Research Title：Adult and adolescent hemophilia patients treated with Marstacimab: a patient Experience Registry (AMBER) Protocol Number：94250855 Protocol Version Number：version 0.3 Date：15-November-2024 Leading site：Institution of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Sciences Active Pharmaceutical Ingredient：a human IgG1 monoclonal antibody that targets the tissue factor pathway inhibitor (TFPI) Drug Name Generic name: marstacimab-hncq Purpose of the Study Primary Objectives:To quantify patient preferences for subcutaneous versus intravenous (IV) injection using the Marstacimab-Patient Preference Questionnaire (M-PPQ) after 1 month of marstacimab for routine prophylaxis treatment.
To assess the treatment burden using the Hemophilia Treatment Experience Measure (Hemo-TEM) in hemophilia patients after 6 months of marstacimab for routine prophylaxis treatment.
Exploratory Objectives:
To evaluate the annualized bleeding rate (ABR) of different types of bleeds after 6 months of subcutaneous marstacimab injections in hemophilia patients.
To assess changes in joint status scores between baseline and the final visit after 6 months of subcutaneous marstacimab injections in hemophilia patients, using the Hemophilia Early Arthropathy Detection with Ultrasound in China (HEAD-US-C).
To evaluate patient preferences regarding treatment experience via M-PPQ after 6 months

开始日期2026-02-01

申办/合作机构-

NCT06703606

/ Recruiting临床1期

A PHASE 1B OPEN-LABEL NON-RANDOMIZED STUDY TO ASSESS THE SAFETY, PHARMACOKINETICS, AND PHARMACODYNAMICS OF MARSTACIMAB TREATMENT FOLLOWING THE DISCONTINUATION OF EMICIZUMAB THERAPY IN ADOLESCENT AND ADULT PARTICIPANTS WITH SEVERE HEMOPHILIA A WITHOUT INHIBITORS.

The purpose of the study is to learn about safety, how the body processes marstacimab and how it works in patients with severe hemophilia A without inhibitors. Hemophilia A is rare bleeding disorder where the blood doesn't clot normally. This causes a person to bleed a lot, even from a small cut.
These patients who are on emicizumab medicine for routine prophylaxis for at least 6 months, and desire to switch to marstacimab medicine. Inhibitors are antibodies that the immune system develops because it sees the infused clotting factor as a foreign substance that needs to be destroyed. Antibodies are proteins that eat up the activated factor before it has time to stop the bleeding. Prophylaxis are preventive medicines.
This study is seeking for participants:
* with severe Hemophilia A withouth inhibitors who are on emicizumab treatment for at least 6 months.
* must be 12 to less than 75 years old
* must have a body weight of at least 35 kilograms. The results from this study will serve as a guide to doctors and their hemophilia A patients who will change their medicines in the real-world clinical setting. Patients who can take part in the study will receive marstacimab medicine as weekly injections under the skin of 150 milligrams for 4 months. Study treatment with marstacimab will be initiated no earlier than 14 days after last dose of emicizumab. The study can last up to 6 months. The sponsor will provide marstacimab. Patients will continue their usual treatment with the infused clotting factor for their bleeds when taking part in the study. Roll-over into an optional study treatment extension period will be available to participants who wish to continue prophylaxis with marstacimab in countries where it is not commercially available.

开始日期2025-05-13

申办/合作机构

Pfizer Inc.

NCT05611801

/ Recruiting临床3期

AN OPEN-LABEL STUDY IN PEDIATRIC (<18 YEARS OF AGE), SEVERE HEMOPHILIA A PARTICIPANTS (COAGULATION FACTOR ACTIVITY <1%) WITH OR WITHOUT INHIBITORS OR MODERATELY SEVERE TO SEVERE HEMOPHILIA B PARTICIPANTS (COAGULATION FACTOR ACTIVITY =2%) WITH OR WITHOUT INHIBITORS COMPARING 12 MONTHS OF HISTORICAL STANDARD TREATMENT TO MARSTACIMAB PROPHYLAXIS

The purpose of this clinical trial is to learn about the safety and effects of the study medicine (called marstacimab) for the potential treatment of hemophilia in pediatric patients.
This study will enroll pediatric participants from ages 1 to 17 years in a sequential manner. The study will open enrollment to adolescent participants aged 12 to 17 years first. Then children aged 6 to 11 years will be permitted to enroll. Lastly, children aged 1 to 5 years will be permitted to enroll.
This study will enroll participants who:
* have severe Hemophilia A or moderately severe to severe Hemophilia B (with or without inhibitors)
* have accurate historical records documenting all factor VIII, factor IX, or bypass agent infusions and hemophilia bleed events for at least 1 year prior to entering the study
* if a non-inhibitor patient, must be on a stable routine prophylaxis regimen with factor VIII or factor IX replacement products for at least 12 months prior to study entry
* if an inhibitor patient, must be on an on-demand bypass treatment regimen during the 12 months prior to study entry
All participants in this study will receive marstacimab to use prophylactically. Marstacimab will be given once a week as a subcutaneous (under the skin) shot. The first dose of marstacimab will be given at the study site by the study site staff. During the 12-month treatment period, weekly doses of marstacimab can be given at home, or if preferred, the doses may be given by the study site staff.
To help us determine if the study medicine is safe and effective, we will compare participant experiences when they are taking the study medicine to a historical period when they were not. Researchers want to see if the study medicine works to prevent the bleeding episodes commonly experienced by patients with Hemophilia.
Participants will be in this study for about 14 months (approximately 1 month in a Screening period, 12 months receiving treatment, and 1 month in a follow-up period) during which they will visit the study site at least 10 times. If preferred, and if local regulations allow it, 2 of the study visits can be completed at the participant's home instead of at the study site. There will also be 6 scheduled telephone calls approximately every 2 months.

开始日期2022-12-09

申办/合作机构

Pfizer Inc.

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项与马塔西单抗相关的文献（医药）

2025-08-03·Expert Review of Hematology

Safety and efficacy of marstacimab in patients with hemophilia A and B: a systematic review and meta-analysis

Review

作者: Mokresh, Sena ; Mokresh, Muhammed Edib ; Caliskan, Cagla Sumeyye ; Ilter, Merve Kabasakal ; Alomari, Omar

BACKGROUND:

Hemophilia A and B are life-threatening congenital bleeding disorders traditionally managed with frequent factor replacement therapies, often complicated by breakthrough bleeds and inhibitor development. Marstacimab, a monoclonal antibody targeting TFPI, has emerged as a novel prophylactic treatment to reduce bleeding episodes in patients without inhibitors.

METHODS:

A systematic review was conducted following PRISMA guidelines. A comprehensive literature search was performed across multiple databases. Meta-analysis was conducted using R, applying a random-effects model.

RESULTS:

Nine manuscripts were included. Marstacimab significantly reduced the annualized bleeding rate (mean difference: -16.30; 95% CI: [-18.46, -14.15], p < 0.001) and showed a favorable safety profile with most adverse events being mild or moderate, and no thrombotic events reported. The use of a prefilled pen device further highlighted the therapeutic benefits and ease-of-use of Marstacimab.

CONCLUSIONS:

This meta-analysis reinforces the efficacy and safety of Marstacimab in reducing bleeding rates in severe hemophilia A and B. The findings support its role as a promising, transformative alternative to conventional factor replacement therapies.

REGISTRATION:

The study protocol for this systematic review was registered in the International Prospective Registry of Systematic Reviews (PROSPERO) database (www.crd.york.ac.uk/prospero/), and it was allocated the PROSPERO identification number CRD42024620215.

2025-07-03·Expert Review of Hematology

Anti-tissue factor pathway inhibitors for hemophilia: are these treatments the answer to overcoming current treatment limitations?

Review

作者: Mahlangu, Johnny

INTRODUCTION:

Non-factor therapies were developed to address the shortcomings of clotting factor concentrates (CFCs) used for hemophilia bleed management. These CFC unmet needs include high treatment burden, immunogenicity, inconsistent hemostatic cover, poor treatment outcomes, and musculoskeletal progression despite adequate prophylactic treatment. Anti-tissue factor pathway inhibitors (anti-TFPIs) that have completed Phase 3 clinical studies are concizumab and marstacimab. The role of these anti-TFPIs in the hemophilia treatment armamentarium remains unclear.

AREAS COVERED:

This review critically appraises data published in PubMed, World of Science, and peer-reviewed congress presentations to determine whether anti-TFPIs merely supplement current treatment options or represent a disruptive shift in the treatment paradigm for hemophilia. It underscores the unmet needs of replacement therapies and compares the pharmacokinetic, efficacy, and safety data of anti-TFPIs and selected FVIII and FIX products.

EXPERT OPINION:

As hemophilia treatment goals continue to evolve, the role of currently developed anti-TFPIs is still not fully defined. This review comprehensively summarizes the clinical trial data, which shows that anti-TFPIs are not intended to replace the standard of care CFCs but to expand the therapeutic arsenal for patients with hemophilia treated with these therapeutic agents.

2025-04-01·The Journal of the Association of Physicians of India

Revolutionizing Treatment Strategies through Inhibition of Tissue Factor Pathway Inhibitor: A Promising Therapeutic Approach for Hemophilia Management.

Review

作者: Shaikh, Bilal Ahmed ; Aggarwal, Sunita ; Seth, Tulika ; Nigam, Rajendra K ; Baveja, Avriti ; Jain, Ankur ; Atri, Sudhir ; Sharma, Vishnu ; Digra, Sanjeev Kumar ; Vyas, Bhadresh ; Gupta, Naresh

Hemophilia, an X-linked genetic bleeding disorder, is caused by the deficiency of coagulation factors VIII (hemophilia A) or IX (hemophilia B). Regular replacement therapy with the missing clotting factor is an effective standard-of-care treatment. However, it comes with a significant fallout of frequent intravenous dosing with poor compliance, the risk of inhibitor development, and a substantial treatment burden. Research has progressed from missing clotting factors and factor VIII mimetics to the most recent rebalancing therapy that suppresses tissue factor pathway inhibitor (TFPI). Thrombin generation is restricted by TFPI, which inhibits the tissue factor-mediated activation of factor VII. This promising therapeutic approach rebalances hemostasis by inhibiting TFPI, a critical regulator of the extrinsic coagulation pathway, thereby increasing thrombin generation. Novel monoclonal antibodies (concizumab and marstacimab) enhance thrombin generation by blocking TFPI to restore hemostasis. Clinical trials have demonstrated good clinical efficacy and safety of these anti-TFPI, besides their convenient subcutaneous administration using pen devices. These innovative therapies have the potential to enhance the quality of life (QoL) of people with hemophilia. This review provides a comprehensive overview of the clinical development, therapeutic potential, challenges, and prospects of anti-TFPI in the management of hemophilia.

161

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2025-08-01

·Firstwordpharma

FDA okays broader indications for Novartis’ lipid-lowering drug, Novo’s haemophilia prophylatic

The FDA cleared additional indications for Novartis’s cholesterol-lowering drug Leqvio (inclisiran) and Novo Nordisk's haemophilia prophylactic Alhemo (concizumab-mtci), broadening their use to wider patient populations.Leqvio gets go-ahead as montherapyNovartis's twice-yearly injectable siRNA drug Leqvio was approved by the FDA as monotherapy alongside diet and exercise to lower low-denisty lipoprotein cholesterol (LDL-C) in adults with hypercholesterolaemia, thereby eliminating the previous requirement for concurrent statin therapy.“With this new indication enabling Leqvio's use as monotherapy…we now have the potential to help even more patients achieve their LDL-C lowering goals earlier in their treatment journey," said Victor Bultó, president of Novartis US.The US regulator said Leqvio’s broader indication was approved based on strong LDL-C-lowering data from PCSK9-targeting therapies. Additional label updates include replacing "primary hyperlipidaemia" terminology with the more specific "hypercholesterolaemia" designation throughout the prescribing information.Novartis noted the decision also aligns with the 2025 American College of Cardiology/American Heart Association Clinical Practice Guidelines, recommending more aggressive treatment approaches for patients with acute coronary syndrome.Leqvio was discovered by Alnylam and co-developed with The Medicines Company, which Novartis acquired in 2020 for $9.7 billion. First approved in the EU for hypercholesterolaemia or mixed dyslipidaemia in 2020, the siRNA’s US approval faced initial delays owing to manufacturing issues, but was subsequently cleared by the FDA in 2021 for patients on maximally tolerated statin therapy.The Swiss drugmaker expects Leqvio not only to surpass $1 billion in sales this year, but potentially outperform consensus forecasts long term. For more, read – Spotlight On: Novartis' fastest growing drugs.Alhemo cleared for patients without inhibitorsThe FDA also okayed an expanded indication for Novo Nordisk's Alhemo (concizumab-mtci), extending its prophylactic use to adult and paediatric patients aged 12 and older with haemophilia A or B without inhibitors. The latest approval builds upon the tissue factor pathway (TFPI) inhibitor antagonist’s approval last year for patients with inhibitors after being rejected by the agency on the first attempt.Anna Windle, senior VP of clinical development, medical & regulatory affairs at Novo Nordisk, said the latest approval “marks a meaningful step forward for people with haemophilia A or B without inhibitors who are looking for a new prophylaxis treatment option."The label expansion was supported by the Phase III explorer8 study, wherein Alhemo achieved an 86% reduction in annualised bleeding rate for patients with haemophilia A and 79% reduction for those with haemophilia B versus no prophylaxis.Following a positive recommendation by the EU drug advisory body last week, the anti-TFPI monoclonal antibody is also on the verge of approval in Europe for the same indication.With the additional indication, Alhemo emerges as a potential rival to Pfizer’s once-weekly Hympavzi (marstacimab-hncq), approved last year for patients without inhibitors. Pfizer also seeks Hympavzi’s expansion to patients with inhibitors following a successful Phase III study.

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·摩熵医药

6月154款新药获批临床！77款纳入特殊审评，涉及恒瑞、信达、康方生物…

注：本文不构成任何投资意见和建议，以官方/公司公告为准；本文仅作医疗健康相关药物介绍，非治疗方案推荐（若涉及），不代表平台立场。任何文章转载需要得到授权。6月全球在研新药月报内容亮点6月国内新药获批临床情况6月共有154款新药获批临床（共计234个受理号），较上个月增加了13款；6月获批临床受理号数量最多的新药为抗肿瘤药和免疫机能调节药物，有106个，占比为45%；6月获批剂型主要为注射剂与片剂，分别有101个，87个。全球获孤儿药/突破性/快速通道资格认定品种盘点6月共有77款药物获孤儿药/突破性/快速通道资格认定品种。全球在研创新药积极/失败临床结果最新动态速递信达生物创新抗体偶联药物IBI343最新1期临床数据公布，针对晚期胰腺癌；强生公司公布niraparib联合疗法3期临床试验积极数据；默沙东公布PCSK9抑制剂enlicitide decanoate双项3期试验积极结果；百时美施贵宝TYK2抑制剂deucravacitinib达3期试验主要终点。每月相关创新药最新政策速递、国内新药注册申报、全球获孤儿药/突破性/快速通道资格认定品种盘点、全球在研创新药积极/失败临床结果TOP20、全球创新药研发进展TOP20等，摩熵咨询团队基于真实可溯源的全球医药数据，将每月推出「全球在研新药月报」，以期帮助业内人士通盘把握每月全球范围内的新药开发情况，全面而深入的了解全球新药开发动态。016月国内新药获批临床/上市情况根据摩熵医药数据库统计，2025年6月共有154款新药获批临床（共计234个受理号），较上个月增加了13款，其中包括82款化药，69款生物制品，3款中药。截图来源：摩熵咨询月报6月获批临床受理号数量最多的新药为抗肿瘤药和免疫机能调节药物，有106个，占比为45%，消化系统与代谢药物获批数量也比较多，有16个。获批剂型主要为注射剂与片剂，分别有101个，87个。截图来源：摩熵咨询月报在6月份，国内医药市场迎来了16款创新药物的获批上市，涉及诺华的布西珠单抗注射液，用于治疗糖尿病黄斑水肿（DME）适应症；海创药业自主研发的1类新药氘恩扎鲁胺软胶囊，用于治疗接受醋酸阿比特龙及化疗后出现疾病进展，且既往未接受新型雄激素受体抑制剂的转移性去势抵抗性前列腺癌（mCRPC）成人患者；复星医药1类新药芦沃美替尼片（曾用名：复迈替尼片），适用于朗格汉斯细胞组织细胞增生症（LCH）和组织细胞肿瘤成人患者；恒瑞医药的苹果酸法米替尼胶囊，联合注射用卡瑞利珠单抗用于既往接受含铂化疗治疗失败但未接受过贝伐珠单抗治疗的复发或转移性宫颈癌患者，此外恒瑞医药还有注射用磷罗拉匹坦帕洛诺司琼和注射用瑞康曲妥珠单抗均已获批上市。截图来源：摩熵咨询月报02全球获孤儿药/突破性/快速通道资格认定品种盘点6月共有77款药物获孤儿药/突破性/快速通道资格认定。其中值得注意的是：和铂医药（上海）有限责任公司、四川科伦药业股份有限公司共同研发的 tagitanlimab 批准上市，适应症包括鼻咽癌、实体瘤、T细胞淋巴瘤、非霍奇金淋巴瘤、非小细胞肺癌、肝细胞癌等；此外，赛诺菲的 rilzabrutinib 正在申请上市，用于治疗哮喘、荨麻疹、免疫性血小板减少性紫癜、结节性痒疹等病症。6月本月全球孤儿药/突破性/快速通道资格认定品种（部分）一览表备注：完整数据识别文末二维码下载查看03全球在研创新药积极/失败临床结果TOP20在6月全球创新药研发动态中，6月3日，信达生物在2025年美国临床肿瘤学会（ASCO）年会上口头报道了 IBI343（创新型抗CLDN18.2 ADC）治疗晚期胰腺癌临床1期研究数据更新。本项研究为一项1a/1b期剂量递增和剂量扩展研究（NCT05458219），本次大会公布了扩展队列的最新研究数据：截至2025年3月14日，共有83例胰腺癌患者接受了至少一次IBI343治疗，中位随访时间为11.1个月。截至数据截止日期，6mg/kg剂量组CLDN18.2 1+2+3+≥60%的44例受试者中，确认的客观缓解率（cORR）为 22.7%，疾病控制率（DCR）为 81.8%，中位PFS为5.4 个月；中位OS为9.1个月。其中既往仅接受过一线治疗受试者（N=17）的中位PFS为5.4个月，中位OS长达12.1个月；既往仅接受过两线治疗受试者（N=18）的中位PFS为5.3个月，中位OS为9.1个月。安全性方面，总体耐受性良好，消化道毒性低，未出现新的安全信号。98.8%受试者发生治疗期间不良事件 (TEAE），常见的TEAE为贫血、中性粒细胞计数减少、白细胞计数减少。≥3级恶心、呕吐发生率均为0。根据新闻稿，在经过更长时间的随访后，基于更成熟的PFS和OS数据，IBI343 在治疗CLDN18.2表达阳性的晚期胰腺癌患者中展现出优秀的治疗潜力，有望在这一难治癌种中实现突破。截图来源：摩熵医药全球药物研发数据库此外，6月27日，辉瑞（Pfizer）宣布其评估TFPI靶向抗体Hympavzi（marstacimab）用于体内存有抑制物的血友病A或血友病B成人和青少年患者的3期BASIS研究获得积极顶线结果。该研究达到了主要终点和关键的次要出血终点，结果显示每周一次皮下注射 Hympavzi 在改善关键出血结局方面优于按需治疗方案。辉瑞计划与监管机构讨论这些数据，目标是启动 Hympavzi 用于伴抑制物血友病患者的监管申请。分析显示，在伴抑制物的严重血友病A或B患者中，Hympavzi 的预防性治疗可显著降低需要治疗的年化出血率（ABR），在统计学和临床上均具有显著意义。在48名血友病患者接受为期12个月的 Hympavzi 治疗后，与接受按需治疗的患者相比，其ABR降低93%（1.39 vs 19.78；p<0.0001）。Hympavzi 在所有出血相关次要终点（自发性出血、关节出血、靶关节出血和总出血）上也显示出优势。该疗法总体耐受性良好，与过去的1/2期结果一致，未报告死亡或血栓栓塞事件。全球在研创新药积极/失败临床结果TOP20备注：完整数据识别文末二维码下载查看04全球创新药研发进展TOP20在6月全球创新药研发进展TOP20榜单中，亮点纷呈。6月4日，中国国家药监局（NMPA）官网公示，康方生物研发的PD-1/CTLA-4双特异性抗体卡度尼利单抗注射液的一项新适应症上市申请已获得批准。该药本次获批的适应症为：卡度尼利单抗加含铂化疗联合/不联合贝伐珠单抗用于一线治疗持续、复发或转移性宫颈癌。卡度尼利是康方生物自主研发的PD-1/CTLA-4双特异性肿瘤免疫治疗药物，最早于2022年6月获NMPA批准，用于既往接受含铂化疗治疗失败的复发或转移性宫颈癌患者的治疗。2024年9月，该药再次获NMPA批准联合奥沙利铂和卡培他滨（XELOX）一线治疗不可手术切除的局部晚期复发或转移性胃或胃食管结合部（G/GEJ）腺癌。相关临床研究数据显示，卡度尼利与PD-1单抗联合CTLA-4单抗的联合疗法相比，毒性显著降低，具有明显的安全性和疗效优势。截图来源：摩熵医药全球药物研发数据库6月12日，中国国家药监局（NMPA）官网公示，晖致医药（Viatris）申报的瑞维那新吸入溶液（曾用名：雷芬那辛吸入溶液，revefenacin）的上市申请已获得批准。公开资料显示，这是一款治疗慢性阻塞性肺病（COPD）的每日一次、长效雾化支气管扩张剂，此前已经于2018年获得美国FDA批准上市（商品名：YUPELRI）。瑞维那新是一种长效毒蕈碱拮抗剂（LAMA）的雾化剂。它具有高度的肺特异性，在每天只用一次的情况下，能够保持长久的支气管扩张，同时防止毒蕈碱拮抗剂的全身性副作用，例如口干、便秘和尿潴留等。瑞维那新吸入溶液最初由Theravance Biopharma开发，晖致医药在2019年通过合作获得了该产品的雾化剂型在大中华区的独家开发和商业化权。截图来源：摩熵医药全球药物研发数据库2025年6月全球在研新药月报完整报告识别下方二维码获取完整《2025年6月全球在研新药月报》PDF版本报告在每月初定期发布，敬请期待END本文为原创文章，转载请留言获取授权近期热门资源获取中国临床试验趋势与国际多中心临床展望-2025052024年医药企业综合实力排行榜-202505中国带状疱疹疫苗行业分析报告-2025052023H2-2024H1中国药品分析报告-202504数据透视：中药创新药、经典验方、改良型新药、同名同方的申报、获批、销售情况-2025032024年中国1类新药靶点白皮书-202503中国AI医疗健康企业创新发展百强榜单-202502解码护肤抗衰：消费偏好洞察与市场格局分析-2025022024年FDA批准上市的新药分析报告-202501小分子化药白皮书（上）-2025012024年中国医疗健康投融资全景洞察报告-2025012024年医保谈判及市场分析报告-202501近期更多摩熵咨询热门报告，识别下方二维码领取联系我们，体验摩熵医药更多专业服务会议合作园区服务数据库咨询定制服务媒体合作👆👆👆点击上方图片，即可开启摩熵化学数据查询点击阅读原文，申请摩熵医药企业版免费试用！

临床3期孤儿药突破性疗法临床结果申请上市

2025-06-30

·辉瑞制药

辉瑞新型抗菌药物思福诺®在华获批，用于多重耐药性感染和治疗药物选择有限的患者

思福诺®是新型β-内酰胺类抗生素/β-内酰胺酶抑制剂复方制剂，用于治疗由多重耐药的革兰阴性菌引起的成人严重感染思福诺®是首个能够覆盖碳青霉烯耐药肠杆菌目细菌（CRE）全酶型的β-内酰胺类抗生素/β-内酰胺酶抑制剂复方制剂，通过固定复方设计，思福诺®不仅能够覆盖肺炎克雷伯菌碳青霉烯酶（KPC）和OXA-48碳青霉烯酶，还有效针对产金属β-内酰胺酶（MBL）的细菌，具有广谱抑酶活性辉瑞公司今日宣布，新型抗菌药物思福诺®（注射用氨曲南阿维巴坦钠，以下简称“氨曲南/阿维巴坦”）获得国家药品监督管理局正式批准，用于治疗由革兰阴性菌引起的治疗药物选择有限或无替代治疗的成人复杂性腹腔内感染（cIAI），医院获得性肺炎（HAP），包括呼吸机相关性肺炎（VAP）1。思福诺®是首个能够覆盖CRE全酶型的β-内酰胺类抗生素/β-内酰胺酶抑制剂复方制剂，通过固定复方设计，思福诺®不仅能够覆盖KPC及OXA-48碳青霉烯酶，还有效针对产MBL的细菌，克服了既往β-内酰胺酶抑制剂复方制剂不能覆盖MBL的问题。其具有广谱抑酶活性，在改善患者治疗结局的同时降低耐药发生风险。此次获批有助于进一步遏制细菌耐药趋势，提升新型抗菌药物对CRE全酶型感染的精准覆盖，助力抗感染治疗迈向精准治疗新时代，对于公共卫生及创新治疗范式具有重要意义。Jean-Christophe Pointeau辉瑞中国区总裁、RDPAC执行委员会主席抗菌药物耐药是全世界面临的共同挑战，辉瑞公司在抗感染领域深耕多年，始终致力于创新抗感染药物的研发，以助力临床应对耐药菌感染，守护生命健康。此次思福诺®的获批将再一次拓宽碳青霉烯类耐药革兰阴性菌（CRO）的治疗边界，为临床提供目标治疗的创新方案，帮助更多感染患者临床获益，遏制耐药趋势的进一步发展。我们感谢国家药品监督管理局和其他相关部门对新型抗菌药物的支持，辉瑞将持续致力于应对抗菌药物耐药带来的健康挑战，不断践行‘为患者带来改变其生活的突破创新’使命。抗菌药物耐药是指病原体在进化过程中找到了抵抗抗菌药物作用的方法。耐药病原体会存活、生长并传播其耐药性，从而使常规治疗无效，造成感染持续存在或扩散。这些耐药感染，尤其是由革兰阴性菌引起的耐药感染，被广泛认为是当今全球面临的最大健康威胁之一2。临床数据显示，产MBL的耐碳青霉烯肠杆菌目细菌（MBL-CRE）感染患者死亡率高达55.3%3。在中国，MBL-CRE细菌的传播态势尤为严峻。CHINET数据显示：2005年-2023年，肺炎克雷伯菌对碳青霉烯类抗菌药物如美罗培南的耐药率从2.9%上升到26.0%，大肠埃希菌对碳青霉烯类抗菌药物如美罗培南的耐药率分从1.4%上升到2.0%，CRE耐药检出率呈上升趋势并始终保持高位，2023 年我国MBL-CRE菌株在CRE菌株中占比高达 39.3%4。因产MBL肠杆菌能够水解几乎所有β-内酰胺类抗生素，而且耐药菌株突变速度非常快，临床上尚无针对性药物，给临床治疗感染性疾病带来了严峻的挑战5。由于产MBL的病原体的传播和死亡率不断增加，世界卫生组织（WHO）将其列为关键病原体6。亟需针对性药物，以遏制这类超级细菌的进一步扩散。思福诺®由氨曲南和阿维巴坦组成。氨曲南是一种单环β-内酰胺类抗生素7，对多种革兰阴性菌具有抗菌活性。氨曲南与青霉素结合蛋白（PBP）结合后，可抑制细菌细胞壁肽聚糖合成，导致细菌细胞裂解和死亡。氨曲南对B类酶（金属β-内酰胺酶）稳定。阿维巴坦是非β-内酰胺类β-内酰胺酶抑制剂，与相应的水解酶形成共价结合物后起作用。阿维巴坦可抑制Ambler A类和C类β-内酰胺酶以及某些D类β-内酰胺酶，包括超广谱β-内酰胺酶（ESBL）、KPC和OXA-48以及AmpC酶，阿维巴坦不会抑制B类酶，并且不能抑制许多D类酶1。两项国际多中心III期临床试验REVISIT和ASSEMBLE用于支持思福诺®获批，试验评估了思福诺®在治疗因革兰阴性菌（包括产MBL的多重耐药菌）引起的严重感染中的疗效、安全性和耐受性。在治疗多重耐药革兰阴性菌（包括产MBL菌）引起的复杂性腹腔感染和医院获得性肺炎中，REVISIT试验组微生物应答率76%8。ASSEMBLE研究表明，氨曲南/阿维巴坦±甲硝唑治疗产金属酶革兰阴性菌感染的cIAI、HAP/VAP、复杂尿路感染（cUITI）或血流感染（BSI）患者，治愈访视（TOC）时的临床治愈率为41.7%，高于对照组接受最佳可用治疗（BAT）治疗的患者（临床治愈率0%），28天全因死亡率明显低于BAT治疗组（8.3% vs. 33.3%）9。目前，思福诺®已获得欧盟、英国等全球多个国家或地区的上市许可，并被国内外多个权威指南一致推荐用于产MBL的耐碳青霉烯肠杆菌目细菌（CRE）感染患者的治疗10,11,12,13,14。关于思福诺®（氨曲南/阿维巴坦）滑动阅览思福诺®适用于治疗18岁及以上患者由敏感革兰阴性菌引起的下列感染：与甲硝唑联合治疗药物选择有限或无替代治疗的复杂性腹腔内感染（cIAI），治疗药物选择有限或无替代治疗的医院获得性肺炎（HAP）和呼吸机相关性肺炎（VAP）1。思福诺®由氨曲南（一种单环β-内酰胺类抗生素）和阿维巴坦（一种非β-内酰胺的β-内酰胺酶抑制剂）组合而成15,16。金属β-内酰胺酶（MBL）作为一类当前β-内酰胺酶抑制剂无法抑制的β-内酰胺酶，可水解几乎所有β-内酰胺类抗生素，但对氨曲南等单环类β-内酰胺抗生素无作用。然而，MBL常同时会与其他β-内酰胺酶表达，这些β-内酰胺酶会水解氨曲南，限制了氨曲南单药治疗的临床应用17。氨曲南与阿维巴坦的联合使用可恢复氨曲南对同时产MBL和其他β-内酰胺酶的细菌的抗菌活性，为多重耐药革兰阴性菌提供耐受性良好且有效的治疗方案14。这些具有多重耐药性的革兰阴性菌已被世界卫生组织列为重点优先病原体，包括耐碳青霉烯肠杆菌目细菌（包括产MBL的肠杆菌）和嗜麦芽窄食单胞菌18,19。关于辉瑞：为患者带来改变其生活的突破创新滑动阅览在辉瑞，我们通过科学和全球资源为人们提供治疗方案，以延长其生命，显著改善其生活。在医疗卫生产品的探索、研发和生产过程中，辉瑞始终致力于奉行严格的质量、安全和价值标准。我们在全球的产品组合包括创新药品和疫苗。每天，辉瑞在发达和新兴市场的员工都在推进人类健康，推动疾病的预防、治疗和治愈，以应对挑战我们这个时代的顽疾。辉瑞还与医疗卫生服务方、政府和社区合作，支持并促进世界各地的人们能够获得更为可靠和可承付的医疗卫生服务。这与辉瑞作为一家全球卓越的创新生物制药公司的责任是一致的。175年来，辉瑞一直致力于为所有依赖我们的人带来改变。如需了解更多信息，请登录www.pfizer.com.cn。免责声明滑动阅览本新闻稿仅在于传递科学前沿信息，不构成且在任何情况下均不应被视为对任何药物或治疗方案的推荐或推广。本新闻稿中所含信息仅截至发布日期。本新闻稿中的所有行动和声明均应遵守不时修订的适用法律法规。没有任何迹象表明与所述内容有关的所有信息均包含在本新闻稿中。不管是由于新信息、未来事件还是进展，辉瑞一概不承担对前瞻性声明进行更新的义务。对于因使用、依赖本新闻稿中包含的任何信息或任何信息遗漏而直接或间接产生的任何损失，辉瑞不承担任何责任。如果分发或使用其中的信息违反了适用的法律或法规，任何司法管辖区或国家的任何个人或实体均不得分发或使用这些信息。建议在使用本新闻稿或进行相关行动之前，向有关部门进行独立咨询/建议，并进行必要的尽职调查和调研等。如您有药物或治疗方面的问题，请咨询医疗卫生专业人士。滑动查看1 注射用氨曲南阿维巴坦钠说明书（2025年6月24日）2 World Health Organization. Antibiotic resistance factsheet. November 2023. https://www.who.int/news-room/fact-sheets/detail/antibiotic-resistance. Accessed October 16, 2024.3 de Jager P, Chirwa T, Naidoo S, Perovic O, Thomas J. Nosocomial outbreak of New Delhi metallo-β-lactamase-1-producing Gram-negative bacteria in South Africa: a case-control study. PLoS One. 2015;10:e0123337.4 CHINET数据云. http://www.chinets.com/Data/GermYear 2023年全年细菌耐药监测结果5 lactamase inhibitors: an unattended global threat. Lancet Infect Dis. 2022 Jan;22(1):e28-e34.6 Tacconelli E, et al. Discovery, research, and development of new antibiotics: the WHO priority list of antibiotic-resistant bacteria and tuberculosis. Lancet Infect Dis. 2018;18:318–27.7 Wright H, et al. Clin Microbiol Infect 2017;23:704–12.8 Carmeli Y, Cisneros JM, Paul M, et al. Aztreonam-avibactam versus meropenem for the treatment of serious infections caused by Gram-negative bacteria (REVISIT): a descriptive, multinational, open-label, phase 3, randomised trial. Lancet Infect Dis. 2025;25(2):218-230. doi:10.1016/S1473-3099(24)00499-79 Daikos GL, et al. 34th European Society of Clinical Microbiology and Infectious Diseases(ECCMID); Barcelona, Spain: 2024.2024.10 SANFORD GUIDELINES热病. 2025.01.05. 09:46pm更新11 Hellenic Society for Infectious Diseases: 22 July 202412 中国碳青霉烯耐药肠杆菌科细菌感染诊治与防控专家共识编写组, 中华医学杂志, 2021,13 《细菌药物敏感试验执行标准和典型报告解读》（第三版）2025年6月14 中华血液学杂志2025年6月第46卷第6期 Chin J Hematol，June 2025，Vol. 46，No. 615 Rossolini GM, Stone G, Kantecki M, et al. In vitro activity of aztreonam/avibactam against isolates of Enterobacterales collected globally from ATLAS in 2019. J Glob Antimicrob Resist. 2022;30:214–221. Rossolini GM, Stone G, Kantecki M, et al. In vitro activity of aztreonam/avibactam against isolates of Enterobacterales collected globally from ATLAS in 2019. J Glob Antimicrob Resist. 2022;30:214–221.16 Cornely OA, Cisneros JM, Torre-Cisneros J, et al. Pharmacokinetics and safety of aztreonam/avibactam for the treatment of complicated intra-abdominal infections in hospitalized adults: results from the REJUVENATE study. J Antimicrob Chemother. 2020;75(3):618–627.17 Rossolini GM, Stone G, Kantecki M, et al. In vitro activity of aztreonam/avibactam against isolates of Enterobacterales collected globally from ATLAS in 2019. J Glob Antimicrob Resist. 2022;30:214–22118 Boyd SE, Livermore DM, Hooper DC, et al. Metallo-β-lactamases: Structure, function, epidemiology, treatment options, and the development pipeline. Antimicrob Agents Chemother. 2020;64(10):e00397-20. 19 World Health Organization. WHO publishes list of bacteria for which new antibiotics are urgently needed. February 2017. Available at: https://www.who.int/news/item/27-02-2017-who-publishes-list-of-bacteria-for-which-new-antibiotics-are-urgently-needed. Last accessed April 2024. 往期 · 推荐遏制耐药，拯救生命，我们在行动！靶向免疫疗法易瑞欧®（埃纳妥单抗）首处落地，深度缓解实现多发性骨髓瘤患者“更长生存”辉瑞血友病创新疗法马塔西单抗落地海南博鳌乐城先行区【一键通达】药品问询不再难，医学信息解忧烦!

临床研究微生物疗法放射疗法

100 项与马塔西单抗相关的药物交易

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外链

KEGG	Wiki	ATC	Drug Bank
D11261	-	-	-

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
血友病A	美国	Pfizer Inc.	2024-10-11
血友病B	美国	Pfizer Inc.	2024-10-11

未上市

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适应症	最高研发状态	国家/地区	公司	日期
血友病	申请上市	中国	Pfizer Inc.	2024-08-13

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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


NCT03938792 (BASIS, BusinessWire) 人工标引	临床3期	血友病B \| 血友病A	-	Hympavzi	顧願遞獵鏇鏇齋糧憲蓋(鹽餘窪襯簾鑰醖艱鑰鹹) = 簾獵蓋鏇夢獵夢糧鏇鹹願願獵廠餘積觸構衊醖 (網顧蓋壓蓋繭餘築餘餘 ) 达到	积极	2025-06-26
				on-demand treatment	顧願遞獵鏇鏇齋糧憲蓋(鹽餘窪襯簾鑰醖艱鑰鹹) = 積鏇鹽鹹醖鬱蓋獵築構願願獵廠餘積觸構衊醖 (網顧蓋壓蓋繭餘築餘餘 ) 达到
ASH2024	N/A	血友病B \| 血友病A	-	Marstacimab 150 mg SC QW	鑰積網鬱憲簾觸廠夢範(鏇艱簾艱壓襯憲艱願選) = 艱憲製鬱獵獵廠構築餘築齋壓鑰繭廠選壓夢鹽 (獵築衊製夢鬱願衊鹹遞 )	-	2024-12-09
				Factor replacement therapy (OD or RP)	鑰積網鬱憲簾觸廠夢範(鏇艱簾艱壓襯憲艱願選) = 鬱餘獵網製選獵餘餘構築齋壓鑰繭廠選壓夢鹽 (獵築衊製夢鬱願衊鹹遞 )
NCT05145127 (FDA_CDER) 人工标引	临床3期	血友病B \| 血友病A	-	On-Demand Factor-Based Therapy	鑰淵積簾網窪膚艱願窪(鬱蓋夢夢鬱遞餘鏇製膚) = 鏇壓鹹鹹簾觸憲淵淵夢鹽膚餘齋鹹獵遞齋窪糧 (網網獵廠齋蓋築築蓋顧, 5.09 ~ 10.61) 更多	积极	2024-10-11
				HYMPAVZI Prophylaxis	鑰淵積簾網窪膚艱願窪(鬱蓋夢夢鬱遞餘鏇製膚) = 艱鑰構選簾壓繭願積網鹽膚餘齋鹹獵遞齋窪糧 (網網獵廠齋蓋築築蓋顧, 3.40 ~ 6.77) 更多
NCT03938792 (FDA_CDER) 人工标引	临床3期	血友病B \| 血友病A	66	On-Demand Factor-Based Therapy	製艱觸範鏇醖願積鹽膚(選觸憲顧衊齋鬱顧憲選) = 艱鹽蓋積築顧憲鏇鏇獵齋齋夢餘築醖願選襯觸 (窪鹽鬱壓憲網齋顧構艱, 31.03 ~ 46.54) 更多	积极	2024-10-11
				HYMPAVZI Prophylaxis	製艱觸範鏇醖願積鹽膚(選觸憲顧衊齋鬱顧憲選) = 憲鹽鹹齋蓋衊鬱廠鹹淵齋齋夢餘築醖願選襯觸 (窪鹽鬱壓憲網齋顧構艱, 2.09 ~ 4.85) 更多
NCT03938792 (BASIS, ASH2023) 人工标引	临床3期	血友病B \| 血友病A	128	Marstacimab (On Demand) (Observational Phase)	憲憲範淵選積鑰積鏇遞(顧製鹽選積衊鑰鹽艱範) = 繭鑰觸鹽鏇憲簾鑰壓遞醖襯選廠鏇襯鑰憲願繭 (醖範鬱鑰廠遞觸構鏇鏇, 31.03 ~ 46.54) 更多	积极	2023-12-09
				Marstacimab (Routine Prophylaxis) (Observational Phase)	憲憲範淵選積鑰積鏇遞(顧製鹽選積衊鑰鹽艱範) = 壓壓窪獵鏇鹹製憲網簾醖襯選廠鏇襯鑰憲願繭 (醖範鬱鑰廠遞觸構鏇鏇, 5.09 ~ 10.61) 更多
NCT04878731 (CTgov)	临床1期	血友病	6	PF-06741086 (Marstacimab)	廠鹹構構糧築艱衊齋艱 = 鹹鏇簾鏇製構餘構構範憲膚憲積網齋觸衊膚範 (憲選憲構醖範鬱襯構網, 鹹齋選膚廠鹹遞齋淵獵 ~ 製鑰簾獵繭鬱願糧窪獵) 更多	-	2023-07-07
NCT03363321 (Pubmed) 人工标引	临床2期	血友病A	18	Marstacimab	膚繭鑰壓鏇網醖艱願壓(淵鹽廠獵製願構艱繭鑰) = 廠蓋齋範廠糧鑰繭醖鬱簾齋遞憲衊壓淵蓋醖積 (獵網簾積願顧鬱醖網遞 )	积极	2022-10-11
NCT02974855 (CTgov)	临床2期	血友病	27	PF-06741086 (PF-06741086 300 mg SC QW Non-Inhibitor)	鑰鏇觸積窪鑰衊壓衊築 = 鹹憲願淵簾鑰醖鹽鬱鏇遞網鏇願築獵製壓糧淵 (夢夢觸遞網糧衊網醖廠, 壓鹽淵窪鹹鏇積網膚窪 ~ 遞獵選遞壓壓願壓選簾) 更多	-	2019-12-04
				PF-06741086 (PF-06741086 300 mg SC Loading + 150 mg SC QW Non-Inhibitor)	鑰鏇觸積窪鑰衊壓衊築 = 廠鹹選齋齋夢顧鹽獵廠遞網鏇願築獵製壓糧淵 (夢夢觸遞網糧衊網醖廠, 觸餘製齋積願繭壓壓壓 ~ 構構糧淵積廠憲願獵膚) 更多