芬妥司兰钠(Fitusiran) - 药物靶点：AT III_在研适应症：血友病A，血友病B，血友病_专利_临床

概要

基本信息

药物类型

siRNA

别名

Fitusiran Sodium、费妥赛仑、ALN-57213

+ [5]

靶点

AT III

作用方式

抑制剂

作用机制

AT III抑制剂(抗凝血酶-III抑制剂)

治疗领域

遗传病与畸形血液及淋巴系统疾病其他疾病

在研适应症

血友病A血友病B血友病

非在研适应症-

原研机构

Alnylam Pharmaceuticals, Inc.

在研机构

Genzyme Corp.

赛诺菲（中国）投资有限公司

Sanofi

+ [2]

非在研机构-

权益机构

Alnylam Pharmaceuticals, Inc.

Soleo Health Holdings, Inc.

Genzyme Corp.

+ [1]

最高研发阶段批准上市

首次获批日期

美国 (2025-03-28),

血友病A血友病B

最高研发阶段(中国)批准上市

特殊审评突破性疗法 (美国)、孤儿药 (美国)、优先审评 (中国)、快速通道 (美国)

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结构/序列

使用我们的RNA技术数据为新药研发加速。

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Sequence Code 30076949

来源: *****

Sequence Code 432002385

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关联

11

项与芬妥司兰钠相关的临床试验

NCT07285460

/ Recruiting临床3期

An Open-label, Parallel, Phase 3, Two-arm Study to Investigate the Efficacy and Safety of Fitusiran Prophylaxis in Male Participants Aged 1 to Less Than 12 Years With Hemophilia A or B With or Without Inhibitory Antibodies to Factors VIII or IX

This is a parallel, Phase 3, two-arm, open-label study to evaluate the efficacy and safety of treatment with fitusiran prophylaxis administered to male pediatric participants (aged 1 to <12 years) who have severe hemophilia A or B, with or without inhibitory antibodies to FVIII or FIX.
Number of participants:
Approximately 85 participants will be enrolled into the study:
* Approximately 60 fitusiran-naïve participants with severe hemophilia A or B, with or without inhibitors (fitusiran-naïve arm), and
* Approximately 25 participants with severe hemophilia A or B with inhibitors rolling over from the EFC15467* dose confirmation study (roll-over arm).
* Fitusiran has been investigated in the pediatric population in study EFC15467, which enrolled male participants aged 1 to <12 years with hemophilia A or B with inhibitors to examine the safety and tolerability of fitusiran in the pediatric population.
Participants will be enrolled into 1 of 2 arms:
* Fitusiran-naïve: these participants have not previously received fitusiran, and they will undergo screening and study eligibility assessments. Once enrolled, they will go through a 24-week standard of care (SOC) period before starting fitusiran prophylaxis.
* Roll-over participants from the EFC15467 study: only participants who are still on active treatment in study EFC15467 and consenting to study EFC17905 will be eligible to roll over. They will not need to undergo screening or further eligibility assessments. They will directly enroll into the fitusiran treatment period and continue treatment on their current fitusiran dose.
The duration of fitusiran treatment will be up to 160 weeks for the fitusiran-naïve arm and up to 60 weeks for the roll-over arm.

开始日期2025-12-18

申办/合作机构

Sanofi

NCT06145373

/ Recruiting临床4期

An Open-label, Single-arm Treatment Study to Investigate the Safety and Tolerability of Switching From Emicizumab to Fitusiran Prophylaxis in Male Participants Aged ≥12 Years of Age With Severe Hemophilia A, With or Without Inhibitors

This is an exploratory, single group, Phase 4, study to assess treatment with fitusiran prophylaxis after switching from emicizumab prophylaxis.
This study aims to evaluate the safety and tolerability of switching to fitusiran after a transition period from the last dose of emicizumab. The study will be conducted in male participants with severe hemophilia A, with or without inhibitors, aged ≥12 years, who were previously receiving emicizumab prophylaxis.
Study details include:
* The study duration will be up to approximately 28 months:
* There will be an approximately 2-month screening period.
* There will be an approximately 2-month period before fitusiran treatment starts (pre-fitusiran treatment period)
* The fitusiran treatment duration will be up to 18-months (fitusiran treatment period)
* The antithrombin (AT) follow-up (FU) period will be approximately 6 months after the last dose of fitusiran (during which the AT activity level will be monitored at approximately monthly intervals following the final fitusiran dose until AT activity levels return to at least 60%).
* The study site visits are scheduled at monthly/ every 2 months intervals of 28 days (4 weeks) / 56 days (8 weeks), respectively, during the fitusiran treatment period.

开始日期2024-03-01

申办/合作机构

Sanofi

NCT05662319

/ Active, not recruiting临床3期

A Phase 3, Single-arm, Multicenter, Multinational, Open-label, One-way Crossover Study to Investigate the Efficacy and Safety of Fitusiran Prophylaxis in Male Participants Aged ≥ 12 Years With Severe Hemophilia A or B With or Without Inhibitory Antibodies to Factor VIII or IX

This is a multicenter, multinational, open-label, one-way cross-over, Phase 3, single-arm study for treatment of hemophilia.
The purpose of this study is to measure the frequency of treated bleeding episodes with fitusiran in male adult and adolescent (≥12 years old) participants with hemophilia A or B, with or without inhibitory antibodies to factor VIII or IX who have switched from their prior standard of care treatment.
The total study duration will be up to approximately 50 months (200 weeks, 1 study month is equivalent to 4 weeks) and will include:
* A screening period up to approximately 60 days,
* A standard of care (SOC) period of approximately 6 study months (24 weeks),
* A fitusiran treatment period of approximately 36 study months (144 weeks),
* An antithrombin (AT) follow-up period of approximately 6 study months (24 weeks) but may be shorter or longer depending on individual participants AT recovery.
The frequency for telephone visits will be approximately every 2 weeks. For site visits the frequency will be approximately every 8 weeks during the SOC period and approximately every 4 weeks during the fitusiran treatment period. If applicable and if allowed by local regulation, home and/or remote visits may be conducted during the study

开始日期2023-02-01

申办/合作机构

Sanofi

100 项与芬妥司兰钠相关的临床结果

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100 项与芬妥司兰钠相关的转化医学

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100 项与芬妥司兰钠相关的专利（医药）

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39

项与芬妥司兰钠相关的文献（医药）

2026-02-01·AMERICAN JOURNAL OF MANAGED CARE

Redefining hemophilia management: treatment goals, nonfactor replacement therapies, and the role of fitusiran

Article

作者: Pipe, Steven W

Hemophilia is a rare, but debilitating congenital bleeding disorder. Current therapeutic guidelines, last updated in 2020, recommend recombinant clotting factor concentrates as the standard replacement therapy, with emicizumab offered as an alternative nonfactor standard of care. Since the publication of those guidelines, approvals of new gene-based and nonfactor therapies have reshaped the therapeutic landscape. This supplement describes the safety and efficacy of those therapies approved after the 2020 guidelines were published. It also presents managed care considerations describing how opportunities for resource optimization, therapeutic efficiency, and alternative administration approaches may advance progress toward aspirational treatment goals. A main focus is on fitusiran, the most recently approved nonfactor therapy, which introduced a novel mechanism of action and dosing strategy to the hemophilia therapeutic landscape in addition to its broad patient applicability. As this and other therapeutic advancements redefine the standard of care for patients with hemophilia, managed care and policy frameworks must adapt to ensure timely access and affordability. Likewise, updates to clinical guidelines and care pathways are essential to capture the rapidly evolving therapeutic landscape.

2026-01-01·CLINICAL AND APPLIED THROMBOSIS-HEMOSTASIS

Comparative Efficacy and Safety of Non-Clotting Factor Prophylaxis Versus. on-Demand Therapy in Hemophilia: A Meta-Analysis of Randomized Controlled Trials

Review

作者: Danish, Hamza ; Ali, Manahil ; Ahuja, Laksh ; Talpur, Abdul Subhan ; Khan, M Raafe Ali ; Ishaque, Rehan ; Reddy, Aswanth ; Rayamajhi, Supratik ; Ikram, Amna ; Memon, Huda ; Janjua, Hira ; Janjua, Hamza ; El joujou, Raghad ; Siddiqui, Muhammad Ahsan ; Reya, Fnu

Introduction:

Hemophilia A and B are among the most common inherited bleeding disorders in humans with lifelong risk of spontaneous and recurrent bleeding. While prophylaxis with clotting factors has improved outcomes, challenges like inhibitor development and treatment burden persist. We aim for a comparative effectiveness between these agents and traditional therapy with this study.

Methods:

A systematic review and meta-analysis of randomized controlled trials (RCTs) was conducted. Studies were identified through PubMed, Cochrane Central, and ClinicalTrials.gov up to May 30th, 2025. Eligible studies were double-arm RCTs comparing non-clotting factor prophylaxis (emicizumab, fitusiran, concizumab) and on-demand therapy in patients with hemophilia A or B.

Results:

Non-clotting factor prophylaxis significantly reduced the annualized bleeding rate (ABR) for all treated bleeds [ n = 399, (RR = 0.13; 95% CI: 0.09–0.19), I2 = 63.8%, p = 0.0107], spontaneous bleeds [RR = 0.08; 95% CI: (0.06, 0.11), I2 = 0.0%, p = 0.5933)] and joint bleeds [RR = 0.09; 95% CI: (0.06, 0.14), : I2 = 26.2%, p = 0.2468 ]. Haem-A-QoL total score improved with prophylaxis [MD = −11.08 [−16.34, −5.83], I2 = 57.5%, p = 0.0949]. Prophylaxis increased the likelihood of achieving zero treated bleeds [RR = 4.11; 95% CI: (1.48%, 11.45%), I2 = 88.5%, p < 0.0001]. An exploratory network meta-analysis comparing fitusiran, emicizumab, and concizumab reported no statistically significant difference in the ABR for all treated bleeds.

Conclusion:

Compared to on-demand therapy, non-factor prophylactic therapies significantly reduce bleeding episodes, improve quality of life, and increase the likelihood of zero bleeds in patients with hemophilia.

2025-11-01·HAEMOPHILIA

Utilising Thrombin Generation Assay to Guide Co‐Administration of Factor Therapies With Fitusiran

Letter

作者: Dericquebourg, Amy ; Rezigue, Hamdi ; Jourdy, Yohann ; Lienhart, Anne ; Nougier, Christophe ; Leuci, Alexandre ; Dargaud, Yesim ; Desage, Stephanie

418

项与芬妥司兰钠相关的新闻（医药）

2026-04-20

·蒲公英Ouryao

参比制剂（第104批）名单发布

转自：国家药监局编辑：随风摘要：2026年04月20日，国家药监局发布了第104批次仿制药一致性评价参比制剂目录，新增品种39个。经国家药品监督管理局仿制药质量和疗效一致性评价专家委员会审核确定，现发布仿制药参比制剂目录（第一百零四批），特此通告。新增品种：盐酸托鲁地文拉法辛缓释片、芬妥司兰钠注射液、阿夫凯泰片、依降钙素注射液、依降钙素注射液、高血糖素注射液、卡泊三醇泡沫剂、硫酸阿托品滴眼液、盐酸羟考酮口服溶液、盐酸羟考酮口服溶液、氟比洛芬含片、氟[18F]西洛维注射液、乳果糖散、奥洛他定莫米松鼻喷雾剂、阿达帕林过氧苯甲酰凝胶、盐酸哌甲酯缓释干混悬剂、卡匹色替片、西诺氨酯片、玛巴洛沙韦干混悬剂、盐酸曲唑酮口服溶液、美沙拉秦缓释颗粒、米氮平口崩片、硝酸甘油喷雾剂等；仿制药参比制剂目录（第104批）

2026-04-17

·药筛

已有11款血友病药物申请上市，12款进入III期

4月17日是世界血友病日，今天带大家了解下血友病。血友病是一种由于凝血因子Ⅷ或凝血因子Ⅸ的基因缺陷引起的，X染色体连锁的隐性遗传性出血性疾病，属于国家第一批罕见病目录病种。凝血功能异常使得患者终生易于出血。主要临床表现为，出血及出血相关症状，如出血部位的疼痛、活动障碍等。主要出血部位为关节和肌肉。颅内出血不常见，但是死亡率高。分为两类主要类型：血友病A（FⅧ缺乏）和血友病B（FⅨ缺乏）。根据患者体内残存的FⅧ活性（FⅧ:C）及FⅨ活性（FⅨ:C），血友病可以分为：重型（活性<1%）、中间型（1%~5%）及轻型（5%~40%）。血友病患者主要的治疗方式有凝血因子替代治疗、非因子治疗、基因治疗。凝血因子替代治疗血友病A首选基因重组FⅧ制剂或病毒灭活的血源性FⅧ制剂，无上述条件时可选用冷沉淀或新鲜冰冻血浆等。血友病B首选基因重组FⅨ制剂或病毒灭活的血源性凝血酶原复合物（PCC），无上述条件时可选用新鲜冰冻血浆等。非因子治疗艾美赛珠单抗：通过双特异性单克隆抗体结构，同时结合活化凝血因子Ⅸ（FⅨa）和因子Ⅹ（FⅩ），模拟活化凝血因子Ⅷ的辅因子功能，促进FⅨa对FⅩ的活化，从而显著增加凝血酶生成，实现出血部位止血。抗AT制剂（Fitusiran）：Fitusiran是一种抗凝血酶导向的siRNA，高效靶向结合抗凝血酶mRNA，抑制其在肝脏中的生成，从而实现凝血平衡。抗TFPI制剂：组织因子途径抑制物（TFPI）是一种调控外源性凝血途径的天然抗凝蛋白，TFPI通过与凝血因子Xa结合形成复合物，抑制Ⅶa/TF复合物的活性。目前已上市的有Concizumab、Marstacimab（马塔西单抗）。基因治疗目前血友病的基因治疗方案主要利用腺相关病毒（adeno-associated virus, AAV）载体来递送所需的基因。目前已有4款药物上市：其他药物治疗 1-去氨基-8-D-精氨酸加压素（DDAVP）：适于轻型血友病A患者、女性血友病A携带者及1型vWD患者，对重型患者及血友病B患者无效；抗纤溶药物：常用的有氨甲环酸及氨基己酸。可以作为血友病的辅助治疗。临床研究现状我搜索了最高研发阶段在申报临床-III期之间的药物，共有51款。企业布局比较分散，约一半是国内企业。我们重点看看已进入III期的药物，有12款，其中有1款基因治疗药物（中国医学科学院血液病医院的GS-1191），4款凝血因子，7款非因子类药物另外值得高兴的是，已有11款药物处于申请上市状态，凝血因子有4款，基因工程药物有1款（Biocad Ltd的arvenacogene dezaparvovec），其他是非因子类的，研发企业主要是国内企业。科普血友病，不止于世界血友病日这一天。国内企业的深耕的背后，是对血友病患者需求的回应，更是医学科技守护生命的底气。未来，随着基因治疗、双抗等技术的不断成熟，相信血友病的治疗将迎来更多突破，让“终生出血”的困境逐渐成为过去，也让每一位患者都能卸下疾病的枷锁，自由拥抱生活。免责声明：本文仅为科普参考，不构成医疗建议，具体诊断、治疗方案请咨询专业医护人员。插图数据来源：摩熵医药数据库

2026-04-17

·杨晴的注册研习社

2024年全球原创新药盘点：50款FDA新药、48款中国1类新药，细胞基因疗法创纪录

2024年，全球生物医药行业在技术创新与临床需求的协同推动下稳步前行。美国、欧盟、日本和中国继续深化新药创制战略，在肿瘤、罕见病等存在未满足临床需求的疾病领域取得重要突破。监管机构的加速审批机制进一步提升了创新药物的上市效率。本文基于《药学进展》2025年发表的《2024年全球获批上市的原创新药：回顾与展望》一文，为您系统梳理2024年全球原创新药获批情况，并展望2025年发展趋势。01 美国FDA：50款新药，近半首创，细胞基因疗法创纪录 2024年，美国FDA药品评估和研究中心（CDER）共批准了50款新药，其中24款（48%）为首创类药物，小分子药物与抗体药物成为两大主力军。关键数字：孤儿药：26款（52%）以孤儿药资格获批加速审评：22款获快速通道资格，18款获突破性疗法认定，28款获优先审评，7款获加速审批 66%的新药使用了至少一种加速程序 68%的新药率先在美国获得批准治疗领域分布：肿瘤仍是新药获批最多的疾病领域，罕见病领域亦有突出表现。细胞与基因疗法创纪录：2024年，FDA生物制品评价与研究中心（CBER）批准了19款产品，包括4款细胞疗法和3款基因疗法，获批数量创历史新高。多款“首款”里程碑：首款间充质干细胞疗法：remestemcel-L（Ryoncil）首款用于特定异染性脑白质营养不良的基因疗法：atidarsagene autotemcel（Lenneldy）首款肿瘤浸润淋巴细胞疗法：lifileucel（Amtagvi），也是首款获批用于实体瘤的T细胞疗法这些疗法的问世，为长期缺乏有效治疗手段的患者群体带来了希望。02 欧盟：46款新活性物质，肿瘤领域最多 2024年，欧洲EMA发布了“Human Medicines in 2024”报告，共有114款产品获得积极意见，其中：新活性物质：46款优先药物计划（PRIME）认定：6款孤儿药：15款先进技术治疗医学产品（ATMP）：1款适应证分布：肿瘤领域最多（27款），其次为免疫学/风湿病学/移植（13款）和血液学/止血学（12款）。首次在欧盟获批的创新药包括：insulin icodec（Awigli，长效胰岛素）、polhexanide（Akanitor，角膜感染）、odronextamab（Ordspono，双抗淋巴瘤）和lutetium(177Lu)chloride（Theralugand，放射性药物前体）。03 日本：6款首次获批，多款与中美同步 2024年，日本PMDA共批准147款产品，同比增长40%。其中6款为首次在日本获批的创新药物，包括3款化学药和3款抗体药物。值得关注的是，danicopan（Voydeya）和zolbetuximab（Vyloy）均于同年获美国FDA批准上市；zolbetuximab于2024年12月31日在中国获批，datopotamab deruxtecan也已在中国提交上市申请，有望2025年获批。04 中国：48款1类创新药，肿瘤占近半 2024年，我国共有48款1类创新药获批，其中：化学药品：23款治疗用生物制品：22款中药：3款治疗领域分布：肿瘤领域最多，共21款（43.8%）；内分泌疾病5款（10.4%）。加速审评成效显著：优先审评审批：17款（35.4%）附条件批准：11款（22.9%）突破性治疗药物程序：13款（27.1%）此外，NMPA还批准了罕见病用药55款、儿童用药106款、境外已上市境内未上市药品89款，数量较2023年均有增加。中国药企国际化步伐加快：百济神州的tislelizumab（替雷利珠单抗）和贝达药业的ensartinib（恩沙替尼）相继获美国FDA批准上市。2025年初，迪哲医药的舒沃替尼新药上市申请获FDA受理并被授予优先审评资格。这些进展彰显了我国新药研发实力的提升。05 2025年展望：技术迭代与临床价值双驱动展望2025年，生物医药领域将呈现技术平台迭代与临床价值验证的双驱动发展：基因编辑技术向临床纵深推进，从概念突破转向临床验证细胞与免疫疗法由个性化定制迈向规模化生产，开启“精准化+自动化”时代 AI制药从算法优化升级至算力基建，进入“数据驱动+硬件加速”新阶段值得关注的潜力产品（根据Evaluate和科睿唯安预测）： VanzaTriple（Vertex，囊性纤维化三联疗法） datopotamab deruxtecan（第一三共/阿斯利康，ADC） suzetrigine（Vertex，Nav1.8通道阻滞剂） Cobenfy（M1/M4毒蕈碱受体激动剂，精神分裂症） fitusiran（siRNA疗法，血友病） vepdgestrant（ER PROTAC降解剂，乳腺癌）结语 2024年，全球生物医药行业在创新与突破的浪潮中持续演进。各国监管机构凭借加速审评机制，推动了多款原创新药上市，成果丰硕。细胞与基因疗法创下历史新高，中国创新药国际化步伐显著加快。展望2025年，跨学科技术将深度重塑药物研发范式，推动创新疗法向着更低成本、更高效率的目标迈进。随着政策支持力度加大、研发投入增加及国际认可度提升，期待更多具有突破性疗效和新颖作用机制的新药好药问世，为全球人类健康事业贡献“中国方案”。撰写依据：杨臻峥, 王孜涵, 王昀, 等. 2024年全球获批上市的原创新药：回顾与展望[J]. 药学进展, 2025, 49(18): 1915-1922.

100 项与芬妥司兰钠相关的药物交易

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外链

KEGG	Wiki	ATC	Drug Bank
-	-	-	DB15002

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	Genzyme Corp.	2025-03-28
血友病B	美国	Genzyme Corp.	2025-03-28

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
血友病	临床3期	美国	Sanofi	2025-12-18
血友病	临床3期	比利时	Sanofi	2025-12-18
血友病	临床3期	巴西	Sanofi	2025-12-18
血友病	临床3期	罗马尼亚	Sanofi	2025-12-18

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临床结果

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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


NCT02554773 (OLE, ASH2025)	临床2/3期	血友病B \| 血友病A	71	Fitusiran	範觸觸壓膚廠艱遞醖鑰(衊鹹蓋鹹醖繭餘壓繭遞) = 簾製顧選衊鬱築廠製窪廠夢鏇壓鏇憲憲構顧膚 (鑰構獵憲遞餘鬱鬱選醖 ) 更多	积极	2025-12-06
NCT03417245 \| NCT03417102 \| NCT03754790 (ATLAS-OLE, FDA_CDER) 人工标引	临床3期	血友病B \| 血友病A	-	QFITLIA AT-DR (prior bypassing agent)	遞蓋築築廠選鑰艱築鹹(醖窪繭齋鏇餘襯獵願鹹) = 顧網膚鹽範觸鏇遞簾襯選糧鬱積憲獵繭蓋築餘 (範積獵獵繭廠鹽夢蓋鏇, 2.8 ~ 9.5) 更多	积极	2025-03-28
				On-Demand BPA (prior bypassing agent)	遞蓋築築廠選鑰艱築鹹(醖窪繭齋鏇餘襯獵願鹹) = 廠範鹽鹹糧壓築鏇糧鹹選糧鬱積憲獵繭蓋築餘 (範積獵獵繭廠鹽夢蓋鏇, 11.8 ~ 31.0) 更多
NCT02554773 (Pubmed) 人工标引	临床2期	血友病B \| 血友病A	34	fitusiran (AT-based dose regimen)	遞鏇鬱餘積憲築鏇膚醖(鏇襯構膚鑰簾網獵顧鹽) = 廠糧鹽鑰蓋壓築糧製製艱鏇鏇襯積積膚繭網鹹 (積繭願窪遞選積築衊鏇 )	积极	2024-12-06
				fitusiran (the original dose regimen)	遞鏇鬱餘積憲築鏇膚醖(鏇襯構膚鑰簾網獵顧鹽) = 廠鏇襯夢築淵壓醖艱窪艱鏇鏇襯積積膚繭網鹹 (積繭願窪遞選積築衊鏇 )
NCT02554773 (OLE, CTgov)	临床1/2期	血友病B \| 血友病A	34	fitusiran (Original Dose Regimen (SAS 1))	鑰衊網鬱鹹夢繭積繭壓 = 築簾鹽範範鹹餘網鬱糧獵憲鹽鹹願壓襯繭憲鹹 (積範範憲艱鏇艱築壓簾, 廠選選簾願繭獵壓鑰鬱 ~ 鹹製鑰簾製範鹽襯窪蓋) 更多	-	2024-06-05
				fitusiran (AT-Based Dose Regimen (SAS 2))	鑰衊網鬱鹹夢繭積繭壓 = 鹽繭構鹹衊膚築製鹽醖獵憲鹽鹹願壓襯繭憲鹹 (積範範憲艱鏇艱築壓簾, 艱蓋網襯積膚夢願壓襯 ~ 鬱鏇壓窪糧構鬱範糧網) 更多
NCT03417102 (ATLAS-INH, Pubmed) 人工标引	临床3期	血友病B \| 血友病A	57	fitusiran	網餘願鹹淵醖淵壓獵選(觸鹹網憲網夢壓窪鑰艱) = 蓋衊壓蓋製壓遞願鑰鬱範廠艱鏇鏇襯顧觸構獵 (範繭窪醖淵鏇獵積製艱, 1.0 ~ 2.7) 更多	积极	2023-04-29
				bypassing agents	網餘願鹹淵醖淵壓獵選(觸鹹網憲網夢壓窪鑰艱) = 繭餘鹹鑰顧顧齋鹹繭鹹範廠艱鏇鏇襯顧觸構獵 (範繭窪醖淵鏇獵積製艱, 10.6 ~ 30.8) 更多
NCT03549871 (ATLAS-PPX, GlobeNewswire) 人工标引	临床3期	出血 \| 血友病B	80	Fitusiran	製鏇簾餘淵鏇膚遞遞艱(窪鑰齋網選遞鏇鹹餘糧) = 鹽糧膚襯淵衊繭壓構醖網網顧網蓋鹽膚窪齋憲 (積鏇廠選製壓獵簾鏇範 ) 更多	积极	2022-07-10
NCT03417245 \| NCT03417102 (ATLAS-INH, ISTH2022)	临床3期	血友病 inhibitors	118	Fitusiran prophylaxis	構壓糧鹽鬱築鹹蓋構糧(鑰鏇顧網膚衊鹹顧鑰簾) = 壓淵範鬱遞鏇襯壓構製窪築襯淵範糧襯積顧醖 (製齋簾夢艱夢鹹製衊窪 ) 更多	-	2022-07-09
NCT03549871 (ATLAS-PPX, ISTH2022)	临床3期	血友病	217	fitusiran	壓鬱顧衊壓艱餘膚齋襯(鬱蓋憲鬱鹹糧顧鹽憲糧) = 網憲憲鬱顧繭選襯淵鑰獵齋餘鬱製範遞壓廠製 (繭憲範簾夢範淵選構簾 )	-	2022-07-09
NCT03417102 (ISTH2022)	临床3期	血友病A	-	Fitusiran prophylaxis	積構夢餘構糧淵壓衊鹹(觸網範膚鹽繭淵網餘範) = 淵蓋淵鬱顧顧淵顧鏇衊夢糧糧鏇選鏇淵衊獵繭 (選衊選蓋憲糧壓觸艱膚 ) 更多	-	2022-07-09
NCT03417245 (ATLAS-A/B, CTgov)	临床3期	血友病B \| 血友病A	120	factor concentrates (Factor On-demand)	製淵鏇構繭構鹽繭簾衊 = 壓淵獵淵遞鑰獵壓製選積鑰鬱鹽構壓餘壓蓋顧 (醖繭網艱壓夢選遞襯築, 築鑰壓鹽蓋艱蓋憲襯鏇 ~ 顧艱築窪築鏇獵淵觸糧) 更多	-	2022-02-04
				factor concentrates+fitusiran (Fitusiran 80 mg Prophylaxis)	製淵鏇構繭構鹽繭簾衊 = 壓顧築鑰構糧遞窪鹹築積鑰鬱鹽構壓餘壓蓋顧 (醖繭網艱壓夢選遞襯築, 鑰選衊鹽衊積積齋餘範 ~ 顧網齋窪糧餘構製顧醖) 更多