Coagulation factor IX recombinant immunoglubulin g1 fusion protein、Coagulation factor IX recombinant immunoglubulin G1 fusion protein;、Coagulation Factor IX(Long-Acting, Recombinant Fc Fusion Protein)

+ [17]

靶点

factor IX

作用方式

刺激剂

作用机制

factor IX刺激剂(凝血因子IX刺激剂)、凝血途径激活剂、凝血因子替代物

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B出血

非在研适应症-

原研机构

Sobi Analytics Ltd.

在研机构

Swedish Orphan Biovitrum AB

Bioverativ, Inc.

Bioverativ Therapeutics, Inc.

+ [3]

非在研机构-

权益机构

Bioverativ Therapeutics, Inc.

Biogen, Inc.

最高研发阶段批准上市

首次获批日期

美国 (2014-03-28),

出血

最高研发阶段(中国)批准上市

特殊审评孤儿药 (美国)、临床急需境外新药 (中国)

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结构/序列

Sequence Code 169138

来源: *****

Sequence Code 616723820

来源: *****

关联

项与艾诺凝血素α 相关的临床试验

NCT06158334

/ RecruitingN/AIIT

Study of Surgical Practices in Patient With Haemophilia A or B Treated With an Extended Half-life Recombinant Factor VIII-Fc or IX-Fc (ELOCTA®, ALPROLIX®)

Haemophilia A and haemophilia B are inherited bleeding disorders resulting from the absence or deficiency of coagulation factors VIII and IX, respectively. The peri-operative period of people with haemophilia is commonly managed with replacement therapy.
In phase 3 studies of Elocta® (extended half-life recombinant factor VIII-Fc) and Alprolix® (extended half-life recombinant factor IX-Fc), haemostatic efficacy was demonstrated to be good or excellent, close to the haemostatic efficacy usually seen in people without haemophilia, with maintenance and stability of circulating FVIII and FIX levels compatible with the surgical procedure, while reducing the frequency of infusions and consumption of therapeutic units. In 2019, the National Protocol for Diagnosis and Care in haemophilia recommended 2 methods for managing patients with haemophilia in the peri-operative period, either discontinuous injections of standard or extended half-life factor VIII/IX or a continuous infusion of FVIII/IX.
Many countries, including France, have adopted these rFVIII/IXFc therapeuitic products and recommended their use in the surgical management of patients. However, the use of these two products in real life during surgery in haemophilic A and B patients has not been described in detail. It seems therefore relevant to better document their use in order to progressively specify their use during surgeries with varied bleeding risks.

开始日期2022-06-01

申办/合作机构

Hospices Civils de Lyon

NCT03901755

/ CompletedN/A

A 24-month Prospective, Non-interventional, International, Multicentre Study to Describe the Real-world Effectiveness and Usage of Alprolix in Patients With Haemophilia B

Alprolix (rFIXFc) is a recombinant extended half-life coagulation factor product. The purpose of this non-interventional study is to describe the real-world usage and effectiveness of Alprolix in the on-demand and prophylactic treatment of haemophilia B.

开始日期2019-09-12

申办/合作机构

Swedish Orphan Biovitrum AB

NCT03655340

/ CompletedN/A

A 24-month Prospective, Non-interventional, Multicentre Study to Evaluate the Real-world Effectiveness and Usage of Alprolix in Patients With Haemophilia B in France

开始日期2018-09-12

申办/合作机构

Swedish Orphan Biovitrum AB

100 项与艾诺凝血素α 相关的临床结果

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100 项与艾诺凝血素α 相关的转化医学

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100 项与艾诺凝血素α 相关的专利（医药）

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100

项与艾诺凝血素α 相关的文献（医药）

2025-03-01·EUROPEAN JOURNAL OF HAEMATOLOGY

ORPHEE: A Real‐World Study on rIX‐FP Prophylaxis Use in Adolescent/Adult Patients With Hemophilia B

Article

作者: Frotscher, Birgit ; Petesch, Brigitte Pan ; Fournel, Alexandra ; Lebreton, Aurélien ; Hassoun, Abel ; Dargaud, Yesim ; Desprez, Dominique ; Gillet, Benjamin ; Lauvray, Thomas ; Berger, Claire ; Harroche, Annie ; Chamouni, Pierre ; de Raucourt, Emmanuelle ; Valentin, Jean‐Baptiste ; d'Oiron, Roseline ; Biron‐Andreani, Christine ; Reynes, Claire ; Rauch, Antoine ; Martin, Cédric ; Guillet, Benoit ; Volot, Fabienne ; Castet, Sabine ; Catovic, Hasan ; Cussac, Vincent ; Tardy, Brigitte

ABSTRACT:

Objectives:

To assess the real‐world efficacy and safety of recombinant factor IX albumin fusion protein (rIX‐FP) in patients with hemophilia B (HB) in France.

Methods:

Data on dosing frequency, weekly consumption, and bleeds before‐and‐after switching to rIX‐FP, were collected from December 2021 to February 2024. Annualized (spontaneous) bleeding rates [A(s)BRs] were calculated only in patients on prophylaxis with a follow‐up ≥ 6 months.

Results:

This interim analysis focused on 77 patients ≥ 12 years; 62 (81%) had severe HB. After switching to rIX‐FP, the infusion interval was 14 (7–14) days. Weekly consumption was 43 (35.5–53) IU/kg. ABRs and AsBRs were 0.5 (0–1.9) and 0 (0–0.7) (n = 63) at 18.2 (12.3–21.9) months of follow‐up. Prophylactic efficacy of rIX‐FP was considered ‘Excellent’/‘Good’ in 65/68 (95%) patients. Among the 43 patients previously treated with rFIXFc, 21 increased the infusion interval from 7 (7–11) days with rFIXFc to 14 (7–14) days with rIX‐FP; 33/43 (77%) reduced weekly factor IX (FIX) consumption from 59.95 (46.35–77.93) to 42.5 (35.88–50.25) IU/kg. Patients maintained good protection against bleeds.

Conclusion:

This analysis confirmed that switching to rIX‐FP allows for reducing injection frequency and FIX consumption while maintaining good bleed protection.

2025-01-01·Therapeutic Advances in Hematology

Real-world usage and effectiveness of recombinant factor IX Fc in haemophilia B from the B-SURE study in France

Article

作者: Santagostino, Elena ; Trossaërt, Marc ; Harroche, Annie ; Susen, Sophie ; Gay, Valérie ; Vanderbecken, Stéphane ; Biron-Andréani, Christine ; Genre-Volot, Fabienne ; Lebreton, Aurélien ; Palmborg, Helena ; Desprez, Dominique ; Petesch, Brigitte Pan ; Oudot, Caroline ; Wibaut, Bénédicte ; Barbay, Virginie ; Stoven, Cécile ; Chamouni, Pierre ; Gandossi, Corinne ; Zidi, Meriem ; Negrier, Claude ; Repesse, Yohann ; Nüesch, Eveline ; Repessé, Yohann ; Lavigne-Lissalde, Geraldine ; Guillet, Benoit ; Castet, Sabine Marie ; Chambost, Hervé ; Puyade, Mathieu ; Fouassier, Marc

Background::

More real-world data are needed to complement existing phase III studies on the efficacy and safety of recombinant factor IX Fc fusion protein (rFIXFc) in people with haemophilia B.

Objectives::

We report final data from the B-SURE study, evaluating the real-world usage and effectiveness of rFIXFc in France.

Methods::

Previously treated patients (all ages/severities) received on-demand or prophylactic rFIXFc during B-SURE. Annualised bleeding rate (ABR), injection frequency (IF) and factor consumption (FC) were prospectively evaluated for patients on rFIXFc prophylaxis (primary endpoints). Six months of retrospective factor IX (FIX) data were collected for comparison; patients with ⩾3 months of treatment pre- and post-switch to rFIXFc were analysed.

Design::

B-SURE was a 24-month, prospective, non-interventional, real-world study across haemophilia treatment centres in France.

Results::

Ninety-one male patients enrolled across 21 centres (34% <18 years, 89% severe haemophilia B). Eighty-four patients received prophylaxis at rFIXFc initiation; mean prospective observation period was 21.5 months. Sixty-eight of 84 patients had prior FIX prophylaxis; on rFIXFc prophylaxis, these patients achieved low median ABR (1.2), IF (47.45 injections/year) and mean FC (2844 IU/kg/year). Compared with previous FIX, mean ABR was reduced by 40% ( n = 63); mean IF and FC were reduced by 38.20 injections/year and 1008 IU/kg/year ( n = 57). In patients with prior FIX on-demand ( n = 15), mean ABR reduced by 84% on rFIXFc prophylaxis ( n = 14), mean IF reduced by 2.13 injections/year and mean FC increased by 381.8 IU/kg/year ( n = 15). Most physicians and patients were satisfied/highly satisfied with rFIXFc prophylaxis. rFIXFc was well tolerated with no new safety concerns.

Conclusion::

Findings support the safety and effectiveness of rFIXFc, with reduced IF and FC while maintaining/improving bleed protection.Trial registration : NCT03655340.

2024-10-01·EUROPEAN JOURNAL OF HAEMATOLOGY

Prophylaxis with recombinant factor IX Fc fusion protein reduces the risk of bleeding and delays time to first spontaneous bleed event in previously untreated patients with haemophilia B: A post hoc analysis of the PUPs B‐LONG study

Article

作者: Shapiro, Amy ; Recht, Michael ; Nolan, Beatrice ; Rendo, Pablo ; Rauch, Antoine ; Foster, Meredith ; Casiano, Sandra ; Falk, Aletta

Abstract:

Objectives:

Haemophilia B (HB), characterised by deficient factor IX (FIX), leads to spontaneous bleeds. Severe cases require prophylactic FIX replacement. This post hoc analysis assessed the first spontaneous bleeds among previously untreated patients (PUPs) with HB treated with recombinant FIX Fc fusion protein (rFIXFc) (NCT02234310) to identify factors influencing bleeds.

Methods:

Subjects included paediatric PUPs with HB (≤2 IU/dL endogenous FIX). Analyses described treatment patterns (on demand [OD] vs. prophylaxis) and prophylaxis type (started on vs. switched to prophylaxis). Kaplan–Meier analyses assessed the time to first spontaneous bleed, including median time to event and fitting models with predictors for treatment regimen and/or baseline age.

Results:

PUPs B‐LONG enrolled 33 subjects. Baseline age did not influence the time to first spontaneous bleed for any rFIXFc regimen. Those who started on prophylaxis with rFIXFc (n = 11), compared with those treated OD (n = 22), had an extended time to first spontaneous bleed. Starting prophylaxis afforded a 93% reduced risk of first spontaneous bleed versus starting OD (hazard ratio [95% confidence interval]: 0.071 [0.009–0.592]) (p = .015).

Conclusion:

rFIXFc prophylaxis, particularly starting early, reduced the risk of bleeding and delayed time to first spontaneous bleed compared with rFIXFc OD. Hence, initial treatment regimens impact bleed patterns in paediatric PUPs.

项与艾诺凝血素α 相关的新闻（医药）

2025-04-24

·FiercePharma

Sanofi and Regeneron's high-flying Dupixent on course for an 'inflection year' in COPD

Sanofi said that the launch of Dupixent in COPD is off to a promising start. The company called 2025 an 'inflection year," for uptake of the product in the indication. As Sanofi anticipates sales of immunotherapy juggernaut Dupixent reaching a major $22 billion by the end of the decade, fulfilling that goal will hinge on its uptake in chronic obstructive pulmonary disorder (COPD).So far, so good.After gaining FDA approval in the indication in September of last year, the company is on a “record-setting pace,” as it relates to coverage, according to Brian Foard, Sanofi’s head of specialty care, speaking at the company’s first quarter conference call.Medicare and commercial coverage are at 90% and 88% respectively, Foard said, adding that “as you look at initiations we’re seeing, it’s our most rapid respiratory initiation launch so far.”COPD represents a significant opportunity for Sanofi and its U.S. drug partner Regeneron as Dupixent is the first biologic approved for the condition, which is the fourth-leading cause of death in the world. With many patients having “resigned themselves to their condition,” according to Sanofi CEO Paul Hudson, the company is busy educating pulmonologists and patients about the benefits of the treatment.Sanofi has launched Dupixent for COPD in eight countries, said Hudson, who added that 2025 will be the "inflection year," for the treatment in the indication. Last week, the FDA approved Dupixent in its seventh indication—the hives disorder chronic spontaneous urticaria (CSU). The company expects another nod this year for Dupixent to treat the skin disease bullous pemphigoid (BP).The company reported Q1 sales of Dupixent at 3.5 billion euros ($4 billion), which was an impressive increase of 20% year over year. In 2024, Dupixent sales came in at $14.1 billion, which was an increase of 22% from 2023.Dupixent’s trajectory contributed heavily to Sanofi’s strong quarterly performance. The company racked up sales of 9.9 billion euros ($11.3 billion) in the first quarter, which was a 10% increase year-over-year. Also playing a role was hemophilia A treatment Altuviiio, which made a 100% leap in sales to 251 million euros ($285 million) and “has the potential to become our next blockbuster this year,” Hudson said.Hudson added that Altuviiio, which was approved in early 2023, is “benefiting from patient switches,” as they move away from factor VIII replacement medicines, such as the company’s aging Eloctate, which saw a 21% decline in sales to 70 million euros ($80 million) in the quarter.Meanwhile, sales of Sanofi’s factor VIX replacement treatment Alprolix—which was approved for hemophilia B patients three months before Eloctate in 2014—grew by 20% to 160 million euros ($182 million).

上市批准免疫疗法临床结果生物类似药

2025-04-23

·PRNewswire

Sobi presents clinical data at WFH 2025 Comprehensive Care Summit

STOCKHOLM, April 23, 2025 /PRNewswire/ -- Sobi® (STO: SOBI) presents clinical data aimed at updating the global haemophilia community attending this year's WFH 2025 Comprehensive Care Summit in Dubai from the 23 – 25 April. New and updated outcomes and analyses will be presented from the XTEND phase 3clinical program testing the effectiveness of Altuvoct® (efanesoctocog alfa) treatment for haemophilia A, including updates on patients' joint health and surgical outcomes in patients. "At WFH 2025 CCS, Sobi will present highlights from the XTEND phase 3 program and discuss the new treatment paradigm with FVIII levels in the non-haemophilia range. Sharing these clinical outcomes with the wider haemophilia community will help to ensure the latest treatment approaches are widely known and understood," said Lydia Abad-Franch, MD, MBA, Head of Research, Development, and Medical Affairs (RDMA), and Chief Medical Officer at Sobi. Key data to be presented at WFH 2025 Comprehensive Care Summit, Dubai, UAE Altuvoct® (efanesoctocog alfa) About efanesoctocog alfa Efanesoctocog alfa [Antihemophilic Factor (Recombinant), Fc-VWF-XTEN Fusion Protein] (formerly BIVV001) is a novel and significant part of the week factor VIII therapy. It has the potential to deliver near-normal factor activity levels for significant parts of the week, improving bleed protection in a once-weekly dose for people with haemophilia A. Efanesoctocog alfa builds on the innovative Fc fusion technology by adding a region of von Willebrand factor and XTEN® polypeptides to extend its time in circulation. It is the only therapy that has been shown to break through the von Willebrand factor ceiling, which imposes a half-life limitation on current factor VIII therapies. It was approved as ALTUVIIIO™ [Antihemophilic Factor (Recombinant), Fc-VWF-XTEN Fusion Protein-ehtl] by Sanofi in the US in February 2023. About the Sobi and Sanofi collaboration Sobi and Sanofi collaborate on the development and commercialisation of Alprolix® and Elocta®/Eloctate®. The companies also collaborate on the development and commercialisation of efanesoctocog alfa, referred to as ALTUVIIIO™ in the USA and ALTOVUCT in Europe. An investigational factor VIII therapy with the potential to provide high sustained factor activity levels with once-weekly dosing for people with haemophilia A. Sobi has final development and commercialisation rights in the Sobi territory (essentially Europe, North Africa, Russia and most Middle Eastern markets). Sanofi has final development and commercialisation rights in North America and all other regions in the world excluding the Sobi territory. Sobi® Sobi is a specialised international biopharmaceutical company transforming the lives of people with rare diseases. Providing sustainable access to innovative medicines in the areas of haematology, immunology and specialty care, Sobi has approximately 1,800 employees across Europe, North America, the Middle East and Asia. In 2022, revenue amounted to SEK 18.8 billion. Sobi's share (STO:SOBI) is listed on Nasdaq Stockholm. More about Sobi at sobi.com, LinkedIn and YouTube. Contact For details on how to contact the Sobi Investor Relations Team, please click here. For Sobi Media contacts, click here. This information was brought to you by Cision The following files are available for download: WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

临床3期上市批准生物类似药

2025-04-07

·ChenFJMU

重磅预防｜全球第七款siRNA药物Fitusiran 获批，用于血友病 A/B出血发作，每两月皮下注射一次！

ChenFJMU团队编辑（原创，全链条硬核解读：从结构剂型，到临床药理，到合理用药）思语湖畔QuoteYou were born to win, but to be a winner you must plan to win, prepare to win, and expect to win. — Zig Ziglar Fitusiran，01Qfitlia（Fitusiran，芬妥司兰），由赛诺菲（Sanofi）和Alnylam制药公司联合研发。于2025年3月28日获美国FDA批准，用于常规预防或减少12 岁及以上患有血友病 A 或血友病 B 的成人和儿童患者的出血发作频率，无论是否有凝血因子 VIII 或 IX 抑制剂。作为首个抗凝血酶（AT）降低寡核苷酸疗法，被证明通过抑制肝脏中抗凝血酶的生成发挥作用。在中国，CDE已于2024年5月受理了Fitusiran注射液的新药上市申请，并将其纳入《以患者为中心的罕见疾病药物研发试点工作计划》，拟用于作为常规预防治疗，用于有或无凝血因子VIII或IX抑制物的血友病A或B的成人和12岁以上患者。这款“First-in-class”创新siRNA疗法，有望在五年内成为重磅的革命性预防手段，将有望造福所有血友病患者。血友病 A/B 是罕见的遗传性终身出血疾病，因 VIII 或 IX 因子缺乏导致凝血功能受损，引发过度出血和自发性关节出血。因此，患有血友病的患者无法正常凝血，在受伤或手术后出血时间可能比正常情况更长，他们还可能出现肌肉、关节和器官自发性出血，导致关节损伤、慢性疼痛并显著影响生活质量。这些出血事件通常需要使用含有 FVIII 或 FIX 的产品或模拟因子按需、间歇性治疗或预防性治疗来控制。Qfitlia的获批不仅是血友病治疗的里程碑，也为siRNA疗法在其他疾病中的应用开辟了道路。这种精准的基因治疗技术有望在未来改变更多罕见病患者的命运。【黑框警告】Qfitlia可能会引发血栓事件（血液凝固）、急性和复发性胆囊疾病（部分患者需要切除胆囊）和肝毒性。警告患者需要在基线时进行肝血检测，并在开始使用 Qfitlia 治疗或增加剂量后至少六个月内每月监测一次。【结构与剂型处方】Fitusiran序列：正义链GGUUAACACCAUUUACUUCAA；反义链GACCAAUUGUGGUAAAUGAAGUU。采用Alnylam公司增强稳定化学ESC-GalNAc共轭技术，序列中包括硫代磷酸酯PS（6个）、2’-OMe（Am、Cm、Gm、Um）、2’-F（Af、Cf、Gf、Uf）、GalNAc靶头修饰（L96）。Qfitlia注射液中的有效成分为Fitusiran，这是一种抗凝血酶导向的双链小干扰核糖核酸（siRNA），与GalNAc部分的配体共价连接。Fitusiran钠盐的分子式为C520H636F21N175Na43O309P43S6，分子量为17193 Da。该原料药为白色至淡黄色粉末，易溶于水和磷酸盐缓冲盐溶液，在50 mM KCl中1%水溶液的pH值为5.4（4.4-7.3）。结构序列如下：Qfitlia是一种无菌、无防腐剂、透明、无色至淡黄色的溶液，用于皮下注射（SC）给药。每支50 mg单剂量预充笔在0.5 mL中输送50 mg Fitusiran（相当于53.0 mg Fitusiran-Na），还含有磷酸一氢钠0.585 mg、磷酸二氢钠0.044 mg、氯化钠2.455 mg和USP注射用水。每个20 mg单剂量小瓶在0.2 mL中输送20 mg Fitusiran（相当于21.2 mg Fitusiran-Na），还含有磷酸一氢钠0.234 mg、磷酸二氢钠0.018 mg、氯化钠0.982 mg和USP注射用水。可以加入磷酸和氢氧化钠调节pH至7.0。【作用机制】Fitusiran是一种抗凝血酶（AT）降低疗法，其不能替代缺失的凝血因子，而是通过降低抗凝血酶水平，促进凝血酶生成，从而实现止血平衡。它利用小干扰RNA（siRNA）技术，靶向结合肝脏中的抗凝血酶mRNA基因SERPINC1并阻断其翻译，抑制肝脏中抗凝血酶的产生，从而重新平衡止血功能。AT降低疗法示意图（Okaygoun D, et al. Journal of Biomedical Science, 2021, 28(1):100741.）Fitusiran的作用机制（Young, G et al. Research and Practice in Thrombosis and Haemostasis, 2023, 7(4):100179.）【药效学及药代动力学】药效学：在血友病患者的临床研究中，主要药效学（PD）观测指标是血浆AT的活性。该数据降低与年化出血率（ABR）减少相关，但AT活性持续低于15%可能增加血栓风险。在LTE15174研究中，AT活性平均值为24%（标准差4.64）。药代动力学：在血友病A或B患者服用后，评估Fitusiran的药代动力学（PK）特性，如下表所示。Fitusiran PD是由肝脏PK驱动而非血浆PK驱动，给药策略基于每月或每两个月给药10、20或50 mg，将血浆AT活性水平维持在15-35%之间。特殊人群用药：1、孕妇：尚无孕妇使用Fitusiran评估药物相关的重大出生缺陷、流产或其他不利的母亲或胎儿结局风险的可用数据。尚未使用Fitusiran对怀孕动物进行生殖研究。尚不清楚孕妇服用Fitusiran是否会对胎儿造成伤害或者影响生殖能力。2、哺乳期：没有关于母乳中存在Fitusiran或其代谢物、对母乳喂养的儿童的影响或对产奶量的影响的数据。尚不清楚在母乳喂养期间使用Fitusiran是否安全。3、儿童患者：在12岁及以上的儿童患者中，使用或不使用因子VIII或IX抑制剂的Fitusiran治疗血友病A或B的安全性和有效性已经得到证实，12岁以下儿童患者使用的安全性和有效性尚未确定。4、老年患者：临床研究没有纳入足够数量的65岁及以上患者，以确定他们的反应是否与年轻患者不同。5、肝功能损伤患者：已确诊肝功能损害(Child-Pugh A、B和C级)的患者应避免使用Fitusiran。6、避孕女性：使用激素避孕药的女性使用Fitusiran可能会增加血栓形成事件的风险。基于雌激素的激素避孕药是遗传性AT缺乏症女性血栓形成的既定危险因素。建议使用激素避孕药的患者在开始接受Fitusiran治疗之前和接受Fitusiran治疗期间使用替代非激素避孕药。免疫原性：在四项长达250周的Qfitlia治疗研究中，290名血友病成年人中有10名（3.4%）出现了抗药物抗体（ADA）。所有患者的ADA滴度均较低，大多数患者有短暂的ADA。没有发现ADA对Qfitlia的PK、PD、安全性或有效性有临床显著影响。【非临床毒理学】Fitusiran尚未对卵巢和雄性生殖器官影响的可逆性评估。无致癌性、致突变性、致染色体断裂性和对雄性生育力指标无不良影响。【临床研究】在两项3期研究中评估了Qfitlia对A型血友病或B型血友病的12岁及以上儿科及成人患者的疗效和安全性：研究ATLAS-INH（NCT03417102），针对具有抑制性抗体的A型或B型血友病患者；研究ATLAS-A/B（NCT03417245），针对不具有抑制性抗体的A型或B型血友病患者。研究受试者被随机分配接受每月一次的Qfitlia预防治疗或或根据需要接受常规按需治疗（旁路药物或凝血因子浓缩物），持续9个月。完成这两项研究的患者转入长期扩展研究ATLAS-OLE（NCT03754790），该研究中患者接受了基于抗凝血酶活性定期测量的可调剂量的Qfitlia。通过将长期扩展研究期间使用Qfitlia给药方案的患者与来自两项随机临床试验的按需对照数据进行比较，证实了Qfitlia的疗效。Qfitlia疗效的主要衡量指标是治疗出血的估计年化出血率（ABR）。在扩展研究中，接受基于抗凝血酶的Qfitlia给药方案的具有抑制物的受试者，与接受旁路药物按需治疗的受试者相比，估计年化出血率降低了73%（估计均值：5.1vs.19.1，p=0.0006）。在接受基于抗凝血酶的Qfitlia给药方案的无抑制物受试者中，与接受凝血因子浓缩物按需治疗的受试者相比，估计年化出血率降低了71%（估计均值：9.0vs.31.4，p<0.0001）。表5：在大于12岁使用抑制剂的患者中，Qfitlia预防治疗出血与按需旁路药物（BPA）治疗出血的年化出血率表6：在年龄大于12岁且无抑制剂的患者中，Qfitlia预防治疗出血与按需凝血因子浓缩物（CFC）治疗出血的年化出血率【用药指南】《使用手册》A. Qfitlia™（Fitusiran） injection（50 mg/0.5 mL）B.Qfitlia™（Fitusiran） injection（20 mg/0.2 mL）血友病治疗及Fitusiran展望（ChenFJMU）作为首款AT降低siRNA疗法，与传统的凝血因子替代疗法（如用于治疗A型血友病的VIII因子DDAVP®、Stimate®和Obizur®等、用于治疗B型血友病的IX因子Alprolix®和Nonacog alfa等）不同，Qfitlia通过一种新颖的机制发挥作用——降低AT水平以重新平衡止血。该药物是一种AT定向双链siRNA，通过皮下注射给药，每两个月一次，旨在通过RNA干扰导致AT mRNA降解，从而降低血浆抗凝血酶水平。与传统的凝血因子注射相比，Qfitlia的优势显而易见。它每年只需注射六次，极大地降低了患者的治疗负担，为血友病患者提供了一种低频给药、广泛适用、治疗效果显著的新选择，推动血友病治疗从“替代”向“根治”迈进。虽然基因治疗能够使血友病A/B患者恢复产生凝血因子，即只需要通过单次给药就可获得长期、稳定的治疗效果。但其昂贵的价格并非普通患者能够承担，据悉每剂Roctavian（valoctocogene roxaparvovec，用于治疗血友病A）定价为150万美元，而用于治疗血友病B的Hemgenix（etranacogene dezaparvovec）定价350万美元，刷新“全球最昂贵药物”的纪录。因此，Qfitlia这款“First-in-class”创新siRNA疗法，有望在五年内成为重磅的革命性预防手段，造福所有血友病患者。此外，单抗药物，如诺和诺德的Alhemo和辉瑞的Hympavzi都能在很大程度上造福血友病患者（详见公众号往期推文，2024年10月和12月FDA批准药物）。往期回顾重试重试编辑：廖源榕排版：周纯情补充和审核：陈昌买推送：ChenFJMU（欢迎个人转发分享，企业及其他公众号需授权后转载，并注明出处！）

siRNA寡核苷酸

100 项与艾诺凝血素α 相关的药物交易

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外链

KEGG	Wiki	ATC	Drug Bank
D10522	-	B02BD04	DB11608

研发状态

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B	澳大利亚	Sanofi-Aventis Australia Pty Ltd.	2014-05-01
出血	美国	Bioverativ Therapeutics, Inc.	2014-03-28

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临床结果

适应症

分期

评价

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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


NCT01027364 (B-LONG, ISTH2023)	临床3期	血友病B	-	Recombinant Factor IX Fc Fusion Protein	襯鏇醖醖鏇壓鏇壓糧鹽(鹹鏇蓋糧獵獵繭膚窪膚) = 鬱淵餘鬱窪壓範餘遞願獵齋鏇積衊選築艱簾遞 (鹽壓築鑰繭鹹顧襯鏇鹽 )	-	2023-06-24
NCT01027364 (B-LONG, ISTH2023)	临床3期	血友病B	-	Recombinant Factor IX Fc Fusion Protein	襯鏇醖醖鏇壓鏇壓糧鹽(鹹鏇蓋糧獵獵繭膚窪膚) = 構願艱構餘餘餘衊蓋衊獵齋鏇積衊選築艱簾遞 (鹽壓築鑰繭鹹顧襯鏇鹽 )	-	2023-06-24
NCT03901755 (B-MORE, ISTH2022)	N/A	血友病B	117	Prophylactic rFIXFc treatment	範製遞築蓋鏇夢壓鏇餘(糧遞觸鬱獵窪夢願憲餘) = 選顧廠築獵構餘窪鑰積繭積衊築鬱觸選選觸醖 (網鹽鹽獵觸窪窪廠遞簾 )	-	2022-07-09
ISTH2022	N/A	血友病B	-	SHL FIX	簾願蓋夢製網願壓夢醖(製繭網繭繭糧廠糧製齋) = 襯簾淵構簾網齋網糧築窪鹹選襯齋積遞觸繭壓 (鬱蓋壓醖膚醖製窪觸鑰 )	-	2022-07-09
ISTH2022	N/A	血友病B	-	rFIXFc	簾願蓋夢製網願壓夢醖(製繭網繭繭糧廠糧製齋) = 窪鑰淵糧壓鏇糧鬱築廠窪鹹選襯齋積遞觸繭壓 (鬱蓋壓醖膚醖製窪觸鑰 ) 更多	-	2022-07-09
ISTH2021	N/A	出血	38	rFIXFc	積構積範壓廠製簾繭網(壓願遞壓夢淵鏇糧積鏇) = 遞糧憲蓋範鬱構鹽淵憲顧夢糧壓範繭憲積鏇蓋 (艱範衊蓋顧簾醖鹹獵願 )	-	2021-07-17
ISTH2021	N/A	出血	38	SHL FIX	積構積範壓廠製簾繭網(壓願遞壓夢淵鏇糧積鏇) = 衊夢網築淵夢淵願窪憲顧夢糧壓範繭憲積鏇蓋 (艱範衊蓋顧簾醖鹹獵願 )	-	2021-07-17
NCT02234310 (PUPs B-LONG, Pubmed \| Blood Adv) 人工标引	临床3期	血友病B	33	rFIXFc	構艱鑰窪艱積範憲廠醖(襯糧廠鏇繭窪顧壓鏇繭) = 獵鬱膚淵製簾繭鑰衊鏇繭鏇餘憲膚廠醖鹹構膚 (齋鹽憲鬱積窪願繭餘鏇, 0.08 ~ 15.76) 更多	积极	2021-07-13
NCT02234310 (PUPs B-LONG, CTgov)	临床3期	出血 \| 血友病B	33	rFIXFc	遞範鹽壓衊窪遞醖艱顧 = 蓋夢築遞獵獵選鬱鹹積製鬱簾艱遞壓衊夢簾網 (淵鑰襯積淵繭淵鹽壓製, 鬱鬱膚淵簾觸艱範繭製 ~ 築衊製簾鹹淵獵夢鏇窪) 更多	-	2020-07-31
ISTH2020	N/A	血友病B	23	Recombinant Factor IX Fc Fusion Protein Concentrate	積獵鬱選襯繭獵齋觸窪(餘製顧鏇膚遞齋鏇鹹獵) = 鏇構鹽網構鑰艱襯夢積艱顧製網衊積夢鑰襯願 (繭範壓積鏇繭鏇窪膚廠 ) 更多	-	2020-07-12
ISTH2020	N/A	血友病B	40	N9-GP	憲簾襯襯夢鑰糧憲窪壓(廠鹽願鬱艱蓋齋衊蓋積) = 壓鏇襯觸襯顧廠簾鏇窪齋憲鹹獵艱窪窪餘鏇廠 (觸鹽衊淵淵網網淵鹽繭 )	-	2020-07-12
ISTH2020	N/A	-	-	rFIXFc	網艱鹽齋獵網構網鏇簾(鏇醖選鬱顧簾鏇壓鬱繭) = 遞遞網鑰顧壓蓋簾鑰積鏇構範願鏇鹽製鏇獵簾 (衊憲憲遞淵餘鹽廠遞網 ) 更多	-	2020-07-12
NCT01425723 (B-YOND, CTgov)	临床3期	出血 \| 血友病B	120	rFIXFc (rFIXFc (9HB02PED [<6 Years Old]))	艱衊糧衊選願願餘築範 = 積範鹹繭製憲範壓窪觸鑰願糧齋壓範遞憲網鏇 (廠餘醖蓋鏇顧膚願構願, 顧鑰鬱糧壓願觸衊範構 ~ 蓋製獵繭憲簾願鏇夢夢)	-	2018-11-23
NCT01425723 (B-YOND, CTgov)	临床3期	出血 \| 血友病B	120	rFIXFc (rFIXFc (9HB02PED [6 to <12 Years]))	艱衊糧衊選願願餘築範 = 觸築鹹顧糧艱廠願衊鑰鑰願糧齋壓範遞憲網鏇 (廠餘醖蓋鏇顧膚願構願, 鏇窪鑰遞襯醖範鬱壓鹽 ~ 餘齋壓範齋遞衊餘顧鹽)	-	2018-11-23