菲卓替尼(Fedratinib Hydrochloride) - 药物靶点：FLT3 x JAK2_在研适应症：原发性血小板增多症后骨髓纤维化，真性红细胞增多症后骨髓纤维化，原发性骨髓纤维化_专利_临床

概要

基本信息

药物类型

小分子化药

别名

FEDR、Fedratinib、INREBIC

+ [4]

靶点

FLT3 x JAK2

作用方式

抑制剂

作用机制

FLT3抑制剂(酪氨酸蛋白激酶受体FLT3抑制剂)、JAK2抑制剂(酪氨酸蛋白激酶JAK2抑制剂)

治疗领域

肿瘤血液及淋巴系统疾病其他疾病

在研适应症

原发性血小板增多症后骨髓纤维化真性红细胞增多症后骨髓纤维化原发性骨髓纤维化+ [8]

非在研适应症

晚期恶性实体瘤急性转化期慢性粒细胞性白血病肝损伤+ [3]

原研机构

TargeGen, Inc.

在研机构

Bristol Myers Squibb Co.

Celgene Corp.Bristol-Myers Squibb Pharma EEIG

+ [2]

非在研机构

TargeGen, Inc.

最高研发阶段批准上市

首次获批日期

美国 (2019-08-16),

骨髓纤维化真性红细胞增多症原发性血小板增多症

最高研发阶段(中国)临床3期

特殊审评孤儿药 (美国)、孤儿药 (韩国)

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结构/序列

分子式C27H40Cl2N6O4S

InChIKeyQAFZLTVOFJHYDF-UHFFFAOYSA-N

CAS号1374744-69-0

关联

30

项与菲卓替尼相关的临床试验

NCT05883904

/ RecruitingN/AIIT

Observational Study Protocol REALFed - Real World Evidence of Fedratinib Effectiveness in Myelofibrosis

This is a multicenter prospective and retrospective observational clinical study in patients with primary or post polycythemia vera or post essential thrombocythemia myelofibrosis to test the efficacy of fedratinib in the rea world. Participants will be managed according to the clinical practice of the participating Center. All Centers will be Italian Hematology Units belonging to the GIMEMA Organization in Italy.

开始日期2024-01-29

申办/合作机构

Gruppo Italiano Malattie EMatologiche dell'Adulto

NCT06073847

/ RecruitingN/A

Inrebic® (Fedratinib) Post-Marketing Surveillance in Korean Patients With Myelofibrosis

The purpose of this study is to assess the real-world safety of fedratinib for the treatment of adult participants with primary myelofibrosis (PMF), post polycythemia vera myelofibrosis (post-PV MF), or post essential thrombocythemia myelofibrosis (post-ET MF) who were previously treated with ruxolitinib. Participants will represent the overall patient population with PMF, post-PV MF or post-ET MF who lost adequate response to and/or are intolerant to ruxolitinib. Inadequate response definitions will follow Ministry of Food and Drug Safety-approved label and reimbursement criteria of the Health Insurance Review & Assessment Service.

开始日期2023-06-13

申办/合作机构

Bristol Myers Squibb Co.

ISRCTN88102629

/ Recruiting临床2期IIT

A phase II study to evaluate the tolerability, safety and activity of fedratinib combined with ropeginterferon alfa-2b in patients with myelofibrosis

开始日期2022-09-12

申办/合作机构

University of Birmingham

100 项与菲卓替尼相关的临床结果

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100 项与菲卓替尼相关的转化医学

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100 项与菲卓替尼相关的专利（医药）

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522

项与菲卓替尼相关的文献（医药）

2025-04-01·MOLECULAR DIVERSITY

Targeting AFP-RARβ complex formation: a potential strategy for treating AFP-positive hepatocellular carcinoma

Article

作者: Dev, Radul R ; Soman, Sowmya ; Banjan, Bhavya ; Raju, Rajesh ; Vishwakarma, Riya ; Kalath, Haritha ; Rehman, Niyas ; Revikumar, Amjesh ; Kumar, Pankaj ; Abhinand, Chandran S ; Ramakrishnan, Krishnapriya

Alpha-fetoprotein (AFP) is a glycoprotein primarily expressed during embryogenesis, with declining levels postnatally. Elevated AFP levels correlate with pathological conditions such as liver fibrosis, cirrhosis, and hepatocellular carcinoma (HCC). Recent investigations underscore AFP's intracellular role in HCC progression, wherein it forms complexes with proteins like Phosphatase and tensin homolog (PTEN), Caspase 3 (CASP3), and Retinoic acid receptors and Retinoid X receptors (RAR/RXR). RAR and RXR regulate gene expression linked to cell death and tumorigenesis in normal physiology. AFP impedes RAR/RXR dimerization, nuclear translocation, and function, promoting gene expression favoring cancer progression in HCC that provoked us to target AFP as a drug candidate. Despite extensive studies, inhibitors targeting AFP to disrupt complex formation and activities remain scarce. In this study, employing protein-protein docking, amino acid residues involved in AFP-RARβ interaction were identified, guiding the definition of AFP's active site for potential inhibitor screening. Currently, kinase inhibitors play a significant role in cancer treatment and, the present study explores the potential of repurposing FDA-approved protein kinase inhibitors to target AFP. Molecular docking with kinase inhibitors revealed Lapatinib as a candidate drug of the AFP-RARβ complex. Molecular dynamics simulations and binding energy calculations, employing Mechanic/Poisson-Boltzmann Surface Area (MM-PBSA), confirmed Lapatinib's stability with AFP. The study suggests Lapatinib's potential in disrupting the AFP-RARβ complex, providing a promising avenue for treating molecularly stratified AFP-positive HCC or its early stages.

2025-04-01·MOLECULAR AND CELLULAR BIOCHEMISTRY

Amyloid beta-induced signalling in leptomeningeal cells and its impact on astrocyte response

Article

作者: Stanton, Janelle E ; Abubaker, Mannthalah ; Mulvihill, John J E ; Newport, David ; Grabrucker, Andreas M ; Mahon, Olwyn

The pathological signature of Alzheimer's disease (AD) includes the accumulation of toxic protein aggregates, mainly consisting of amyloid beta (Aβ). Recent strides in fundamental research underscore the pivotal role of waste clearance mechanisms in the brain suggesting it may be an early indication of early onset AD. This study delves into the involvement of leptomeningeal cells (LMCs), crucial components forming integral barriers within the clearance system, in the context of AD. We examined the inflammatory cytokine responses of LMCs in the presence of Aβ, alongside assessments of LMC growth response, viability, oxidative stress, and changes in vimentin expression. The LMCs showed no changes in growth, viability, oxidative stress, or vimentin expression in the presence of Aβ, indicating that LMCs are less susceptible to Aβ damage compared to other CNS cells. However, LMCs exhibited a unique pro-inflammatory response to Aβ when compared to an LPS inflammatory control, showing an mRNA expression of pro-inflammatory cytokines such IL-6, IL-10 and IL-33 but no changes in IL-1α and IL-1β. Furthermore, LMCs influenced the astrocyte response to Aβ, as conditioned media from Aβ-treated LMCs was observed to downregulate somatic S100β in astrocytes. We also investigated whether the JAK/STAT3 pathway was involved in the Aβ response of the LMCs, as this pathway has been shown to be activated in astrocytes and neurons in the presence of Aβ. JAK/STAT3 activation was assessed through phosphorylated STAT3, revealing that JAK/STAT3 was not active in the cells when in the presence of Aβ. However, when JAK1 and JAK2 were inhibited, cytokine protein levels of IL7, IL10, IL15 and IL33 levels, which had shown alteration when LMCs were treated with Aβ, returned to base levels. This indicates that although JAK1/STAT3 and JAK2/STAT3 are not the direct pathway for Aβ response in LMCs, JAK1 and JAK2 may still play a role in regulating cytokine levels, potentially through indirect means or crosstalk. Overall, our findings reveal that LMCs are resilient to Aβ toxicity and suggest that JAK1/STAT3 and JAK2/STAT3 does not play a central role in the inflammatory response, providing new insights into the cellular mechanisms underlying AD.

2025-02-19·Future Oncology

Real-world treatment patterns and health outcomes for patients with myelofibrosis treated with fedratinib

Article

作者: Korgaonkar, Siddhi ; Rombi, Julien ; Jones, Shalon ; Parikh, Rohan C ; Yucel, Aylin ; Zissler, Dorothy ; Davis, Keith L ; Slaff, Samantha ; Chevli, Manoj ; Passamonti, Francesco

AIM:

Assess real-world fedratinib (FEDR) treatment patterns and clinical outcomes in patients with primary or secondary myelofibrosis following discontinuation of ruxolitinib (RUX).

PATIENTS & METHODS:

This study was a retrospective, noninterventional medical record review of patients in Canada, Germany, and the United Kingdom (UK). A total of 70 physicians (primarily hematologist-oncologists [78.6%]) provided data for 196 eligible patients.

RESULTS:

Patients were mostly male (62.8%) with primary myelofibrosis (76.5%) and initiated FEDR at a mean age of 67.7 years. Median treatment duration was 11.5 months (median follow-up, 13.8 months), and nearly half (49.5%) of patients initiated FEDR at the label-indicated dose of 400 mg daily. Six months post-initiation, 77.7% and 66.8% of patients experienced symptom and spleen response, respectively. Kaplan-Meier estimates of median progression-free and overall survival from initiation were 23.8 months (95% CI, 21.1-27.6) and 29.8 months (95% CI, 23.9-NE), respectively.

CONCLUSION:

These findings demonstrate real-world FEDR effectiveness among patients with myelofibrosis who discontinued RUX.

67

项与菲卓替尼相关的新闻（医药）

2025-03-12

·微信

AJH丨梅奥经验：脾切除术在JAK2抑制剂治疗的骨髓纤维化患者中的结果

点击蓝字关注我们编者按骨髓纤维化（MF）是一种以脾脏肿大和骨髓功能衰竭为特征的骨髓增殖性肿瘤。JAK2V617F突变的发现和JAK2抑制剂（JAKi）的开发彻底改变了MF的治疗格局。然而，JAKi治疗的益处通常是短暂的，许多患者最终会因耐药而停药。为此，梅奥诊所Naseema Gangat教授等探讨了脾切除术在JAK2抑制剂治疗的骨髓纤维化患者中的应用，并于近期发表于American Journal of Hematology。本文特整理研究主要内容，以飨读者。骨髓纤维化（MF）是一种以脾脏肿大和骨髓功能衰竭为特征的骨髓增殖性肿瘤。JAK2V617F突变的发现和JAK2抑制剂（JAKi）的开发彻底改变了MF的治疗格局。目前，美国食品药品监督管理局（FDA）已批准四种JAKi用于MF的治疗：芦可替尼（Ruxolitinib）、菲卓替尼（Fedratinib）、帕瑞替尼（Pacritinib）和莫洛替尼（Momelotinib）。这些药物能够有效缓解脾脏肿大和全身症状。然而，JAKi治疗的益处通常是短暂的，许多患者最终会因耐药而停药。对于JAKi耐药的患者，脾切除术或脾脏放疗通常被建议作为异体造血干细胞移植（ASCT）的桥梁。本研究回顾性分析了梅奥诊所34例接受JAKi治疗后进行脾切除术的MF患者。纳入标准包括：（1）根据国际共识分类诊断为MF；（2）有JAKi治疗记录并随后进行了脾切除术；（3）有术前和术后的详细信息。最终纳入34例患者，其中29例接受开腹脾切除术，5例接受腹腔镜脾切除术。患者特征 34例MF患者中，原发性MF（PMF）18例，真性红细胞增多症后MF （post-PV MF）15例，原发性血小板增多症后MF（post-ET MF）1例。中位年龄为63岁，59%为男性，中位脾脏长度为25.1厘米（范围14.7-36厘米）。21例（62%）检测到JAK2V617F突变，9例（26%）检测到CALR突变，1例（3%）检测到MPL突变，3例（9%）为三阴性。26例（76%）接受过一种JAKi治疗，6例（18%）接受过两种JAKi治疗，2例（6%）接受过三种JAKi治疗。具体JAKi包括芦可替尼（Ruxolitinib，79%）、莫洛替尼（Momelotinib，15%）、菲卓替尼（Fedratinib，9%）、帕瑞替尼（Pacritinib，9%）和BMS 911543（15%）。术后并发症 21例（62%）患者报告了术后并发症，包括重大出血（29%）、血栓形成（26%）和其他并发症（感染、肾损伤、气胸）。PMF与post-PV/ET MF患者并发症发生率之间无统计学差异（78% vs. 50%，P=0.09），使用一种与两种或更多JAKi患者并发症发生率之间亦无统计学差异（71% vs. 50%，P=0.29），停药与继续使用JAKi患者并发症发生率之间也无统计学差异（64% vs. 61%，P=0.88）。生存率和移植结果脾切除术后中位随访1.7年（范围0.01-10.2年），15例（44%）患者死亡。脾切除术后中位生存期为4.3年，30天和60天死亡率分别为6%和15%。 △脾切除术后患者生存情况 14例患者在接受脾切除术后准备移植，其中12例（86%）成功桥接至ASCT，中位时间为3个月（范围1.5-26个月）。移植患者的中位生存期未达到，而非移植患者为2.6年（5年生存率65% vs. 22%，P=0.02）。移植后中位随访2.6年（范围0.3-9.8年），7例（58%）患者仍存活且完全缓解，2例（17%）复发，3例（25%）死于感染和移植物抗宿主病。 △脾切除术后移植或未移植患者生存情况血液学改善 22例未接受ASCT的患者中，10例（45%）在脾切除术时有输血依赖性贫血，其中5例（50%）在术后至少12周内不再需要输血，中位输血独立反应持续时间为2.8年（范围0.3-7.1年）。5例基线血小板计数低于50×10^9/L的患者中，4例（80%）在术后3个月内血小板计数超过100×10^9/L，中位血小板计数为170×10^9/L（范围119-279×10^9/L）。 △脾切除术、脾切除术后 6 周至 3 个月和 1 年的中位血细胞计数结论脾切除术在JAKi治疗后的MF患者中具有重要的治疗价值，既可作为桥接至ASCT的手段，也可作为改善输血依赖性贫血和血小板减少症的姑息治疗策略。尽管术后并发症发生率较高，但大多数患者能够成功桥接至ASCT，并在移植后长期存活且完全缓解。未来的研究需要进一步确定术后并发症的预测因素，以指导管理策略，并确定脾切除术后和围移植期JAKi使用的最佳方法。参考文献： Carey P, Pardanani A, Starlinger P, Tefferi A, Gangat N. Outcome of Splenectomy in JAK2 Inhibitor Treated Patients With Myelofibrosis: A Mayo Clinic Experience in 34 Consecutive Cases. Am J Hematol. 2025 Mar;100(3):527-530. doi: 10.1002/ajh.27570. Epub 2024 Dec 26. PMID: 39723660. （来源：《肿瘤瞭望–血液时讯》编辑部）声明凡署名原创的文章版权属《肿瘤瞭望》所有，欢迎分享、转载。本文仅供医疗卫生专业人士了解最新医药资讯参考使用，不代表本平台观点。该等信息不能以任何方式取代专业的医疗指导，也不应被视为诊疗建议，如果该信息被用于资讯以外的目的，本站及作者不承担相关责任。

临床结果

2025-02-20

·药筛

专利未到，首仿已获批

今天继续推荐关注品种：磷酸芦可替尼片。原研全球市场销售一路高歌猛进，2024年超过50亿美元，还有增长潜力。国内市场，原研诺华2017年进口，2019年进入医保目录，目前独家在售。医院市场年销售额超过5亿，加上院外市场，总规模约10亿元。原研国内核心专利2026年到期，国内已有多个企业申报仿制。成都苑东首家报产获批上市，但不能上市销售。正大天晴、重庆华森等，多个企业申报，竞争激烈以下是产品简介，由摩熵医药AI生成。磷酸芦可替尼片（Ruxolitinib Phosphate）详细分析一、药物基本信息通用名：磷酸芦可替尼（Ruxolitinib Phosphate）商品名：捷恪卫（Jakavi，美国商品名Jakafi）靶点：JAK1/JAK2抑制剂（选择性抑制Janus激酶通路）适应症：中高危骨髓纤维化（MF）；真性红细胞增多症（PV）对羟基脲耐药或不耐受；急性移植物抗宿主病（GVHD）；特应性皮炎（部分国家获批）研发公司：由Incyte Corporation研发，诺华（Novartis）负责美国以外市场商业化。二、作用机制与药理学特点 JAK-STAT通路抑制：芦可替尼通过抑制JAK1和JAK2激酶，阻断促炎细胞因子（如IL-6、IFN-γ）和促增殖信号（如EPO、TPO）的传导。在骨髓纤维化中，抑制异常活化的JAK2 V617F突变信号，减少炎症因子释放和髓外造血。选择性优势：对JAK2的选择性高于JAK3（避免过度免疫抑制），但对JAK1的抑制可能增加感染风险。三、药代动力学（PK/PD）吸收：口服生物利用度约95%，达峰时间1-2小时，食物对吸收无显著影响。代谢：主要经CYP3A4代谢，次要经CYP2C9。半衰期：约3小时，需每日两次给药。排泄：74%经尿液（主要为代谢物），22%经粪便。特殊人群：肝肾功能不全者需调整剂量（如Child-Pugh B/C患者剂量减半）。四、临床疗效数据骨髓纤维化（MF）： 41.9%患者脾脏体积缩小≥35%（vs. 0.7%安慰剂组）； 45.9%患者总症状评分改善≥50%（vs. 5.3%安慰剂组）。 COMFORT-I/II试验（III期）：生存获益：5年随访显示芦可替尼组生存期显著延长（HR=0.69）。真性红细胞增多症（PV）： 21%患者达到完全血液学缓解（vs. 1%对照组）； 60%患者无需放血治疗（vs. 20%对照组）。 RESPONSE试验： GVHD： REACH试验：芦可替尼治疗激素难治性急性GVHD，总缓解率（ORR）达57%。五、安全性及不良反应常见不良反应：血液系统：贫血（44%）、血小板减少（38%）、中性粒细胞减少（16%）。感染：带状疱疹（7%）、尿路感染（10%）。其他：头晕、头痛、血脂升高。黑框警告：严重感染（结核、真菌、细菌性肺炎等）；恶性肿瘤风险（如淋巴瘤、非黑色素瘤皮肤癌）；血栓事件（深静脉血栓、肺栓塞）。六、药物政策与市场表现国内现状： 2017年在中国获批骨髓纤维化适应症，2020年进入国家医保（乙类）。价格：约500元/5mg/片（医保后自付比例约30%）。全球市场： 2022年全球销售额：Jakafi（美国）20.3亿美元，Jakavi（海外）14.6亿美元。核心竞争产品：Fedratinib（JAK2抑制剂）、Momelotinib（JAK1/2/ACVR1抑制剂）。七、研发进展与未来方向适应症拓展：白癜风：III期试验显示52周后面部复色率≥75%的患者达30%；特应性皮炎：欧盟已批准，美国处于III期临床。联合疗法：与BCL-2抑制剂（Venetoclax）联用探索骨髓纤维化治疗；与PD-1抑制剂联用于实体瘤（早期临床）。用摩熵药筛小程序，快速查询产品申报进展！

专利到期临床3期

2024-12-20

·PRNewswire

Vanda Pharmaceuticals Announces Orphan Drug Designation Granted for VGT-1849A, a Novel and Selective Antisense Oligonucleotide Candidate for the Treatment of Polycythemia Vera

WASHINGTON, Dec. 20, 2024 /PRNewswire/ -- Vanda Pharmaceuticals Inc. (Vanda) (Nasdaq: VNDA) today announced the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation for VGT-1849A, a selective antisense oligonucleotide (ASO)-based JAK2 inhibitor for the treatment of polycythemia vera (PV), a form of a rare hematologic malignancy that is estimated to affect 1 in 2000 Americans.1 PV is a chronic myeloproliferative disorder characterized by aberrant hematopoiesis of myeloid lineage with exuberant red cell production and increased release of pro-inflammatory cytokines. More than 95% of PV patients harbor the JAK2 V617F gain-of-function mutation leading to aberrant JAK2 production.2 Inhibiting JAK2 acts to suppress hematopoiesis, consequently reducing red blood cell, neutrophil, platelet, and lymphocyte production. JAK2 inhibitors have been shown to be efficacious in treating various JAK-dependent hematologic malignancies, including the treatment of PV. By selective reduction of JAK2 levels, the ASO VGT-1849A has the potential to reduce JAK2V617F-driven pathogenic signaling, ultimately suppressing the malignant proliferation and survival of hematopoietic cells. Currently available small molecule inhibitors targeting the JAK2 protein kinase, such as Jakafi®, Inrebic®, Ojjaara®, and Vonjo®, lack sole selectivity for the target protein, which can result in off target effects. The adverse side effects that may occur from JAK inhibition emphasize the importance of selectively targeting JAK2 while avoiding inhibition of other JAK family members. By specifically targeting JAK2, Vanda seeks to reduce the risk of infection and toxic effects that are seen with inhibitors also blocking JAK1, JAK3, TYK2, or other kinases outside of the JAK family. If approved, VGT-1849A could offer targeted efficacy with an improved safety profile and convenient dosing. "This orphan designation for VGT-1849A is an important milestone in precision medicine-based therapeutics in the space of hematological malignancies. This milestone marks the second precision medicine therapeutic for Vanda following the development of VCA-894A for Charcot-Marie-Tooth3 that is expected to begin clinical testing in the coming months," said Mihael H. Polymeropoulos, M.D., Vanda's President, CEO and Chairman of the Board. VGT-1849A is a novel ASO treatment candidate for PV and other JAK2-driven hematologic malignancies. By selectively targeting JAK2, VGT-1849A reduces downstream signaling and JAK2V617F-driven autonomous cell proliferation, without any off-target kinase effects. The ability of VGT-1849A to reduce JAK2 activity may alleviate the disease burden that patients with PV face with a favorable safety profile, resulting in a higher quality of life for patients. Orphan Drug Designation is granted by the FDA to investigational therapies addressing rare medical conditions and provides benefits to drug developers. References: Grunwald, M. R.; Stein, B. L.; Boccia, R. V.; Oh, S. T.; Paranagama, D.; Parasuraman, S.; Colucci, P.; Mesa, R. Clinical and Disease Characteristics From REVEAL at Time of Enrollment (Baseline): Prospective Observational Study of Patients With Polycythemia Vera in the United States. Clin Lymphoma Myeloma Leuk 2018, 18 (12), 788-795.e2. . P. Gou, W. Zhang, and S. Giraudier, "Insights into the Potential Mechanisms of JAK2V617F Somatic Mutation Contributing Distinct Phenotypes in Myeloproliferative Neoplasms," Myeloproliferative Neoplasms. Int. J. Mol. Sci, vol. 2022, p. 1013, 2022, S. Smieszek, C. Tyner, A. Kaden, C. Johnson, C. Polymeropoulos, G. Birznieks, M. Polymeropoulos. Potential treatment for CMT2S caused by IGHMBP2 cryptic splice variant, with ASO based therapeutic [abstract]. Mov Disord. 2023; 38 (suppl 1). . About Vanda Pharmaceuticals Inc. Vanda is a leading global biopharmaceutical company focused on the development and commercialization of innovative therapies to address high unmet medical needs and improve the lives of patients. For more on Vanda Pharmaceuticals Inc., please visit and follow us on X @vandapharma. About VGT-1849A VGT-1849A is an antisense oligonucleotide (ASO) that selectively targets JAK2, reducing increased activity of JAK2 that may cause hematologic malignancies. ASOs have broad applicability in addressing a number of disorders caused by genetic variants. CAUTIONARY NOTE REGARDING FORWARD-LOOKING STATEMENTS Various statements in this press release, including, but not limited to statements regarding the estimated prevalence of PV, the potential therapeutic effects of VGT-1849A, the timing of the initiation of clinical testing of VCA-894A and the potential benefits of VGT-1849A, are "forward-looking statements" under the securities laws. Forward-looking statements are based upon current expectations that involve risks, changes in circumstances, assumptions and uncertainties. Important factors that could cause actual results to differ materially from those reflected in Vanda's forward-looking statements include, among others, the accuracy of the reporting and diagnosis of PV cases, the ability of VGT-1849A to safely and effectively treat PV, Vanda's ability to begin clinical testing of VCA-894A during the specified timeframe and Vanda's ability to successfully complete the clinical development of, and obtain regulatory approval for, VGT-1849A in the treatment of PV. There can be no assurance that the actual results or developments anticipated by Vanda will be realized or, even if substantially realized, that they will have the expected consequences to, or effects on, Vanda. Therefore, no assurance can be given that the outcomes stated in such forward-looking statements and estimates will be achieved. Forward-looking statements in this press release should be evaluated together with the various risks and uncertainties that affect Vanda's business and market, particularly those identified in the "Cautionary Note Regarding Forward-Looking Statements", "Risk Factors" and "Management's Discussion and Analysis of Financial Condition and Results of Operations" sections of Vanda's Annual Report on Form 10-K for the fiscal year ended December 31, 2023, as updated by Vanda's subsequent Quarterly Reports on Form 10-Q, Current Reports on Form 8-K and other filings with the U.S. Securities and Exchange Commission, which are available at . All written and verbal forward-looking statements attributable to Vanda or any person acting on its behalf are expressly qualified in their entirety by the cautionary statements contained or referred to herein. Vanda cautions investors not to rely too heavily on the forward-looking statements Vanda makes or that are made on its behalf. The information in this press release is provided only as of the date of this press release, and Vanda undertakes no obligation, and specifically declines any obligation, to update or revise publicly any forward-looking statements, whether as a result of new information, future events or otherwise, except as required by law. Corporate Contact: Kevin Moran Senior Vice President, Chief Financial Officer and Treasurer Vanda Pharmaceuticals Inc. 202-734-3400 [email protected] Jim Golden / Jack Kelleher / Dan Moore Collected Strategies [email protected] SOURCE Vanda Pharmaceuticals Inc. WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

孤儿药寡核苷酸

100 项与菲卓替尼相关的药物交易

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外链

KEGG	Wiki	ATC	Drug Bank
-	菲卓替尼	L01XE57	DB12500

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
原发性血小板增多症后骨髓纤维化	欧盟	Bristol-Myers Squibb Pharma EEIG	2021-02-08
原发性血小板增多症后骨髓纤维化	冰岛	Bristol-Myers Squibb Pharma EEIG	2021-02-08
原发性血小板增多症后骨髓纤维化	列支敦士登	Bristol-Myers Squibb Pharma EEIG	2021-02-08
原发性血小板增多症后骨髓纤维化	挪威	Bristol-Myers Squibb Pharma EEIG	2021-02-08
真性红细胞增多症后骨髓纤维化	欧盟	Bristol-Myers Squibb Pharma EEIG	2021-02-08
真性红细胞增多症后骨髓纤维化	冰岛	Bristol-Myers Squibb Pharma EEIG	2021-02-08
真性红细胞增多症后骨髓纤维化	列支敦士登	Bristol-Myers Squibb Pharma EEIG	2021-02-08
真性红细胞增多症后骨髓纤维化	挪威	Bristol-Myers Squibb Pharma EEIG	2021-02-08
原发性骨髓纤维化	欧盟	Bristol-Myers Squibb Pharma EEIG	2021-02-08
原发性骨髓纤维化	冰岛	Bristol-Myers Squibb Pharma EEIG	2021-02-08
原发性骨髓纤维化	列支敦士登	Bristol-Myers Squibb Pharma EEIG	2021-02-08
原发性骨髓纤维化	挪威	Bristol-Myers Squibb Pharma EEIG	2021-02-08
脾肿大	欧盟	Bristol-Myers Squibb Pharma EEIG	2021-02-08
脾肿大	冰岛	Bristol-Myers Squibb Pharma EEIG	2021-02-08
脾肿大	列支敦士登	Bristol-Myers Squibb Pharma EEIG	2021-02-08
脾肿大	挪威	Bristol-Myers Squibb Pharma EEIG	2021-02-08
骨髓纤维化	美国	Bristol Myers Squibb Co.	2019-08-16
真性红细胞增多症	美国	Bristol Myers Squibb Co.	2019-08-16
原发性血小板增多症	美国	Bristol Myers Squibb Co.	2019-08-16

未上市

10 条进展最快的记录,

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适应症	最高研发状态	国家/地区	公司	日期
血液肿瘤	临床3期	美国	Bristol Myers Squibb Co.	2011-12-01
血液肿瘤	临床3期	澳大利亚	Bristol Myers Squibb Co.	2011-12-01
血液肿瘤	临床3期	奥地利	Bristol Myers Squibb Co.	2011-12-01
血液肿瘤	临床3期	比利时	Bristol Myers Squibb Co.	2011-12-01
血液肿瘤	临床3期	巴西	Bristol Myers Squibb Co.	2011-12-01
血液肿瘤	临床3期	加拿大	Bristol Myers Squibb Co.	2011-12-01
血液肿瘤	临床3期	法国	Bristol Myers Squibb Co.	2011-12-01
血液肿瘤	临床3期	德国	Bristol Myers Squibb Co.	2011-12-01
血液肿瘤	临床3期	匈牙利	Bristol Myers Squibb Co.	2011-12-01
血液肿瘤	临床3期	爱尔兰	Bristol Myers Squibb Co.	2011-12-01

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临床结果

适应症

分期

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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


NCT03952039 (FREEDOM2, Pubmed \| Lancet Haematol) 人工标引	临床3期	骨髓纤维化二线	201	Fedratinib 400 mg per day	選獵積窪齋簾襯醖壓選(窪繭簾製鏇糧網觸憲遞) = 糧憲鏇網觸築膚鹽齋鬱鏇鏇淵艱鏇蓋艱簾鬱範 (簾壓餘廠選襯顧願製艱 ) 更多	积极	2024-09-01
				Best Available Therapy (BAT)	選獵積窪齋簾襯醖壓選(窪繭簾製鏇糧網觸憲遞) = 襯鹽窪醖鹹繭蓋艱範鏇鏇鏇淵艱鏇蓋艱簾鬱範 (簾壓餘廠選襯顧願製艱 ) 更多
NCT03755518 (FREEDOM, Pubmed \| Leuk Lymphoma)	临床3期	骨髓纤维化	38	Fedratinib	遞齋顧鏇廠選鏇蓋衊選(鏇積簾醖獵願遞範積壓) = 網積憲醖鹽願淵鹽構夢衊壓製鬱築鏇繭鬱餘衊 (願築壓齋餘鬱蓋鏇簾顧, 12.5 ~ 43.3) 更多	积极	2024-07-28
NCT03952039 (FREEDOM2, EHA2024) 人工标引	临床3期	骨髓纤维化二线	201	Fedratinib 400mg daily	廠製顧夢蓋窪衊鬱蓋願(製築鏇製憲遞網艱衊築) = 艱觸遞衊淵淵繭艱選襯膚膚窪齋夢範網選鑰願 (範夢襯艱廠壓製襯構淵 ) 更多	积极	2024-05-14
				Best Available Therapy (BAT)	廠製顧夢蓋窪衊鬱蓋願(製築鏇製憲遞網艱衊築) = 構壓壓鑰襯蓋醖鹽襯觸膚膚窪齋夢範網選鑰願 (範夢襯艱廠壓製襯構淵 ) 更多
Tandem Meetings ASTCT and CIBMTR2024	N/A	骨髓纤维化	18	Fedratinib 400 mg once daily	遞顧願網衊憲窪艱齋鹽(窪簾範糧鹹網廠艱鏇淵) = n=4 (22.2%) deaths were recorded as of May 2023. Two of the 4 deaths were related to MF and both patients had disease transformation to blast phase MPN before HSCT. A median duration of 14.5 months elapsed between FEDR initiation and death among these 4 patients 觸遞壓製繭壓積觸餘夢 (鬱築夢夢糧製鹹鹽醖範 ) 更多	积极	2024-02-01
NCT03952039 (FREEDOM2, CTgov)	临床3期	原发性血小板增多症后骨髓纤维化 \| 真性红细胞增多症后骨髓纤维化 \| 原发性骨髓纤维化	202	Best Available Therapy (BAT)	鬱選製膚夢壓顧鹽鹹繭 = 鹹構繭廠構鑰壓鹽糧衊餘願糧觸獵壓鹹齋構顧 (壓獵艱築範簾憲憲顧網, 築窪鏇窪醖鑰襯簾製醖 ~ 築積積繭觸窪廠窪窪艱) 更多	-	2024-01-30
NCT04817007 (CA011-023, ASH2023)	临床1/2期	原发性骨髓纤维化	40	BMS-986158+Ruxolitinib	壓廠壓觸鬱繭願壓蓋鏇(觸蓋膚範繭鑰網艱積壓) = 憲餘醖網鹹觸積蓋遞遞觸糧淵鏇蓋蓋遞醖繭顧 (繭顧蓋簾糧夢憲窪糧糧 )	-	2023-12-10
				BMS-986158+Fedratinib	壓廠壓觸鬱繭願壓蓋鏇(觸蓋膚範繭鑰網艱積壓) = 窪範壓觸蓋顧蓋夢製襯觸糧淵鏇蓋蓋遞醖繭顧 (繭顧蓋簾糧夢憲窪糧糧 )
NCT03952039 (FREEDOM2, ASH2023)	临床3期	骨髓纤维化	201	Fedratinib 400 mg/day	醖鹹製願鑰網鑰構繭製(膚繭壓簾鑰範餘範蓋鏇) = 糧築網糧糧夢壓鑰觸鹽顧壓蓋積糧廠築鹽夢鑰 (鏇廠餘繭範鹹淵夢醖觸 ) 更多	积极	2023-12-10
				Best Available Therapy (BAT)	醖鹹製願鑰網鑰構繭製(膚繭壓簾鑰範餘範蓋鏇) = 築醖製範糧衊鹹願顧糧顧壓蓋積糧廠築鹽夢鑰 (鏇廠餘繭範鹹淵夢醖觸 ) 更多
ASH2023	N/A	二线	-	Ruxolitinib	襯簾鬱衊鹽鑰選膚鑰襯(獵醖鑰艱糧淵鬱築簾積) = 積壓選鏇構襯積醖廠衊網觸窪窪構鑰鬱遞窪鹹 (糧蓋鏇淵衊醖糧窪壓衊 ) 更多	-	2023-12-09
				Fedratinib	襯簾鬱衊鹽鑰選膚鑰襯(獵醖鑰艱糧淵鬱築簾積) = 遞憲顧繭遞壓鬱廠獵壓網觸窪窪構鑰鬱遞窪鹹 (糧蓋鏇淵衊醖糧窪壓衊 )
ASH2023	N/A	原发性骨髓纤维化	58	Fedratinib	窪夢簾獵築鬱糧遞範醖(遞鹹繭壓壓窪鏇艱艱獵) = 鹹願鹽鹹齋廠蓋衊範襯鹹製蓋醖製糧衊齋顧壓 (襯範蓋鹹餘廠積齋鹹網 )	-	2023-12-09
NCT05177211 (ASH2023)	临床2期	慢性中性粒细胞白血病 \| 骨髓增生性疾病	10	Fedratinib	襯艱醖餘選膚襯淵蓋遞(蓋鏇顧構鹽獵夢觸襯窪) = 獵窪齋築觸夢積窪夢淵獵襯願齋齋積壓簾窪願 (願遞觸觸鹹繭膚襯選遞 ) 更多	-	2023-12-09