Open-label Safety Study in Adults and Adolescents With Haemophilia A With and Without FVIII Inhibitors Switching Directly From Emicizumab Prophylaxis to NNC0365-3769 (Mim8) Prophylaxis

This study is looking at how safe it is to switch from emicizumab to Mim8, in people with haemophilia A. Mim8 is a new medicine that is used to prevent bleeding episodes in people with haemophilia A. Mim8 works by replacing the function of the missing clotting factor VIII (FVIII). Mim8 will be injected under the skin using a pen-injector either once every week, once every two weeks or once every month. The participants will be trained in using the pen injector. The participants can choose themselves, in collaboration with the study doctor how often they get Mim8 in this study. When the participant will get their first Mim8 injection depends on their current treatment with emicizumab. The participants will get their first Mim8 injection at Visit 2. Participants will have between 6 and 27 Mim8 injections. The total number of injections participants will have depends on their dosing frequency. The study will last for about 6-12 months. While taking part in this study, there are some restrictions about what medicine participant can use. The study doctor will tell the participants more about this. In case the participants experience bleeds, these can be treated with additional haemostatic medicine as agreed with the study doctor. Female participants cannot take part if they are pregnant, breast-feeding or plan to get pregnant during the study period.

开始日期2023-06-26

申办/合作机构

Novo Nordisk A/S

NCT05685238

/ Active, not recruiting临床3期

Open-label, Long-term Safety and Efficacy Study of Mim8 in Participants With Haemophilia A With or Without Inhibitors

This study is looking at how Mim8 works in people with haemophilia A, who either have inhibitors or do not have inhibitors. Mim8 is a new medicine that will be used to avoid bleeding episodes. Mim8 works by replacing the function of the missing clotting factor VIII (FVIII). The study will last for up to 5.5 years. The duration of the study depends on when the participant enrolled in this study. The study will end if Mim8 is approved and marketed in participant's country during the study, or the study will end in June 2028, whichever comes first. Participants will get up to 262 injections; the number of injections depends on how often participants will get injections and how long time participants take part in the study. While taking part in this study, there are some restrictions about what medicine participants can use. The study doctor will tell the participants more about this. In case the participants experience bleeds, these can be treated with additional haemostatic medicine as agreed with the study doctor. Female participants cannot take part if they are pregnant, breast-feeding or plan to get pregnant during the study period.

开始日期2023-02-13

申办/合作机构

Novo Nordisk A/S

NCT05681845

/ Completed临床1期

A Study to Demonstrate Bioequivalence of a Single Subcutaneous Administration of NNC0365-3769 (Mim8) With the DV3407-C1 Pen Injector Versus a Syringe and Enhanced Cartridge in Healthy Male Participants

Novo Nordisk is developing the study medicine Mim8 for the treatment of haemophilia A. The study aims to show similar levels of Mim8 in blood when using a new pen injector, called DV3407-C1 pen injector, and when using a syringe and cartridge. The new pen injector is intended to facilitate the administration of Mim8 for patients with haemophilia A. The participants will get Mim8 as injection under the skin (subcutaneously) of the belly using the DV3407-C1 pen injector and a needle (hereinafter referred to as pen injector) or using a needle and syringe from a cartridge (hereafter called syringe and cartridge). The participants will receive one injection with Mim8, either with the DV3407-C1 pen injector or with a syringe and cartridge. The study participation will last up to 20 weeks. Only healthy men can take part in the study.

开始日期2023-01-02

申办/合作机构

Novo Nordisk A/S

100 项与 Denecimig 相关的临床结果

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100 项与 Denecimig 相关的专利（医药）

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项与 Denecimig 相关的文献（医药）

2025-12-31·mAbs

Antibodies to watch in 2025

Review

作者: Kapoor, Vaishali ; Kaplon, Hélène ; Crescioli, Silvia ; Visweswaraiah, Jyothsna ; Reichert, Janice M. ; Wang, Lin

The commercial development of antibody therapeutics is a global enterprise involving thousands of biopharmaceutical firms and supporting service organizations. To date, their combined efforts have resulted in over 200 marketed antibody therapeutics and a pipeline of nearly 1,400 investigational product candidates that are undergoing evaluation in clinical studies as treatments for a wide variety of diseases. Here, we discuss key events in antibody therapeutics development that occurred during 2024 and forecast key events related to the late-stage clinical pipeline that may occur in 2025. In particular, we report on 21 antibody therapeutics granted a first approval in at least one country or region during 2024, including bispecific antibodies tarlatamab (IMDELLTRA®), zanidatamab (Ziihera®), zenocutuzumab (BIZENGRI®), odronextamab (Ordspono®), ivonescimab (®), and antibody-drug conjugate (ADC) sacituzumab tirumotecan (®). We also discuss 30 investigational antibody therapeutics for which marketing applications were undergoing review by at least one regulatory agency, as of our last update on December 9, 2024, including ADCs datopotamab deruxtecan, telisotuzumab vedotin, patritumab deruxtecan, trastuzumab botidotin, becotatug vedotin, and trastuzumab rezetecan. Of 178 antibody therapeutics we include in the late-stage pipeline, we summarize key data for 18 for which marketing applications may be submitted by the end of 2025, such as bi- or multispecific antibodies denecimig, sonelokimab, erfonrilimab, and anbenitamab. Key trends in the development and approval of antibody formats such as bispecifics and ADCs, as well as clinical-phase transition and global approval success rates for these antibody formats, are reported.

2025-10-01·Research and Practice in Thrombosis and Haemostasis

FRONTIER1 multiple ascending dose extension: a safety, tolerability, pharmacokinetics, and pharmacodynamics study of Mim8 in people with hemophilia A

Article

作者: Laursen, Peter Nørkjær ; López-Jaime, Francisco J ; Mahlangu, Johnny ; Chowdary, Pratima ; Ong Clausen, Wan Hui ; Lentz, Steven R ; Windyga, Jerzy ; Gil, Lidia

Background:

Mim8 (denecimig) is a next-generation, factor VIIIa mimetic, human bispecific antibody for hemophilia A. Mim8 was well tolerated in the phase 1/2 FRONTIER1 (NCT04204408) dose-escalation part.

Objectives:

Report Mim8 long-term safety in the FRONTIER1 multiple ascending dose (MAD) extension phase.

Methods:

People (≥12 years) with severe hemophilia A with/without inhibitors who completed the 12-week FRONTIER1 MAD main phase enrolled in the extension and continued Mim8 once weekly or once every 4 weeks for up to 148 weeks; patients could switch doses or transition to maintenance doses designed to achieve target exposure in phase 3 trials. Primary endpoint: treatment-emergent adverse events (TEAEs). Secondary endpoints: injection-site reactions, anti-Mim8 antibodies.

Results:

Forty-one patients entered the extension; n = 39 switched to Mim8 maintenance doses administered using tiered dosing by body weight (30-<60 kg or ≥60 kg): n = 31 received once-weekly dosing (6 or 11 mg); n = 9 received once-every 4-weeks dosing (30 or 46 mg); n = 1 patient switched between doses. Total exposure: 69.5 patient-years. Thirty-five patients experienced 174 TEAEs. Seven serious TEAEs occurred (none Mim8-related). TEAE rate, type, and severity were not dose-dependent. Of 3128 injections, 6 (0.2%) injection-site reactions were recorded in 6 (14.6%) patients. There was no evidence of anti-Mim8 antibodies. Mim8 exposure and peak thrombin levels remained consistent across cohorts and maintenance doses. No dose-dependent changes in D-dimer, prothrombin fragment 1 + 2, or fibrinogen levels were observed. Most patients experienced no bleeds during maintenance dosing.

Conclusion:

Mim8 was well tolerated, with no safety concerns nor anti-Mim8 antibodies during the FRONTIER1 MAD extension, supporting phase 3 development of Mim8 once weekly and once every 4 weeks.

2025-10-01·Research and Practice in Thrombosis and Haemostasis

Comparative ex vivo analysis of Mim8 in thrombin generation assays triggered by activated factor XI or tissue factor

Article

作者: Latendorf, Ties ; Ezban, Mirella ; Stehr, Rielana ; Ong Clausen, Wan Hui ; Lund, Jacob

Background:

Mim8 is a next-generation activated factor (F)VIII-mimetic, bispecific antibody, currently in clinical development for the prophylactic treatment of hemophilia A. To evaluate the ex vivo pharmacodynamic effects of Mim8, a thrombin generation assay (TGA) using activated FXI (FXIa) as trigger was employed in the phase 1/2 FRONTIER1 (NCT04204408) study, whereas tissue factor (TF) was used in the phase 3 FRONTIER2 (NCT05053139) study.

Objectives:

To compare the pharmacodynamic results from FRONTIER1 and FRONTIER2 by evaluating Mim8 ex vivo and in vitro thrombin generation (TG) responses in TGAs triggered by either FXIa or TF.

Methods:

TG was assessed ex vivo in 125 plasma samples from 34 participants with hemophilia A from FRONTIER1 and FRONTIER2 studies, using 2 TGAs triggered with either 1 pM TF or 8 mU/mL FXIa. Additionally, TG was assessed in vitro in FVIII-deficient plasma spiked with Mim8 in both TGAs. Bland-Altman plots were utilized to compare TG responses.

Results:

Within the tested Mim8 concentration range, a constant TG response ratio was observed between the 2 triggers. TF-triggered peak thrombin levels were approximately 3 to 4 times lower than those triggered by FXIa. Both triggers exhibited similar endogenous thrombin potential responses, although FXIa showed slightly higher values. These differences were consistent in both ex vivo and in vitro TG assessments.

Conclusion:

While both assays are considered relevant for assessing Mim8 pharmacodynamic response, systematic and consistent differences between TGAs triggered by FXIa and TF were observed, indicating different kinetics in the intrinsic and extrinsic pathways.

项与 Denecimig 相关的新闻（医药）

2025-12-05

·BioPharmaDive

Hemophilia gene therapies are struggling on the market, even as innovation soars

Hemophilia gene therapies arrived a few years ago with blockbuster expectations. But now, one therapy has been pulled from the market while two are fighting an uphill battle for reimbursement and a solid footing, according to Michael Recht, chief medical and scientific officer for the National Bleeding Disorders Foundation.Analysts predicted Biomarin Pharmaceuticals hemophilia A gene therapy Roctavian would hit peak sales of $2.2 billion. Instead, Biomarin is now looking to offload the drug due to weak commercial uptake.In a similar position, Pfizer pulled its hemophilia B treatmentBeqvez from the market while CSL Behrings Hemgenix, which was first to market, is also significantly underperforming.Dismal sales came as a surprise to many who saw hemophilia gene therapies as a major scientific leap forward and a promising one-time alternative that could free patients from frequent clotting factor infusions or injections.But gene therapies come with a high price tag. Hemgenix became the worlds most expensive drug when it was approved in 2022, with a list price of $3.5 million per dose. Still, treatment could be worth the sticker shock, and CSL estimates that Hemgenix could save up to $5.8 million per person by eliminating the need for once- or twice-weekly clotting factor injections.Its a value-laden intervention, Recht said. Its just shockingly expensive.Reimbursement hurdlesPayers have balked at the bill for gene therapy treatments, although Recht said all of his interested patients have been able to get them.As part of an advocacy organization, when weve spoken to the payers, what we've heard is, Well, the average time someone is on a specific health plan, since the Affordable Care Act has been around, is under two years. So, why should we pay for lifetime therapy when that person might be on our plan for under two years? Recht said.Still, an NBDF data analysis found that rapid plan hopping is not as common as insurers claim. Since the introduction of the Affordable Care Act, people typically only change plans when they change jobs, he said.Im very hopeful there will be products to address the unmet needs of the inheritable bleeding disorders population.Dr. Michael RechtChief medical and scientific officer, NBDFBeyond reimbursement, structural disincentives further complicate adoption. The Hemophilia Treatment Center 340B Factor Program offers certified organizationsdiscounts for medications and supplies. The centers can then reinvest those savings back into facility programs.The money for gene therapy, however, typically gets paid to a hospital, according to Recht.So the hospital would get all the income from the gene therapy, and the treatment center could lose $500,000 to $750,000 a year in revenues from someone on prophylaxis.This discourages partaking in gene therapy, he added.To me, that is not a compelling reason at all, and potentially an unethical reason if this is the right drug [for a patient], Recht said. But this is what I've heard from some of my colleagues.Making the gene therapy leapWhile gene therapy isnt the best option for all patients, it could be for a subset. But many can afford to take a wait-and-see approach because other treatments are effective.There are some really great non-gene therapy options for people with hemophilia A, Recht said. [Hemlibra] is a great drug, and has changed the way Ive treated people.Many patients also dont qualify for treatment. Hemgenix targets hemophilia B, which makes up only about 15% of overall cases, and some patients, including children and those with certain liver conditions, are excluded.For Beqvez, uptake obstacles proved too difficult to overcome.Beqvez was a perfectly good product, Recht said. But as the second to market with the results of their Phase 3 maybe not as robust as the Hemgenix clinical trial, I think that played a big role.Roctavian, a hemophilia A option, targets a broader pool of patients, but has also faced challenges.The durability is the biggest issue, Recht said, noting that its still uncertain how long the treatment will last, although recent data showed it still worked after five years. To get 10 years of not having hemophilia, that's still 10 years of not having hemophilia.The markets futureDespite the market struggles, the future for hemophilia patients is promising.I'm very hopeful there will be products to address the unmet needs of the inheritable bleeding disorders population, Recht said.Novo Nordisk, for example, recently submitted anapplication for Mim8, a drug that Recht predicts could be as safe and effective as Hemlibra, but potentially more appealing to patients because it uses a pen injector.Interest in gene therapy hasnt waned, and companies are still looking for a way in. Be Biopharma is in Phase 1 with BE-101, a cell therapy with a twist.Theyre putting the vector into a persons own B cells, Recht said. The fascinating part about this is if this is safe and it works, it will be re-dosable because you wont have an immune response like you do with an adeno-associated virus vector.Startups are also testing gene editing for hemophilia. However, many of these treatments are years from reaching patients.While determining the best uses for all the existing medications remains a challenge, there are more options than ever.Where we are today in the 30-ish years of my career is a miracle, he said. '

基因疗法临床3期临床1期细胞疗法

2025-12-03

·LONGPORT

由于血友病患病率的增加、新药的强劲吸收以及高成本基因疗法的出现，预计血友病 A 市场将在预测期（2025-2034 年）以 2

血友病 A 市场预计在 2025 年至 2034 年间以 2.9% 的年均增长率增长，主要受患病率上升、新药物采用和高成本基因疗法的推动。诺和诺德公司和辉瑞等主要参与者正在开发有前景的治疗方案。2024 年市场规模为 122 亿美元，美国占据最大市场份额。基因疗法的进展和监管批准正在扩展治疗选择，包括非因子疗法和 siRNA 药物。重点是通过按需和预防性疗法来减少出血并发症血友病 A 市场的动态预计将主要因患病率的增加和即将推出的疗法（如 Mim8（诺和诺德）、DTX201（Pebocotocogene camaparvovec, BAY2599023 [Ultragenyx Pharmaceutical]）、giroctocogene fitelparvovec（SB-525 [Sangamo Therapeutics]）等）而发生变化。 , /PRNewswire/ -- DelveInsight 的血友病 A 市场洞察报告包括对当前治疗实践、血友病 A 新兴药物、各个疗法的市场份额以及 2020 年至 2034 年的当前和预测市场规模的全面理解，市场细分为主要市场 [美国、EU4（德国、法国、意大利和西班牙）、英国和日本]。血友病 A 市场概述 2024 年，血友病 A 的市场规模在主要市场 [美国、EU4（德国、法国、意大利和西班牙）、英国和日本] 中达到了 122 亿美元。美国占据了最大的血友病 A 治疗市场规模，约占 2024 年 7MM 总市场规模的 48%，相比其他主要市场，包括 EU4 国家、英国和日本。 HEMLIBRA 的增长曲线（美国 2025 年第一季度同比销售）在美国保持平稳，表明来自新疗法的竞争加剧。 2024 年，7MM 中被诊断的血友病 A 患者数量约为 49,500 例。领先的血友病 A 公司，如诺和诺德、Ultragenyx Pharmaceutical、Sangamo Therapeutics、ASC Therapeutics、罗氏、中外制药等，正在开发新的血友病 A 治疗药物，这些药物将在未来几年内进入血友病 A 市场。临床试验中有前景的血友病 A 疗法包括 Mim8、DTX201（Peboctocogene camaparvovec, BAY2599023）、Giroctocogene fitelparvovec（SB-525）、ASC618、NXT007/RG6512 等。发现新的血友病 A 治疗 @ 血友病 A 治疗市场推动血友病 A 市场增长的关键因素血友病 A 目标患者群体的增加预计血友病 A 的诊断患病率将从 2024 年的 49K 上升到 2034 年的 50K。这一病例增加主要是由于诊断能力的提高和对疾病认识的增强。基因治疗的进展基因治疗已成为血友病 A 治疗中的一种变革性方法。值得注意的是，BioMarin 的 ROCTAVIAN 已在美国和欧盟获得批准，提供一次性输注，能够长期纠正 VIII 因子缺乏症。辉瑞的基因治疗 giroctocogene fitelparvovec 在血友病 A 的晚期试验中也显示出良好的结果，显著减少出血事件，并优于传统治疗。监管批准与血友病 A 市场扩展最近的监管批准增强了血友病 A 治疗的可用性。例如，美国 FDA 批准了辉瑞的每周一次注射 HYMPAVZI（于 2024 年 10 月），用于 12 岁及以上的血友病 A 患者，旨在预防或减少出血事件。同样，赛诺菲的 QFITLIA（于 2025 年 3 月）是一种每两个月注射一次的皮下治疗，已获批准用于 12 岁及以上的血友病 A 或 B 患者。非因子血友病 A 疗法的出现血友病 A 的护理已扩展到两种前沿的非因子方法：抗 TFPI 疗法（辉瑞的 HYMPAVZI 和诺和诺德的 ALHEMO）和 siRNA 疗法（赛诺菲的 QFITLIA）。强劲的血友病 A 临床试验活动多家血友病 A 公司正在积极开发新兴疗法，包括诺和诺德（Mim8、ALHEMO）、Sangamo Therapeutics（Giroctocogene fitelparvovec）、罗氏/中外制药（NXT007）、ASC Therapeutics（ASC-618）、Ultragenyx（DTX201）等。血友病 A 市场分析当前血友病 A 治疗策略的主要目标是最小化因出血导致的关节、组织或器官并发症。随着技术的不断进步和医学理解的提高，患者现在可以获得多种治疗选择。治疗通常以 “按需” 或 “预防” 方式进行，预防性治疗越来越成为首选方法。美国 FDA 已批准多种重组 VIII 因子产品用于血友病 A 管理，包括 HELIXATE FS、RECOMBINATE、KOGENATE FS、ADVATE、REFACTO、ELOCTATE、NUWIQ、ADYNOVATE、KOVALTRY、JIVI 和 XYNTHA。此外，血浆衍生的 VIII 因子产品，如 MONARC-M、MONOCLATE-P、HEMOFIL M 和 KOATE-DVI 仍然可用。最新的补充产品 QFITLIA（fitusiran）是一种于 2025 年批准的 siRNA 基础疗法，通过降低抗凝血酶水平来增强血块形成。它每年仅需六次注射，且对抑制剂和非抑制剂患者均有效。 HEMLIBRA 已成为血友病 A 患者（有抑制剂）的首选预防性治疗，尽管免疫耐受诱导（ITI）治疗仍然是金标准。对于那些在 ITI 中遇到困难的患者，HEMLIBRA 提供了一种替代方案，在 ITI 期间需要更少频率的 FVIII 给药。展望未来，血友病管理正向延长半衰期因子疗法和前沿模式转变，包括 siRNA 药物、双特异性抗体和基因治疗。随着新型长效因子产品和下一代治疗技术的推出，竞争格局预计将扩大。血友病 A 竞争格局血友病 A 的临床试验领域有多种药物处于中期和晚期开发阶段，预计将在不久的将来获得批准。新兴的市场提供了多样化的治疗选择，包括 Mim8（诺和诺德）、DTX201（Pebocotocogene camaparvovec）、BAY2599023（Ultragenyx Pharmaceutical）、giroctocogene fitelparvovec（SB-525，Sangamo Therapeutics）等。诺和诺德的 Mim8 是一种先进的 FVIIIa 模拟双特异性抗体，旨在为血友病 A 患者提供持续的止血控制，方便的每周或每月一次的预防性给药，无论是否存在抑制剂。Mim8 通过皮下注射给药，激活后通过桥接激活的 IXa 和 X 因子（FIXa/FX）来发挥作用，有效补偿 FVIII 的缺失。这恢复了正常的凝血酶生成，促进有效的血液凝固。 Ultragenyx Pharmaceutical 的 DTX201 是首个临床阶段的血友病 A 基因疗法，采用来自 AAVhu37 血清型的腺相关病毒（AAV）载体。它是一种非复制的 AAV 载体，携带编码 B 域缺失 FVIII 的单链 DNA 基因组，由肝脏特异性启动子和增强子序列驱动，优化以实现强大的转基因表达。AAVhu37 外壳是肝向性 E 类家族的一部分，因其在临床前研究中显示出能够有效实现肝脏靶向 FVIII 基因递送、最佳生物分布和持久的 FVIII 表达而被选中。这些新兴疗法的预期推出将改变未来几年血友病 A 市场的格局。随着这些前沿疗法的不断成熟和获得监管批准，预计它们将重塑血友病 A 市场，提供新的护理标准，并为医疗创新和经济增长开辟机会。了解更多关于 FDA 批准的血友病 A 基因疗法的信息 @ FDA 批准的 AAV 基因疗法血友病 A 血友病 A 市场的最新动态在 2025 年 7 月，诺和诺德宣布 FDA 已批准 ALHEMO 注射剂用于每日一次的预防性使用，以防止或减少 12 岁及以上血友病 A 或 B（HA/HB）患者的出血事件。此批准将该药物的适应症扩展至最初于 2024 年 12 月批准的有抑制剂的 HA/HB 患者。在 2025 年 6 月，辉瑞公司报告了其第 3 期 BASIS 试验（NCT03938792）的积极顶线结果，该试验评估了在有抑制剂的血友病 A 或 B 成人和青少年中使用 HYMPAVZI（marstacimab）。在 **2025 年 5 月，**FDA 批准了抗血友病因子（重组），PEG 化-aucl（Jivi；拜耳）用于治疗 7 岁及以上的血友病 A 儿童患者。该批准得到了 Alfa-PROTECT 和 PROTECT Kids 试验结果的支持。在 2025 年 3 月，Alnylam Pharmaceuticals 宣布 FDA 批准 Qfitlia™（fitusiran），这是美国批准的第六种 Alnylam 发现的 RNAi 治疗药物。它是首个也是唯一一个降低抗凝血酶（AT）的治疗，旨在促进凝血酶生成，重新平衡止血，并防止出血。什么是血友病 A？血友病 A 是一种遗传性出血性疾病，由于凝血因子 VIII 的缺乏或低水平引起。多年来，标准治疗一直是 FVIII 替代疗法。这始于从捐赠的全血中提取 FVIII，后来发展为血浆衍生的 FVIII，现在主要通过重组人 FVIII（rFVIII）产品进行管理，这些产品改变了血友病的护理。虽然该病通常在出生时被诊断，但也可能在生命后期获得，当免疫系统产生中和凝血因子的抗体时，这是一种罕见的情况，称为获得性血友病。血友病 A 流行病学细分血友病 A 流行病学预测部分提供了对历史和当前血友病 A 患者群体的洞察，以及主要市场的预测趋势。在 7 个主要市场中，美国的血友病 A 确诊病例最多，预计在 2024 年将达到近 14,900 例。这些病例预计在未来几年将增加。血友病 A 治疗市场报告提供了 2020 年至 2034 年研究期间的流行病学分析，细分为：血友病 A 的总确诊病例有抑制剂或无抑制剂的血友病 A 确诊病例按年龄细分的血友病 A 确诊病例按严重程度细分的血友病 A 确诊病例血友病 A 市场报告指标详细信息研究期间 2020–2034 覆盖范围 7 个主要市场 [美国、EU4（德国、法国、意大利和西班牙）、英国和日本]。血友病 A 市场年均增长率 2.9 % 2024 年血友病 A 市场规模 122 亿美元主要血友病 A 公司诺和诺德、Ultragenyx Pharmaceutical、Sangamo Therapeutics、ASC Therapeutics、罗氏、中外制药、赛诺菲、Alnylam Pharmaceuticals、辉瑞、基因泰克、生物马林制药、HEMA 生物制剂、LFB 制药、Octapharma、CSL Behring、Octapharma、拜耳、武田等主要血友病 A 疗法 Mim8、DTX201（Pebocotocogene camaparvovec、BAY2599023）、Giroctocogene fitelparvovec（SB-525）、ASC618、NXT007/RG6512、QFITLIA、HYMPAVZI、ALHEMO、HEMLIBRA、ALTUVIIIO、ROCTAVIAN、SEVENFACT/CEVENFACTA、ESPEROCT、WILATE、JIVI、ADYNOVATE、AFSTYLA、NUWIQ、FEIBA、KOVALTRY、OBIZUR、ELOCTATE/Elocta、XYNTHA 等血友病 A 市场报告的范围治疗评估：血友病 A 当前市场及新兴疗法血友病 A 市场动态：新兴血友病 A 药物的关键市场预测假设及市场前景竞争情报分析： SWOT 分析及市场进入策略未满足的需求、KOL 观点、分析师观点、血友病 A 市场准入及报销下载报告以评估血友病治疗公司 HEMLIBRA 在女性血友病中的应用 @ 皇家血友病 A 或 B 目录 1 血友病 A 市场关键洞察 2 血友病 A 市场报告介绍 3 执行摘要 4 关键事件 5 流行病学及市场预测方法论 6 血友病 A 市场概览 6.1 2020 年血友病 A 按疗法的市场份额（%）分布 6.2 2034 年血友病 A 按疗法的市场份额（%）分布 6.3 2020 年血友病 A 按抑制剂的市场份额（%）分布 6.4 2034 年血友病 A 按抑制剂的市场份额（%）分布 6.5 2020 年血友病 A 按非抑制剂的市场份额（%）分布 6.6 2034 年血友病 A 按非抑制剂的市场份额（%）分布 7 血友病 A：疾病背景及概述 7.1 介绍 7.2 血友病 A 的症状和体征 7.3 血友病 A 的遗传模式 7.4 血友病 A 的分子发病机制 7.5 血友病 A 的病理生理学 7.6 血友病 A 的风险因素 7.7 血友病 A 的诊断 8 血友病 A 治疗算法、当前治疗及医疗实践 9 流行病学及患者人群 9.1 关键发现 9.2 假设及理由：7MM 9.3 7MM 中血友病 A 的总诊断流行病例 9.4 美国 9.4.1 美国血友病 A 的总诊断流行病例 9.4.2 美国有抑制剂和无抑制剂的血友病 A 诊断流行病例 9.4.3 美国血友病 A 的年龄特定诊断流行病例 9.4.4 美国血友病 A 的严重程度特定流行病例 9.5 EU4 及英国 9.6 日本流行病学 10 血友病 A 患者旅程 11 已上市的血友病 A 疗法 11.1 已上市疗法的关键交叉 11.2 QFITLIA（fitusiran）：赛诺菲/阿尔尼拉姆制药 11.2.1 产品描述 11.2.2 监管里程碑 11.2.3 其他开发活动 11.2.4 临床开发 11.2.4.1 临床试验信息 11.2.5 安全性和有效性 11.3 HYMPAVZI（marstacimab-hncq）：辉瑞 11.4 ALHEMO（concizumab）：诺和诺德 11.5 HEMLIBRA（emicizumab-kxwh）：中外制药/基因泰克/罗氏 11.6 ALTUVIIIO（抗血友病因子 [重组]，Fc-VWF-XTEN 融合蛋白-ehtl）：赛诺菲/索比 11.7 ROCTAVIAN（valoctocogene roxaparvovec）：百奥明制药 11.8 SEVENFACT/CEVENFACTA [凝血因子 VIIa（重组）-jncw]: HEMA 生物制药/LFB 制药 11.9 ESPEROCT（N8-GP; Turoctocog alfa pegol）：诺和诺德 11.10 WILATE：Octapharma 11.11 JIVI（BAY94-9027）：拜耳 11.12 ADYNOVATE（Adynovi; BAX 855）：武田制药 11.13 AFSTYLA（Lonoctocog alfa）：CSL 贝赫林 11.14 NUWIQ（simoctocog alfa）：Octapharma 11.15 FEIBA：武田 11.16 KOVALTRY（BAY 81-8973）：拜耳 11.17 OBIZUR（susoctocog alfa）：武田 11.18 ELOCTATE/Elocta（efmoroctocog alfa）：赛诺菲/索比 11.19 XYNTHA（ReFacto AF）：辉瑞 12 新兴血友病 A 药物 12.1 关键交叉竞争 12.2 Mim8：诺和诺德 12.2.1 产品描述 12.2.2 其他开发活动 12.2.3 临床开发 12.2.3.1 临床试验信息 12.2.4 安全性和有效性 12.2.5 分析师观点 12.3 DTX201（Peboctocogene camaparvovec，BAY2599023）：Ultragenyx 制药 12.4 Giroctocogene fitelparvovec（SB-525）：Sangamo Therapeutics 12.5 ASC618：ASC Therapeutics 12.6 NXT007/RG6512：罗氏/中外制药 13 血友病 A 市场：7MM 分析 13.1 关键发现 13.2 血友病 A 市场前景 13.3 血友病 A 市场关键预测假设 13.4 联合分析 13.5 7MM 中血友病 A 的总市场规模按国家划分 13.6 美国血友病 A 市场规模 13.6.1 美国血友病 A 的总市场规模 13.6.2 美国血友病 A 按疗法的市场规模 13.7 EU4 及英国血友病 A 市场规模 13.8 日本血友病 A 市场规模 14 血友病 A 市场未满足的需求 15 血友病 A 市场 SWOT 分析 16 KOL 对血友病 A 的观点 17 血友病 A 市场准入及报销 17.1 美国 17.2 EU4 及英国 17.3 日本 17.4 血友病 A 的市场准入及报销 18 参考文献 19 血友病 A 市场报告方法论相关报告血友病 A 临床试验分析血友病 A 管线洞察 – 2025 报告提供了关于管线景观、管线药物概况的全面洞察，包括临床和非临床阶段产品，以及关键的血友病 A 公司，包括江苏基因科学公司、武田、Gritgen Therapeutics 有限公司、苏州阿尔法玛生物科技有限公司、诺和诺德、辉瑞、SK Plasma 有限公司、霍夫曼 - 拉罗氏、上海信念生物医药有限公司、ASC Therapeutics、Biocad、Expression Therapeutics, LLC、GCBiopharma、Amarna Therapeutics、Cabaletta Bio 等。获得性血友病 A 市场获得性血友病 A 市场洞察、流行病学和市场预测 – 2032 报告提供了对该疾病的深入理解，包括历史和预测的流行病学，以及市场趋势、市场驱动因素、市场障碍和主要获得性血友病 A 公司，包括辉瑞、诺和诺德、Hema Biologics、霍夫曼 - 拉罗氏、武田等。血友病市场血友病市场洞察、流行病学和市场预测 – 2032 报告提供了对该疾病的深入理解，包括历史和预测的流行病学，以及市场趋势、市场驱动因素、市场障碍和主要血友病公司，包括 Centessa Pharmaceuticals、Alnylam Pharmaceuticals、ASC Therapeutics、Spark Therapeutics、Freeline Therapeutics、BioMarin Pharmaceutical、Staidson Beijing BioPharmaceuticals、辉瑞、Sangamo Therapeutics、Amunix、Bioverativ、诺和诺德等。血友病 B 市场血友病 B 市场洞察、流行病学和市场预测 – 2032 报告提供了对该疾病的深入理解，包括历史和预测的流行病学，以及市场趋势、市场驱动因素、市场障碍和主要血友病 B 公司，包括 UniQure Biopharma B.V.、CSL Behring、辉瑞、Spark Therapeutics、Genzyme（赛诺菲公司）、Alnylam Pharmaceuticals、诺和诺德、UniQure Biopharma B.V.、ApcinteX Ltd、Freeline Therapeutics、Sangamo Therapeutics 等。关于 DelveInsight DelveInsight 是一家领先的商业咨询和市场研究公司，专注于生命科学领域。它通过提供全面的端到端解决方案来支持制药公司，提高其业绩。通过我们的订阅平台 PharmDelve，轻松访问所有医疗保健和制药市场研究报告。联系我们 Shruti Thakur [email protected] +14699457679 www.delveinsight.com Logo - https://mma.prnewswire.com/media/1082265/3528414/DelveInsight_Logo.jpg 来源 DelveInsight Business Research, LLP 血友病 A 市场的动态预计将主要因患病率的增加和即将推出的疗法（如 Mim8（诺和诺德）、DTX201（Pebocotocogene camaparvovec, BAY2599023 [Ultragenyx Pharmaceutical]）、giroctocogene fitelparvovec（SB-525 [Sangamo Therapeutics]）等）而发生变化。 , /PRNewswire/ -- DelveInsight 的血友病 A 市场洞察报告包括对当前治疗实践、血友病 A 新兴药物、各个疗法的市场份额以及 2020 年至 2034 年的当前和预测市场规模的全面理解，市场细分为主要市场 [美国、EU4（德国、法国、意大利和西班牙）、英国和日本]。血友病 A 市场概述 2024 年，血友病 A 的市场规模在主要市场 [美国、EU4（德国、法国、意大利和西班牙）、英国和日本] 中达到了 122 亿美元。美国占据了最大的血友病 A 治疗市场规模，约占 2024 年 7MM 总市场规模的 48%，相比其他主要市场，包括 EU4 国家、英国和日本。 HEMLIBRA 的增长曲线（美国 2025 年第一季度同比销售）在美国保持平稳，表明来自新疗法的竞争加剧。 2024 年，7MM 中被诊断的血友病 A 患者数量约为 49,500 例。领先的血友病 A 公司，如诺和诺德、Ultragenyx Pharmaceutical、Sangamo Therapeutics、ASC Therapeutics、罗氏、中外制药等，正在开发新的血友病 A 治疗药物，这些药物将在未来几年内进入血友病 A 市场。临床试验中有前景的血友病 A 疗法包括 Mim8、DTX201（Peboctocogene camaparvovec, BAY2599023）、Giroctocogene fitelparvovec（SB-525）、ASC618、NXT007/RG6512 等。发现新的血友病 A 治疗 @ 血友病 A 治疗市场推动血友病 A 市场增长的关键因素血友病 A 目标患者群体的增加预计血友病 A 的诊断患病率将从 2024 年的 49K 上升到 2034 年的 50K。这一病例增加主要是由于诊断能力的提高和对疾病认识的增强。基因治疗的进展基因治疗已成为血友病 A 治疗中的一种变革性方法。值得注意的是，BioMarin 的 ROCTAVIAN 已在美国和欧盟获得批准，提供一次性输注，能够长期纠正 VIII 因子缺乏症。辉瑞的基因治疗 giroctocogene fitelparvovec 在血友病 A 的晚期试验中也显示出良好的结果，显著减少出血事件，并优于传统治疗。监管批准与血友病 A 市场扩展最近的监管批准增强了血友病 A 治疗的可用性。例如，美国 FDA 批准了辉瑞的每周一次注射 HYMPAVZI（于 2024 年 10 月），用于 12 岁及以上的血友病 A 患者，旨在预防或减少出血事件。同样，赛诺菲的 QFITLIA（于 2025 年 3 月）是一种每两个月注射一次的皮下治疗，已获批准用于 12 岁及以上的血友病 A 或 B 患者。非因子血友病 A 疗法的出现血友病 A 的护理已扩展到两种前沿的非因子方法：抗 TFPI 疗法（辉瑞的 HYMPAVZI 和诺和诺德的 ALHEMO）和 siRNA 疗法（赛诺菲的 QFITLIA）。强劲的血友病 A 临床试验活动多家血友病 A 公司正在积极开发新兴疗法，包括诺和诺德（Mim8、ALHEMO）、Sangamo Therapeutics（Giroctocogene fitelparvovec）、罗氏/中外制药（NXT007）、ASC Therapeutics（ASC-618）、Ultragenyx（DTX201）等。血友病 A 市场分析当前血友病 A 治疗策略的主要目标是最小化因出血导致的关节、组织或器官并发症。随着技术的不断进步和医学理解的提高，患者现在可以获得多种治疗选择。治疗通常以 “按需” 或 “预防” 方式进行，预防性治疗越来越成为首选方法。美国 FDA 已批准多种重组 VIII 因子产品用于血友病 A 管理，包括 HELIXATE FS、RECOMBINATE、KOGENATE FS、ADVATE、REFACTO、ELOCTATE、NUWIQ、ADYNOVATE、KOVALTRY、JIVI 和 XYNTHA。此外，血浆衍生的 VIII 因子产品，如 MONARC-M、MONOCLATE-P、HEMOFIL M 和 KOATE-DVI 仍然可用。最新的补充产品 QFITLIA（fitusiran）是一种于 2025 年批准的 siRNA 基础疗法，通过降低抗凝血酶水平来增强血块形成。它每年仅需六次注射，且对抑制剂和非抑制剂患者均有效。 HEMLIBRA 已成为血友病 A 患者（有抑制剂）的首选预防性治疗，尽管免疫耐受诱导（ITI）治疗仍然是金标准。对于那些在 ITI 中遇到困难的患者，HEMLIBRA 提供了一种替代方案，在 ITI 期间需要更少频率的 FVIII 给药。展望未来，血友病管理正向延长半衰期因子疗法和前沿模式转变，包括 siRNA 药物、双特异性抗体和基因治疗。随着新型长效因子产品和下一代治疗技术的推出，竞争格局预计将扩大。血友病 A 竞争格局血友病 A 的临床试验领域有多种药物处于中期和晚期开发阶段，预计将在不久的将来获得批准。新兴的市场提供了多样化的治疗选择，包括 Mim8（诺和诺德）、DTX201（Pebocotocogene camaparvovec）、BAY2599023（Ultragenyx Pharmaceutical）、giroctocogene fitelparvovec（SB-525，Sangamo Therapeutics）等。诺和诺德的 Mim8 是一种先进的 FVIIIa 模拟双特异性抗体，旨在为血友病 A 患者提供持续的止血控制，方便的每周或每月一次的预防性给药，无论是否存在抑制剂。Mim8 通过皮下注射给药，激活后通过桥接激活的 IXa 和 X 因子（FIXa/FX）来发挥作用，有效补偿 FVIII 的缺失。这恢复了正常的凝血酶生成，促进有效的血液凝固。 Ultragenyx Pharmaceutical 的 DTX201 是首个临床阶段的血友病 A 基因疗法，采用来自 AAVhu37 血清型的腺相关病毒（AAV）载体。它是一种非复制的 AAV 载体，携带编码 B 域缺失 FVIII 的单链 DNA 基因组，由肝脏特异性启动子和增强子序列驱动，优化以实现强大的转基因表达。AAVhu37 外壳是肝向性 E 类家族的一部分，因其在临床前研究中显示出能够有效实现肝脏靶向 FVIII 基因递送、最佳生物分布和持久的 FVIII 表达而被选中。这些新兴疗法的预期推出将改变未来几年血友病 A 市场的格局。随着这些前沿疗法的不断成熟和获得监管批准，预计它们将重塑血友病 A 市场，提供新的护理标准，并为医疗创新和经济增长开辟机会。了解更多关于 FDA 批准的血友病 A 基因疗法的信息 @ FDA 批准的 AAV 基因疗法血友病 A 血友病 A 市场的最新动态在 2025 年 7 月，诺和诺德宣布 FDA 已批准 ALHEMO 注射剂用于每日一次的预防性使用，以防止或减少 12 岁及以上血友病 A 或 B（HA/HB）患者的出血事件。此批准将该药物的适应症扩展至最初于 2024 年 12 月批准的有抑制剂的 HA/HB 患者。在 2025 年 6 月，辉瑞公司报告了其第 3 期 BASIS 试验（NCT03938792）的积极顶线结果，该试验评估了在有抑制剂的血友病 A 或 B 成人和青少年中使用 HYMPAVZI（marstacimab）。在 **2025 年 5 月，**FDA 批准了抗血友病因子（重组），PEG 化-aucl（Jivi；拜耳）用于治疗 7 岁及以上的血友病 A 儿童患者。该批准得到了 Alfa-PROTECT 和 PROTECT Kids 试验结果的支持。在 2025 年 3 月，Alnylam Pharmaceuticals 宣布 FDA 批准 Qfitlia™（fitusiran），这是美国批准的第六种 Alnylam 发现的 RNAi 治疗药物。它是首个也是唯一一个降低抗凝血酶（AT）的治疗，旨在促进凝血酶生成，重新平衡止血，并防止出血。什么是血友病 A？血友病 A 是一种遗传性出血性疾病，由于凝血因子 VIII 的缺乏或低水平引起。多年来，标准治疗一直是 FVIII 替代疗法。这始于从捐赠的全血中提取 FVIII，后来发展为血浆衍生的 FVIII，现在主要通过重组人 FVIII（rFVIII）产品进行管理，这些产品改变了血友病的护理。虽然该病通常在出生时被诊断，但也可能在生命后期获得，当免疫系统产生中和凝血因子的抗体时，这是一种罕见的情况，称为获得性血友病。血友病 A 流行病学细分血友病 A 流行病学预测部分提供了对历史和当前血友病 A 患者群体的洞察，以及主要市场的预测趋势。在 7 个主要市场中，美国的血友病 A 确诊病例最多，预计在 2024 年将达到近 14,900 例。这些病例预计在未来几年将增加。血友病 A 治疗市场报告提供了 2020 年至 2034 年研究期间的流行病学分析，细分为：血友病 A 的总确诊病例有抑制剂或无抑制剂的血友病 A 确诊病例按年龄细分的血友病 A 确诊病例按严重程度细分的血友病 A 确诊病例血友病 A 市场报告指标详细信息研究期间 2020–2034 覆盖范围 7 个主要市场 [美国、EU4（德国、法国、意大利和西班牙）、英国和日本]。血友病 A 市场年均增长率 2.9 % 2024 年血友病 A 市场规模 122 亿美元主要血友病 A 公司诺和诺德、Ultragenyx Pharmaceutical、Sangamo Therapeutics、ASC Therapeutics、罗氏、中外制药、赛诺菲、Alnylam Pharmaceuticals、辉瑞、基因泰克、生物马林制药、HEMA 生物制剂、LFB 制药、Octapharma、CSL Behring、Octapharma、拜耳、武田等主要血友病 A 疗法 Mim8、DTX201（Pebocotocogene camaparvovec、BAY2599023）、Giroctocogene fitelparvovec（SB-525）、ASC618、NXT007/RG6512、QFITLIA、HYMPAVZI、ALHEMO、HEMLIBRA、ALTUVIIIO、ROCTAVIAN、SEVENFACT/CEVENFACTA、ESPEROCT、WILATE、JIVI、ADYNOVATE、AFSTYLA、NUWIQ、FEIBA、KOVALTRY、OBIZUR、ELOCTATE/Elocta、XYNTHA 等血友病 A 市场报告的范围治疗评估：血友病 A 当前市场及新兴疗法血友病 A 市场动态：新兴血友病 A 药物的关键市场预测假设及市场前景竞争情报分析： SWOT 分析及市场进入策略未满足的需求、KOL 观点、分析师观点、血友病 A 市场准入及报销下载报告以评估血友病治疗公司 HEMLIBRA 在女性血友病中的应用 @ 皇家血友病 A 或 B 目录 1 血友病 A 市场关键洞察 2 血友病 A 市场报告介绍 3 执行摘要 4 关键事件 5 流行病学及市场预测方法论 6 血友病 A 市场概览 6.1 2020 年血友病 A 按疗法的市场份额（%）分布 6.2 2034 年血友病 A 按疗法的市场份额（%）分布 6.3 2020 年血友病 A 按抑制剂的市场份额（%）分布 6.4 2034 年血友病 A 按抑制剂的市场份额（%）分布 6.5 2020 年血友病 A 按非抑制剂的市场份额（%）分布 6.6 2034 年血友病 A 按非抑制剂的市场份额（%）分布 7 血友病 A：疾病背景及概述 7.1 介绍 7.2 血友病 A 的症状和体征 7.3 血友病 A 的遗传模式 7.4 血友病 A 的分子发病机制 7.5 血友病 A 的病理生理学 7.6 血友病 A 的风险因素 7.7 血友病 A 的诊断 8 血友病 A 治疗算法、当前治疗及医疗实践 9 流行病学及患者人群 9.1 关键发现 9.2 假设及理由：7MM 9.3 7MM 中血友病 A 的总诊断流行病例 9.4 美国 9.4.1 美国血友病 A 的总诊断流行病例 9.4.2 美国有抑制剂和无抑制剂的血友病 A 诊断流行病例 9.4.3 美国血友病 A 的年龄特定诊断流行病例 9.4.4 美国血友病 A 的严重程度特定流行病例 9.5 EU4 及英国 9.6 日本流行病学 10 血友病 A 患者旅程 11 已上市的血友病 A 疗法 11.1 已上市疗法的关键交叉 11.2 QFITLIA（fitusiran）：赛诺菲/阿尔尼拉姆制药 11.2.1 产品描述 11.2.2 监管里程碑 11.2.3 其他开发活动 11.2.4 临床开发 11.2.4.1 临床试验信息 11.2.5 安全性和有效性 11.3 HYMPAVZI（marstacimab-hncq）：辉瑞 11.4 ALHEMO（concizumab）：诺和诺德 11.5 HEMLIBRA（emicizumab-kxwh）：中外制药/基因泰克/罗氏 11.6 ALTUVIIIO（抗血友病因子 [重组]，Fc-VWF-XTEN 融合蛋白-ehtl）：赛诺菲/索比 11.7 ROCTAVIAN（valoctocogene roxaparvovec）：百奥明制药 11.8 SEVENFACT/CEVENFACTA [凝血因子 VIIa（重组）-jncw]: HEMA 生物制药/LFB 制药 11.9 ESPEROCT（N8-GP; Turoctocog alfa pegol）：诺和诺德 11.10 WILATE：Octapharma 11.11 JIVI（BAY94-9027）：拜耳 11.12 ADYNOVATE（Adynovi; BAX 855）：武田制药 11.13 AFSTYLA（Lonoctocog alfa）：CSL 贝赫林 11.14 NUWIQ（simoctocog alfa）：Octapharma 11.15 FEIBA：武田 11.16 KOVALTRY（BAY 81-8973）：拜耳 11.17 OBIZUR（susoctocog alfa）：武田 11.18 ELOCTATE/Elocta（efmoroctocog alfa）：赛诺菲/索比 11.19 XYNTHA（ReFacto AF）：辉瑞 12 新兴血友病 A 药物 12.1 关键交叉竞争 12.2 Mim8：诺和诺德 12.2.1 产品描述 12.2.2 其他开发活动 12.2.3 临床开发 12.2.3.1 临床试验信息 12.2.4 安全性和有效性 12.2.5 分析师观点 12.3 DTX201（Peboctocogene camaparvovec，BAY2599023）：Ultragenyx 制药 12.4 Giroctocogene fitelparvovec（SB-525）：Sangamo Therapeutics 12.5 ASC618：ASC Therapeutics 12.6 NXT007/RG6512：罗氏/中外制药 13 血友病 A 市场：7MM 分析 13.1 关键发现 13.2 血友病 A 市场前景 13.3 血友病 A 市场关键预测假设 13.4 联合分析 13.5 7MM 中血友病 A 的总市场规模按国家划分 13.6 美国血友病 A 市场规模 13.6.1 美国血友病 A 的总市场规模 13.6.2 美国血友病 A 按疗法的市场规模 13.7 EU4 及英国血友病 A 市场规模 13.8 日本血友病 A 市场规模 14 血友病 A 市场未满足的需求 15 血友病 A 市场 SWOT 分析 16 KOL 对血友病 A 的观点 17 血友病 A 市场准入及报销 17.1 美国 17.2 EU4 及英国 17.3 日本 17.4 血友病 A 的市场准入及报销 18 参考文献 19 血友病 A 市场报告方法论相关报告血友病 A 临床试验分析血友病 A 管线洞察 – 2025 报告提供了关于管线景观、管线药物概况的全面洞察，包括临床和非临床阶段产品，以及关键的血友病 A 公司，包括江苏基因科学公司、武田、Gritgen Therapeutics 有限公司、苏州阿尔法玛生物科技有限公司、诺和诺德、辉瑞、SK Plasma 有限公司、霍夫曼 - 拉罗氏、上海信念生物医药有限公司、ASC Therapeutics、Biocad、Expression Therapeutics, LLC、GCBiopharma、Amarna Therapeutics、Cabaletta Bio 等。获得性血友病 A 市场获得性血友病 A 市场洞察、流行病学和市场预测 – 2032 报告提供了对该疾病的深入理解，包括历史和预测的流行病学，以及市场趋势、市场驱动因素、市场障碍和主要获得性血友病 A 公司，包括辉瑞、诺和诺德、Hema Biologics、霍夫曼 - 拉罗氏、武田等。血友病市场血友病市场洞察、流行病学和市场预测 – 2032 报告提供了对该疾病的深入理解，包括历史和预测的流行病学，以及市场趋势、市场驱动因素、市场障碍和主要血友病公司，包括 Centessa Pharmaceuticals、Alnylam Pharmaceuticals、ASC Therapeutics、Spark Therapeutics、Freeline Therapeutics、BioMarin Pharmaceutical、Staidson Beijing BioPharmaceuticals、辉瑞、Sangamo Therapeutics、Amunix、Bioverativ、诺和诺德等。血友病 B 市场血友病 B 市场洞察、流行病学和市场预测 – 2032 报告提供了对该疾病的深入理解，包括历史和预测的流行病学，以及市场趋势、市场驱动因素、市场障碍和主要血友病 B 公司，包括 UniQure Biopharma B.V.、CSL Behring、辉瑞、Spark Therapeutics、Genzyme（赛诺菲公司）、Alnylam Pharmaceuticals、诺和诺德、UniQure Biopharma B.V.、ApcinteX Ltd、Freeline Therapeutics、Sangamo Therapeutics 等。关于 DelveInsight DelveInsight 是一家领先的商业咨询和市场研究公司，专注于生命科学领域。它通过提供全面的端到端解决方案来支持制药公司，提高其业绩。通过我们的订阅平台 PharmDelve，轻松访问所有医疗保健和制药市场研究报告。联系我们 Shruti Thakur [email protected] +14699457679 www.delveinsight.com Logo - https://mma.prnewswire.com/media/1082265/3528414/DelveInsight_Logo.jpg 来源 DelveInsight Business Research, LLP

基因疗法siRNA临床研究

2025-12-03

·PRNewswire

Hemophilia A Market to Exhibit Growth at a CAGR of 2.9% During the Forecast Period (2025-2034) Owing to the Rising Hemophilia Prevalence, Strong Uptake of New Drugs, and Emergence of High-cost Gene Therapies | DelveInsight

The dynamics of the hemophilia A market are expected to change mainly due to the increase in prevalence and the launch of upcoming therapies such as Mim8 (Novo Nordisk), DTX201 (Pebocotocogene camaparvovec, BAY2599023 [Ultragenyx Pharmaceutical]), giroctocogene fitelparvovec (SB-525 [Sangamo Therapeutics]), and others during the forecast period. LAS VEGAS, Dec. 3, 2025 /PRNewswire/ -- DelveInsight's Hemophilia A Market Insights report includes a comprehensive understanding of current treatment practices, hemophilia A emerging drugs, market share of individual therapies, and current and forecasted market size from 2020 to 2034, segmented into leading markets [the United States, the EU4 (Germany, France, Italy, and Spain), the United Kingdom, and Japan]. Hemophilia A Market Summary The market size for hemophilia A was found to be USD 12.2 billion in the leading markets [the United States, the EU4 (Germany, France, Italy, and Spain), the United Kingdom, and Japan] in 2024. The United States accounted for the largest hemophilia A treatment market size, approximately 48% of the total market size in the 7MM in 2024, compared to other major markets, including the EU4 countries, the United Kingdom, and Japan. HEMLIBRA's growth curve (US Q1 2025 year-over-year sales) in the United States was flat, indicating increased competition from newer therapies. The total number of diagnosed prevalent cases of Hemophilia A in the 7MM was around 49,500 in 2024. Leading hemophilia A companies, such as Novo Nordisk, Ultragenyx Pharmaceutical, Sangamo Therapeutics, ASC Therapeutics, Roche, Chugai, and others, are developing new hemophilia A treatment drugs that can be available in the hemophilia A market in the coming years. The promising hemophilia A therapies in clinical trials include Mim8, DTX201 (Peboctocogene camaparvovec, BAY2599023), Giroctocogene fitelparvovec (SB-525), ASC618, NXT007/RG6512, and others. Discover the new hemophilia A treatment @ Hemophilia A Treatment Market Key Factors Driving the Growth of the Hemophilia A Market Rising Hemophilia A Target Patient Pool The diagnosed prevalence of Hemophilia A is expected to rise from 49K in 2024 to 50K by 2034. Improved diagnostic capabilities and increased disease awareness mainly drive this increase in cases. Advancements in Gene Therapy Gene therapy has emerged as a transformative approach in hemophilia A treatment. Notably, BioMarin's ROCTAVIAN has been approved in the U.S. and EU, offering a one-time infusion that provides long-term correction of the factor VIII deficiency. Pfizer's gene therapy giroctocogene fitelparvovec for hemophilia A has also demonstrated promising results in late-stage trials, significantly reducing bleeding episodes and outperforming traditional treatments. Regulatory Approvals and Hemophilia A Market Expansion Recent regulatory approvals have bolstered the availability of hemophilia A treatments. For instance, the U.S. FDA approved Pfizer's once-weekly injection, HYMPAVZI (in October 2024), for hemophilia A patients aged 12 and above, aiming to prevent or reduce bleeding episodes. Similarly, Sanofi's QFITLIA (in March 2025), a subcutaneous treatment administered every two months, was approved for patients with Hemophilia A or B aged 12 and older. Emergence of Non-Factor Hemophilia A Therapies Expanding beyond traditional therapies, hemophilia A care now includes two cutting-edge non-factor approaches: anti-TFPI therapies (Pfizer's HYMPAVZI and Novo Nordisk's ALHEMO) and siRNA therapies (Sanofi's QFITLIA). Robust Hemophilia A Clinical Trial Activity Several hemophilia A companies are actively developing emerging therapies, including Novo Nordisk (Mim8, ALHEMO), Sangamo Therapeutics (Giroctocogene fitelparvovec), Roche/Chugai (NXT007), ASC Therapeutics (ASC-618), Ultragenyx (DTX201), and others. Hemophilia A Market Analysis The primary goal of current treatment strategies for hemophilia A is to minimize complications caused by bleeding into joints, tissues, or organs. With ongoing technological advancements and improved medical understanding, patients now have access to a range of therapeutic options. Treatment is typically administered either "on-demand" or as "prophylaxis", with prophylactic therapy increasingly becoming the preferred approach. The U.S. FDA has approved several recombinant Factor VIII products for hemophilia A management, including HELIXATE FS, RECOMBINATE, KOGENATE FS, ADVATE, REFACTO, ELOCTATE, NUWIQ, ADYNOVATE, KOVALTRY, JIVI, and XYNTHA. Additionally, plasma-derived Factor VIII products, such as MONARC-M, MONOCLATE-P, HEMOFIL M, and KOATE-DVI, remain available. The latest addition, QFITLIA (fitusiran), a siRNA-based therapy approved in 2025, works by lowering antithrombin levels to enhance clot formation. It offers the convenience of only six injections per year and is effective for both inhibitor and non-inhibitor patients. HEMLIBRA has emerged as a first-line prophylactic therapy for Hemophilia A patients with inhibitors, although immune tolerance induction (ITI) therapy continues to be the gold standard. For those who encounter difficulties with ITI, HEMLIBRA provides an alternative, requiring less frequent FVIII dosing during ITI. Looking ahead, hemophilia management is shifting toward extended half-life factor therapies and cutting-edge modalities, including siRNA agents, bispecific antibodies, and gene therapy. The competitive landscape is expected to expand with the introduction of new long-acting factor products and next-generation therapeutic technologies. Hemophilia A Competitive Landscape The hemophilia A clinical trial landscape features several drugs in mid- and late-stage development that are expected to be approved in the near future. The emerging landscape offers a diverse range of therapeutic alternatives for treatment, including Mim8 (Novo Nordisk), DTX201 (Pebocotocogene camaparvovec, BAY2599023 [Ultragenyx Pharmaceutical]), giroctocogene fitelparvovec (SB-525 [Sangamo Therapeutics]), and others. Novo Nordisk's Mim8 is an advanced FVIIIa-mimicking bispecific antibody designed to provide sustained hemostatic control with convenient once-weekly or once-monthly prophylactic dosing for individuals with Hemophilia A, regardless of inhibitor status. Delivered subcutaneously, Mim8 functions by bridging activated Factors IXa and X (FIXa/FX) upon activation, effectively compensating for the absence of FVIII. This restores normal thrombin generation and promotes effective blood clotting. Ultragenyx Pharmaceutical's DTX201 represents the first clinical-stage gene therapy for Hemophilia A utilizing an Adeno-associated Virus (AAV) vector derived from the AAVhu37 serotype. It is a non-replicating AAV vector carrying a single-stranded DNA genome that encodes a B-domain–deleted FVIII, driven by a liver-specific promoter and enhancer sequence optimized for strong transgene expression. The AAVhu37 capsid, part of the hepatotropic clade E family, was chosen for its demonstrated ability in preclinical studies to enable efficient liver-targeted FVIII gene delivery, optimal biodistribution, and long-lasting FVIII expression. The anticipated launch of these emerging therapies are poised to transform the hemophilia A market landscape in the coming years. As these cutting-edge therapies continue to mature and gain regulatory approval, they are expected to reshape the hemophilia A market landscape, offering new standards of care and unlocking opportunities for medical innovation and economic growth. Discover more about the FDA approved gene therapy hemophilia A @ FDA Approved AAV Gene Therapy Hemophilia A Recent Developments in the Hemophilia A Market In July 2025, Novo Nordisk announced that the FDA has approved ALHEMO injection for once-daily prophylactic use to prevent or reduce bleeding episodes in adults and children aged 12 years and older with hemophilia A or B (HA/HB) without inhibitors. This approval broadens the drug's indication beyond its initial December 2024 approval for patients with HA/HB with inhibitors. In June 2025, Pfizer Inc. reported positive topline findings from its Phase 3 BASIS trial (NCT03938792), which evaluated HYMPAVZI (marstacimab) in adults and adolescents with hemophilia A or B who have inhibitors. In May 2025, the FDA approved Antihemophilic factor (recombinant), PEGylated-aucl (Jivi; Bayer) for treating pediatric patients aged 7 and older with hemophilia A. The approval was supported by results from the Alfa-PROTECT and PROTECT Kids trials. In March 2025, Alnylam Pharmaceuticals announced the FDA's approval of Qfitlia™ (fitusiran), the sixth Alnylam-discovered RNAi therapeutic approved in the U.S. It is the first and only treatment to lower antithrombin (AT), aiming to promote thrombin generation, rebalance hemostasis, and prevent bleeds. What is Hemophilia A? Hemophilia A is an inherited bleeding disorder caused by a deficiency or low levels of clotting factor VIII. For many years, the standard treatment has been replacement therapy with FVIII. This began with FVIII obtained from donated whole blood, later advanced to plasma-derived FVIII, and is now primarily managed with recombinant human FVIII (rFVIII) products that have transformed hemophilia care. While the condition is typically diagnosed at birth, it can also be acquired later in life when the immune system develops antibodies that neutralize clotting factors, a rare condition known as acquired hemophilia. Hemophilia A Epidemiology Segmentation The hemophilia A epidemiology forecast section provides insights into the historical and current hemophilia A patient pool and forecasted trends for the leading markets. In the 7MM, the highest diagnosed prevalent cases of Hemophilia A were in the United States, accounting for nearly 14,900 cases in 2024. These cases are anticipated to increase in the upcoming years. The hemophilia A treatment market report proffers epidemiological analysis for the study period 2020–2034 in the leading markets, segmented into: Total Diagnosed Prevalent Cases of Hemophilia A Diagnosed Prevalent Cases of Hemophilia A with Inhibitors or without Inhibitors Age-Specific Diagnosed Prevalent Cases of Hemophilia A Severity-specific Diagnosed Prevalent Cases of Hemophilia A Scope of the Hemophilia A Market Report Therapeutic Assessment: Hemophilia A current marketed and emerging therapies Hemophilia A Market Dynamics: Key Market Forecast Assumptions of Emerging Hemophilia A Drugs and Market Outlook Competitive Intelligence Analysis: SWOT analysis and Market entry strategies Unmet Needs, KOL's views, Analyst's views, Hemophilia A Market Access and Reimbursement Download the report to evaluate the hemophilia A treatment company HEMLIBRA on hemophilia in women @ Royal Hemophilia A or B Table of Contents Related Reports Hemophilia A Clinical Trial Analysis 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 A companies, including Jiangsu Gensciences Inc., Takeda, Gritgen Therapeutics Co., Ltd., Suzhou Alphamab Co., Ltd., Novo Nordisk, Pfizer, SK Plasma Co., Ltd., Hoffmann-La Roche, Shanghai Belief-Delivery BioMed Co., Ltd, ASC Therapeutics, Biocad, Expression Therapeutics, LLC, GCBiopharma, Amarna Therapeutics, Cabaletta Bio, among others. Acquired Hemophilia A Market Acquired Hemophilia A Market Insights, Epidemiology, and Market Forecast – 2032 report delivers an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key acquired hemophilia A companies, including Pfizer, Novo Nordisk, Hema Biologics, Hoffman-La-Roche, Takeda, among others. Hemophilia Market Hemophilia Market Insights, Epidemiology, and Market Forecast – 2032 report delivers an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key hemophilia companies, including Centessa Pharmaceuticals, Alnylam Pharmaceuticals, ASC Therapeutics, Spark Therapeutics, Freeline Therapeutics, BioMarin Pharmaceutical, Staidson Beijing BioPharmaceuticals, Pfizer, Sangamo Therapeutics, Amunix, Bioverativ, Novo Nordisk, among others. Hemophilia B Market Hemophilia B Market Insights, Epidemiology, and Market Forecast – 2032 report delivers an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key hemophilia B companies, including UniQure Biopharma B.V., CSL Behring, Pfizer, Spark Therapeutics, Genzyme, a Sanofi Company, Alnylam Pharmaceuticals, Novo Nordisk, UniQure Biopharma B.V., ApcinteX Ltd, Freeline Therapeutics, Sangamo Therapeutics, 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 . Contact Us Shruti Thakur [email protected] +14699457679 Logo - SOURCE DelveInsight Business Research, LLP 21% more press release views with Request a Demo

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适应症	最高研发状态	国家/地区	公司	日期
血友病A	申请上市	美国	Novo Nordisk A/S	2025-09-30

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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


NCT04204408 (FRONTIER1, Pubmed \| Res Pract Thromb Haemost)	临床1/2期	血友病A	41	Mim8 maintenance doses	築膚醖鹽選廠積壓窪糧(鑰構積築鹹齋願顧蓋鬱) = 遞積夢顧廠襯範夢夢選鹹膚醖艱齋顧膚鑰選觸 (窪簾構選餘鏇範顧鑰糧 ) 更多	积极	2025-10-01
FRONTIER3 (GlobeNewswire) 人工标引	临床3期	血友病A	70	Mim8	糧選淵簾獵築觸範鹹網(廠廠膚觸衊襯蓋餘鏇繭) = 築築廠選醖憲鹹範網築築鹹鬱襯顧淵積顧艱襯 (繭鏇衊齋膚簾夢遞遞醖 ) 更多	积极	2025-02-07
NCT05685238 (FRONTIER4, ASH2024)	-	血友病A	37	Mim8	選鏇艱膚遞糧鏇獵窪鹹(鏇繭範簾遞築積鹽獵構) = 遞鏇醖築醖醖壓製鏇淵衊醖範壓網製膚觸糧積 (顧衊鏇糧窪醖簾廠襯製 )	积极	2024-12-09
NCT05053139 (Frontier 2, Corporate Publications) 人工标引	临床3期	血友病A	254	Mim8 once-weekly prophylaxis (no prior prophylaxis treatment)	壓鑰鑰繭廠鑰鬱夢遞範(淵糧齋齋蓋築顧鏇糧選) = 網壓獵鑰衊繭築壓獵艱齋廠積淵繭壓製鬱淵簾 (製糧廠齋衊選鏇壓觸廠 ) 更多	积极	2024-06-23
NCT05053139 (Frontier 2, Corporate Publications) 人工标引	临床3期	血友病A	254	Mim8 once-monthly prophylaxis (no prior prophylaxis treatment)	壓鑰鑰繭廠鑰鬱夢遞範(淵糧齋齋蓋築顧鏇糧選) = 鏇窪繭齋夢糧獵獵鹹廠齋廠積淵繭壓製鬱淵簾 (製糧廠齋衊選鏇壓觸廠 ) 更多	积极	2024-06-23
NCT05053139 (FRONTIER 2, GlobeNewswire) 人工标引	临床3期	血友病A	254	Mim8 once-weekly	鑰憲蓋繭襯艱壓夢鹽顧(糧衊膚膚選鹽製淵糧鹹) = 壓積膚膚壓鹽醖選淵築簾獵餘憲蓋鏇築壓簾夢 (壓鬱淵觸觸壓齋夢簾夢 ) 达到更多	积极	2024-05-13
NCT05053139 (FRONTIER 2, GlobeNewswire) 人工标引	临床3期	血友病A	254	Mim8 once-monthly	鑰憲蓋繭襯艱壓夢鹽顧(糧衊膚膚選鹽製淵糧鹹) = 範衊衊廠蓋顧觸夢鏇齋簾獵餘憲蓋鏇築壓簾夢 (壓鬱淵觸觸壓齋夢簾夢 ) 达到更多	积极	2024-05-13
EUCTR2019-000465-20-AT \| NCT04204408 (FRONTIER 1, ASH 12022 \| ASH 22022) 人工标引	临床1/2期	血友病A	42	Mim 8	襯選窪餘醖簾積夢壓壓(艱窪廠鹽鑰遞窪觸繭糧) = 繭齋鹽齋網鹹餘構繭簾鹹鹹齋艱簾糧窪醖製鹽 (願願齋簾繭顧淵鏇選鏇 ) 更多	积极	2022-11-15
EUCTR2019-000465-20-AT \| NCT04204408 (FRONTIER1, ISTH2022) 人工标引	临床1/2期	-	-	Mim 8 (SAD phase)	網襯餘衊壓積膚網簾鹽(膚鬱製憲繭淵鏇醖製憲) = Mim8 was well tolerated following both single and multiple dosing, and no thromboembolic events or related serious adverse events were reported 鏇範淵醖艱鹹壓淵簾蓋 (淵顧鹹醖積蓋壓醖鹹繭 )	积极	2022-07-11
	临床1/2期	-	-	Mim 8 (MAD phase)		积极	2022-07-11
NCT04204408 (FRONTIER1, ISTH2022)	临床1/2期	血友病A	-	Mim8	鹹鏇憲鹹簾簾衊壓餘壓(淵築簾齋醖艱鹹鬱淵鑰) = 築鏇膚範積鬱遞繭膚鑰艱鹹鏇襯醖積選鬱膚積 (積鹹蓋窪範構鹽淵製衊 ) 更多	-	2022-07-09