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HEMLIBRA has strong market potential due to its unique, subcutaneous prophylactic treatment for Hemophilia A, including patients with and without factor VIII inhibitors. With increasing global diagnosis rates and expanding access in emerging markets, HEMLIBRA is poised for continued growth. LAS VEGAS, April 21, 2025 /PRNewswire/ -- DelveInsight's " HEMLIBRA Market Size, Forecast, and Market Insight Report" highlights the details around HEMLIBRA, a bispecific factor IXa- and factor X-directed antibody indicated for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adult and pediatric patients ages newborn and older with hemophilia A (congenital factor VIII deficiency) with or without factor VIII inhibitors. The report provides product descriptions, patent details, and competitor products (marketed and emerging therapies) of HEMLIBRA. The report also highlights the historical and forecasted sales from 2020 to 2034 segmented into 7MM [the United States, the EU4 (Germany, France, Italy, and Spain), the United Kingdom, and Japan]. Chugai/Genentech/Roche's HEMLIBRA (emicizumab) Overview HEMLIBRA is a bispecific antibody that targets both factor IXa and factor X, two key proteins in the body's natural blood clotting process. Bridging these proteins it helps restore clotting function in individuals with hemophilia A. HEMLIBRA is used as a preventive treatment and is administered as a ready-to-use subcutaneous injection. It can be given once a week, every two weeks, or every four weeks—following an initial four-week period of weekly doses. Developed by Chugai Pharmaceutical Co., Ltd., HEMLIBRA is co-developed internationally by Chugai, Roche, and Genentech. In the U.S., Genentech markets the drug under the name HEMLIBRA (emicizumab-kxwh), with "kxwh" assigned per FDA guidance on naming biological products. HEMLIBRA can be prescribed in various dosage regimens. The initial loading dose is 3 mg/kg administered via subcutaneous injection once weekly for the first four weeks. This is followed by one of the following maintenance dosing options: 1.5 mg/kg once every week, 3 mg/kg once every two weeks, or 6 mg/kg once every four weeks The choice of maintenance dose should be guided by the healthcare provider's judgment, taking into account the potential for improved patient adherence with certain regimens. The prophylactic use of bypassing agents should be stopped the day before initiating HEMLIBRA prophylaxis. Factor VIII (FVIII) prophylaxis may be continued during the first week of HEMLIBRA treatment. Learn more about HEMLIBRA projected market size for hemophilia A @ HEMLIBRA Market Potential Hemophilia A is marked by bleeding episodes, though in mild and moderate cases, these initial episodes are often less intense, which can lead to delays in diagnosis as patients may not seek medical attention promptly. Moreover, early detection is frequently hindered by the lack of access to adequate diagnostic facilities. In 2023, the number of treated prevalent Hemophilia A cases across the 7MM was estimated at around 45,000, with projections indicating growth throughout the forecast period, according to DelveInsight. Among hemophilia A patients—whether or not they have inhibitors—those without inhibitors represent a larger portion of the population. The standard treatment approach focuses on replacing the missing clotting factor, FVIII, to support normal clot formation and minimize or prevent bleeding episodes. Initially, FVIII replacement was sourced from whole blood and plasma, but advances in biotechnology led to the development of recombinant FVIII (rFVIII) therapies, significantly enhancing disease management by offering more precise and efficient options. More recently, innovative therapies using novel mechanisms to prevent bleeding are expanding the treatment arsenal. Several approved therapies for hemophilia A include ALTUVIIIO (Sanofi/Sobi), ROCTAVIAN (BioMarin), OBIZUR (Takeda), ALHEMO (Novo Nordisk), HEMLIBRA (Chugai/Genentech/Roche), ESPEROCT (Novo Nordisk), JIVI (Bayer), WILATE (Octapharma), and ADYNOVATE (Takeda), among others. Notably, ROCTAVIAN, a gene therapy developed by BioMarin, represents a major advancement—it offers a one-time treatment by introducing functional FVIII genes into the patient's cells, potentially restoring normal clotting ability and reducing the need for regular injections. With a wholesale acquisition cost of USD 2.9 million, BioMarin has already facilitated reimbursement in Europe and administered treatment to a patient in Germany in Q2 2023 at a discounted price of approximately USD 900,000. Looking ahead, the hemophilia A treatment landscape is expected to evolve significantly from 2024 to 2034, fueled by the introduction of new therapies currently in development. According to DelveInsight, the hemophilia A market across the 7MM is projected to grow from USD 11.1 billion in 2023 at a robust CAGR through 2034. Nonetheless, factors like delayed diagnosis, limited awareness, and high treatment costs may pose barriers to market growth. Discover more about the hemophilia A market in detail @ Hemophilia A Market Report Emerging Competitors of HEMLIBRA Emerging therapies such as SPK-8011 (Roche [Spark Therapeutics]), fitusiran (Sanofi), marstacimab (Pfizer), Mim8 (Novo Nordisk), and others are being developed to provide safe and effective treatment options. Companies like GeneVentiv Therapeutics are also working on advancements like Adeno-associated Virus (AAV)-based universal gene therapy for hemophilia (GENV-HEM [AAV8.Fva]). Fitusiran is a subcutaneously administered small interfering RNA (siRNA) therapy under development for the prophylactic treatment of hemophilia A and B, regardless of the presence of inhibitors. Its mechanism involves reducing levels of antithrombin—a protein that inhibits clot formation—to enhance thrombin generation, restore hemostatic balance, and prevent bleeding episodes. In January 2018, Sanofi and Alnylam Pharmaceuticals restructured their RNAi therapeutics collaboration to better align and accelerate the development and commercialization of select treatments for rare genetic diseases. Through this revised agreement, Sanofi obtained global rights to develop and commercialize fitusiran. Marstacimab (PF-06741086) is a monoclonal IgG1 antibody that targets the Kunitz 2 domain of the Tissue Factor Pathway Inhibitor (TFPI). It is being developed to prevent or reduce bleeding events in patients with severe hemophilia A or B (defined as <1% activity of Factor VIII or IX), whether or not they have inhibitors. The FDA granted Fast Track Designation to marstacimab in September 2019 for its potential use in patients with inhibitors. In December 2023, Pfizer announced that the FDA had accepted its Biologics License Application (BLA) for marstacimab, supported by data from the Phase III BASIS study involving patients without FVIII or FIX inhibitors. BBM-H803 is Belief BioMed's first gene therapy candidate for hemophilia A and its second product to gain Investigational New Drug (IND) approval from China's National Medical Products Administration (NMPA). In December 2022, the U.S. FDA granted Orphan Drug Designation to BBM-H803. To know more about the number of competing drugs in development, visit @ HEMLIBRA Market Positioning Compared to Other Drugs Key Milestones of HEMLIBRA In June 2022, Chugai received approval in Japan for acquired Hemophilia A based on a domestic Phase III AGEHA study. In October 2018, HEMLIBRA was approved by the US FDA for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adults and children, ages newborn and older, with hemophilia A without factor VIII inhibitors (FVIII). However, this therapy, which was co-developed by Genentech, Chugai, and Roche, was already FDA-approved in 2017 for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adults and children with hemophilia A with FVIII inhibitors In April 2018, the US FDA granted breakthrough therapy designation to Genentech's HEMLIBRA for people with hemophilia A without factor VIII inhibitors. In October 2014, the US FDA granted orphan drug designation to HEMLIBRA for the treatment of Hemophilia A. Discover how HEMLIBRA is shaping the hemophilia A treatment landscape @ HEMLIBRA Cost HEMLIBRA Market Dynamics HEMLIBRA developed by Roche/Genentech, has significantly reshaped the hemophilia A treatment landscape since its approval. Designed as a bispecific monoclonal antibody, HEMLIBRA mimics the function of factor VIII by bridging activated factor IX and factor X, offering prophylactic treatment for patients with hemophilia A with or without factor VIII inhibitors. Its subcutaneous administration and less frequent dosing schedule (weekly, biweekly, or monthly) offer considerable convenience compared to traditional intravenous factor replacement therapies, making it a preferred option for both patients and caregivers. The market dynamics for HEMLIBRA have been largely influenced by its clinical efficacy, improved patient compliance, and strong safety profile. Since its launch, it has rapidly gained market share, especially among inhibitor patients where treatment options were limited and complex. In non-inhibitor patients as well, the shift away from frequent IV infusions has led to strong adoption rates, allowing HEMLIBRA to cannibalize traditional recombinant factor VIII therapies. Furthermore, its entry has pressured pricing and market share dynamics across the hemophilia therapeutic space, triggering strategic shifts by legacy players like Takeda, Sanofi, and Bayer. However, HEMLIBRA's market leadership is being challenged by the evolving competitive landscape. The emergence of gene therapies, which offer the potential for long-term or even curative outcomes, introduces a new dimension to treatment decisions. While some gene therapies are still in early stages of adoption and face challenges related to durability, safety, and patient eligibility, they represent a looming threat to the long-term dominance of HEMLIBRA, particularly in younger, eligible patient populations. Going forward, the market trajectory for HEMLIBRA will depend on several factors: expanding label indications (such as pediatric use), long-term safety and efficacy data, continued pricing negotiations with payers, and how effectively Roche can defend its position against next-generation therapies. Additionally, with increasing emphasis on patient-centric outcomes and value-based care, HEMLIBRA's ability to demonstrate long-term cost-effectiveness versus both traditional and novel competitors will be key to sustaining its market share. Dive deeper to get more insight into HEMLIBRA's strengths & weaknesses relative to competitors @ HEMLIBRA Market Drug Report Table of Contents Related Reports Hemophilia A Market Hemophilia A Market Insights, Epidemiology, and Market Forecast – 2034 report deliver an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key hemophilia A companies, including BioMarin Pharmaceutical, Roche (Spark Therapeutics), ApcinteX, Novo Nordisk, Sanofi (Genzyme), Alnylam Pharmaceuticals, Pfizer, Sangamo Therapeutics, Bayer, Ultragenyx Pharmaceutical, among others. Hemophilia A Pipeline Hemophilia A Pipeline Insight – 2025 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key hemophilia B companies, including UniQure Biopharma B.V., CSL Behring, Pfizer, Spark Therapeutics, Genzyme, a Sanofi Company, Alnylam Pharmaceuticals, Novo Nordisk, ApcinteX Ltd, Freeline Therapeutics, Sangamo Therapeutics, among others. Hemophilia B Market Hemophilia B Market Insight, Epidemiology, and Market Forecast – 2034 report delivers an in-depth understanding of market trends, market drivers, market barriers, and key hemophilia B companies such as Gem Pharmaceuticals, Lytix Biopharma, Incyte Corporation, Daiichi Sankyo, NantPharma, Iovance Biotherapeutics, Agenus, Eli Lilly and Company, Adaptimmune, Aadi, AVEO Pharmaceuticals, Amgen, among others. Hemophilia B Pipeline Hemophilia B Pipeline Insight – 2025 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key hemophilia B companies, including Shanghai Vitalgen BioPharma, Belief BioMed, TiumBio, Jiangsu Gensciences, Centessa Pharmaceuticals, Takeda, Sanofi, GC Biopharma Corp, Equilibra Bioscience LLC, Regeneron Pharmaceuticals, ISU Abxis, among others. About DelveInsight DelveInsight is a leading Business Consultant and Market Research firm focused exclusively on life sciences. It supports pharma companies by providing comprehensive end-to-end solutions to improve their performance. Get hassle-free access to all the healthcare and pharma market research reports through our subscription-based platform PharmDelve . 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▎药明康德内容团队报道今日（4月17日）是“世界血友病日”。血友病是一种罕见的遗传性出血性疾病，主要分为血友病A和血友病B，分别由体内缺乏凝血因子VIII和IX所致。由于凝血功能障碍，患者会出现关节内反复出血，即使轻微磕碰也可能引发持久的出血风险。血友病患者也被称为“玻璃人”，他们日常生活中承受着极大的身体痛苦和不便，同时面临着沉重的心理负担和用药负担。而随着新药研发的进展，近年来血友病的治疗也取得了极大的突破，多种类型的创新疗法迎来监管机构批准，为患者带来了全新的治疗选择。公开资料显示，目前全球范围内有近40款新药已经获批治疗不同类型的血友病，包括凝血因子替代疗法、靶向TFPI的单抗药物，以及基因治疗药物、小干扰RNA（siRNA）药物等等。今年以来，该领域也接连迎来新进展，比如4月10日，中国NMPA宣布批准信念医药研发的基因治疗药物波哌达可基注射液，用于治疗中重度血友病B。今年3月，赛诺菲（Sanofi）联合开发的“first-in-class”siRNA疗法fitusiran获美国FDA批准治疗血友病。除了已经获批上市的新药，全球范围内还有更多血友病创新疗法正在持续开发中。其中有约20款新药已经在中国进入临床研究或上市申请阶段，有望在不久的将来为更多患者造福。本文我们将看看这些新药有哪些特点。“潜在重磅”siRNA疗法fitusiran已经在中国申报上市赛诺菲和Alnylam联合开发的siRNA疗法fitusiran注射液已经获FDA批准治疗有或无抑制物的血友病A或血友病B患者，同时该药在中国也已经申报上市。这款疗法通过抑制肝脏中抗凝血酶的生产，可以治疗多种不同类型的血友病，也无需考虑患者体内是否存在凝血因子抑制物，大大扩展了它的应用范围。在两项3期临床研究中，与对照组相比，每月一次皮下注射fitusiran预防治疗组患者的年化出血率降低了90%。值得一提的是，业内知名机构科睿唯安（Clarivate）在其发布的Drugs to Watch报告中指出，这款有潜力变革所有血友病患者治疗的创新寡核苷酸疗法，有望在五年内成为重磅。 近10款血友病抗体疗法正在中国开展临床研究在抗体药物治疗领域，辉瑞（Pfizer）马塔西单抗注射液（marstacimab）已经获FDA批准，用于常规预防或减少12岁及以上血友病A和血友病B成人和儿童患者的出血。同时，该产品也已经在中国申报上市。马塔西单抗是一种靶向组织因子途径抑制物（TFPI）K2结构域的单克隆抗体。TFPI是一种天然抗凝蛋白，具有防止血栓形成和恢复止血的功能。马塔西单抗的作用机制与因子替代药品不同，预期疗效不受抑制物存在的影响，有望改变临床治疗上抑制物产生这一大痛点。且其每周一次皮下注射给药的方式也更加便利，有望改善依从性。在关键性3期BASIS研究中，在不伴抑制物的血友病A及血友病B受试者中，经过马塔西单抗12个月的积极治疗期后，与既往因子预防和按需治疗相比，其经治疗出血的ABR（年化出血率）分别降低了35%和92%。值得一提的是，该产品已经于今年2月落地海南博鳌乐城先行区。苏州康宁杰瑞在研的TFPI单克隆抗体KN057也已经在中国进入关键3期临床研究阶段，针对适应症为伴或不伴抑制物的血友病A和血友病B预防治疗。该产品具有半衰期长、每周一次皮下注射等特点，其已经获得FDA授予的三项孤儿药资格，分别用于治疗血友病A、血友病B和血管性血友病（vWD）。此外，还有多款凝血因子替代疗法以及针对其他靶点的单抗新药也正在中国开展临床研究，比如：赛诺菲研发的血友病A疗法efanesoctocog alfa（BIVV001）正在中国开展3期临床研究。这是一款高持久性凝血因子VIII（FVIII）替代疗法，其将将FVIII、血管性血友病因子（vWF）部分区域、XTEN多肽与创新Fc融合蛋白结合形成复合体。与标准和延长半衰期FVIII因子产品相比半衰期延长3至4倍，从而允许每周一次的给药。诺和诺德（Novo Nordisk）在研的长效血友病A疗法Mim8（一款模拟凝血因子VIIIa的双特异性抗体）也正在开展国际多中心（含中国）3期临床研究，该产品旨在为血友病A患者提供持续止血效果，可用于每周一次、每两周一次或每月一次的预防性治疗。今年2月，Mim8针对1~11岁血友病A儿童所进行的3期临床试验FRONTIER3取得积极中期结果。诺和诺德预计在2025年递交Mim8的监管申请。晟斯生物培重组人凝血因子Ⅷ-Fc融合蛋白FRSW117，这是一款超长效重组八因子产品，能够满足“一周一次”给药频率，目前正在3期临床研究中评估治疗血友病A。除了FRSW117，晟斯生物还有多款针对血友病的在研抗体疗法，比如长效重组七因子产品SS109、超长效重组九因子SS327均正在进行临床研究，针对不同类型的血友病患者。上海莱士和Equilibra Bioscience正在共同开发的一款特异性抑制人活化蛋白C抗凝血功能的单克隆抗体SR604注射液，该产品已进入2期临床研究阶段，拟用于血友病A/B及先天性凝血因子Ⅶ缺乏症患者出血发作的预防治疗。人活化蛋白C具有抗血栓、抗炎和纤溶特性，SR604拟先开展每2周一次的皮下注射预防治疗临床试验。至少6款血友病基因治疗药物正在中国开展注册临床研究基因治疗药物具有“一次给药、长期有效”的潜力，有望帮助血友病患者摆脱需要终生频繁静脉注射用药的困扰。通过公开渠道梳理，除了信念医药的波哌达可基注射液，在中国还有至少6款针对血友病的基因治疗药物正在中国开展注册临床研究。其中，进展较快的产品已经进入3期临床研究阶段，为来自华毅乐健研发的GS1191-0445注射液。这是一款用于血友病A的AAV基因治疗在研药物，通过单次静脉输注，将人凝血因子FⅧ基因导入血友病A患者体内，从而提高并长期维持患者体内凝血因子水平，以期达到对因治疗及预防出血的效果。今年2月，华毅乐健宣布GS1191-0445正式进入3期临床研究阶段。在该产品此前的注册1/2期临床研究以及IIT研究中（共19例受试者），目标给药剂量（3E12vg/kg）及以上受试者1年内的FVIII活性水平始终在有效控制出血水平及以上（即＞5ILU/dL），未出现需要外源性FVIII替代治疗的出血事件（手术相关出血除外），相较于GS1191-0445注射液给药前，给药后ABR（年化出血率）降低约99%。此外，还有多款产品处于1期或2期临床研究阶段。包括：信念医药在研的AAV基因治疗药物BBM-H803注射液，拟用于治疗血友病A；方拓生物新型rAAV载体基因治疗药物FT-004注射液，拟开发治疗血友病B；至善唯新在研的rAAV基因治疗药物ZS802注射液和ZS801注射液，分别拟用于治疗血友病A和血友病B；诺洁贝生物和中国医学科学院血液病医院（血液学研究所）共同开发的NGGT003注射液，拟用于治疗血友病A。除了上述产品，全球范围内还有数十款血友病新药正在临床前研究中进行探索。限于篇幅，此处不再一一介绍。期待更多的创新疗法在临床中取得突破，早日为患者造福。参考资料：[1] 各公司官方新闻稿及公开资料本文来自药明康德内容团队，欢迎个人转发至朋友圈，谢绝媒体或机构未经授权以任何形式转载至其他平台。转载授权及其他合作需求，请联系wuxi_media@wuxiapptec.com。免责声明：药明康德内容团队专注介绍全球生物医药健康研究进展。本文仅作信息交流之目的，文中观点不代表药明康德立场，亦不代表药明康德支持或反对文中观点。本文也不是治疗方案推荐。如需获得治疗方案指导，请前往正规医院就诊。