The purpose of the aPCC-emicizumab safety study is to prospectively investigate the safety and hemostatic efficacy of a personalized dose of aPCC in children and adults with hemophilia A and inhibitors on emicizumab prophylaxis during acute bleeding events or prior to procedures.

开始日期2025-02-01

申办/合作机构

Emory University

[+1]

CTRI/2024/08/071826 / Recruiting临床4期IIT

Outcome of Emicizumab Prophylaxis in Patients of Haemophilia A with or without Inhibitor An Early Experience from A Tertiary Care Hospital From Eastern India - NIL

开始日期2024-08-15

申办/合作机构-

NCT05248594 / RecruitingN/AIIT

Real World Use of Emicizumab in Infants and Children Ages 0-3 Years With Hemophilia A

We have developed a questionnaire to elucidate the dosing, frequency and indication for the use of emicizumab in patients with Hemophilia A (mild, moderate or severe) ages 0-3 years. We are also collecting data on any pre-, peri and post surgical practices while on emicizumab. More importantly, we are asking if pediatricians are planning to introduce factor 8 to children who are already on emicizumab for primary prophylaxis as well as how and when they are planning to do so. We hope that this data will help inform understanding of current use of emicizumab in infants and young children as a form of primary prophylaxis, especially when venous access has historically been a limiting factor.

开始日期2024-08-01

申办/合作机构

Montefiore Medical Center

[+1]

100 项与艾美赛珠单抗相关的临床结果

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100 项与艾美赛珠单抗相关的转化医学

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100 项与艾美赛珠单抗相关的专利（医药）

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594

项与艾美赛珠单抗相关的文献（医药）

2024-12-31·mAbs

A pivotal decade for bispecific antibodies?

Article

作者: Surowka, Marlena ; Klein, Christian

Bispecific antibodies (bsAbs) are a class of antibodies that can mediate novel mechanisms of action compared to monospecific monoclonal antibodies (mAbs). Since the discovery of mAbs and their adoption as therapeutic agents in the 1980s and 1990s, the development of bsAbs has held substantial appeal. Nevertheless, only three bsAbs (catumaxomab, blinatumomab, emicizumab) were approved through the end of 2020. However, since then, 11 bsAbs received regulatory agency approvals, of which nine (amivantamab, tebentafusp, mosunetuzumab, cadonilimab, teclistamab, glofitamab, epcoritamab, talquetamab, elranatamab) were approved for the treatment of cancer and two (faricimab, ozoralizumab) in non-oncology indications. Notably, of the 13 currently approved bsAbs, two, emicizumab and faricimab, have achieved blockbuster status, showing the promise of this novel class of therapeutics. In the 2020s, the approval of additional bsAbs can be expected in hematological malignancies, solid tumors and non-oncology indications, establishing bsAbs as essential part of the therapeutic armamentarium.

2024-12-01·PEDIATRIC BLOOD & CANCER

CHIEF: A retrospective self‐control study of children with severe hemophilia A without inhibitors comparing emicizumab to FVIII prophylaxis

Article

作者: Malvar, Jemily ; Carneiro, Kendall ; Mashiach, Daniel ; Young, Guy ; Mead, Patrice ; Knight, Susan

Abstract:

Background:

Hemophilia A (HA) is an X‐linked bleeding disorder diagnosed by a deficiency in factor VIII (FVIII). For severe HA (SHA), prophylaxis clotting factor concentrates (CFC) has become the standard of care; however, it imparts a high treatment burden and typically results in an annualized bleeding rate (ABR) of 2–6. Emicizumab, a subcutaneously administered FVIII substitute, has become the de facto standard‐of‐care prophylaxis for children with SHA in many countries. Previous clinical trials of emicizumab have assessed ABR in patients greater than 12 years without inhibitors, and in children less than 12 years with inhibitors; however, there is little information published regarding the ABR of emicizumab compared to CFC in non‐inhibitor SHA children.

Methods:

Using a retrospective electronic medical record chart review, we conducted a self‐control analysis of 15 patients less than 12 years of age during equivalent periods of CFC versus emicizumab prophylaxis.

Results:

The mean ABR on CFC and emicizumab was 1.79 and 1.13 (p = .092), respectively, with a substantially decreased rate of joint bleeds (CFC 0.94; emicizumab 0.33; p = .001) and spontaneous bleeds (CFC 0.79; emicizumab 0.23; p = .008). No safety events were recorded for patients while administering emicizumab. The mean annual cost of CFC prophylaxis was $515,340 (SD $199,540), compared to $328,410 (SD $137,230) for emicizumab prophylaxis (p < .001).

Conclusion:

Emicizumab resulted in an improved ABR compared to CFC, especially for joint and spontaneous bleeds, had fewer administration complications, and was substantially less expensive compared to CFC prophylaxis; however, more research is necessary for a complete understanding of the effect of emicizumab on joint health and muscle bleeds.

2024-12-01·Blood Research

Real-world insights into the management of hemophilia A in Italy: treatment patterns and healthcare resource utilization

Article

作者: Cappuccilli, Maria ; Leogrande, Melania ; Perrone, Valentina ; Esposti, Luca Degli

Abstract:

Purpose:

This real-world analysis described the Hemophilia A (HA) population in Italy, evaluating drug utilization and consumption of factor VIII (FVIII) products of patients under prophylaxis and on-demand therapy.

Methods:

From Jan-2017 to Jun-2022, male patients with HA were identified through prescriptions of FVIII products [extended half-life FVIII, standard half-life recombinant FVIII, and plasma-derived FVIII (EHL FVIII, SHL rFVIII, and pdFVIII, respectively)], or emicizumab or FVIII plus von Willebrand factor or HA-related hospitalization using administrative flows of Italian healthcare entities. Patients on treatment with FVIII products during 2021–2022 were stratified by treatment regimen (prophylaxis/on-demand). The mean annual consumption expressed in International Units (IU) of EHL FVIII and SHL FVIII in patients treated during 2021–2022 having at least 12-month follow-up were assessed.

Results:

Among included HA patients, 145 (39.5%) received EHL FVIII and 222 (60.5%) SHL FVIII. Of 165 patients on prophylaxis, 105 (64%) received an EHL FVIII and 60 (36%) an SHL FVIII. The mean annual consumption of FVIII was 336,700 IU (median 319,000 IU) for EHL FVIII and 440,267 IU (median 360,500 IU) for SHL FVIII. Specifically, for patients on EHL FVIII, the most common drugs were efmoroctocog alfa (N = 51) and damoctocog alfa pegol (N = 50), followed by turoctocog alfa pegol (N = 25) and rurioctocog alfa pegol (N = 19). Of 702 HA patients initially treated with FVIII products, 74 (10.5%) switched to emicizumab during follow-up.

Conclusion:

These findings revealed an extensive use of EHL FVIII products, suggesting growing efforts from clinicians to optimize prophylactic strategies and achieve better bleeding protection.

349

项与艾美赛珠单抗相关的新闻（医药）

2024-12-23

·美通社

罗氏又一款CD20/CD3双特异性抗体中国获批，T细胞疗法将给复发难治性滤泡性淋巴瘤带来临床新选择

上海 2024年12月23日 /美通社/ -- 2024年12月23日，罗氏制药中国宣布，中国国家药品监督管理局（NMPA）正式批准皓罗华 ® （英文商品名：Lunsumio ® ，中英文通用名：莫妥珠单抗/mosunetuzumab）单药用于治疗既往接受过至少两线系统性治疗的复发或难治性滤泡性淋巴瘤成人患者。作为全球首款 [1] 获批用于滤泡性淋巴瘤（FL）治疗的CD20/CD3双特异性抗体（以下简称"双抗"），莫妥珠单抗是继格菲妥单抗之后，罗氏在中国连续第二年获批的第二款CD20/CD3双抗药物。区别于格菲妥单抗独特的2:1结构，莫妥珠单抗是典型的1:1结构，两款结构不同的双抗分别覆盖不同的淋巴瘤亚型，给不同分型的淋巴瘤患者带来获益。治疗持久战：滤泡性淋巴瘤复发困局待解滤泡性淋巴瘤是非霍奇金淋巴瘤（NHL）中最常见的惰性亚型之一，我国滤泡性淋巴瘤的发病率占B细胞NHL的8%~23% [2] 。近年来，随着中国老龄化程度的增加，滤泡性淋巴瘤发病率也呈逐年上升的趋势 [3] 。目前滤泡性淋巴瘤仍是一种无法治愈的疾病，大多数患者会经历反复复发、进展，严重影响着患者的生存和生活质量。约有20%的患者接受免疫化疗后24个月内发生疾病进展（POD24） [4] ，随着复发次数的增加，后续治疗难度也不断升级，患者的缓解时间和生存时间也会缩短。滤泡性淋巴瘤患者因为疾病易复发、难治愈的特点长期承受着身心压力，影响生存质量。《2020中国滤泡性淋巴瘤患者生存状况白皮书》（下文简称《白皮书》）调查显示，滤泡性淋巴瘤患者深受疾病需要反复治疗的困扰，怀有对复发的恐惧，复发患者感受到更多的疼痛、焦虑、沮丧，生活质量远低于普通人群，绝大多数受访患者均表示对治疗滤泡性淋巴瘤的新药有非常迫切的需求 [5] 。进一步减少复发，获得更长时间和更高质量的生存是滤泡性淋巴瘤患者最为迫切的希望。哈尔滨血液病肿瘤研究所所长马军教授谈到："近年来淋巴瘤的治疗发展迅速，各类新型疗法和药物不断涌现，患者的生存状况得到了显著改善。然而，对于以滤泡性淋巴瘤为代表的惰性淋巴瘤而言，临床上仍存在巨大的未尽之需。虽然FL临床进展较为缓慢，病程较长，但临床治疗中约20%的患者可发生早期进展，这部分患者5年总生存（OS）率仅50%，死亡风险是非POD24患者的6到7倍。除早期进展，在一次次疾病复发的过程中，每年可能有2-3%的滤泡性淋巴瘤患者转化为其它侵袭性更强的淋巴瘤类型，这使得治疗变得更加复杂和困难，患者的生存期也随之缩短。" 改写复发或难治性滤泡性淋巴瘤治疗格局，莫妥珠单抗带来治疗新选择近年来，T细胞介导的免疫疗法在血液肿瘤治疗领域持续展现出巨大潜力，双特异性抗体是近年来最受关注的T细胞免疫疗法的一个研究方向，为传统治疗手段疗效不佳的患者带来了新的治疗选择 [5] 。由于滤泡性淋巴瘤具有高度异质性，分类复杂、诊断困难，需要为每个患者制定个体化的治疗方案，因此推动其规范化诊疗发展，让患者得到及时、有效的诊治就显得至关重要。"传统治疗的客观缓解率（ORR）和完全缓解（CR）率相对较低，复发或难治性滤泡性淋巴瘤患者一旦再次复发，会迫切需要创新疗法，T细胞介导的免疫疗法是我们学界关注的重点，我们临床中不断证实了双抗对于淋巴瘤后线患者治疗获益。" 苏州大学附属第一医院血液科主任吴德沛教授说道。莫妥珠单抗是IgG1样人源化双特异性抗体，能够同时靶向B细胞表面的CD20抗原和T细胞表面的CD3抗原，重定向T细胞并清除恶性B细胞。此次莫妥珠单抗中国获批是基于多中心、开放标签I/II期GO29781研究的积极结果。该研究旨在评估固定疗程莫妥珠单抗对既往接受两种或两种以上治疗的复发或难治性滤泡性淋巴瘤（R/R FL）患者的安全性和抗肿瘤活性。 GO29781研究的关键结果显示，患者的客观缓解率（ORR）为80%，完全缓释率（CR）为60%。高危POD24亚组的CR率为60%，与总体人群一致。最常见的不良事件（AE）为细胞因子释放综合征（CRS），主要为低级别（1-2级），中位随访37.4个月时，患者的3年OS率为82.4% [6] 。2024年CSCO大会上公布的数据显示，莫妥珠单抗在中国患者中的ORR高达88.2%，CR率为64.7%，与GO29781研究相符 [7] 。北京大学肿瘤医院党委书记朱军教授表示："如何延长滤泡性淋巴瘤患者的无病生存时间，改善复发或难治性患者预后，实现高质量长期生存仍是目前亟待攻破的难题。GO29781研究成果令人惊喜。我们中心入组患者情况与总体研究结果一致，经长期随访，使用莫妥珠单抗的患者获得了较高的完全缓解率，显著延长患者总生存期，且未在研究期间观察到严重不良事件的发生，证实了莫妥珠单抗在高效抗肿瘤的同时耐受性佳、安全性良好。更令人欣喜的是，我们观察到POD24患者同样能获得持久缓解和PFS获益，且越早使用莫妥珠单抗的患者能有更好的生存获益。"基于研究，莫妥珠单抗此前已在美国和欧盟获批，并被纳入2024年版《CSCO淋巴瘤诊疗指南》，推荐其用于复发或难治性滤泡性淋巴瘤（FL1~3a级）的治疗（II级推荐，2A类）。上海交通大学医学院附属瑞金医院副院长赵维莅教授表示："试验结果清晰展现，莫妥珠单抗不仅能够为复发或难治性滤泡性淋巴瘤患者带来长期生存获益，同时作为无需定制的即用型免疫疗法，莫妥珠单抗也为患者带来了门诊固定周期的全新治疗选择。结合中国临床现状，需要治疗的患者远比实际入院的患者多，莫妥珠单抗能够为因各种现实因素限制无法住院的患者提供了更为灵活、便利的用药场景和便捷的治疗体验，也为日后复发或难治性滤泡性淋巴瘤患者的规范化诊疗提供了新的可能。" 群星璀璨，罗氏引领中国血液疾病诊疗作为双抗研发领域的先行者，罗氏血液领域的第一个双特异性单克隆抗体艾美赛珠单抗于2019年进入中国，填补了A型血友病抑制物患者预防治疗的临床空白，作为非因子预防治疗创新疗法让A型血友病患者实现"零"出血 [8] 。在血液研发以外，罗氏已陆续布局多个双抗技术平台，将双抗技术应用到更多领域，全球首个眼科双抗药物 [9] 法瑞西单抗也已在中国获批多个适应症。罗氏全球药品开发中国中心负责人厉文泓："罗氏血液研发始终关注患者最迫切的需求，此次获批的莫妥珠单抗正是我们以创新研发满足患者治疗需求的又一力证。作为全球双抗研发领域的先行者，罗氏通过不同的技术平台，将产品差异化定位深植于分子设计，为患者带来多个具有独特临床价值的双抗治疗解决方案。未来，我们将继续专注研发，不断突破创新，为中国患者提供更加多元化的血液疾病创新产品。" 罗氏制药中国总裁边欣："在过去的四年时间里，皓罗华是继佳罗华、优罗华、高罗华之后罗氏给中国带来的第四款血液肿瘤创新药。如皓字含义一般，我们希望皓罗华如‘红日初升，其道大光'，给更多惰性淋巴瘤患者带来新的治疗选择。秉承‘先患者之需而行'的理念，罗氏将持续携手各个生态圈伙伴，科研助力学科发展，开创患者服务新模式，共同携手为中国血液肿瘤患者治愈而努力，坚定实践‘健康中国2030'宏伟蓝图。" [1] Cao, Y., Marcucci, E. C., & Budde, L. E. (2023). Mosunetuzumab and lymphoma: latest updates from 2022 ASH annual meeting. Journal of Hematology & Oncology, 16(69). https://doi.org/10.1186/s13045-023-01462-0 [2] 中国抗癌协会血液肿瘤专业委员会, 中华医学会血液学分会淋巴细胞疾病学组, 中国滤泡淋巴瘤工作组, et al. 中国滤泡性淋巴瘤诊断与治疗指南（2023年版） [J]. 中华血液学杂志, 2023, 44(7): 529-34. [3] 中国抗癌协会淋巴瘤专业委员会、中华医学会血液学分会，中国滤泡性淋巴瘤诊断与治疗指南（2020年版） [4] Casulo C, Byrtek M, Dawson KL,et al. Early relapse of follicular lymphoma after rituximab pluscyclophosphamide, doxorubicin, vincristine, and prednisone defines patients athigh risk for death: an analysis from the National LymphoCare Study [correctionpublished in J Clin Oncol. 2016;34(12):1430]. J Clin Oncol. 2015;33(23):2516-2522. [5] 《2020中国滤泡性淋巴瘤患者生存白皮书》 [6] Stephen J. Schuster, et al., ASH 2023. oral 603. [7] LI Z , SHIYONG; LIU, YANYAN, et al. Safety and efficacy of mosunetuzumab monotherapy in Chinese patients with relapsed or refractory follicular lymphoma [J]. 2024 CSCO, 2024. [8] Callaghan, Michael U et al. "Long-term outcomes with emicizumab prophylaxis for hemophilia A with or without FVIII inhibitors from the HAVEN 1-4 studies." Blood vol. 137,16 (2021): 2231-2242. [9] Faricimab is the first bispecific antibody designed for intraocular use. In January 2022 faricimab received its first approvals（Drugs (2022) 82:825–830 https://doi.org/10.1007/s40265-022-01713-3）

细胞疗法免疫疗法上市批准

2024-12-23

·罗氏制药

罗氏又一款CD20/CD3双特异性抗体中国获批！

2024年12月23日，罗氏制药中国宣布，中国国家药品监督管理局（NMPA）正式批准皓罗华®（英文商品名：Lunsumio®，中英文通用名：莫妥珠单抗/mosunetuzumab）单药用于治疗既往接受过至少两线系统性治疗的复发或难治性滤泡性淋巴瘤成人患者。作为全球首款[1]获批用于滤泡性淋巴瘤（FL）治疗的CD20/CD3双特异性抗体（以下简称“双抗”），莫妥珠单抗是继格菲妥单抗之后，罗氏在中国连续第二年获批的第二款CD20/CD3双抗药物。区别于格菲妥单抗独特的2:1结构，莫妥珠单抗是典型的1:1结构，两款结构不同的双抗分别覆盖不同的淋巴瘤亚型，给不同分型的淋巴瘤患者带来获益。治疗持久战：滤泡性淋巴瘤复发困局待解滤泡性淋巴瘤是非霍奇金淋巴瘤（NHL）中最常见的惰性亚型之一，我国滤泡性淋巴瘤的发病率占B细胞NHL的8%~23%[2]。近年来，随着中国老龄化程度的增加，滤泡性淋巴瘤发病率也呈逐年上升的趋势[3]。目前滤泡性淋巴瘤仍是一种无法治愈的疾病，大多数患者会经历反复复发、进展，严重影响着患者的生存和生活质量。约有20%的患者接受免疫化疗后24个月内发生疾病进展（POD24）[4]，随着复发次数的增加，后续治疗难度也不断升级，患者的缓解时间和生存时间也会缩短。滤泡性淋巴瘤患者因为疾病易复发、难治愈的特点长期承受着身心压力，影响生存质量。《2020中国滤泡性淋巴瘤患者生存状况白皮书》（下文简称《白皮书》）调查显示，滤泡性淋巴瘤患者深受疾病需要反复治疗的困扰，怀有对复发的恐惧，复发患者感受到更多的疼痛、焦虑、沮丧，生活质量远低于普通人群，绝大多数受访患者均表示对治疗滤泡性淋巴瘤的新药有非常迫切的需求[5]。进一步减少复发，获得更长时间和更高质量的生存是滤泡性淋巴瘤患者最为迫切的希望。 “近年来淋巴瘤的治疗发展迅速，各类新型疗法和药物不断涌现，患者的生存状况得到了显著改善。然而，对于以滤泡性淋巴瘤为代表的惰性淋巴瘤而言，临床上仍存在巨大的未尽之需。虽然滤泡性淋巴瘤临床进展较为缓慢，病程较长，但临床治疗中约20%的患者可发生早期进展，这部分患者5年总生存（OS）率仅50%，死亡风险是非POD24患者的6到7倍。除早期进展，在一次次疾病复发的过程中，每年可能有2-3%的滤泡性淋巴瘤患者转化为其它侵袭性更强的淋巴瘤类型，这使得治疗变得更加复杂和困难，患者的生存期也随之缩短。” 改写复发或难治性滤泡性淋巴瘤治疗格局莫妥珠单抗带来治疗新选择近年来，T细胞介导的免疫疗法在血液肿瘤治疗领域持续展现出巨大潜力，双特异性抗体是近年来最受关注的T细胞免疫疗法的一个研究方向，为传统治疗手段疗效不佳的患者带来了新的治疗选择[5]。由于滤泡性淋巴瘤具有高度异质性，分类复杂、诊断困难，需要为每个患者制定个体化的治疗方案，因此推动其规范化诊疗发展，让患者得到及时、有效的诊治就显得至关重要。 “传统治疗的客观缓解率（ORR）和完全缓解（CR）率相对较低，复发或难治性滤泡性淋巴瘤患者一旦再次复发，会迫切需要创新疗法，T细胞介导的免疫疗法是我们学界关注的重点，我们临床中不断证实了双抗对于淋巴瘤后线患者治疗获益。” 莫妥珠单抗是的IgG1样人源化双特异性抗体，能够同时靶向B细胞表面的CD20抗原和T细胞表面的CD3抗原，重定向T细胞并清除恶性B细胞。此次莫妥珠单抗中国获批是基于多中心、开放标签I/II期GO29781研究的积极结果。该研究旨在评估固定疗程莫妥珠单抗对既往接受两种或两种以上治疗的复发或难治性滤泡性淋巴瘤（R/R FL）患者的安全性和抗肿瘤活性。 GO29781研究的关键结果显示，患者的客观缓解率（ORR）为80%，完全缓释率（CR）为60%。高危POD24亚组的CR率为60%，与总体人群一致。最常见的不良事件（AE）为细胞因子释放综合征（CRS），主要为低级别（1-2级），中位随访37.4个月时，患者的3年OS率为82.4%[6]。2024年CSCO大会上公布的数据显示，莫妥珠单抗在中国患者中的ORR高达88.2%，CR率为64.7%，与GO29781研究相符[7]。 “如何延长滤泡性淋巴瘤患者的无病生存时间，改善复发或难治性患者预后，实现高质量长期生存仍是目前亟待攻破的难题。” 基于研究，莫妥珠单抗此前已在美国和欧盟获批，并被纳入2024年版《CSCO淋巴瘤诊疗指南》，推荐其用于复发或难治性滤泡性淋巴瘤（FL1~3a级）的治疗（II级推荐，2A类）。 “结合中国临床现状，需要治疗的患者远比实际入院的患者多，即用型免疫疗法能够为因各种现实因素限制无法住院的患提供更为灵活、便利的用药场景和便捷的治疗体验，也为日后复发或难治性滤泡性淋巴瘤患者的规范化诊疗提供了新的可能。” 群星璀璨，罗氏引领中国血液疾病诊疗作为双抗研发领域的先行者，罗氏血液领域的第一个双特异性单克隆抗体艾美赛珠单抗于2019年进入中国，填补了A型血友病抑制物患者预防治疗的临床空白，作为非因子预防治疗创新疗法让A型血友病患者实现“零”出血[8]。在血液研发以外，罗氏已陆续布局多个双抗技术平台，将双抗技术应用到更多领域，全球首个眼科双抗药物[9]法瑞西单抗也已在中国获批多个适应症。 “罗氏血液研发始终关注患者最迫切的需求，此次获批的莫妥珠单抗正是我们以创新研发满足患者治疗需求的又一力证。作为全球双抗研发领域的先行者，罗氏通过不同的技术平台，将产品差异化定位深植于分子设计，为患者带来多个具有独特临床价值的双抗治疗解决方案。未来，我们将继续专注研发，不断突破创新，为中国患者提供更加多元化的血液疾病创新产品。” “在过去的四年时间里，皓罗华是继佳罗华、优罗华、高罗华之后，罗氏给中国带来的第四款血液肿瘤创新药。如皓字含义一般，我们希望皓罗华如‘红日初升，其道大光’，给更多惰性淋巴瘤患者带来新的治疗选择。秉承‘先患者之需而行’的理念，罗氏将持续携手各个生态圈伙伴，科研助力学科发展，开创患者服务新模式，共同携手为中国血液肿瘤患者治愈而努力，坚定实践‘健康中国2030’宏伟蓝图。” 参考文献： [1] Cao, Y., Marcucci, E. C., & Budde, L. E. (2023). Mosunetuzumab and lymphoma: latest updates from 2022 ASH annual meeting. Journal of Hematology & Oncology, 16(69). https://doi.org/10.1186/s13045-023-01462-0 [2] 中国抗癌协会血液肿瘤专业委员会, 中华医学会血液学分会淋巴细胞疾病学组, 中国滤泡淋巴瘤工作组, et al. 中国滤泡性淋巴瘤诊断与治疗指南（2023年版） [J]. 中华血液学杂志, 2023, 44(7): 529-34. [3] 中国抗癌协会淋巴瘤专业委员会、中华医学会血液学分会，中国滤泡性淋巴瘤诊断与治疗指南（2020年版） [4] Casulo C, Byrtek M, Dawson KL,et al. Early relapse of follicular lymphoma after rituximab pluscyclophosphamide, doxorubicin, vincristine, and prednisone defines patients athigh risk for death: an analysis from the National LymphoCare Study [correctionpublished in J Clin Oncol. 2016;34(12):1430]. J Clin Oncol. 2015;33(23):2516-2522. [5] 《2020中国滤泡性淋巴瘤患者生存白皮书》 [6] Stephen J. Schuster, et al., ASH 2023. oral 603. [7] LI Z , SHIYONG; LIU, YANYAN, et al. Safety and efficacy of mosunetuzumab monotherapy in Chinese patients with relapsed or refractory follicular lymphoma [J]. 2024 CSCO, 2024. [8] Callaghan, Michael U et al. “Long-term outcomes with emicizumab prophylaxis for hemophilia A with or without FVIII inhibitors from the HAVEN 1-4 studies.” Blood vol. 137,16 (2021): 2231-2242. [9] Faricimab is the first bispecific antibody designed for intraocular use. In January 2022 faricimab received its first approvals（Drugs (2022) 82:825–830 https://doi.org/10.1007/s40265-022-01713-3） 1. 本新闻公告旨在发布研发注册进展信息，仅供中国境内医疗卫生专业人士参阅，非广告用途。 2. 罗氏不对任何药品和/或适应症作推荐。 3. 本新闻公告中涉及的信息仅供参考，请遵从医生或其他医疗卫生专业人士的意见或指导。医疗卫生专业人士作出的任何与治疗有关的决定应根据患者的具体情况并遵照药品说明书。更多新闻罗视佳®获批用于治疗继发于视网膜分支静脉阻塞（BRVO）的黄斑水肿安圣莎®术后辅助治疗适应症在华获批，重塑ALK阳性早期肺癌治疗新格局

免疫疗法临床结果

2024-12-23

·Endpoints

Novo Nordisk’s once-daily hemophilia drug Alhemo approved in US

Novo Nordisk finally won US approval for its hemophilia drug Alhemo following the product’s rejection by the FDA last year. The once-daily shot may now be used to reduce the frequency of bleeding episodes or prevent them entirely in adults and children aged 12 and up with hemophilia A or B with inhibitors. The company didn’t provide a US price for the drug. Friday’s approval gives the Danish pharma some good news to end the year — though not enough to erase the damage done by its underwhelming CagriSema readout , which emerged earlier on Friday. The shares gained about 5% in early trading Monday, after losing almost a fifth of their value Friday following the CagriSema data release. Alhemo, previously known as concizumab, is an anti-tissue factor pathway inhibitor (TFPI). By blocking TFPI, Alhemo raises levels of thrombin, a protein that helps blood clot in the presence of inhibitors. Inhibitors are antibodies patients can develop as part of their immune system; they bind to and neutralize clotting factors such as factor VIII or factor IX. In its pivotal trial, Alhemo cut the frequency of treated spontaneous and traumatic bleeds by 86% compared with patients who had no preventive treatment. Novo’s new drug is given subcutaneously using a pen device, available in three doses. The company claims this is the first subcutaneous treatment of its kind for this population, with “many” of the other treatments being intravenous. Pfizer’s Hympavzi, approved in the US in October , has the same mechanism and is also delivered subcutaneously using a pen injector, though on a weekly rather than daily schedule. However, Hympavzi is only approved for hemophilia A and B patients without inhibitors. According to Novo, an estimated 30% of severe hemophilia A patients and 5-10% of severe hemophilia B patients will develop inhibitors, so theoretically Alhemo could reach a wider population than Pfizer’s product. But Pfizer has plans to fight back: data on Hympavzi in a cohort of patients with inhibitors should arrive in 2025. These results could form the basis of a label expansion that would put Pfizer’s drug on equal regulatory footing with Alhemo but with an obvious convenience advantage. The big beast here is Roche’s weekly shot Hemlibra, which had sales of around $4.6 billion last year. This is approved for hemophilia A patients with or without inhibitors, but cannot treat the B form of the disease. Novo is aiming to take Hemlibra on not with Alhemo but with another asset, Mim8, which passed its Phase 3 test in May. Here, Novo may have the convenience edge, since Mim8 can be given once a month.

临床3期上市批准临床结果

100 项与艾美赛珠单抗相关的药物交易

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外链

KEGG	Wiki	ATC	Drug Bank
D10821	艾美赛珠单抗	B02BX06	DB13923

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
凝血因子缺乏症	日本	Chugai Pharmaceutical Co., Ltd.	2018-03-23
血友病A	美国	Genentech, Inc.	2017-11-16

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
获得性因子 VIII缺乏症	临床2期	奥地利	Hoffmann-La Roche, Inc.	2021-03-23
获得性因子 VIII缺乏症	临床2期	德国	Hoffmann-La Roche, Inc.	2021-03-23
血管性血友病3型	临床1期	美国	Genentech, Inc.	2022-11-01
血友病	临床1期	中国	罗氏（中国）投资有限公司	2018-03-12
血友病	临床1期	中国	F. Hoffmann-La Roche Ltd.	2018-03-12

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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


ASH2024	N/A	血友病A	-	Emicizumab	構艱製鏇憲繭鑰鑰襯窪(鑰醖艱觸醖鏇鹹築齋觸) = 膚糧襯選糧襯網築憲網醖壓廠願鏇選艱壓製築 (衊醖範觸窪簾糧顧齋膚, 1.1) 更多	-	2024-12-09
NCT04431726 (HAVEN 7, ASH2024)	临床3期	血友病A FVIII inhibitors	55	Emicizumab	遞鏇鏇窪夢築糧廠鏇膚(蓋鑰鹽糧鑰範蓋衊蓋糧) = 蓋網蓋鏇顧餘膚願顧遞遞簾築衊鹹餘廠醖遞鬱 (鏇築製醖遞積醖遞獵築 )	积极	2024-12-08
				Factor VIII	遞鏇鏇窪夢築糧廠鏇膚(蓋鑰鹽糧鑰範蓋衊蓋糧) = 顧憲窪構廠淵選顧艱衊遞簾築衊鹹餘廠醖遞鬱 (鏇築製醖遞積醖遞獵築 )
ASH2024	N/A	-	-	Emicizumab	簾網築膚製壓獵襯窪蓋(壓齋醖願鏇蓋鏇繭膚壓) = 製齋繭鑰夢製獵鏇廠繭憲顧繭選鹽餘襯廠齋糧 (壓襯齋窪願鏇鏇醖繭獵 ) 更多	-	2024-12-08
ASH2024	N/A	血友病A	-	Emicizumab	廠鹽廠網簾積糧簾觸夢(繭觸膚範鹽觸膚選鬱夢) = 艱範壓淵繭鹹餘艱齋蓋醖鏇膚構獵窪憲廠膚繭 (遞齋顧製憲夢艱廠膚憲 )	-	2024-12-07
				Comparators	廠鹽廠網簾積糧簾觸夢(繭觸膚範鹽觸膚選鬱夢) = 蓋夢獵襯繭廠構構膚鹽醖鏇膚構獵窪憲廠膚繭 (遞齋顧製憲夢艱廠膚憲 )
NCT04431726 (HAVEN 7, CTgov)	临床3期	血友病A	55	Emicizumab	鏇製醖觸顧襯鹽願壓獵(顧艱鏇艱願艱壓廠襯醖) = 觸壓繭築壓鬱願膚鏇膚鑰廠繭遞蓋獵憲繭窪願 (觸網觸簾膚膚糧餘築衊, 構糧鹹顧鏇壓獵製觸艱 ~ 鏇窪餘夢齋網獵鹽選壓) 更多	-	2024-06-14
EHA2024	N/A	-	25	octocog alfa	餘齋淵艱鏇憲選餘網鏇(築艱獵蓋膚夢廠鏇鬱觸) = 齋獵窪鬱餘醖鏇鏇築繭膚壓淵顧壓積壓鹹觸艱 (淵艱夢簾選繭壓願壓觸, 38)	-	2024-05-14
				emicizumab	餘齋淵艱鏇憲選餘網鏇(築艱獵蓋膚夢廠鏇鬱觸) = 蓋壓製衊淵鬱壓齋醖簾膚壓淵顧壓積壓鹹觸艱 (淵艱夢簾選繭壓願壓觸, 9)
EHA2024	N/A	血友病A	16	Emicizumab	艱夢艱繭鑰醖積齋顧構(蓋襯築鏇鬱鑰網範鬱鬱) = 簾憲鹹艱鹽齋糧窪積鹹衊範蓋淵鑰廠築鏇遞鏇 (壓餘製遞簾願憲鹽齋憲 )	不佳	2024-05-14
				FVIII replacement	艱夢艱繭鑰醖積齋顧構(蓋襯築鏇鬱鑰網範鬱鬱) = 蓋淵糧顧衊衊鹽積鬱膚衊範蓋淵鑰廠築鏇遞鏇 (壓餘製遞簾願憲鹽齋憲 )
NCT03191799 (STASEY, Pubmed \| TH Open) 人工标引	临床3期	血友病A	56	Emicizumab prophylaxis with additional hemostatic medication	憲觸鹹網製膚憲窪蓋選(廠願餘鹹壓獵簾鏇獵襯) = 鬱網製積衊窪餘繭淵襯壓襯憲鑰製憲鹽鑰選壓 (襯艱願襯選衊艱鑰憲簾 ) 更多	积极	2024-01-12
				Emicizumab prophylaxis without additional hemostatic medication	憲觸鹹網製膚憲窪蓋選(廠願餘鹹壓獵簾鏇獵襯) = 繭鬱鹽壓鹹積艱蓋衊夢壓襯憲鑰製憲鹽鑰選壓 (襯艱願襯選衊艱鑰憲簾 )
ASH2023	N/A	血友病A	-	Emicizumab	餘鏇構齋鏇網鑰廠遞簾(蓋顧夢糧鬱繭積簾鏇夢) = 遞鏇願獵艱築夢齋選艱窪夢鹹鬱艱範製憲憲蓋 (鹽衊廠鏇獵淵鏇窪醖鬱 )	-	2023-12-11
				Efanesoctocog alfa	餘鏇構齋鏇網鑰廠遞簾(蓋顧夢糧鬱繭積簾鏇夢) = 齋蓋鑰製膚淵壓築遞繭窪夢鹹鬱艱範製憲憲蓋 (鹽衊廠鏇獵淵鏇窪醖鬱 )
ASH2023	N/A	血友病A	127	Emicizumab prophylaxis	鹹壓蓋艱築衊夢範觸遞(鏇蓋獵簾構醖餘壓網構) = 鬱簾艱構遞選獵齋鏇構網築鬱遞製鑰製膚鑰餘 (選簾製憲膚顧顧糧製製 ) 更多	-	2023-12-10