾莉莎·约翰森，医学博士，哲学博士

, Global Therapeutic Area Head, Immunology and Oncology Development at Sanofi. “This underscores our commitment to multiple myeloma, a disease for which we have acquired strong expertise with the development of another widely used and approved immunotherapy treatment.”

赛诺菲免疫学和肿瘤学开发全球治疗领域负责人。“这强调了我们对多发性骨髓瘤的承诺，通过开发另一种广泛使用和获批的免疫治疗药物，我们已经在这方面积累了丰富的专业知识。”

The safety and efficacy of SAR446523 has not been evaluated by any regulatory authority and is still under investigation.

SAR446523 的安全性和有效性尚未经过任何监管机构的评估，仍在研究中。

About SAR446523

关于SAR446523

SAR446523 is an investigational IgG1-based mAb designed to target GPRC5D, which is highly expressed on plasma cells, with an engineered fragment crystallizable domain to enhance antibody dependent cell-mediated cytotoxicity. This innovative approach aims to improve the efficacy of treatment for MM, a rare and challenging cancer of plasma cells.

SAR446523 是一种基于 IgG1 的研究性单克隆抗体，旨在靶向在浆细胞上高度表达的 GPRC5D，并通过工程化改造的可结晶片段域来增强抗体依赖性细胞介导的细胞毒性。这一创新方法旨在提高治疗多发性骨髓瘤（MM）的疗效，这是一种罕见且具有挑战性的浆细胞癌症。

Subcutaneous SAR446523 is currently being evaluated in an ongoing phase 1, first-in-human study in patients with R/R MM (clinical study identifier: .

目前，正在进行一项针对复发/难治性多发性骨髓瘤（R/R MM）患者的1期、首次人体研究，评估皮下注射SAR446523（临床研究标识符：）。

NCT06630806

NCT06630806

). SAR4465523 originates from Sanofi Research in Vitry-sur-Seine, France.

). SAR4465523 源自法国维特里苏伊纳的赛诺菲研究所。

About multiple myeloma

关于多发性骨髓瘤

Multiple myeloma is considered a rare disease, yet MM is the second most common hematologic malignancy with more than 180,000 people diagnosed with MM each year, globally. Despite available treatments, MM remains an incurable malignancy with an estimated 62% five-year survival rate for newly diagnosed patients.

多发性骨髓瘤被认为是一种罕见病，但多发性骨髓瘤是第二常见的血液系统恶性肿瘤，全球每年有超过18万人被诊断患有多发性骨髓瘤。尽管有可用的治疗方法，多发性骨髓瘤仍然是一种无法治愈的恶性肿瘤，据估计，新诊断患者的五年生存率为62%。

There is a need for new frontline therapeutic options for all patients, especially for those who are transplant ineligible, due to high attrition rates in subsequent lines of therapy. Since MM does not have a cure, most patients will relapse and stop responding to therapies they have received..

所有患者都需要新的前线治疗选择，尤其是那些因后续治疗中高流失率而无法进行移植的患者。由于多发性骨髓瘤（MM）无法治愈，大多数患者将会复发，并对之前接受的治疗停止响应。

At Sanofi, we are building on a long-standing commitment to oncology as we continue to chase the miracles of science to improve the lives of those living with cancer. We are committed to transforming cancer care by developing innovative, first and best-in-class immunological and targeted therapies for rare and difficult-to-treat cancers with high unmet need..

在赛诺菲，我们秉承对肿瘤学的长期承诺，继续追寻科学的奇迹，以改善癌症患者的生活。我们致力于通过开发创新的、同类首创和最佳的免疫疗法及靶向疗法，来改变癌症治疗的现状，特别是针对那些需求高度未满足的罕见且难以治疗的癌症。

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、以人工智能为支撑的生物制药公司，致力于改善人们的生活并实现令人瞩目的增长。我们凭借对免疫系统的深刻理解，开发药物和疫苗，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的影响。

Sanofi is listed on EURONEXT: SAN and NASDAQ: SNY

赛诺菲在 EURONEXT（欧元区泛欧证券交易所）上市，代码为 SAN；并在 NASDAQ（纳斯达克）上市，代码为 SNY。