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2025-09-01

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

N/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

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100 项与罗特西普相关的转化医学

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100 项与罗特西普相关的专利（医药）

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261

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 ; Gavrilov, Svetlana ; Ball, Brian J.

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·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 ; Zhang, Li ; Yang, Wenrui ; 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.

2025-12-01·Current Hematologic Malignancy Reports

Emerging Therapeutic Approaches for Anemia in Myelofibrosis

Review

作者: Harrington, Patrick ; Palumbo, Giuseppe A ; Chifotides, Helen T ; Duminuco, Andrea ; Torre, Elena ; Bose, Prithviraj ; Vetro, Calogero

PURPOSE OF REVIEW:

In this review, we highlight conventional agents and novel emerging therapeutic strategies to treat anemia in MF.

RECENT FINDINGS:

Anemia is a common and challenging feature of myelofibrosis (MF). The pathobiology of anemia is multifactorial, including progressive bone marrow fibrosis, decreased erythropoiesis due to high hepcidin levels leading to iron sequestration in the reticuloendothelial system, hypersplenism, erythropoiesis inhibition by myelosuppressive JAK inhibitors (ruxolitinib, fedratinib), and others. MF-associated anemia has a negative impact on survival. Conventional agents to manage anemia include erythropoiesis-stimulating agents, danazol, corticosteroids, and immunomodulatory agents, but responses are infrequent and lack durability. Notable advancements have emerged in developing novel treatments for anemia in MF, including the regulatory approval of momelotinib (ACVR1/JAK1/2 inhibitor) in 2023 and development of novel promising agents targeting hemojuvelin and activins. Momelotinib and pacritinib (ACVR1/JAK2 inhibitor) are the preferred JAK inhibitors for patients with cytopenias (anemia, thrombocytopenia). Luspatercept and elritercept are activin receptor ligand traps, promoting erythroid maturation and late-stage erythropoiesis. Currently, luspatercept is being evaluated in a phase 3 trial (INDEPENDENCE™) for anemia in MF patients who are on a JAK2 inhibitor and require transfusions, and in a phase 2 trial (ODYSSEY) in combination with momelotinib in MF patients who are transfusion dependent, whether or not on a JAK inhibitor. Interim results of the RESTORE trial demonstrated that elritercept significantly decreased transfusions in MF patients. DISC-0974 is a first-in-class anti-hemojuvelin (positive hepcidin regulator) monoclonal antibody that decreased hepcidin expression, increased serum iron, and enhanced erythropoiesis in anemic patients with MF in a phase 1b/2 study. Burgeoning studies of novel anemia-targeted agents and combinations are significantly improving the quality of life and outcomes of patients with MF. The recent approval of momelotinib to treat MF with anemia and the emerging novel anemia-directed strategies in early and advanced clinical development have ushered in a new era in the treatment of MF-related anemia.

322

项与罗特西普相关的新闻（医药）

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！

2025-09-03

·PRNewswire

Myelofibrosis Market is Going to Grow at a CAGR of 9% in the Next Ten Years (2025-2034) | DelveInsight

The launch of emerging therapies like BESREMi (PharmaEssentia and AOP Orphan Pharmaceuticals), INCB057643 (Incyte), XPOVIO (Karyopharm Therapeutics), RYTELO (Geron), REBLOZYL (Bristol Myers Squibb), Navtemadlin (Kartos Therapeutics), Pelabresib (Novartis), and others is going to shift the myelofibrosis market. LAS VEGAS, Sept. 3, 2025 /PRNewswire/ -- DelveInsight's Myelofibrosis Market Insights report includes a comprehensive understanding of current treatment practices, emerging myelofibrosis drugs, market share of individual therapies, and current and forecasted myelofibrosis market size from 2020 to 2034, segmented into leading markets (the US, EU4, UK, and Japan). Myelofibrosis Market Summary The total myelofibrosis treatment market size in the leading markets (the US, EU4, UK, and Japan) was USD 2.2 billion in 2024. The United States accounts for the largest market size of myelofibrosis, in comparison to EU4 (Germany, Italy, France, and Spain) and the UK, and Japan. Based on DelveInsight's assessment in 2024, the 7MM had approximately 56K patient pool (prevalent cases) of myelofibrosis. Key myelofibrosis companies, including PharmaEssentia, AOP Orphan Pharmaceuticals, Incyte, Karyopharm Therapeutics, Geron, Bristol Myers Squibb, Kartos Therapeutics, Novartis, Merck, Telios Pharma, Ryvu Therapeutics, Sumitomo Pharma, Syntara, Disc Medicine, Menarini Group, and others, are actively working on innovative myelofibrosis drugs. Some of the key myelofibrosis therapies in clinical trials include BESREMi (ropeginterferon alfa-2b-njft/P-1101), INCB057643, XPOVIO (NEXPOVIO/selinexor/KPT-330), RYTELO (imetelstat), REBLOZYL (luspatercept/ACE-536), Navtemadlin (KRT-232), Pelabresib (DAK539), Bomedemstat (IMG-7289/MK-3543), TL-895, RVU120 ( SEL-120), TP-3654 (nuvisertib), SNT-5505 (PXS-5505), DISC-0974, ELZONRIS (tagraxofusp/SL-401), and others. These novel myelofibrosis therapies are anticipated to enter the myelofibrosis market in the forecast period and are expected to change the market. By 2034, among all the therapies, the highest revenue is expected to be generated by OJJAARA/OMJJARA. Discover which myelofibrosis therapies are expected to grab the largest market share @ Myelofibrosis Market Report Key Factors Driving the Growth of the Myelofibrosis Market Novel Therapies Drive Myelofibrosis Treatment Advancements Currently, four JAK inhibitors have been approved by the US FDA for the treatment of myelofibrosis, including JAKAFI (ruxolitinib), INREBIC (fedratinib), VONJO (pacritinib), and OJJAARA (momelotinib). No drug therapy can cure myelofibrosis. As myelofibrosis primarily affects older adults, stem cell transplantation is not a treatment option for most myelofibrosis patients. The launch of another JAK inhibitor, BESREMi, will further change the dynamics of the myelofibrosis market. Rich clinical pipeline and emerging non-JAK approaches driving the myelofibrosis landscape Beyond JAK inhibitors, active clinical development includes BET inhibitors (Incyte's INCB057643; Novartis' Pelabresib), XPO1 inhibitor (Karyopharm Therapeutics' XPOVIO), Telomerase inhibitor (Geron's RYTELO), MDM2 protein inhibitor (Kartos Therapeutics' Navtemadlin), Tyrosine kinase inhibitors (Telios Pharma's TL-895), PIM1 kinase inhibitor (Sumitomo Pharma's TP-3654), LOX inhibitor (Syntara's SNT-5505), and others, raising market potential by promising new label expansions, second-line options, and higher-value therapies should any prove to be disease-modifying. An aging population & increasing incidence with better survival Myelofibrosis primarily affects older adults; population aging increases the absolute number of patients. As treatments improve symptoms and (in some cases) survival, prevalent patient pools grow, supporting longer-term market demand. In the US in 2024, the 70+ years of age group had the highest number of cases, accounting for approximately 60% of the total prevalent cases, while the ≤39 years of age group accounted for just ~2%. Diagnostic & genomic testing improvements are driving myelofibrosis patient pool, leading to the growth of myelofibrosis market Wider use of molecular profiling (JAK2, CALR, MPL, and others) enables earlier and more accurate MF diagnosis and better patient stratification for targeted therapies. This both expands the diagnosed population and helps match patients to appropriate, often higher-value, treatments. Myelofibrosis Market Analysis JAK inhibitors have become the cornerstone of treatment for patients with myelofibrosis, offering significant benefits such as spleen reduction, symptom relief, and improved quality of life, which can also extend survival in those with advanced disease. All approved JAK inhibitors primarily target JAK2, particularly the wild-type form, but they differ in their activity against other JAK family members. For instance, JAKAFI myelofibrosis inhibits both JAK1 and JAK2; INREBIC selectively inhibits JAK2 while sparing JAK1 and also affects FLT3 and other targets; VONJO myelofibrosis inhibits JAK2 while sparing JAK1 but additionally impacts FLT3, IRAK, and ACVR1; and OJJAARA, approved through a different pathway, targets JAK1/JAK2 and ACVR1, mainly for myelofibrosis patients with anemia. These varying mechanisms lead to distinct patient outcomes. JAKAFI myelofibrosis continues to see strong demand and is expected to grow further, maintaining its position as the standard of care in myelofibrosis. Myelofibrosis will remain the largest segment of JAKAFI's patient population until polycythemia vera cases eventually increase. However, market growth may be limited due to patent expirations of key therapies, with JAKAFI patents set to expire in 2027 for Novartis and 2028 for Incyte, presenting potential opportunities for competitors. In response, Incyte is exploring combination trials with novel drugs to extend JAKAFI's therapeutic lifespan. Learn more about the treatment options for myelofibrosis @ Myelofibrosis Therapy The myelofibrosis treatment landscape offers significant opportunities for innovation, focusing on several key areas: Treating Lower-Risk Patients: Developing safe and effective therapies for patients with lower-risk myelofibrosis, allowing for earlier and more favorable interventions. Enhancing First-Line Treatments: Improving existing first-line therapies for intermediate- to high-risk patients through novel drugs or combination strategies. Managing Cytopenia: Developing targeted therapies to address cytopenia, a significant unmet need in patient care. Myelofibrosis Competitive Landscape The myelofibrosis clinical trial pipeline includes several drugs in mid- and late-stage development that are expected to enter the market during the forecast period. The emerging landscape offers a diverse range of therapeutic alternatives for treatment, including XPOVIO (Karyopharm Therapeutics), Elitracet (Takeda/Keros Therapeutics), Navtemadlin (Kartos Therapeutics), Pelabresib (Novartis), INCB057643 (Incyte), Bomedemstat (Merck), and others, all of which are used in various lines of treatment. The expected launch of these therapies is expected to have a further positive impact on the market. INCB57643 is an orally administered small molecule. Bromodomain and extra-terminal (BET) proteins act as epigenetic readers that control the expression of key oncoproteins implicated in the development of myelofibrosis and other hematologic malignancies, including B-lymphoma-2, nuclear factor kappa, and c-Myc. In a prior Phase I/II clinical trial, the oral BET inhibitor INCB057643, tested both as a monotherapy and in combination with ruxolitinib, demonstrated favorable tolerability and promising clinical activity in patients with advanced cancers. In its Q2 2025 financial report, the company announced that the combination of INCB057643 with ruxolitinib and INCB57643 (a JAK1/JAK2 and BET inhibitor) is currently being evaluated in a Phase II trial for myelofibrosis. RYTELO (imetelstat) is an investigational telomerase inhibitor that targets telomerase, selectively eliminating malignant stem and progenitor cells in the bone marrow, which drive diseases like myelodysplastic syndromes (MDS) and myelofibrosis. By blocking the proliferation of these malignant cells, imetelstat supports the recovery of healthy bone marrow and blood cell production and has shown disease-modifying effects and clinical benefits in Phase III trials for myelofibrosis. This mechanism sets imetelstat apart from other approved or investigational therapies for these blood cancers. In January 2025, Geron reported achieving 75% enrollment in the Phase III IMpactMF trial, which is comparing imetelstat to Best Available Therapy (BAT) in intermediate-2 or high-risk myelofibrosis patients who have relapsed or are refractory to JAK inhibitor treatment. Elritercept is a late-stage investigational activin inhibitor aimed at treating anemia associated with hematologic malignancies, including MDS and myelofibrosis. It is currently undergoing Phase II evaluation in patients with myelofibrosis. In December 2024, Keros Therapeutics presented updated data from this ongoing Phase II trial at ASH 2024. Additionally, in December 2024, Takeda announced an exclusive licensing agreement with Keros to further develop, manufacture, and commercialize elritercept globally, excluding mainland China, Hong Kong, and Macau. The anticipated launch of these emerging myelofibrosis therapies are poised to transform the myelofibrosis market landscape in the coming years. As these cutting-edge myelofibrosis therapies continue to mature and gain regulatory approval, they are expected to reshape the myelofibrosis market landscape, offering new standards of care and unlocking opportunities for medical innovation and economic growth. To know more about new myelofibrosis treatment, visit @ Myelofibrosis Treatment Market Recent Developments in the Myelofibrosis Market In July 2025, Incyte announced that the Phase I data in patients with myelofibrosis as monotherapy and in combination with ruxolitinib are anticipated in the second half of 2025. In June 2025, QIAGEN and Incyte announced a new global collaboration to develop a novel diagnostic panel to support Incyte's extensive portfolio of investigational therapies for patients with myeloproliferative neoplasms (MPNs), including Incyte's monoclonal antibody INCA033989. In January 2025, Karyopharm Therapeutics stated that it expects to report top-line results from the Phase III SENTRY trial in the second half of 2025, which could represent a potentially transformative opportunity to establish a new treatment paradigm in myelofibrosis. What is Myelofibrosis? Myelofibrosis is a rare blood cancer marked by the accumulation of scar tissue, or 'fibrosis', in the bone marrow. This excess scar tissue prevents the bone marrow from producing enough healthy blood cells. It belongs to a group of blood cancers called 'myeloproliferative neoplasms (MPNs)', in which the blood cells produced by the bone marrow grow and function abnormally. When myelofibrosis arises independently, without being caused by another bone marrow disorder, it is referred to as primary myelofibrosis. In other cases, it can develop from another MPN, such as polycythemia vera or essential thrombocythemia. When this occurs, it is called secondary myelofibrosis, sometimes specifically termed post-polycythemia vera myelofibrosis or post-essential thrombocythemia myelofibrosis. Myelofibrosis Epidemiology Segmentation The myelofibrosis epidemiology section provides insights into the historical and current myelofibrosis patient pool and forecasted trends for the leading markets (the US, EU4, UK, and Japan). It helps recognize the causes of current and forecasted patient trends by exploring numerous studies and views of key opinion leaders. The myelofibrosis market report proffers epidemiological analysis for the study period 2020–2034 in the leading markets (the US, EU4, UK, and Japan) segmented into: Total Prevalent Cases of Myelofibrosis Type-specific Cases of Myelofibrosis Myelofibrosis Cases Based on Risk Stratification Age-specific Prevalent Cases of Myelofibrosis Myelofibrosis Cases Based on Molecular Alterations Download the report to understand which factors are driving myelofibrosis epidemiology trends @ Myelofibrosis Treatment Drugs Scope of the Myelofibrosis Market Report Myelofibrosis Therapeutic Assessment: Myelofibrosis current marketed and emerging therapies Myelofibrosis Market Dynamics: Conjoint Analysis of Emerging Myelofibrosis Drugs Competitive Intelligence Analysis: SWOT analysis and Market entry strategies Myelofibrosis Market Unmet Needs, KOL's views, Analyst's views, Myelofibrosis Market Access and Reimbursement Discover more about myelofibrosis drugs in development @ Myelofibrosis Clinical Trials Table of Contents Related Reports JAK Inhibitors Market JAK Inhibitors Market Size, Target Population, Competitive Landscape, and Market Forecast – 2034 report delivers an in-depth understanding of the market trends, market drivers, market barriers, and key JAK inhibitors companies, including Pfizer, AbbVie, Galapagos, Sierra Oncology, Theravance Biopharma, Dizal Pharmaceutical, Aclaris Therapeutics, Celon Pharma, Incyte Corporation, Gilead Sciences, Reistone Biopharma, Jiangsu Hengrui Medicine Co., MaxiNovel Pharmaceuticals, among others. Myelofibrosis Clinical Trial Analysis Myelofibrosis Pipeline Insight – 2025 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key myelofibrosis companies, including Geron Corporation, Merck, Ryvu Therapeutics SA, Morphic Therapeutic, iOnctura, Pharmaxis, Nippon Shinyaku, Active Biotech, Incyte Corporation, Sumitomo Pharma America, Inc., Cellenkos, Disc Medicine, among others. Polycythemia Vera Market Polycythemia Vera Market Insights, Epidemiology, and Market Forecast – 2034 report delivers an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key polycythemia vera companies, including Incyte, Novartis, PharmaEssentia, AOP Orphan Pharmaceuticals, Protagonist Therapeutics, Merck (Imago BioSciences), Italfarmaco, Ionis Pharmaceutical, Silence Therapeutics, Perseus Proteomics, AbbVie, Johnson & Johnson Innovative Medicine, Mabwell (Shanghai) Bioscience, Disc Medicine, GluBio Therapeutics, among others. Multiple Myeloma Market Multiple Myeloma Market Insights, Epidemiology, and Market Forecast – 2034 report delivers an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key multiple myeloma companies, including Sanofi, Karyopharm Therapeutics, AbbVie, Takeda Pharmaceutical, Celgene, Bristol-Myers Squibb, RAPA Therapeutics, Pfizer, Array Biopharma, Cellectar Biosciences, BioLineRx, Celgene, Aduro Biotech, ExCellThera, Janssen Pharmaceutical, Precision BioSciences, Takeda, Glenmark (Ichnos Sciences SA), Poseida Therapeutics, Molecular Partners AG, Chipscreen Biosciences, AbbVie, Genentech (Roche), Janssen Biotech, Nanjing Legend Biotech, Merck Sharp & Dohme Corp., among others. About DelveInsight DelveInsight is a leading Business Consultant and Market Research firm focused exclusively on life sciences. It supports pharma companies by providing comprehensive end-to-end solutions to improve their performance. Get hassle-free access to all the healthcare and pharma market research reports through our subscription-based platform PharmDelve . Connect with us on LinkedIn | Facebook | Twitter Contact Us Shruti Thakur [email protected] +14699457679 Logo: SOURCE DelveInsight Business Research, LLP 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期引进/卖出临床1期上市批准临床结果

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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,

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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

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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

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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	憲構築壓鬱襯齋鏇齋鑰(網網窪鑰製艱遞淵網鬱): 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	壓壓齋簾壓衊觸願網襯(壓夢襯簾鏇繭製願廠繭) = 網構構窪積積鑰遞襯淵鏇獵醖壓繭願鑰鬱窪構 (獵鹽鑰壓襯鬱觸襯襯衊 ) 更多	积极	2025-05-30
				Erythropoiesis-stimulating agents	壓壓齋簾壓衊觸願網襯(壓夢襯簾鏇繭製願廠繭) = 簾鏇淵積襯壓夢鬱顧餘鏇獵醖壓繭願鑰鬱窪構 (獵鹽鑰壓襯鬱觸襯襯衊 ) 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	簾獵夢餘壓築製窪鬱製(積製憲網觸簾遞鏇衊網) = 壓繭範遞鏇構顧範鬱窪範夢鑰築鑰廠齋淵蓋範 (醖憲壓衊糧憲憲艱網憲 ) 更多	积极	2025-05-30
				Epoetin alfa	簾獵夢餘壓築製窪鬱製(積製憲網觸簾遞鏇衊網) = 積遞廠齋簾獵遞築窪選範夢鑰築鑰廠齋淵蓋範 (醖憲壓衊糧憲憲艱網憲 ) 更多
ASCO2025	N/A	骨髓增生异常综合征	2,614	Luspatercept	鑰齋願膚繭繭範鬱鹹構(觸鬱夢夢夢壓壓鏇鏇簾) = 願簾範壓顧膚積製願醖積膚膚夢餘獵襯艱蓋範 (膚壓膚窪廠繭製積鏇艱, 31.7 ~ 60.2) 更多	积极	2025-05-30
				Placebo	鑰齋願膚繭繭範鬱鹹構(觸鬱夢夢夢壓壓鏇鏇簾) = 繭觸獵獵築鬱夢餘繭願積膚膚夢餘獵襯艱蓋範 (膚壓膚窪廠繭製積鏇艱, 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	網憲積壓網選繭構壓淵(觸壓網衊壓廠範壓遞艱) = 製鏇繭餘憲憲築願夢鑰範顧壓製窪願簾壓觸衊 (顧齋積鏇鑰夢積壓蓋鑰 ) 更多	积极	2025-05-22
				Epoetin alfa	網憲積壓網選繭構壓淵(觸壓網衊壓廠範壓遞艱) = 顧鏇餘餘顧簾鏇觸築製範顧壓製窪願簾壓觸衊 (顧齋積鏇鑰夢積壓蓋鑰 ) 更多
NCT03682536 (COMMANDS, Pubmed \| Adv Ther)	临床3期	骨髓增生异常综合征	-	Luspatercept	壓遞膚醖構壓齋憲醖衊(齋網壓鹽鹽餘艱製鏇廠) = Rates of treatment-emergent adverse events, including asthenia and hypertension, generally decreased over time in both arms 願遞築顧鹽膚繭網艱膚 (壓壓遞積築積願願範觸 ) 更多	积极	2025-05-16
				Epoetin alfa
NCT03682536 (COMMANDS, EHA2025)	临床3期	骨髓增生异常综合征	350	Luspatercept	積鑰築齋齋壓醖鏇網鹹(餘顧獵網醖築繭淵淵糧) = 鹹選顧憲鬱遞憲憲鹽膚膚齋鹽齋鬱構鹹淵築憲 (製餘積觸齋鹹廠夢襯淵 ) 更多	积极	2025-05-14
				Epoetin Alfa	積鑰築齋齋壓醖鏇網鹹(餘顧獵網醖築繭淵淵糧) = 衊廠艱獵窪衊觸鏇衊簾膚齋鹽齋鬱構鹹淵築憲 (製餘積觸齋鹹廠夢襯淵 ) 更多
ACE-536-MF-001 (EHA2025)	临床2期	骨髓纤维化	36	Luspatercept	遞顧網憲齋襯襯積遞觸(願鑰膚範襯簾衊觸鑰鹹) = achieving a Hb level of ≥10 g/dL or increase of ≥1.5 g/dL was associated with a meaningful improvement in patient-reported anemia and fatigue symptoms 醖築蓋醖襯蓋鹽築遞鬱 (膚構顧願範襯鹹鑰壓鬱 ) 更多	-	2025-05-14
QOL-ONE PHOENIX (EHA2025)	N/A	输血依赖性贫血 \| 骨髓增生异常综合征	22	Luspatercept 1.0 mg/kg	構鑰觸鑰鑰蓋構觸繭遞(壓鏇餘醖簾艱窪遞遞艱) = one non-responder died of pneumonia 艱選窪膚獵廠夢願淵製 (積築遞遞壓醖築繭構製 ) 更多	积极	2025-05-14
COMMANDS (EHA2025)	临床3期	骨髓增生异常综合征一线	-	Luspatercept	網鏇願餘鏇構製鑰餘獵(糧顧鹽膚艱製糧憲襯醖) = 獵鏇選夢膚憲糧觸鏇醖築糧衊遞鏇齋繭淵窪構 (顧願鏇膚構襯襯艱醖淵 )	积极	2025-05-14
				Epoetin alfa	網鏇願餘鏇構製鑰餘獵(糧顧鹽膚艱製糧憲襯醖) = 範製顧觸齋鑰窪範齋顧築糧衊遞鏇齋繭淵窪構 (顧願鏇膚構襯襯艱醖淵 )