Background:
Multiple myeloma (MM) is a type of blood cancer that affects a person s immunity. MM returns after treatment (relapse) in almost all people; MM may also not respond to initial treatment (refractory). Many people with relapsed refractory MM (RRMM) also have changes in their KRAS and NRAS genes. Researchers want to try a new drug treatment that targets cancer with these changed genes.
Objective:
To test 2 drugs (mirdametinib and sirolimus) in people with RRMM.
Eligibility:
People aged 18 and older with RRMM who have changes in their KRAS or NRAS genes.
Design:
Participants will be screened. They will have blood tests and imaging scans. They will have an eye exam and a test of their heart function. They will need to provide proof of their disease status and of their KRAS or NRAS status. If neither is available, the tests will be repeated.
Participants will have a bone marrow biopsy: A needle will be inserted into a hipbone to draw out some soft tissue.
This study will be done in two parts. In the first part of this study, we will find a safe dose of mirdametinib combined with sirolimus. In the second part, we will learn more about how mirdametinib combined with sirolimus may work against RRMM.
Mirdametinib (capsules) and sirolimus (tablets) are taken by mouth. Participants will take both drugs at home on a 4-week cycle. They will take mirdametinib twice a day for the first 3 weeks of each cycle. They will take sirolimus once a day, every day, during each cycle.
Participants will have study visits once a week during the first cycle, and then on the first day of subsequent cycles. Blood, heart, imaging scans, and other tests will be repeated.
Treatment with the study drugs will go on for 1 year. Then participants will have follow-up visits every 3 months for 4 more years.

开始日期2026-08-27

申办/合作机构

National Cancer Institute

CTIS2026-526403-30-00

/ Not yet recruiting临床1期

A Study to Assess the Effects of Probenecid on the Pharmacokinetics of Mirdametinib and its Metabolites in Healthy Participants

开始日期2026-08-03

申办/合作机构

Merck Healthcare KGaA

NCT07521657

/ Not yet recruiting临床2期IIT

Efficacy of Mirdametinib Alone or Combination With Radiotherapy for Germline and Sporadic NF1-Altered High-Grade Glioma

This is a phase 2, open label, parallel multi-arm study of mirdametinib in combination with radiation in participants with recurrent sporadic glioblastoma (GBM) harboring NF1 alterations (Cohort 1); participants with NF1 with a newly diagnosed GBM (Cohort 2); mirdametinib alone in other NF1-associated High-Grade Gliomas (Cohort 3).

开始日期2026-08-01

申办/合作机构

The University of Alabama at Birmingham

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100 项与米达美替尼相关的临床结果

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100 项与米达美替尼相关的转化医学

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100 项与米达美替尼相关的专利（医药）

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903

项与米达美替尼相关的文献（医药）

2026-07-02·MOLECULAR CANCER THERAPEUTICS

Chromobox 2 Inhibition: A Novel Activity of Alisertib, an Aurora A Kinase Inhibitor

Article

作者: Nurmemmedov, Elmar ; Bitler, Benjamin G. ; Elder, Alan M. ; Petkov, Alexander ; Reigan, Philip ; Woodruff, Elizabeth R. ; Brubaker, Lindsay W. ; Eisenmesser, Elan ; Yamamoto, Tomomi M. ; Iwanaga, Ritsuko

Abstract:

Chromobox 2 (CBX2), a subunit of polycomb repressor complex 1 (PRC1), is expressed in high-grade serous carcinoma (HGSC). CBX2-inhibitory peptide (CBX2i) has demonstrated efficacy in a syngeneic mouse model but has limitations. We sought to identify an alternative approach to CBX2 inhibition. A computational-based molecular docking screen was performed using the Selleckchem Bioactive Library to identify inhibitors of CBX2. A similarity screen of top hits against the bound conformation of CBX2i pharmacophore model was performed in parallel. A series of in vitro validation studies evaluated the effect of alisertib on proliferation, a CBX2 target gene, and stemness. CBX2-knockdown cell lines and a syngeneic murine model were utilized to evaluate alisertib response in the context of CBX2 loss. Cell target engagement assay was performed. PRC1 activity was measured by H2AK119ub levels. Immune profiling of treated tumors defined the immune microenvironment. The computational-based screen identified 10 candidate compounds. In vitro validation narrowed compounds of interest to raltitrexed, alisertib, GTX-007, LY315920, and PD0325901. Ultralow dilution assay demonstrated a dramatic decrease in spheroid formation with alisertib, an aurora A kinase (AURKA) inhibitor. Good structural overlap was observed between CBX2i and alisertib. Cell target engagement assay confirmed alisertib selectivity for both AURKA and CBX2. Loss of CBX2 attenuated alisertib efficacy in vitro and in vivo. Treatment with alisertib leads to a decrease in H2AK119ub and shift in the immune tumor microenvironment. Alisertib efficacy in HGSC is dependent on functional CBX2, and cell target engagement confirms selectivity for CBX2, supporting that alisertib activity involves CBX2 inhibition.

2026-06-01·JOURNAL OF CELL BIOLOGY

Bistable Otx2–Id1 circuitry governs a developmental transition state in the pluripotency continuum

Article

作者: Ye, Shou-Dong ; Dong, Baoxing ; Ban, Qian ; Chen, Peng ; Ji, Junxiang ; Zhu, Zhenhua ; Wu, Lefan ; Liu, Wenqiang ; Zhu, Yuqing ; Duan, Junbo

While current pluripotency models capture discrete embryonic stages, they inadequately resolve transitional states during peri-implantation development. Here, we establish rosette-formative intermediate stem cells (rfISCs) from mouse embryonic stem cells using the MEK inhibitor PD0325901, the Wnt inhibitor IWR1, and the PKA activator Forskolin. These cells exhibit transcriptomic/epigenetic profiles mirroring those of E5.0‒5.5 epiblasts, bridging rosette-stage and formative pluripotency. rfISCs demonstrate developmental bipotency, retaining in vitro germline differentiation capacity while generating germline-competent chimeras in vivo. Mechanistically, we identified opposing signaling axes that govern rfISC identification through the regulation of lineage priming: IWR1 stabilizes Tcf7l1 to drive Otx2-mediated rfISC specification and neural priming, whereas Forskolin activates PKA to induce Id1-dependent neural suppression. This creates a bistable regulatory circuit in which Otx2/Id1 synergy maintains pluripotency plasticity under MEK inhibition. Notably, rfISCs can be directly derived from E5.25 epiblasts, confirming their physiological relevance. Our work bridges a fundamental gap in pluripotency modeling by capturing the RSC-to-FSC transition through dynamic signaling equilibria.

2026-06-01·Journal of Thoracic Disease

Comprehensive analysis of POLD2 as a potential biomarker for modulating the immune microenvironment and predicting immunotherapy response in lung adenocarcinoma

Article

作者: Hu, Shaoqing ; Guo, Zhiqin ; Wu, Wanxin ; Wei, Leiyan ; Zhang, Lin ; Chen, Xiangyi ; Zhang, Hong ; Zhu, Wu

Background:

Lung adenocarcinoma (LUAD) is the leading cause of global cancer mortality. Although significant progress has been made in treatment modalities, including surgery, radiation, chemotherapy, targeted agents and immunotherapies, clinical outcomes for patients continue to be unfavorable. Key obstacles include disease recurrence, metastasis and the development of treatment resistance. This study aimed to explore molecular mechanisms driving LUAD progression and identify novel prognostic biomarkers and therapeutic targets.

Methods:

Gene expression data for LUAD were retrieved from The Cancer Genome Atlas (TCGA) and the Genotype-Tissue Expression (GTEx) databases to conduct a preliminary investigation. The differential expression of POLD2 was validated by analyzing gene expression datasets from the Gene Expression Omnibus (GEO). Clinical patient data with survival information from the TCGA and GEO databases were employed to investigate and validate the prognostic significance of POLD2. The association of POLD2 with the tumor immune microenvironment (TIME) was examined through RNA transcript-based single-sample gene set enrichment analysis (ssGSEA) and ESTIMATE algorithm, which quantifies stromal and immune cell components in malignant tumor tissues based on expression data. The Tumor Immune Dysfunction and Exclusion (TIDE) model was used to evaluate the potential therapeutic response of POLD2 to immune checkpoint inhibitors (ICIs). Molecular docking and molecular dynamics (MD) simulations were used to investigate the binding interactions between POLD2 and specific therapeutic agents. In vitro functional assays were performed to validate the role of POLD2 in the progression of LUAD.

Results:

Our study demonstrated that POLD2 is markedly upregulated in LUAD, which is associated with unfavorable patient outcomes and more advanced disease stages. Functional analyses revealed that POLD2 is critically involved in essential biological process (BP), including DNA repair, cell cycle control, and epithelial-mesenchymal transition (EMT), all of which are key contributors to tumor progression. Furthermore, immunological investigations, the core focus of this study, indicated that POLD2 is linked to an immunosuppressive microenvironment, a factor that may impair the efficacy of ICIs and thus compromise immunotherapy response. As an extended exploratory analysis to further explore POLD2's therapeutic potential, molecular docking and MD simulations revealed strong binding interactions between POLD2 and MEK inhibitors, including PD0325901, selumetinib and trametinib. In vitro studies showed that suppressing POLD2 expression significantly decreased the proliferation, colony-forming ability and migratory capacity of A549 LUAD cells.

Conclusions:

Our results identify POLD2 as a promising prognostic biomarker and a potential therapeutic target in LUAD, with particular relevance to predicting immunotherapy response-consistent with the core focus of this study. This finding not only provides important insights into the mechanisms underlying LUAD progression and treatment resistance but also lays a preliminary foundation for subsequent research on both immunotherapeutic optimization and targeted therapy (via MEK inhibitors) for LUAD.

241

项与米达美替尼相关的新闻（医药）

2026-08-12

·赛柏蓝

全球药企TOP20出炉，礼来、艾伯维、强生、阿斯利康……

作者 | 草履虫编辑 | 郑瑶随着2026年上半年跨国药企（MNC）财报季基本落幕，全球制药企业TOP20榜单也随之出炉。从排名来看，礼来以427.73亿美元的制药业务营收位居榜首，同时以49%的同比增速领跑TOP20，展现出强劲的增长势头。艾伯维、强生、阿斯利康、罗氏、默沙东和辉瑞等传统制药巨头仍稳居第一梯队。与此同时，拜耳则基本与去年同期持平，同比增长0.2%（表1）。值得关注的是，头部药企之间的增长分化正进一步加剧。礼来、诺和诺德分别实现49%和18%的同比增长，以GLP-1药物为代表的代谢疾病赛道继续成为全球制药行业最重要的增长引擎之一；诺华和德国默克则成为TOP20中仅有的两家制药业务收入同比下滑的企业，降幅分别为2.0%和0.3%，整体业绩承压明显。从高速增长到逆势下滑，不同药企在产品周期和核心品种表现上的差异，正在越来越直接地反映到半年报成绩上。表1.2026上半年全球TOP20制药企业营收排名数据来源：各公司财报整理 01礼来营收增速双领先前三格局初现礼来2026上半年总营收427.73亿美元，同比增长49%。制药业务收入和增速均位列头部MNC第一，与第二名艾伯维拉开108亿美元差距。其中，中国市场收入16.35亿美元，同比激增73%，增速同样位居已披露企业之首（表2）。上半年，礼来旗下八款核心产品合计收入290.77亿美元，占总营收68%。其中替尔泊肽（Mounjaro和Zepbound）收入276.93亿美元（+88%），与竞争对手司美格鲁肽（175亿美元）拉开102亿美元差距，坐稳全球药王宝座。此外，其口服小分子GLP-1药物奥氟格列隆（Orforglipron，Foundayo）上市首个季度即实现9800万美元销售额。后续第三款GLP-1产品Retatrutide也已完成5项Ⅲ期临床，预计明年一季度递交上市申请，有望进一步巩固其在代谢领域的竞争优势。基于上半年的强劲表现，礼来再次上调全年营收预期至850亿—870亿美元。艾伯维上半年总营收319.92亿美元，同比增长11.2%，位居制药业务营收榜第二名。其中免疫学业务收入160.76亿美元，同比增长15.7%，占公司总营收比例达到45.7%。艾伯维在免疫学领域拥有三款重磅产品，分别是阿达木单抗（Humira）、利生奇珠单抗（Skyrizi）和乌帕替尼（Rinvoq）。其中，作为曾经全球“药王”的Humira，受2023年专利到期及生物类似药竞争影响，销售额持续承压（图1）。今年上半年收入进一步下滑至14.44亿美元，同比下降37.2%。图1.艾伯维3款自免产品销售额变化情况图片来源：艾伯维官网数据整理 Skyrizi和Rinvoq正成为Humira之后艾伯维新的增长支柱。Skyrizi凭借多项适应症的快速拓展实现持续放量，2025年销售额高达175.62亿美元，同比增长49.87%（图1）；2026年上半年Skyrizi进一步实现99.88亿美元营收，同比增长27.3%，与Rinvoq合计销售额达到146.32亿美元，占免疫业务收入91%，占公司总营收46%。在Skyrizi和Rinvoq的强力驱动下，艾伯维成功完成“后修美乐时代”的战略切换（图1），实现了由单一超级产品向多元化免疫平台的平稳过渡。强生上半年总营收493.72亿美元（+8.2%）。其中，创新制药收入318.10亿美元（+9.4%），位居全球制药业务营收榜第三名。强生的创新制药业务主要聚焦肿瘤、自免、神经、肺动脉高压、感染、心血管及代谢等六大板块，肿瘤业务的增长主要由达雷妥尤单抗（Darzalex）、阿帕他胺（Erleada）和西达基奥仑赛（Carvykti）等产品强劲驱动。其中，达雷妥尤单抗（Darzalex）收入81.71亿美元（+20.6%），西达基奥仑赛（Carvykti）收入也强劲增长55.1%至12.54亿美元（2025H1为8.08亿美元）。值得注意的是，强生的3款双抗产品开始发力，特立妥单抗（Tecvayli）、塔奎妥单抗（Talvey）、埃万妥单抗（Rybrevant）合计收入13.34亿美元（2025H1为8.29亿美元），其中后两款产品增速高达70%。自免业务，古塞奇尤单抗（Tremfya）保持涨势，上半年收入36.54亿美元（+70.6%）。神经科学领域，抑郁症药物艾司氯胺酮鼻喷雾剂（Spravato）表现亮眼，上半年销售额达到10.52亿美元（+43.2%）。从半年报成绩来看，强生正在稳步接近年初定下的千亿美元营收预期目标。并且，强生也基于上半年收入进一步上调了全年营收预期，由1003亿—1013亿美元调整至1008亿—1014亿美元。 02赛诺菲、BI、再生元跑出“加速度” 今年上半年，勃林格殷格翰（BI）、再生元和赛诺菲的制药业务均保持较快增长，增幅分别达到20.1%、17.8%和15.7%，明显高于行业整体水平。赛诺菲2026年上半年实现总营收257.42亿美元，同比增长15.7%。其中，度普利尤单抗（Dupixent）仍是公司最核心的增长引擎。作为全球最畅销的自免药物，度普利尤单抗上半年销售额约108.83亿美元，同比增长34.4%，成为继替尔泊肽、司美格鲁肽和K药之后，全球又一款半年度销售额迈入“百亿美元俱乐部”的超级大单品。随着其在特应性皮炎、哮喘、慢阻肺等2型炎症疾病中的持续渗透，赛诺菲预计，到2030年度普利尤单抗销售额有望达到约250亿欧元（约290亿美元），进一步向平台型超级单品迈进。 BI上半年总营收达到183.49亿美元，同比增长16.2%；其中，人用药品业务收入153.1亿美元，同比增长20.1%。恩格列净（Jardiance）一款产品便贡献了66亿美元收入，是公司当前最主要的增长动力。不过，BI并非只依赖一款成熟产品。2025年获批的肺纤维化药物那米司特（Jascayd）和HER2突变非小细胞肺癌药物宗艾替尼（Hernexeos）已经进入商业化阶段。与此同时，GLP-1R/GCGR双激动剂Survodutide已进入Ⅲ期并取得积极数据，公司还启动了三项肿瘤Ⅲ期研究。从半年报表现来看，BI今年总营收有望突破300亿欧元（约346亿美元），恩格列净销售额也有望超过100亿欧元（约115亿美元，不包括礼来收入）。再生元上半年同样保持较快增长，实现营收78.96亿美元，同比增长17.8%。其中，与赛诺菲合作相关收入（主要来自Dupixent共同商业化）达到43.55亿美元，同比增长28%，成为业绩增长的重要驱动力。产品端，眼科业务正加速向高剂量产品切换，阿柏西普（EYLEA HD）上半年全球销售额达到17.64亿美元，同比增长62%，其中美国市场销售额为10.65亿美元。与此同时，传统阿柏西普眼内注射溶液（EYLEA）销售快速下滑，使EYLEA系列合计销售额降至33.45亿美元，同比下降17%。肿瘤产品西米普利单抗（Libtayo）上半年销售额达到9.28亿美元，同比增长40%，已成为继度普利尤单抗和阿柏西普之后，再生元最重要的增长品种之一。整体来看，再生元的增长结构正在发生变化，商业化动力逐步从传统眼科单一支柱转向免疫、眼科和肿瘤业务共同驱动。 03诺华、德国默克逆势承压上半年TOP20企业制药业务合计营收达4385.26亿美元，其中18家实现同比增长，行业整体延续稳健扩张态势（表1）。相比之下，诺华和德国默克成为TOP20中仅有的两家制药业务收入同比下滑的企业，降幅分别为2.0%和0.3%。这两家公司均处于产品更替阶段，部分成熟品种受到专利到期和市场竞争加剧影响。与同期依靠重磅创新药快速放量实现增长的MNC相比，其医药板块整体表现偏弱，短期业绩压力较为明显。 7月21日，诺华发布2026年上半年财报，总营收达275.21亿美元，同比下降2%；核心营业收入为108.37亿美元，同比下降7%。其中，第二季度净销售额为144.08亿美元，同比增长1%，净利润为32.57亿美元，同比下降19%，整体业绩仍明显承压。专利悬崖是拖累诺华业绩的重要因素。上半年，沙库巴曲缬沙坦（Entresto）、艾曲泊帕（Promacta）和Tasigna三款成熟产品明显失速，销售额合计减少超过30亿美元。不过，诺华并非缺少接棒产品。瑞波西利（Kisqali，32.11亿美元，+48%）、奥法妥木单抗（Kesimpta，25.88亿美元，+29%）、阿思尼布（Scemblix，9.95亿美元，+85%）、镥[177Lu]-特昔维匹肽（Pluvicto，12.93亿美元，+55%）、英克司兰（Leqvio，9.32亿美元，+64%）等新产品仍保持高速增长，只是现阶段尚不足以完全填补成熟品种下滑留下的缺口。表2. 2026年上半年 MNC中国区制药收入排名数据来源：各公司财报整理值得注意的是，诺华在中国市场的表现相对突出。上半年，诺华中国区销售额约25亿美元，同比增长7%（表2），是除礼来外中国区业绩唯一保持正增长的头部企业，这一表现主要得益于多款创新产品持续放量。 2026年1月，英克司兰正式进入国家医保；第二季度，该产品全球销售额达到4.80亿美元，同比增长59%。与此同时，瑞波西利在中国早期乳腺癌新增处方市场的份额达到46%；口服慢性自发性荨麻疹药物瑞米布替尼（Rhapsido）已在中国治疗超过3000名患者；镥[177Lu]-特昔维匹肽（Pluvicto）也在持续推进中国市场准入。随着上述产品逐步放量，新产品正成为支撑诺华中国区后续增长的重要力量。与诺华类似，德国默克医药业务也处于产品换挡期。2026H1，德国默克实现营收120.39亿美元，同比增长0.3%，但核心医药Healthcare板块明显承压，上半年收入47.88亿美元，同比下降0.3%。产品销售方面，曾经的增长支柱——多发性硬化症（MS）药物克拉屈滨（Mavenclad）受到美国仿制药竞争冲击，上半年销售额降至6.11亿美元，同比下降10.7%；PD-L1抗体阿维鲁单抗（Bavencio）销售额为3.00亿美元，同比下降17.5%；老牌MS药物重组干扰素β-1α（Rebif）销售额下降15.2%；抗肿瘤西妥昔单抗（Erbitux）则基本持平于6.81亿美元。相比之下，促排卵重组复方制剂注射用人促卵泡激素α、促黄体激素α（Pergoveris）销售额增长14.8%，收购SpringWorks获得的尼罗司他（Ogsiveo）和米达美替尼（Gomekli）也分别贡献1.59亿美元和0.80亿美元收入，成为德国默克医药板块为数不多的增量来源。不过，这些产品目前体量仍相对有限，尚不足以完全抵消核心成熟品种下滑带来的影响，德国默克医药业务的产品接续压力仍然存在。 04结语随着2026年上半年头部MNC财报陆续披露，全球制药行业的增长版图也愈发清晰。过去由肿瘤药长期占据主导的市场格局，正在被GLP-1、自免等赛道快速改写，替尔泊肽、度普利尤单抗等超级单品对药企业绩的拉动愈发显著。可以预见，未来几年，围绕这些高景气赛道和重磅品种的竞争仍将继续，并成为影响全球药企座次变化的重要变量。参考资料：各企业官微、官网等本期素材组图片来源：https://unsplash.com/s/photos/ END

2026-08-11

·ClinTrials Talk+

2025年全年及2026年上半年中美创新药获批上市全景分析

2025年至2026年上半年，全球创新药领域经历了深刻的格局重塑。中国在创新药批准数量上实现历史性跨越，首次在年度批准数量上超越美国；与此同时，美国FDA在保持稳定审批节奏的基础上，2026年上半年呈现显著反弹态势。本文基于官方数据与行业报告，对两国创新药获批情况进行客观梳理与对比分析。一、中国：数量创新高，质量跨越式提升（一）2025年全年：历史性突破2025年是中国创新药审评审批的里程碑之年。国家药品监督管理局（NMPA）全年批准1类创新药76个品种，较2024年的48个增长58.3%，创历史新高。若纳入更广泛的新药统计口径（含进口新药等），全年共有108款新药首次获批上市，其中国产新药59款、进口新药49款，国产新药数量首次超过进口。从药物类型来看，76款创新药中化学药品47个、生物制品23个（含10个抗体药物、4个重组蛋白、3个细胞基因疗法、3个抗体偶联药物、1个基因疗法、1个多肽）及6个中成药。多元化创新疗法在中国加速上市的趋势十分明显。从治疗领域来看，抗肿瘤药物仍是绝对主力，全年共34款抗肿瘤创新药获批，占比达44.74%。其中乳腺癌新药最多（9款），非小细胞肺癌（8款）紧随其后。获批药物涵盖小分子、单抗、ADC（抗体偶联药物）、CAR-T细胞疗法等多种技术路线。值得关注的是源头创新能力的提升。2025年获批的76个创新药中，有11个属于新靶点、新机制药物，其中国产仅占4个。虽然国产占比尚不足一半，但已标志着中国创新药正从“跟跑”向“并跑”迈进。其他亮点包括：· 4款国产流感创新药相继获批 · 3款GLP-1类产品获批，含全球首个GCG/GLP-1双受体激动剂玛仕度肽 · 罕见病领域迎来多个突破，含3款重症肌无力新药、3款血友病新药 · 17款药物获得突破性疗法认定（二）2026年上半年：势头不减，国产“真创新”崛起2026年上半年，中国创新药审批延续高增长态势。国家药监局累计批准38个1类创新药上市，包括20个化学药品、17个生物制品和1个中药。另有更宽口径统计显示，上半年累计批准首次上市的创新药达55款（含1类新药36款、进口创新药25款）。最大的质变在于源头创新：2026年上半年获批的38个1类创新药中，有11个新靶点、新机制药物，且全部为国产药品。对比2025年全年11个新靶点新药仅4个国产的数据，本土源头创新质量实现了跨越式提升。这11款国产新靶点新药覆盖抗肿瘤、抗感染、医学影像诊断、内分泌、血液系统疾病等领域，其中包括全球首个双特异性抗体ADC药物、全球首个治疗实体瘤的CAR-T药物、全球首创靶向乙肝和丁肝的单克隆抗体等。从治疗领域来看，抗肿瘤药物4个、抗感染药物2个、医学影像学药物2个、内分泌系统药物1个、外科及其他药物1个、血液系统疾病药物1个。国际化方面，中国创新药出海势头强劲。2026年上半年，中国创新药完成81笔对外授权交易，合同潜在总额约1100亿美元，约为2025年全年的80%。多款国产创新药实现海外获批：2025年4月康方生物派安普利单抗在美国获批鼻咽癌适应症；2025年7月迪哲医药舒沃替尼获FDA加速批准；2026年5月海思科环泊酚获FDA批准用于全身麻醉诱导。二、美国FDA：稳定输出，2026年上半年显著反弹（一）2025年全年：46款新药获批2025年，美国FDA药物评价与研究中心（CDER）批准了46款“novel drugs” （此前从未在美国获批或上市的新药），其中包括34个新分子实体（NME）和12个生物制品。若计入4种细胞与基因治疗产品，全年共批准49种新医疗疗法。从审批节奏来看，2025年上半年FDA仅批准16款新药，处于历史低位。全年46款的成绩虽延续了每年约50项新批准的稳定态势，但上半年审批速度明显偏慢。2025年获批的代表性药物包括：肿瘤领域：· Datroway（datopotamab deruxtecan-dlnk）：TROP2靶向ADC，用于乳腺癌治疗· Grafapex（treosulfan）：用于造血干细胞移植前的预处理· Romvimza（vimseltinib）：用于腱鞘巨细胞瘤· Imlunestrant：口服选择性雌激素受体降解剂，用于乳腺癌疼痛与神经领域：· Journavx（suzetrigine）：非阿片类疼痛治疗药物遗传病与罕见病：· Gomekli（mirdametinib）：用于1型神经纤维瘤病相关丛状神经纤维瘤21款药物获得优先审评资格，体现了FDA对创新疗法的政策倾斜。（二）2026年上半年：强势反弹，24款创新药获批2026年上半年，FDA累计批准24款创新药，其中新分子实体（NME）19个、生物创新药5个，First-in-class（全球首创）新药达9个。相比2025年同期的16款，同比增长50%。另有统计口径显示上半年共批准26种创新药物。四大趋势尤为突出：1. “首个”标签密集出现。上半年10款药物为各自适应症的首个获批疗法，占比高达42%。代表性药物包括：· Veppanu（vepdegestrant）：全球首个获批的口服雌激素受体（ER）蛋白降解剂（PROTAC），用于ER+/HER2-、ESR1突变的晚期或转移性乳腺癌。临床数据显示，该药将中位无进展生存期从氟维司群的2.1个月延长至5.0个月。· Zycubo（copper histidinate）：Menkes病（一种因铜吸收障碍导致的致命性神经退行性疾病）的首个治疗药物。· Foundayo（orforglipron）：首个口服GLP-1药物。· Otarmeni（lunsotogene parvec-cwha）：首个遗传性耳聋基因疗法。2. 快速审批通道常态化。 Foundayo（50天）和Otarmeni（61天）在CNPV试点项目下极速获批。3. 口服替代注射浪潮加速。多款口服剂型药物获批，回应患者对给药便利性的诉求。4. 中国创新药登上全球舞台。海思科环泊酚成为中国首个获得FDA批准的自主研发麻醉药。获批当日，海思科还与礼来达成最高30.54亿美元的战略合作。三、中美对比与趋势分析（一）数量对比- 2025年全年：中国（1类创新药） 76款，美国（Novel Drugs）46款- 2026年上半年：中国（1类创新药）38款，美国（Novel Drugs）24款中国在年度创新药批准数量上已超越美国。2020至2025年间，中国批准的新型抗癌药数量（94款）也已超过美国（87款）。（二）质量对比· 新靶点/新机制药物：2025年中国11个（国产4个），美国2025年First-in-class数量未有明确官方统计，但46款中21款获优先审评。 · 2026年上半年：中国11个新靶点新药全部为国产；美国9个First-in-class。 · 中国在源头创新上正快速追赶，从2025年新靶点新药国产仅占36%到2026年上半年100%国产，进步显著。（三）治疗领域两国均以抗肿瘤药物为首要领域。中国2025年抗肿瘤药占比44.74%；美国肿瘤领域同样占据最大批准份额。在细分方向上，中国在乳腺癌、非小细胞肺癌领域布局密集，美国则在罕见病、首创机制药物上更具优势。（四）国际化· 中国创新药海外获批数量持续增加 · 对外授权交易额爆发式增长：2025年1356.55亿美元，2026年上半年已达997亿美元 · “反向出海” 趋势明确——国产创新药先在中国获批、再登陆美国市场（五）研发管线截至2025年底，中国在研创新药数量已超越美国，跃居全球首位。2025年全球新启动临床试验5226项，中国内地新开展创新药临床试验2502项，为同期美国的1.5倍。美国研发管线的新化合物总量约1/3来自中国。结语2025年至2026年上半年，全球创新药格局正在发生深刻变化。中国在获批数量上已实现对美国的超越，在源头创新质量上正快速追赶，在对外授权和国际化方面势头强劲。美国FDA则在保持稳定审批节奏的基础上，2026年上半年呈现显著反弹，尤其是在First-in-class药物和快速审评通道方面持续发力。两国创新药审评审批的竞争与合作，最终受益的是全球患者——更多创新疗法以更快速度惠及临床，这无疑是生物医药产业发展的根本价值所在。免责声明本文数据来源于国家药品监督管理局（NMPA）、美国食品药品监督管理局（FDA）官方公开信息，以及Insight数据库、医药魔方NextPharma数据库、界面新闻、新浪财经等第三方行业报道。笔者对数据的准确性已尽合理核实义务，但不保证所有数据的绝对精确。本文内容仅供参考与学术交流，不构成任何投资建议或医疗建议。文中提及的具体药物及企业仅为客观数据呈现，不代表任何推荐或评价。读者据此操作或决策，风险自担。

2026-08-06

·Firstwordpharma

Boosted by new rare disease pillar, Merck KGaA lifts sales outlook for the year

Merck KGaA raised its sales forecast for 2026 after second-quarter results showed its still-young rare diseases business, set up as a new strategic pillar of its Healthcare segment, is already pulling its weight.The company now expects full-year sales of €21 billion to €21.8 billion — up from its earlier guidance of €20.4 billion to €21.4 billion — with organic growth projected at 1% to 3%.Sales in the Healthcare segment reached €2.2 billion in the quarter, representing reported growth of 2.4%, but an organic decline of 3.4% as certain established products lost ground. However, that was partially offset by portfolio contributions from the $3.9-billion acquisition of SpringWorks Therapeutics a year ago.Rare disease medicines contributed €115 million in quarterly sales. The franchise includes Ogsiveo, a treatment for progressing desmoid tumors, and Gomekli, which is approved for neurofibromatosis type 1-associated plexiform neurofibromas. The products, both added through the SpringWorks takeout, generated sales of €75 million and €39 million, respectivelyPimicotinib, an oral CSF-1R inhibitor for tenosynovial giant cell tumour, generated low single-digit million-euro sales in the quarter following its approval in China last December. Merck exercised its option on the drug last year from Abbisko Therapeutics for $85 million.Within Healthcare's specialty care portfolio, Erbitux provided a bright spot, with sales increasing 5.3% to €303 million despite ongoing competition in China, thanks to stronger demand in Europe and Latin America and a recent EU label extension in colorectal cancer.However, Mavenclad sales (-10.3%, €275 million) were down amid generic competition in the US, while sales of Bavencio (-18.3%, €130 million) and Rebif (-10.2%, €107 million) also saw double-digit declines.Tariff refundsRevenues in the Life Science segment increased by 6.4% to € 2.4 billion. Since taking over from Belén Garijo in May, new CEO Kai Beckmann has moved quickly on the dealmaking front. In June, he brokered the company's largest deal in over a decade, agreeing to buy life science firm Bio-Techne for about $11.3 billion.Merck also raised its full-year outlook for both its Healthcare and Life Science businesses. Healthcare is now expected to earn sales of about €8.4 billion to €8.7 billion despite an anticipated 2%-4% organic decline year-over-year, while Life Science sales are forecast at €9.3 billion to €9.6 billion, representing 5%-7% organic growth.The Life Science guidance includes a potential headwind of around €40 million from tariff refunds to customers.€1.00 = $1.15250866

上市批准并购生物类似药

100 项与米达美替尼相关的药物交易

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外链

KEGG	Wiki	ATC	Drug Bank
D11675	-	-	DB07101

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
NF1突变型丛状神经纤维瘤	欧盟	SpringWorks Therapeutics Ireland Ltd.	2025-07-18
NF1突变型丛状神经纤维瘤	冰岛	SpringWorks Therapeutics Ireland Ltd.	2025-07-18
NF1突变型丛状神经纤维瘤	列支敦士登	SpringWorks Therapeutics Ireland Ltd.	2025-07-18
NF1突变型丛状神经纤维瘤	挪威	SpringWorks Therapeutics Ireland Ltd.	2025-07-18
丛状神经纤维瘤	欧盟	-	2025-07-17
丛状神经纤维瘤	冰岛	-	2025-07-17
丛状神经纤维瘤	列支敦士登	-	2025-07-17
丛状神经纤维瘤	挪威	-	2025-07-17
神经纤维瘤病1型	美国	Springworks Therapeutics, Inc.	2025-02-11

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
肿瘤	申请上市	加拿大	EMD Serono, Inc.	2026-06-01
胶质瘤	临床2期	美国	Springworks Therapeutics, Inc.	2026-04-10
去分化脂肪肉瘤	临床2期	美国	Springworks Therapeutics, Inc.	2025-02-19
粘液样脂肪肉瘤	临床2期	美国	Springworks Therapeutics, Inc.	2025-02-19
晚期恶性实体瘤	临床2期	美国	Springworks Therapeutics, Inc.	2023-02-03
晚期恶性实体瘤	临床2期	澳大利亚	Springworks Therapeutics, Inc.	2023-02-03
低级神经胶质瘤	临床2期	美国	Springworks Therapeutics, Inc.	2021-06-21
结直肠癌	临床2期	荷兰	Pfizer Inc.	2014-04-02
KRAS突变非小细胞肺癌	临床2期	荷兰	Pfizer Inc.	2014-04-02
晚期非小细胞肺癌	临床2期	美国	Pfizer Inc.	2005-11-01

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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


NCT03962543 (RENEU, EANO2025)	临床2期	NF1突变型丛状神经纤维瘤	114	Mirdametinib (adults)	衊構醖淵鑰製窪夢築齋(鏇夢顧獵製簾餘鹹糧觸) = 窪醖願鑰齋壓繭構淵壓夢獵顧窪壓鬱顧願艱襯 (憲壓衊範窪繭製衊鹽醖, -90 ~ 13) 更多	积极	2025-10-16
				Mirdametinib (children)	衊構醖淵鑰製窪夢築齋(鏇夢顧獵製簾餘鹹糧觸) = 衊衊壓糧鹹製構製鹽鬱夢獵顧窪壓鬱顧願艱襯 (憲壓衊範窪繭製衊鹽醖, -98 ~ 48) 更多
NCT03962543 (CTNI-21 \| RENEU, CTgov)	临床2期	神经纤维瘤病1型 \| 丛状神经纤维瘤	114	Mirdametinib (PD-0325901) (Treatment Phase - Pediatric Cohort Mirdametinib (PD-0325901))	範壓繭構鹽鑰願蓋觸餘 = 齋壓糧築製範鬱願蓋艱蓋築繭壓餘顧襯遞膚鏇 (築獵範夢壓獵鏇構醖網, 夢鹹齋餘鹹夢範膚構蓋 ~ 範鬱繭顧鬱夢積衊鑰築) 更多	-	2025-08-07
				Mirdametinib (PD-0325901) (Treatment Phase - Adult Cohort Mirdametinib (PD-0325901))	範壓繭構鹽鑰願蓋觸餘 = 壓醖壓衊積衊壓窪襯糧蓋築繭壓餘顧襯遞膚鏇 (築獵範夢壓獵鏇構醖網, 壓鏇衊獵網壓繭憲鏇選 ~ 淵鏇鹹願選鬱積築壓艱) 更多
NCT03962543 (ReNeu, AAN2025) 人工标引	临床2期	NF1突变型丛状神经纤维瘤	114	Mirdametinib (adults)	網遞願遞遞鹹獵積選構(鑰範繭鏇製齋選壓製簾) = 範襯積膚艱膚簾鹽築淵觸艱餘廠製鹽艱顧夢觸 (襯遞鏇遞範獵餘蓋鑰鹹, 29 ~ 55) 更多	积极	2025-04-07
				Mirdametinib (children)	網遞願遞遞鹹獵積選構(鑰範繭鏇製齋選壓製簾) = 醖製簾製蓋築鬱醖壓鹽觸艱餘廠製鹽艱顧夢觸 (襯遞鏇遞範獵餘蓋鑰鹹, 38 ~ 65) 更多
NCT04923126 (SJ901, SNO2024) 人工标引	临床1期	低级神经胶质瘤 BRAF alteration \| FGFR1 alteration \| NF1 alteration ... 更多	23	MIRDAMETINIB	壓簾夢製壓廠餘齋鏇構(築獵壓鹹齋鹹餘獵顧餘) = 選糧衊蓋選襯範齋鬱獵顧窪積窪衊簾製衊廠鬱 (構遞衊糧築蓋廠憲簾築 ) 更多	积极	2024-11-11
NCT03962543 (ReNeu, SNO2024 \| FDA_CDER) 人工标引	临床2期	神经纤维瘤病1型	114	mirdametinib (adults)	夢鹽鑰鹹廠觸餘鹹選繭(構觸糧蓋獵鹹鬱願製齋) = 顧網夢顧簾築願艱獵範膚網齋鏇淵築壓壓憲淵 (壓鑰壓觸獵膚襯鹽鬱鏇, 29 ~ 55) 更多	积极	2024-11-11
				mirdametinib (children)	夢鹽鑰鹹廠觸餘鹹選繭(構觸糧蓋獵鹹鬱願製齋) = 網選窪膚壓遞遞繭壓壓膚網齋鏇淵築壓壓憲淵 (壓鑰壓觸獵膚襯鹽鬱鏇, 38 ~ 65) 更多
NCT03962543 (ReNeu, SNO2024) 人工标引	临床2期	NF1突变型丛状神经纤维瘤	114	MIRDAMETINIB (adults)	夢鑰願簾憲膚醖衊獵選(鏇醖顧選夢膚獵夢醖觸) = Clinically meaningful improvement was defined as change from baseline >9.7 points for adults, >8.5 points for child self-report, and >9.0 points for parent proxy-report. PedsQL-TS improvement (least-squares mean, LSM [SE] change) from baseline at C13 was 3.9 (1.6; P=.018; n=34) for adults, 4.0 (2.4; P=.096; n=38) for children by self-report, and 5.6 (1.9; P=.005; n=43) by parent proxy-report. PedsQL-TS improvements for adults and children by parent proxy-report (not by children self-report) were observed early (at C5 and C3, respectively) and sustained through C24. Clinically meaningful improvement in PedsQL-TS from baseline at C13 was achieved by 37% (10/27) adults, 45% (13/29) children by self-report, and 47% (15/32) children by parent proxy-report (among patients who could have achieved a clinically meaningful change from baseline). Significant (P<.05) improvement from baseline to C13 was reported for physical (adults, children, and parent proxy-report), school/work (adults), and emotional and social (parent proxy-report) subscales. 膚繭網繭鬱醖廠窪醖選 (衊選網獵壓糧憲淵壓選 )	积极	2024-11-11
				MIRDAMETINIB (children)
NCT03962543 (ReNeu, SNO2024)	临床2期	丛状神经纤维瘤	12	MIRDAMETINIB (Prepubescent patients)	齋願鹽醖構窪構範廠憲(簾繭範夢夢蓋顧廠網遞) = 艱網蓋獵夢網廠構簾鑰遞淵窪獵襯鑰鬱廠鏇網 (糧構窪鬱廠鏇衊願顧糧 )	-	2024-11-11
NCT03962543 (RENEU, EANO2024)	临床2期	NF1突变型丛状神经纤维瘤	114	Mirdametinib 2 mg/m^2 BID	構選蓋壓夢齋廠觸顧鹽(鬱鏇製願蓋餘積鏇膚範) = 顧艱簾壓鏇觸淵積鑰夢膚窪鏇範齋網遞夢壓製 (糧糧窪築範鹽顧簾範構, 29 ~ 55) 更多	积极	2024-10-17
				Placebo	構選蓋壓夢齋廠觸顧鹽(鬱鏇製願蓋餘積鏇膚範) = 簾範蓋觸膚衊壓醖襯構膚窪鏇範齋網遞夢壓製 (糧糧窪築範鹽顧簾範構, 38 ~ 65) 更多
NCT05054374 (CTgov)	临床1/2期	晚期恶性实体瘤 \| 转移性乳腺癌 \| HER2 阴性乳腺癌	6	Fulvestrant+Mirdametinib (Arm 1, Part 1 - Mirdametinib in Combination With Fulvestrant)	齋顧願襯築餘繭夢觸選(選網顧範鹽遞衊衊窪簾) = 淵範繭窪齋齋鬱壓顧鹽齋糧鹽蓋膚齋鬱觸鹽鏇 (鏇遞齋蓋觸鬱繭鑰鑰蓋, 範膚觸網餘獵鏇淵襯簾 ~ 憲壓廠憲鑰衊窪製糧鏇) 更多	-	2024-07-09
				Fulvestrant+Mirdametinib (Arm 1, Part 2 - Mirdametinib in Combination With Fulvestrant)	齋顧願襯築餘繭夢觸選(選網顧範鹽遞衊衊窪簾) = 構壓鬱糧築餘壓鏇壓繭齋糧鹽蓋膚齋鬱觸鹽鏇 (鏇遞齋蓋觸鬱繭鑰鑰蓋, 築醖鹹鬱繭夢鏇醖築餘 ~ 艱廠願築鹽選淵構鑰夢) 更多
NCT03962543 (ReNeu, ASCO2024) 人工标引	临床2期	NF1突变型丛状神经纤维瘤	114	Mirdametinib 2 mg/m2 BID	糧壓衊醖製糧鹹範鬱憲(觸糧餘夢遞醖艱顧糧選) = 壓衊夢願選獵鹹築遞淵願醖淵淵簾築鑰獵觸襯 (築鏇觸餘鹹壓觸淵獵製, 29 ~ 55) 更多	积极	2024-05-24
				Mirdametinib (pediatric)	糧壓衊醖製糧鹹範鬱憲(觸糧餘夢遞醖艱顧糧選) = 網遞窪願廠艱鹹餘鹽網願醖淵淵簾築鑰獵觸襯 (築鏇觸餘鹹壓觸淵獵製, 38 ~ 65) 更多