罗特西普(Luspatercept-AAMT) - 药物靶点：ACVR2B x GDF11_在研适应症：非输血依赖性地中海贫血，贫血，铁粒幼细胞性贫血_专利_临床

概要

基本信息

药物类型

Fc融合蛋白

别名

Luspatercept、ACE-536、BMS 986346

+ [7]

靶点

ACVR2B x GDF11

作用方式

抑制剂

作用机制

ACVR2B抑制剂(激活素受体-2B抑制剂)、GDF11 inhibitors(growth differentiation factor 11 inhibitors)

治疗领域

遗传病与畸形血液及淋巴系统疾病其他疾病+ [1]

在研适应症

非输血依赖性地中海贫血贫血铁粒幼细胞性贫血+ [19]

非在研适应症

染色体5q缺失综合征原发性血小板增多症后骨髓纤维化

原研机构

Acceleron Pharma, Inc.

在研机构

Celgene Corp.

Bristol Myers Squibb Co.

Acceleron Pharma, Inc.

+ [6]

非在研机构

新基医药咨询（上海）有限公司

权益机构

Celgene Corp.

Acceleron Pharma, Inc.

最高研发阶段批准上市

首次获批日期

美国 (2019-11-08),

β地中海贫血

最高研发阶段(中国)批准上市

特殊审评快速通道 (美国)、孤儿药 (美国)、孤儿药 (欧盟)、优先审评 (中国)、孤儿药 (韩国)、儿科研究计划 (欧盟)、附条件批准 (中国)、孤儿药 (日本)

登录后查看时间轴

结构/序列

Sequence Code 37465

来源: *****

关联

68

项与罗特西普相关的临床试验

NCT07096297

/ Not yet recruiting临床2期IIT

A Phase II Study of Luspatercept Plus Darbepoetin Alfa in SF3B1 Wild Type Lower-risk Myelodysplastic Syndromes

This is a single arm open-label Phase II trial of luspatercept and darbepoetin alfa in SF3B1 wild type, lower-risk, RBC transfusion dependent MDS participants with an endogenous erythropoietin (EPO) level < 500 IU/L.

开始日期2025-11-01

申办/合作机构

Yale University

[+1]

NCT06964971

/ Not yet recruitingN/AIIT

Treatment of Transfusion-dependent Nonsevere Aplastic Anemia With Luspatercept: a Multicenter Prospective Clinical Study

The goal of this clinical trial is to learn whether Luspatercept alone or in combination with Deferasirox can promote hematopoietic function in patients with transfusion-dependent non-severe aplastic anemia, as well as to assess the safety and efficacy of this treatment approach.
The main questions it aims to answer is:
whether the combination therapy of Luspatercept and Deferasirox can improve hemoglobin levels in these patients.
Participants will receive Luspatercept every 3 to 5 weeks based on hemoglobin response, undergo complete blood counts every 1 to 3 weeks, and receive other necessary evaluations as required.

开始日期2025-05-20

申办/合作机构

浙江中医药大学

ChiCTR2500100409

/ Not yet recruitingN/AIIT

Treatment of transfusion-dependent nonsevere aplastic anemia with Luspatercept: a multicenter prospective clinical study

开始日期2025-04-21

申办/合作机构

浙江中医药大学

[+1]

100 项与罗特西普相关的临床结果

登录后查看更多信息

100 项与罗特西普相关的转化医学

登录后查看更多信息

100 项与罗特西普相关的专利（医药）

登录后查看更多信息

263

项与罗特西普相关的文献（医药）

2025-12-31·JOURNAL OF MEDICAL ECONOMICS

Healthcare resource utilization of patients with lower-risk myelodysplastic syndromes treated with luspatercept versus erythropoiesis-stimulating agents: a United States healthcare claims database study

Article

作者: Mohan, Manasi ; Yenikomshian, Mihran ; Huynh, Lynn ; Swanson, Ashley ; Pinaire, Megan ; Tang, Derek ; Slaff, Samantha ; Zanardo, Enrico ; Song, Rui ; Ball, Brian J. ; Gavrilov, Svetlana

OBJECTIVE:

This study compared the healthcare resource utilization (HRU) of patients with lower-risk myelodysplastic syndromes (LR-MDS) treated with luspatercept versus erythropoiesis-stimulating agents (ESAs).

METHODS:

This real-world retrospective cohort study used claims data from the Symphony Health Integrated Dataverse, a large nationally representative United States database, to identify patients with LR-MDS who initiated luspatercept or ESA between May 1, 2020, and June 30, 2022. Index date was defined as the date of the first claim for luspatercept or ESA. The follow-up period was from the index date to the earliest of the end of clinical activity or end of data availability. All-cause and MDS-related HRU were evaluated for both treatment cohorts and compared using generalized estimating equations with Poisson distribution and robust variance estimator.

RESULTS:

Overall, 243 and 3,515 patients were included in the luspatercept and ESA cohorts, respectively. Patients in both cohorts had a similar median (interquartile range [IQR]) age at index (luspatercept: 77.0 [70.0-79.0] years; ESA: 78.0 [72.0-79.0] years) and median (IQR) follow-up duration (luspatercept: 14.6 [10.4-22.6] months; ESA: 14.4 [9.5-20.9] months). Compared with patients treated with ESA, patients treated with luspatercept had a 26% lower rate of all-cause inpatient visits (adjusted incidence rate ratio [aIRR], 0.74; 95% confidence interval [CI], 0.58-0.93; p < .05) and a 31% lower rate of all-cause outpatient visits (aIRR, 0.69; 95% CI, 0.61-0.79; p < .001). The rate of MDS-related inpatient visits was also 25% lower among the patients treated with luspatercept versus ESA (aIRR, 0.75; 95% CI, 0.56-0.99; p < .05).

CONCLUSION:

This study showed that patients with LR-MDS treated with luspatercept required significantly less HRU than patients treated with ESA. Further research is warranted to evaluate the financial impact of this lower HRU burden.

2025-12-31·JOURNAL OF MEDICAL ECONOMICS

Treatment patterns and outcomes in patients with lower-risk myelodysplastic syndromes treated with erythropoiesis-stimulating agents in a large US community oncology practice: a retrospective chart review

Article

作者: Gavrilov, Svetlana ; Gordan, Lucio N. ; Ming, Amy ; Alajrash, Aya ; Swanson, Ashley ; Griffin, Ernest ; Warner, Amanda ; Slaff, Samantha ; Heritage, Trevor ; Tang, Derek ; Singh, Nisha ; Fonseca, Gustavo

BACKGROUND:

Erythropoiesis-stimulating agents (ESAs) are an established treatment for lower-risk myelodysplastic syndromes (LR-MDS). However, real-world data on the outcomes of patients with LR-MDS treated with ESAs are limited. This study describes treatment patterns and clinical outcomes in this population.

METHODS:

This is a retrospective review of electronic medical records compiled from Florida Cancer Specialists & Research Institute databases. Inclusion criteria were: LR-MDS diagnosis (between January 2018 through December 2022), initiation of ESA treatment, ≥18 years at diagnosis, availability of ≥6 months of data prior to diagnosis, and ≥1 year of follow-up data. Patients treated with luspatercept prior to ESAs or enrolled in clinical trials during the study period were excluded. Patients were followed for 1 year from the initial diagnosis until loss to follow-up/death. Outcomes included treatment patterns (occurrence of and time to ESA failure, and time on ESA treatment after failure), and clinical outcomes related to ESA failure (hematologic improvement and transfusion independence), with ESA failure defined as a < 1.5 g/dL rise in hemoglobin or no decrease in red blood cell transfusions by 6-8 weeks of treatment.

RESULTS:

At baseline, of 359 eligible patients with LR-MDS most were male (65.2%), White (88.0%), non-transfusion dependent (74.9%), and had hemoglobin levels of 8-10 g/dL (56.3%) and ring sideroblasts <5% (59.3%). Most patients (68.0%) experienced ESA failure, with a median of 56.0 days from ESA initiation to failure, and a median of 466.5 days receiving ESA treatment after failure. Hematologic improvement was observed in 33.7% of patients.

CONCLUSIONS:

In this real-world evaluation of ESA treatment in a population with LR-MDS, only one-third of patients experienced hematologic improvement and two-thirds of patients remained on ESA treatment for ≥1 year after failure. These results demonstrate high rates of ESA failure and indicate patients in community oncology settings in the US may be continuing with ESA treatment despite failure.

2025-12-31·Hematology

Luspatercept for the treatment of lower-risk myelodysplastic syndrome with SF3B1 mutation: a real-world single-center research in China

Article

作者: Xiong, Youzhen ; Zhao, Yufei ; Zhou, Kang ; Zhao, Xin ; Li, Yuan ; Peng, Guangxin ; Hu, Xiangrong ; Fan, Huihui ; Li, Jianping ; Liu, Xu ; Xing, Lingxiao ; Hu, Jing ; Ye, Lei ; Yang, Wenrui ; Zhang, Li ; Zhang, Fengkui ; Liang, Weiru ; Zhang, Baohang ; Jing, Liping ; Kang, Rui ; Shi, Yimeng ; Yang, Yang

BACKGROUND:

Luspatercept, approved by the FDA and EMA for patients with transfusion-dependent lower-risk myelodysplastic syndrome (LR-MDS) unresponsive to erythropoiesis-stimulating agents (ESAs), lacks extensive real-world data, particularly in China.

METHODS:

We retrospectively analyzed 14 LR-MDS-SF3B1 patients treated with luspatercept for ≥12 weeks.

RESULTS:

Median age was 60 years (range 47-72); 42.9% were male. Before treatment, 78.6% were transfusion-dependent, and 42.9% had prior ESA therapy. At median 24-week follow-up (range 12-44), erythroid response rates were 71.43% (week 12), 75.00% (week 16), and 62.50% (week 24). Hemoglobin levels significantly improved at weeks 12 and 24 (P = 0.013, P = 0.005). No grade 3-4 adverse events occurred. Hematologic improvement-erythroid (HI-E) patients exhibited higher white blood cells, neutrophils, and reticulocytes at week 12 versus non-HI-E patients. Bone marrow analysis revealed erythroid hyperplasia in HI-E patients, with higher erythrocyte percentage (56.00% vs. 34.00%, P = 0.023), lower myeloid-to-erythroid ratio (0.60 vs. 1.59, P = 0.024), and increased polychromatic erythroblasts (19.50% vs. 10.00%, P = 0.034).

CONCLUSIONS:

Luspatercept demonstrated efficacy and safety in Chinese LR-MDSSF3B1 patients. Greater erythroid hyperplasia correlated with better clinical response.

323

项与罗特西普相关的新闻（医药）

2025-09-26

·药代动力学

速扒一下B司！

上世纪八十年代，肉眼看不见的“细菌”威胁着许多中国老百姓的生命健康。“先锋6号”的出现，不仅成为了中国患者的“守护者”，也在无声中开启了一家跨国药企与中国长达四十余年的命运交织 —— 百时美施贵宝在中国的故事由此展开。从抗感染先锋6号和感冒药日夜百服咛，到抗癌化疗药紫杉醇和老牌降糖药二甲双胍，再到强效低耐药抗乙肝病毒的恩替卡韦……这些由百时美施贵宝引入到中国的经典药物，不仅守护了千千万万中国患者的健康，也是一代中国人药箱里的时代记忆。随着时代更迭，医疗健康的需求不断演变，百时美施贵宝在中国的发展战略也随之演进，但始终未变的是“因时制宜”—— 聚焦满足时代健康需求的创新，将更多创新药物更快地惠及更多中国患者。 2014年，百时美施贵宝决意在中国转型，成为了最早在中国开始向下一代多元化生物制药转型的跨国药企之一。2020年，百时美施贵宝对中国医疗健康与创新做出一项长期承诺，率先推出公司 “中国2030战略”。通过加速引入兼具“广度”与“深度”的全球创新管线，和“内外兼修”的创新模式，充分融入中国蓬勃发展的创新生态系统，致力于“在中国、为中国、为全球”。百时美施贵宝在十多年前转型之时就已经前瞻性地关注到了中国老龄化社会带来的健康医疗挑战。其“中国2030战略”中所聚焦的肿瘤学、血液学、免疫学、心血管也都是中国患者亟待创新疗法的疾病领域，其中，大部分还威胁着数亿老年人群健康。乘着中国大力支持创新的政策东风，百时美施贵宝于过去五年在中国获批上市了多款“同类首创First-in-Class”或“同类最佳Best-in-Class”创新药物和适应症，包括治疗β-地中海贫血和骨髓增生异常综合征的全球首个红细胞成熟剂罗特西普、治疗中重度斑块银屑病的全球首个TYK2抑制剂氘可来昔替尼、治疗梗阻性肥厚性心肌病的全球首个心肌肌球蛋白抑制剂玛伐凯泰等。而以O药为基础的免疫肿瘤治疗方案目前已覆盖肺癌、胃癌、食管癌、肝癌等中国高发瘤种。随着中国癌症早筛覆盖面每年突破上亿人次，癌症治疗重心前移，百时美施贵宝正在加速布局早期肿瘤治疗，满足癌症患者从“晚期救治”向“早诊断、早干预、早治疗”的需求变化。 “加速引入创新产品”只是第一步。在国家致力推动的多层次医疗保障体系的探索中，百时美施贵宝始终是积极参与的先行者。在基本医保层面，过去三年上市的四款创新药，全部实现了上市当年即被纳入国家医保目录。在商保和早期准入方面，从被纳入最多“惠民保”项目药物之一的O药，到落地海南博鳌“先行先试”的玛伐凯泰……每一次的创新突破，都是为了让高度创新且具差异化的药物，能够尽可能快地惠及更多患者，改善患者治疗结局。为了更高效地顺应行业的快速演变、患者需求和行为的转变、以及科学技术的革新，百时美施贵宝选择专注于肿瘤学、免疫学、血液学、心血管和神经科学这五大疾病领域，布局兼具“广度”与“深度”的产品管线。在广度上，百时美施贵宝在药物类型的多样性上进行了全面布局，包括单抗、ADC、细胞治疗、小分子核酸、核酸等核心治疗模态。在深度上，以免疫学相关疾病为例，这类疾病常被患者无奈地称为“不死的癌症”，或许不直接致命，却用漫长的病程将患者的生活拖入泥潭。其中，系统性红斑狼疮（SLE）可能会从轻微皮肤关节痛骤变为核心脏器损伤，而银屑病患者则承受着皮肤和心理的“双重压力”。如何回应患者对创新的期盼？百时美施贵宝的答案，蕴藏在其深厚的研发管线与创新积淀中。第一步，是从“点”突破，最大化“明星分子”的价值。作为全球首个TYK2变构抑制剂，氘可来昔替尼不仅为中度至重度斑块状银屑病患者带来了新的希望，更在银屑病关节炎（PsA）、成人活动性干燥综合征（SjD）和系统性红斑狼疮（SLE）等治疗领域展现潜力。第二步，是连“点”成“线”，为复杂疾病构建“全病程解决方案”。以系统性红斑狼疮（SLE）为例，百时美施贵宝正在布局一整套“组合拳”：前端，有口服小分子药物（如氘可来昔替尼）精准控制炎症；后端，则有前沿的CD19 NEX-T细胞疗法、CD19 TCE平台，旨在通过靶向清除病理性B细胞，有望实现患者免疫系统的功能性重置，实现长期的临床缓解，甚至有望从根源上改变疾病进程。在推进创新管线布局的同时，百时美施贵宝在充分融入并助力推动全球的创新浪潮。这其中，一个显而易见的趋势是 —— 中国生物医药的创新正在重塑全球研发生态。置身于这场创新变革中，百时美施贵宝认为：相较于药物从哪里来，满足患者迫切的、未被满足的需求更为重要。在这一理念的指引下，百时美施贵宝与中国创新生态圈的连接正变得日益紧密。通过与BioNTech、艾博兹、百利天恒SystImmune等一系列的BD合作，百时美施贵宝正在从一个创新引入者，转化成为中国创新生态的共建者和赋能者。从“一代人的药箱”到FIC/BIC创新药，改变的是治疗的需求，是疾病的认知和科学的更多可能，不变的是百时美施贵宝用行动回应对中国患者的创新需求。

免疫疗法上市批准

2025-09-05

·PRNewswire

Florida Cancer Specialists & Research Institute Clinical Expertise And Real-World Evidence Capabilities Advancing Treatment of Blood Cancers

Abstracts Presented at Worldwide Gathering of Hematology ExpertsSOHO 2025 abstracts featuring FCS research: CT087, MM353, MPN566, MM353 FORT MYERS, Fla., Sept. 5, 2025 /PRNewswire/ -- Physicians, clinicians, and senior leaders from Florida Cancer Specialists & Research Institute, LLC (FCS) are among the internationally recognized experts presenting advancements in the treatment of hematologic malignancies at the Society for Hematologic Oncology (SOHO) 2025 annual meeting this week in Houston. Continue Reading Lucio N. Gordan, MD. "We are driving advances in blood cancers by accelerating development of new therapies." Post this Florida Cancer Specialists & Research Institute presenting groundbreaking hematologic research at the annual Society of Hematologic Oncology. FCS President & Managing Physician Lucio N. Gordan, MD. "By combining the care of more than one million patients with our deep clinical, research, and informatics expertise, we are driving meaningful advances in understanding blood cancers and accelerating the development of new therapies." FCS Director of Real-World Evidence Amanda Warner, MS, RN is the presenting author of two abstracts: Myelofibrosis Observational Real-World Experience in Community Oncology (MORE-CO): A Retrospective Analysis of Baseline Clinical Factors Impacting Overall Survival | Co-authors are Lucio N. Gordan, MD, Arsh Singh, MD, FCS hematologist and medical oncologist, and Trevor Heritage, PhD, FCS clinical technology director. Real-World Treatment Patterns and Outcomes of Luspatercept in Treatment-Naïve Patients with Lower-Risk Myelodysplastic Syndromes (LR-MDS) in a Large Community Oncology Practice | Co-authors are Gustavo Fonseca, MD, FACP, FCS director of research & clinical trials, and Christian Okitondo, PhD, FCS real-world evidence biostatistician. "FCS in recent years has made forward-thinking investments in proprietary real-world datasets and advanced data science," noted Heritage. "Our comprehensive approach transforms real-world clinical data into high-quality insights that are driving improvements in clinical decision making, closing gaps in care, and enhancing patient experiences and outcomes." Additionally, the following abstract presentations feature research results with FCS participation: Phase 2 SubQSA Study of Subcutaneous (SC) Isatuximab (Isa) Administered by an On-Body Delivery System With Weekly Carfilzomib and Dexamethasone (Kd) in Patients with Relapsed/Refractory Multiple Myeloma (RRMM) – co-authored by Alexander Philipovskiy, MD, PhD, FCS medical oncologist and hematologist specializing in drug development. Preliminary Insights into CAR-T Therapy for Melanoma: A Systematic Review – co-authored by Uma Iyer, MD, FCS medical oncologist and hematologist. At any point in time, more than 150 active clinical trials are underway at FCS, including early-phase studies at one of three drug development units. Research conducted at FCS is made possible through valuable relationships with Sarah Cannon Research Institute, one of the world's leading clinical research organizations, and Paradigm Health, an AI-enabled clinical trial matching and recruitment platform. In recent years, the majority of new cancer drugs approved for use in the U.S. were studied in clinical trials with FCS participation, prior to approval. About Florida Cancer Specialists & Research Institute, LLC: (FLCancer.com) For more than 40 years, Florida Cancer Specialists & Research Institute (FCS) has embraced innovation to deliver world-class care and drive the dramatic transformation of oncology care through its robust clinical research program. FCS provides patients with access to a wide range of clinical trials, positioning it as a leader in research among private oncology practices in Florida and across the country. In fact, before receiving FDA approval, the majority of new cancer drugs in the U.S. were first made available to patients through participation in clinical trials at FCS. Our outstanding team of highly trained and dedicated physicians is committed to delivering tailored treatment plans that make the best use of cutting-edge precision oncology advancements to enhance patient outcomes. SOURCE Florida Cancer Specialists & Research Institute WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

临床2期

2025-09-03

·丁香园 Insight 数据库

百时美施贵宝朱正缨：中国研发与全球创新「同频」，加速惠及中国患者

今年以来，百时美施贵宝 (下称「BMS」) 已有两款创新产品的四个新适应症在中国获批，在「缩短中国患者与全球获得创新药时间差」方面再度取得多项实质性进展。 2025 年是 BMS「中国 2030 战略」的承上启下之年。在百时美施贵宝副总裁、中国研发负责人朱正缨博士看来，这一战略执行过半，BMS 始终秉持「急患者之所急，需患者之所需」的理念，加速引入创新产品。而搭乘多项政策东风，BMS 中国已逐步实现了新品递交、获批的「全球同步」，甚至「全球第一」。百时美施贵宝副总裁、中国研发负责人朱正缨博士。受访者供图战略半程：数量与速度并进 BMS「中国 2030 战略」的核心是让更多的创新产品更快更广地惠及患者。为达成这一目标，BMS 设立了加速创新引入、加速创新准入、加速创新合作、加速人才发展四大「支柱」。其中朱正缨所负责的研发工作的重点之一，就是「加速引入全球创新产品及适应症」。朱正缨介绍，BMS 始终聚焦患者未满足的治疗需求，过去四年多在肿瘤学、免疫学、血液学和心血管这些 BMS 核心领域引入了多款同类首个 (First-in-Class) 和同类最佳 (Best-in-Class) 的产品和适应症。以骨髓异常综合征 (MDS) 为例，朱正缨介绍说，由于长期缺乏创新药物，许多患者不得不依赖输血进行治疗，严重影响其生活质量。「中国 2030 战略」实施后，这一未尽之需促使 BMS 开创性地通过在中国开展桥接试验、整合海外数据和中国数据并提交注册申请的方式，加速引入全球首个红细胞成熟剂罗特西普，打破了这一领域近 20 年没有创新药物的局面。而在 BMS 拥有科学优势的领域，得益于全球同步研发策略、一系列政策支持以及BMS 中国研发团队的不懈努力，其免疫肿瘤药物在中国高发瘤种中的获批时间不断加速——去年 10 月，免疫双子星 (欧狄沃联合逸沃，简称「O 药+Y 药」) 在中国实现了 MSI-H/dMMR 结直肠癌这一适应症的「全球首发」；今年 3 月，其肝细胞癌 (HCC) 一线适应症的获批几乎与欧盟同步，早于美国。加速甚至同步获批也源于对同步递交的高效执行，朱正缨表示：「『中国 2030 战略』实施以来，在注册递交环节，我们基本已经实现了在全球首个主要市场递交申请之后三个月之内完成在中国市场递交的目标，但我们并不满足于此，希望能把时间差缩得更短。」事实上，BMS 的步伐已经越来越快。今年 5 月，氘可来昔替尼的第二个适应症银屑病关节炎 (PsA) 完成了向国家药品监督管理局药品审评中心 (CDE) 的注册递交，这与首个递交该适应症的市场——美国的注册递交只差了 72 个小时；而此前，中国团队在「免疫双子星」治疗 MSI-H/dMMR 结直肠癌适应症的注册中，还实现了全球首个递交、首个获批。谈及未来愿景时，朱正缨分享说：「目前我们内部有一个更高的目标，即未来能够实现与全球市场『同一天』递交。」管线布局：筑创新基石促人才成长随着战略的深入实施，BMS 中国已逐渐构建起了丰富的研发管线，中国研发团队也开始逐步成为全球研发链条中的关键组成部分。「BMS 的管线布局思路非常明确，关注严重疾病，重点聚焦存在迫切且未被满足治疗需求的领域，」朱正缨说，「只要是能惠及中国患者的产品和适应症，我们都会能入则入，在整个研发链条上实现与全球同步进入。」在管线战略上，BMS 始终强调兼顾广度与深度，力求「以更匹配的治疗模态，更好地达到目标靶点，从而产生我们希望得到的治疗效应。」朱正缨说。她进一步分析，从广度来说，目前 BMS 内部管线覆盖的治疗模态非常广泛，包括 ADC、细胞治疗、核药、小分子核酸等核心治疗模态。另一方面，BMS 也有能力围绕某一疾病实现深度上的突破。以系统性红斑狼疮的研发策略为例，「我们的策略是在疾病发生发展过程的不同阶段进行干预。除氘可来昔替尼外，BMS 的管线中还有处于临床前以及临床开发阶段的靶向 B 细胞的不同治疗模式，包括细胞疗法，我们希望从提高临床缓解率和延长缓解周期这一起点开始，逐步推进，并考虑能否通过免疫系统的『重置』，进一步改善患者的长期预后，」朱正缨说，「这种思路前景非常令人振奋，如果我们能够调整免疫系统恢复到正常工作状态，那么距离一些免疫疾病被治愈的未来就不远了，这是我们的终极目标。」而兼具广度与深度的研发管线布局，也成为 BMS 吸引研发领域人才的重要「磁石」。「研发人才的特点是乐于不断接触和探索新的治疗方式。相较于传统单抗、小分子药物疗法，双特异性抗体、T 细胞连接器、细胞治疗、乃至放射性药物治疗等创新方向的研发路径、药物作用机制、疗效与安全性评估方式都截然不同，有些甚至颠覆了传统医药开发的认知，」朱正缨说，BMS 管线布局的丰富度以及公司对于深度探索的强大支持，使研发团队有机会接触甚至引领新疗法的研发，并通过多种工具的创新组合，对疾病的不同环节、不同的病患群体特征等进行广泛探究，「这是研发人才非常看重，也非常有热情去为之努力的。」此外，BMS 的全球化研发机制亦是促使研发人才迅速成长的沃土。朱正缨指出，当前，中国研发团队已经全面融入了 BMS 全球创新链的每个环节，这意味着中国研发和医学团队的专业能力、项目经验将得以快速提升，持续迈向全球顶尖团队行列。　下个五年：「全球同步」将成常态扎根中国 40 余年，BMS 见证了中国医药创新环境的巨大变迁，深度参与其中的同时，也成为改革创新的受益者和共鸣者。朱正缨表示，BMS 的「中国 2030 战略」能够一再加速引进创新药物，除团队内部的共同努力外，更离不开外部创新环境的利好。「中国现在的政策环境非常鼓励跨国药企的中国团队把创新产品尽快引入中国市场，药监部门等政府机构先后出台了一系列政策。最近，《关于优化创新药临床试验审评审批有关事项的公告》征求意见稿发布，有望将去年试行的 30 天内审评反馈快速通道正式常态化。」朱正缨说，这对中国研发团队参与全球管线开发是非常大的激励，使之能在一期、二期临床研究阶段就有更多的机会与全球同步，「对于加强中国在全球创新药研发中的参与度和数据积累至关重要，而企业也能充分发挥中国患者群体和资源的优势，加速创新药物进入中国市场。」此外，朱正缨谈及，在注册审批环节，中国药监局也同企业一起，更加科学多元地通过不同临床研发阶段的数据对新产品做整体的评估和考量。「助力患者尽早获益，成为研发人员、药企、监管部门、临床医生等共同的目标，」她说，「回顾近两年在同步申报、同步获批方面的实践经验，中国的创新药注册和获批距离『全球同步』已越来越近——这将会变成一种常态。」「中国 2030 战略」下半程即将开启，BMS 还将如何发力创新？朱正缨表示，从管线角度看，未来几年将持续加速引入公司在肿瘤学、血液学、免疫学领域的创新产品。而在神经科学这一领域，中国研发团队也将聚焦未被满足的需求，围绕公司丰富的神经科学早期管线布局，与总部共同积极推进，争取尽早将这些管线引入中国。「公司总体的战略目标是要在 2030 年前实现全球范围内获批 10 个产品、30 个新适应症，对于中国研发团队而言，我们希望，只要是能够惠及中国患者的产品和适应症，全球有的我们一个都不落下，」朱正缨总结道，另一方面，身处日益蓬勃的中国创新生态中，BMS 也希望能够以合作促创新，「未来五年，会有更多源自中国的分子在全球创新药舞台上发光发热。作为跨国药企研发团队，我们也希望和研究者、临床机构等开展广泛合作，大家共同提升、共同成长、共同引领行业发展。」本文首发于「中新网上海」，Insight 数据库获授权转载。封面来源：企业logo 免责声明：本文仅作信息分享，不代表 Insight 立场和观点，也不作治疗方案推荐和介绍。如有需求，请咨询和联系正规医疗机构。编辑：馨药 PR 稿对接：微信 insightxb 投稿：微信 insightxb；邮箱 insight@dxy.cn 多样化功能、可溯源数据…… Insight 数据库网页版等你体验点击阅读原文，立刻解锁！

100 项与罗特西普相关的药物交易

登录后查看更多信息

外链

KEGG	Wiki	ATC	Drug Bank
-	罗特西普	B03XA06	DB12281

研发状态

批准上市

10 条最早获批的记录,

登录

后查看更多信息

适应症	国家/地区	公司	日期
非输血依赖性地中海贫血	欧盟	Bristol-Myers Squibb Pharma EEIG	2023-03-20
非输血依赖性地中海贫血	冰岛	Bristol-Myers Squibb Pharma EEIG	2023-03-20
非输血依赖性地中海贫血	列支敦士登	Bristol-Myers Squibb Pharma EEIG	2023-03-20
非输血依赖性地中海贫血	挪威	Bristol-Myers Squibb Pharma EEIG	2023-03-20
贫血	韩国	Bristol Myers Squibb Co.	2022-05-09
铁粒幼细胞性贫血	欧盟	Bristol-Myers Squibb Pharma EEIG	2020-06-25
铁粒幼细胞性贫血	冰岛	Bristol-Myers Squibb Pharma EEIG	2020-06-25
铁粒幼细胞性贫血	列支敦士登	Bristol-Myers Squibb Pharma EEIG	2020-06-25
铁粒幼细胞性贫血	挪威	Bristol-Myers Squibb Pharma EEIG	2020-06-25
骨髓增生异常综合征	欧盟	Bristol-Myers Squibb Pharma EEIG	2020-06-25
骨髓增生异常综合征	冰岛	Bristol-Myers Squibb Pharma EEIG	2020-06-25
骨髓增生异常综合征	列支敦士登	Bristol-Myers Squibb Pharma EEIG	2020-06-25
骨髓增生异常综合征	挪威	Bristol-Myers Squibb Pharma EEIG	2020-06-25
输血依赖性贫血	欧盟	Bristol-Myers Squibb Pharma EEIG	2020-06-25
输血依赖性贫血	冰岛	Bristol-Myers Squibb Pharma EEIG	2020-06-25
输血依赖性贫血	列支敦士登	Bristol-Myers Squibb Pharma EEIG	2020-06-25
输血依赖性贫血	挪威	Bristol-Myers Squibb Pharma EEIG	2020-06-25
输血依赖性β地中海贫血	欧盟	Bristol-Myers Squibb Pharma EEIG	2020-06-25
输血依赖性β地中海贫血	冰岛	Bristol-Myers Squibb Pharma EEIG	2020-06-25
输血依赖性β地中海贫血	列支敦士登	Bristol-Myers Squibb Pharma EEIG	2020-06-25

未上市

10 条进展最快的记录,

登录

后查看更多信息

适应症	最高研发状态	国家/地区	公司	日期
骨髓纤维化	临床3期	中国	Celgene Corp.	2022-02-23
真性红细胞增多症	临床3期	中国	Celgene Corp.	2022-02-23
原发性血小板增多症	临床3期	中国	Celgene Corp.	2022-02-23
真性红细胞增多症后骨髓纤维化	临床3期	美国	Celgene Corp.	2021-02-25
真性红细胞增多症后骨髓纤维化	临床3期	中国	Celgene Corp.	2021-02-25
真性红细胞增多症后骨髓纤维化	临床3期	日本	Celgene Corp.	2021-02-25
真性红细胞增多症后骨髓纤维化	临床3期	阿根廷	Celgene Corp.	2021-02-25
真性红细胞增多症后骨髓纤维化	临床3期	澳大利亚	Celgene Corp.	2021-02-25
真性红细胞增多症后骨髓纤维化	临床3期	奥地利	Celgene Corp.	2021-02-25
真性红细胞增多症后骨髓纤维化	临床3期	比利时	Celgene Corp.	2021-02-25

登录后查看更多信息

临床结果

适应症

分期

评价

查看全部结果

研究	分期	人群特征	评价人数	分组	结果	评价	发布日期


NCT04717414 (Independence, NEWS) 人工标引	临床3期	贫血 \| 骨髓纤维化	-	Reblozyl	網淵築鬱鑰糧繭襯範範(簾鑰衊憲鑰糧鑰製繭艱): P-Value = 0.0674 未达到	不佳	2025-07-18
				Placebo
COMMANDS trial (ASCO2025)	N/A	骨髓增生异常综合征一线	103	Luspatercept	膚膚憲遞積鬱觸範襯簾(壓窪遞齋淵壓範積獵構) = 廠廠夢醖築襯衊築鹽構糧網觸膚觸夢構壓夢醖 (憲襯糧衊糧夢願網選窪 ) 更多	积极	2025-05-30
				Erythropoiesis-stimulating agents	膚膚憲遞積鬱觸範襯簾(壓窪遞齋淵壓範積獵構) = 遞醖醖壓齋衊願醖製蓋糧網觸膚觸夢構壓夢醖 (憲襯糧衊糧夢願網選窪 ) 更多
NCT03682536 (COMMANDS, ASCO2025)	临床3期	骨髓增生异常性贫血一线 ESA-naive \| LR-MDS	-	Luspatercept	觸衊艱願製壓觸醖蓋淵(鑰構鹹艱齋淵願淵壓鬱) = 齋淵衊窪鑰廠鹹鹽齋衊艱淵衊範壓願築艱醖遞 (襯製膚廠願簾願鹽膚鏇 ) 更多	积极	2025-05-30
				Epoetin alfa	觸衊艱願製壓觸醖蓋淵(鑰構鹹艱齋淵願淵壓鬱) = 蓋淵選糧網顧糧鹹積顧艱淵衊範壓願築艱醖遞 (襯製膚廠願簾願鹽膚鏇 ) 更多
ASCO2025	N/A	骨髓增生异常综合征	2,614	Luspatercept	構願網夢繭鹹糧窪餘範(夢獵繭製襯網壓廠鬱壓) = 艱願蓋窪構窪製獵壓廠淵淵簾蓋醖蓋齋製衊願 (範憲獵範壓醖製夢壓鏇, 31.7 ~ 60.2) 更多	积极	2025-05-30
				Placebo	構願網夢繭鹹糧窪餘範(夢獵繭製襯網壓廠鬱壓) = 願醖網網糧夢廠膚簾範淵淵簾蓋醖蓋齋製衊願 (範憲獵範壓醖製夢壓鏇, 60.7 ~ 74.0) 更多
NCT03682536 (COMMANDS, EHA2025)	临床3期	骨髓增生异常综合征一线 erythropoiesis stimulating agent (ESA)-naive \| transfusion-dependent (TD)	-	Luspatercept	顧鬱願選願蓋積壓築選(糧顧憲築廠構顧鹽襯鬱) = 積願鑰蓋簾醖蓋窪艱醖餘夢廠願構鬱選餘構窪 (鏇積淵艱鏇遞鹽鏇齋鏇 ) 更多	积极	2025-05-22
				Epoetin alfa	顧鬱願選願蓋積壓築選(糧顧憲築廠構顧鹽襯鬱) = 鏇鹹襯襯憲糧淵鬱獵夢餘夢廠願構鬱選餘構窪 (鏇積淵艱鏇遞鹽鏇齋鏇 ) 更多
NCT03682536 (COMMANDS, Pubmed \| Adv Ther)	临床3期	骨髓增生异常综合征	-	Luspatercept	鬱衊觸鏇餘醖顧膚鹽築(憲顧築艱夢顧鑰膚觸鑰) = Rates of treatment-emergent adverse events, including asthenia and hypertension, generally decreased over time in both arms 範鬱簾積觸選蓋憲膚網 (範壓壓窪蓋選糧簾顧淵 ) 更多	积极	2025-05-16
				Epoetin alfa
NCT03682536 (COMMANDS, EHA2025)	临床2期	贫血 \| 骨髓增生异常综合征 SF3B1 mutation	34	Luspatercept 1.0 mg/kg	選願襯獵憲鹽齋襯壓餘(齋築顧窪築遞構積淵壓) = 積簾艱獵願艱鹽範夢製襯壓衊鹹遞鹽選衊膚壓 (餘鹽築醖夢鏇顧選鬱網, 6.7 ~ 9.8) 更多	积极	2025-05-14
NCT03682536 (COMMANDS, EHA2025)	临床3期	骨髓增生异常综合征	350	Luspatercept	繭網餘鹹衊夢憲鹽憲餘(構範構艱簾鬱憲獵構艱) = 餘鑰蓋獵鬱鑰築獵網顧網餘願醖遞獵衊鏇鏇衊 (襯壓構顧襯膚窪齋遞築 ) 更多	积极	2025-05-14
				Epoetin Alfa	繭網餘鹹衊夢憲鹽憲餘(構範構艱簾鬱憲獵構艱) = 簾膚積鏇襯鏇築醖網鏇網餘願醖遞獵衊鏇鏇衊 (襯壓構顧襯膚窪齋遞築 ) 更多
NCT06045689 (MAXILUS, EHA2025)	临床3期	骨髓增生异常综合征 SF3B1 mutation	90	Luspatercept 1.75 mg/kg	構膚衊廠鹽廠觸築顧顧(簾簾艱遞糧艱積製淵願) = 窪繭蓋鑰鏇鹽窪範觸選願糧艱餘廠齋顧醖構艱 (餘夢簾繭繭餘壓築衊願 ) 更多	积极	2025-05-14
				Placebo	構膚衊廠鹽廠觸築顧顧(簾簾艱遞糧艱積製淵願) = 襯範糧膚窪艱鏇襯鬱獵願糧艱餘廠齋顧醖構艱 (餘夢簾繭繭餘壓築衊願 ) 更多
PS2187 (EHA2025)	N/A	输血依赖性β地中海贫血	15	Luspatercept 1 mg/kg	鏇糧襯膚鹹繭獵襯遞夢(顧壓鑰獵醖鬱繭鹹膚窪) = All patients had at least one adverse event (AE); the majority were Grade 1-2 (70%), and four Grade 4 AEs were related to hospitalization. LT was discontinued in two patients due to unresponsiveness at 24 and 72 weeks. Three patients who demonstrated a good to moderate response to LT experienced adverse events, including musculoskeletal pain, fatigue, and headache, at weeks 48, 84, and 72, respectively, and subsequently stopped LT treatment. 遞積蓋觸繭襯鹹觸積齋 (簾襯憲齋艱艱選製遞網 )	-	2025-05-14
				Deferasirox