A Phase 3b Open-label Study Evaluating Physical Activity and Joint Health in Previously Treated Patients ≥12 Years With Severe Haemophilia A Treated With Recombinant Coagulation Factor VIII Fc-von Willebrand Factor-XTEN Fusion Protein (Efanesoctocog Alfa) for 24 Months

FREEDOM is a multicentre, open-label, single-arm, phase 3b study in Europe that aims to enrol approximately 90 previously treated severe haemophilia A patients aged ≥12 years, currently on prophylaxis. After a run-in period of 30-45 days, patients will receive efanesoctocog alfa prophylaxis, 50 IU/kg once-weekly for 24 months (additional preventive dose not permitted). An activity tracker and an electronic patient diary will be used to collect data on physical activity, bleeds, factor dosing, pain, and injuries from screening throughout the study.
The primary objective is to describe changes in physical activities over 24 months on efanesoctocog alfa prophylaxis, with a primary endpoint of change from baseline in International Physical Activity Questionnaire (IPAQ) at month 24.
Secondary objectives include relationship between physical activity and other variables (bleeds, joint status, pain, injuries, and quality of life); changes in joint status as assessed by HEAD-US, HJHS and MRI; occurrence of bleeds, injuries, pain. Safety and tolerability of efanesoctocog alfa will also be evaluated.

开始日期2023-07-31

申办/合作机构

Swedish Orphan Biovitrum AB

[+1]

NCT05911763 / RecruitingN/A

Prospective, Observational , Multicenter Study of Effectiveness of Efanesoctocog Alfa on Long-term Joint Health in Patients With Hemophilia A

This is a prospective, observational, multi-center longitudinal cohort study to describe the real-world effectiveness, safety and treatment usage of efanesoctocog alfa in patients with hemophilia A treated per standard of care in the US and Japan.
Patients will be enrolled in the study after the introduction of efanesoctocog alfa in the hemophilia treatment landscape in each study country. Decision to initiate treatment with commercially available efanesoctocog alfa will be made by the treating physician independently from the decision to include patients in the study. No study medication is provided. The data related to efanesoctocog alfa effectiveness, safety and usage will be collected prospectively during routine visits (expected annual/semi-annual visits) for up to 5 years following enrollment /treatment initiation.

开始日期2023-06-30

申办/合作机构

Sanofi

NCT05662319 / Recruiting临床3期

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

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

开始日期2023-02-01

申办/合作机构

Sanofi

100 项与 Efanesoctocog alfa 相关的临床结果

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100 项与 Efanesoctocog alfa 相关的转化医学

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100 项与 Efanesoctocog alfa 相关的专利（医药）

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项与 Efanesoctocog alfa 相关的文献（医药）

2024-02-15·Haematologica

Efanesoctocog alfa: the renaissance of Factor VIII replacement therapy.

Article

作者: Yesim Dargaud ; Alexandre Leuci ; Alejandra Reyes Ruiz ; Sebastien Lacroix-Desmazes

Efanesoctocog alfa (ALTUVIIIOTM, Sanofi-SOBI) is a B domain-deleted single-chain Factor VIII (FVIII) connected to D'D3 domain of von Willebrand Factor (VWF). Its ingenious design allows efanesoctocog alfa to operate independently of endogenous VWF and results in an outstanding 3-4 times longer half-life compared to standard and extended half-life (EHL) FVIII products. The prolonged half-life ensures sustained high levels of factor activity, maintaining normal to near-normal ranges for the majority of the week, facilitating the convenience of once-weekly administration. Efanesoctocog alfa received regulatory approval in 2023 for application in both adults and children with inherited hemophilia A in the United States and Japan. Its sanctioned use encompasses both prophylaxis and on demand treatment for bleeding episodes. The European Medicines Agency (EMA) is currently undertaking a comprehensive review of ALTUVIIIOTM. This comprehensive review focuses on the immunological profile of efanesoctocog alfa, a highly sophisticated new class of EHL FVIII molecule. The integration of the VWF D'D3 domain, XTEN polypeptides, and potential regulatory T-cell epitopes within various segments of efanesoctocog alfa collectively serves as a mitigating factor against the development of a neutralizing T-cell-mediated immune response. We hypothesize that such distinctive attribute may significantly reduce the risk of neutralizing antibodies, particularly in previously untreated patients. The discussion extends beyond regulatory approval to encompass the preclinical and clinical development of efanesoctocog alfa, including considerations for laboratory monitoring. The review also highlights areas that warrant further investigation to deepen our understanding of this groundbreaking therapeutic agent.

2024-01-15·Seminars in thrombosis and hemostasis

Shifting Paradigms and Arising Concerns in Severe Hemophilia A Treatment.

Article

作者: Rubhan Chandran ; Eusni R Mohd Tohit ; Johnson Stanslas ; Norazlinaliza Salim ; Tuan M T Mahmood ; Mogana Rajagopal

The management of hemophilia A has undergone a remarkable revolution, in line with technological advancement. In the recent past, the primary concern associated with Factor VIII (FVIII) concentrates was the risk of infections, which is now almost resolved by advanced blood screening and viral inactivation methods. Improving patients' compliance with prophylaxis has become a key focus, as it can lead to improved health outcomes and reduced health care costs in the long term. Recent bioengineering research is directed toward prolonging the recombinant FVIII (rFVIII) coagulant activity and synthesising higher FVIII yields. As an outcome, B-domain deleted, polyethylene glycolated, single-chain, Fc-fused rFVIII, and rFVIIIFc-von Willebrand Factor-XTEN are available for patients. Moreover, emicizumab, a bispecific antibody, is commercially available, whereas fitusiran and tissue factor pathway inhibitor are in clinical trial stages as alternative strategies for patients with inhibitors. With these advancements, noninfectious complications, such as inhibitor development, allergic reactions, and thrombosis, are emerging concerns requiring careful management. In addition, the recent approval of gene therapy is a major milestone toward a permanent cure for hemophilia A. The vast array of treatment options at our disposal today empowers patients and providers alike, to tailor therapeutic regimens to the unique needs of each individual. Despite significant progress in modern treatment options, these highly effective therapies are markedly more expensive than conventional replacement therapy, limiting their access for patients in developing countries.

2023-12-23·Blood reviews

Nothing short of a revolution: Novel extended half-life factor VIII replacement products and non-replacement agents reshape the treatment landscape in hemophilia A.

Review

作者: Hussien Ahmed H Abdelgawad ; Rachel Foster ; Mario Otto

Hemophilia A, an X-linked genetic disorder, is characterized by a deficiency or dysfunction of clotting Factor VIII. The treatment landscape has substantially changed by introducing novel extended half-life factor VIII (EHL-FVIII) replacement therapies such as efanesoctocog Alfa and non-factor replacement therapy such as emicizumab. These agents signal a shift from treatments requiring multiple weekly infusions to advanced therapies with long half-lives, offering superior protection against bleeding and improving patient adherence and quality of life. While EHL-FVIII treatment might lead to inhibitor development in some patients, non-factor replacement therapy carries thrombotic risks. Therefore, ongoing research and the generation of robust clinical evidence remain vital to guide the selection of optimal and cost-effective first-line therapies for hemophilia A patients.

118

项与 Efanesoctocog alfa 相关的新闻（医药）

2024-03-19

·药明康德

2024“最具创新力”10大医药公司榜单出炉！

▎药明康德内容团队编辑今日，知名商业媒体《快公司》（Fast Company）公布了“全球最具创新力的10家医药公司名单”。这份名单反映了过往一年治疗顽固性疾病上的重大突破，许多上榜公司为以往难以有效治疗的疾病带来“首款”或“改变游戏规则”的疗法，这些领域包含阿尔茨海默病、血友病、抑郁症等。这些入选公司代表着人类在医药创新路程中的前锋，其所开发的创新科技也代表着未来产业界的潜在方向。图片来源：123RF诺和诺德：释放GLP-1疗法在糖尿病与减重以外的潜力诺和诺德旗下重磅药物司美格鲁肽（semaglutide）分别在2017年与2021年获美国FDA批准上市，用于2型糖尿病患者的血糖控制（商品名：Ozempic）与治疗普通肥胖患者（商品名：Wegovy），该款药物的成功上市推动该公司的整体成长。然而诺和诺德并不止步于此，除了在减重与糖尿病领域持续不断布局外，该公司还积极探索司美格鲁肽在不同疾病的潜在应用，包含用于治疗早期阿尔茨海默病、非肝硬化非酒精性脂肪性肝炎（NASH）/代谢功能障碍相关脂肪性肝炎（MASH），以及降低2型糖尿病和慢性肾病（CKD）患者发生肾病相关事件风险等。这个月，Wegovy再获美国FDA批准，成为获批用于辅助患有心血管疾病及肥胖或超重的成年人预防重大心血管事件的首款减重药物。参考阅读：12周体重下降超13%！诺和诺德下一代减重疗法最新结果与公司开发战略公布Vertex Pharmaceuticals：将首款CRISPR疗法推进上市2023年11月Vertex及其合作伙伴CRISPR Therapeutics共同开发的CRISPR基因编辑疗法Casgevy（exagamglogene autotemcel，exa-cel）在英国获批上市，成为全世界首款获批的CRISPR基因编辑疗法。美国FDA也随后在2023年12月与今年1月先后批准该疗法用于治疗镰刀型细胞贫血病（SCD）与输血依赖性β地中海贫血（TDT）患者。除了专注于基因编辑疗法的开发，Vertex也即将递交其选择性NaV1.8抑制剂VX-548用于治疗中度至重度急性疼痛的新药申请。新闻稿指出，如果获得批准，这将成为数十年来首款具有新作用机制的止痛药。此外，Vertex也在上个月公布其用以治疗囊性纤维化（CF）的新一代三联疗法（vanzacaftor/tezacaftor/deutivacaftor）在三项3期临床试验中获得积极结果，该公司预计在2024年年中向美国、欧盟和加拿大的监管机构递交上市申请。卫材：致力于开发靶向淀粉样蛋白斑块的阿尔茨海默病疗法开发去年1月，由卫材（Eisai）和渤健（Biogen）联合开发的Leqembi（lecanemab）获得美国FDA的加速批准，治疗阿尔茨海默病，成为首款能延缓疾病进程、缓解认知功能衰退的获批疗法。Leqembi并在同年7月进一步获得FDA的完全批准，这是20年来FDA首次完全批准一款阿尔茨海默病药物。这款药物也登上《时代》周刊所发布的2023年度最佳发明的榜单。Leqembi与β淀粉样蛋白的可溶性寡聚体特异性结合。不但可以中和并清除具有毒性的可溶性寡聚体，还能快速降低患者大脑中的淀粉样蛋白沉积。在名为CLARITY AD的3期临床试验中，Leqembi与安慰剂相比，显著降低了试验主要终点CDR-SB的增加速度（CDR-SB评分越高意味着患者临床功能越低）。在第79周时与安慰剂组相比，平均CDR-SB评分的变化降低27%。公司同时也在开发更为方便使用的Leqembi皮下制剂，相关生物制品许可申请（BLA）预计在近日向FDA递交。Sage Therapeutics：开发出更快、更简便的产后抑郁症疗法美国FDA在去年8月批准由Sage Therapeutics和渤健共同开发的Zurzuvae（zuranolone）上市，使其成为首个获批用以治疗产后抑郁症（PPD）成人患者的口服疗法。在此之前，PPD患者的治疗仅可通过医护人员在医疗机构通过静脉注射给药。PPD是妊娠期间和妊娠后最常见的并发症之一。PPD可能给女性带来严重的负面影响，包括严重的功能障碍、情绪低落和/或对新生儿失去兴趣，以及相关的抑郁症状。之前所公布的3期试验结果显示，Zurzuvae达成试验主要终点以及所有的关键次要终点。参与者在使用药物第3天开始便有快速与显著的抑郁症状改善，并一直持续至第45天。Perrigo：推出首款非处方避孕药Perrigo所开发的Opill（norgestrel）片剂在2023年7月获得美国FDA批准，成为首个获批不用处方就可使用的每日口服避孕药。Opill仅含孕激素（progestin）一种有效成分，使用者可在一般药店、便利商店与网上自行购买。在过去，其他经批准的口服避孕药的配方和剂量只能通过医疗人员拿到处方后获得。根据FDA新闻稿，在美国，每年大约有610万例怀孕，其中近一半属于意外怀孕。意外怀孕与不良的孕产结果密切相关，这包括减少获得早期产前护理的机会、增加早产的风险，以及不利于新生儿、儿童发展和健康的后果。使用非处方的Opil可能有助于降低意外怀孕的发生率及其可能带来的负面后果。赛诺菲：保护独特的弱势患者群体在2023年，赛诺菲公司的两款创新疗法获得美国FDA的批准，解决了独特弱势患者群体的未竟医疗需求。2023年2月，赛诺菲的Altuviiio获得美国FDA的批准，这是首款只需每周一次治疗，就可预防血友病A患者出血的疗法。通常血友病A患者需要每周接受3-4次凝血因子VIII输注，这为他们的生活带来极大的不便。在名为XTEND-1的临床试验中，Altuviiio与预防性凝血因子VIII治疗相比，在预防出血方面也表现出优效性，显著降低血友病A患者的年出血率。此外，赛诺菲和阿斯利康联合开发的呼吸道合胞病毒（RSV）抗体Beyfortus（nirsevimab）也在2023年7月获得美国FDA批准上市。Beyfortus是首个只需一剂，就可以为所有婴儿和有感染风险的幼儿提供针对RSV保护的疗法。安斯泰来：开发首款治疗潮热的创新疗法安斯泰来开发的口服药物Veozah（fezolinetant）在2023年5月首次获得美国FDA的批准上市，用于治疗由于更年期引起的中度至重度血管舒缩症状（VMS，即潮热）。Veozah是神经激肽3（NK3）受体拮抗剂，可通过与受体结合以阻断其作用。神经激肽3受体参与大脑调节体温的机制。Veozah不是一种激素，而是靶向引起更年期潮热的神经活动。2023年7月公布的3b期临床试验结果显示，从基线到第24周，与安慰剂组相比，接受45 mg Veozah的患者的中度至重度VMS的发生频率在统计学上显著降低。Blueprint Medicines：为罕见血液疾病提供首款精准疗法2023年5月，Blueprint Medicines公司的Ayvakit（avapritinib）获得美国FDA的批准，治疗惰性系统性肥大细胞增多症（SM）成人患者。它是获批治疗惰性SM的首款精准疗法。Ayvakit是一种靶向激酶激活构象的1型抑制剂，它能够抑制多种携带KIT和PDGFRA基因突变的蛋白激酶。在约95%的SM患者中带有KIT蛋白D816V突变。Ayvakit的批准是基于双盲、安慰剂对照的临床试验PIONEER的数据。在该试验中，惰性SM患者每日一次接受25 mg的Ayvakit治疗并辅以支持性护理，对照组成员则接受安慰剂治疗外加支持性护理。相对于安慰剂治疗，Ayvakit在主要和所有关键次要终点上都显示出了显著的改善，包括总体症状和肥大细胞负荷。安进：扩展自身免疫疾病治疗的可及性2023年10月，安进公司开发的Wezlana获得美国FDA的批准，成为首款可替换重磅药物Stelara的可替换生物类似药（interchangeable biosimilar）。这款药物可治疗斑块状银屑病、银屑病关节炎、克罗恩病和溃疡性结肠炎。Wezlana无需咨询医生就可用于替代Stelara，它的上市有望扩展多种自身免疫疾病治疗的可及性。百济神州：为治疗常见白血病提供新选择2023年，百济神州开发的布鲁顿氏酪氨酸激酶抑制剂Brukinsa（泽布替尼）获得美国FDA的批准，用于治疗慢性淋巴细胞白血病（CLL）或小淋巴细胞淋巴瘤（SLL），这是最常见的白血病类型之一。这个月该款药物再获FDA加速批准，用以治疗既往经过至少二线系统治疗的复发或难治性（R/R）滤泡性淋巴瘤（FL）成人患者。2022年12月在《新英格兰医学杂志》发表的3期临床试验ALPINE的试验结果显示，泽布替尼与活性对照组相比，在无进展生存期（PFS）和总缓解率（ORR）方面均达到优效性标准。24个月时，泽布替尼组研究者评估的无进展生存率为78.4%，活性对照组为65.9%。经独立评审委员会（IRC）评估，在所有主要亚组中均观察到PFS获益，包括携带特定TP53突变的高风险患者亚组。泽布替尼是一款由百济神州科学家自主研发的BTK小分子抑制剂，目前正在全球进行广泛的临床试验项目，作为单药和与其他疗法进行联合用药治疗多种B细胞恶性肿瘤。由于新的BTK会在人体内不断合成，泽布替尼的设计通过优化生物利用度、半衰期和选择性，实现对BTK蛋白完全、持续的抑制。凭借与其他获批BTK抑制剂存在差异化的药代动力学，泽布替尼能在多个疾病相关组织中抑制恶性B细胞增殖。它在2019年首次获得美国FDA批准，迄今为止已获FDA批准5项适应症。大家都在看▲欲了解更多前沿技术在生物医药产业中的应用，请长按扫描上方二维码，即可访问“药明直播间”，观看相关话题的直播讨论与精彩回放参考资料：[1] The most innovative companies in medicines and therapeutics for 2024. Retrieved March 19, 2024 from https://www.fastcompany.com/91037063/medicines-therapeutics-most-innovative-companies-2024[2] FDA Approves First Nonprescription Daily Oral Contraceptive. Retrieved March 19, 2024 from https://www.fda.gov/news-events/press-announcements/fda-approves-first-nonprescription-daily-oral-contraceptive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。版权说明：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。转载授权请在「药明康德」微信公众号回复“转载”，获取转载须知。分享，点赞，在看，聚焦全球生物医药健康创新

上市批准基因疗法临床3期

2024-02-19

·赛柏蓝

2024年｜这13个重磅新药值得关注

特约作者 | 大可责任编辑 | 郑瑶在近期发布的《科睿唯安最值得关注的药物预测》中，列出了最近上市或即将上市的，有可能在五年内改变治疗模式和/或达到重磅炸弹地位的13款药物。01第一名：阿柏西普(8mg)这是一款再生元推出的眼科药物，用于治疗湿性年龄相关性黄斑变性(AMD)、糖尿病性黄斑水肿(DME)或糖尿病视网膜病变(DR)。科睿唯安预计，2027年阿柏西普(8mg)在G7市场湿性AMD患者中的销售额将达到17.7亿美元。这份预测基于“高剂量阿柏西普以更低频率给药就能达到与当前标准治疗(每 8周给药一次的阿柏西普2mg/艾力雅，或者基因泰克生产的每4周给药一次的诺适得®)相似的疗效和安全性。”现有阿柏西普2mg的治疗方案是，固定每8周治疗一次；而8mg阿柏西普是16周治疗一次。48周的数据显示出两者视力改善相当，积液被快速有效控制。诺适得是4周给药一次。02第二名：布地奈德肠溶胶囊Calliditas Therapeutics的布地奈德肠溶胶囊排名第二。这款药的中文商品名是耐赋康，可以说是近期乃至未来一段时间之内的“网红药”。它并不是传统的布地奈德激素，而是一款先进的治疗IgA肾小球肾炎药物，是该疾病唯一对因治疗药物，能够从疾病的源头靶向肠道粘膜B细胞，解决受损IgA产生的问题。在这个领域，亚洲人种是高发群体，而在欧美人群中却较为少见。另一方面，此前IgA肾小球肾炎的治疗几乎是中成药的天下，以抗炎或降血压药物为主，所以不论是患者还是临床，都对这款药物充满期待。03第三名：Datopotamab deruxtecan阿斯利康和第一三共联合开发的癌症治疗药物Datopotamab deruxtecan，也被称为Dato-DXd，是一款ADC药物，靶向Trop2。Trop2是泛癌种靶点，也是近期肿瘤研究的热门。Dato-DXd的首要攻克目标是联合一线治疗不可切除的局部晚期或转移性三阴性乳腺癌，而近期公司又宣布，该药用于晚期非小细胞肺癌患者的TROPION-Lung01 III期试验，已经取得积极结果。就凭借拿下两大癌种，Dato-DXd的关注度从未减弱。2月19日，第一三共/阿斯利康联合宣布，其递交的Dato-DXd生物制品许可申请（BLA）获FDA受理，用于既往接受过全身治疗的局部晚期或转移性非鳞状NSCLC成年患者。04第四名：Efanesoctocog alfaEfanesoctocog alfa是赛诺菲与Sobi共同开发的一种新型融合蛋白药物，基于创新的Fc融合技术开发,每周一次治疗A型血友病，在2023年2月24日，获美国FDA批准上市。这是一种first-in-class高持续性的凝血因子VIII替代疗法，在传统治疗中，患者需要每周接受3-4次凝血因子VIII注射；而Efanesoctocog alfa是每周一次，即能显著降低严重A型血友病患者的年出血率。赛诺菲拥有该产品在美国开发和商业化的权利，Sobi负责该药物在欧洲和其他市场的开发和销售。05第五名：EnsifentrineEnsifentrine是Verona Pharma研发的、一种“first-in-class”的磷酸二酯酶3和4（“PDE3”和“PDE4”）双重抑制剂，在一个化合物中结合了支气管扩张剂和抗炎活性，预期能够减少中重度慢性阻塞性肺病(COPD)的恶化。Ensifentrine用于雾化，不会像当前的口服PDE抑制剂一样产生全身性副作用。如果获得批准，这将是同类首创，也是超过10年来首个可用于维持 COPD治疗的新机制药物。阻塞性气管疾病用药2021年样本医院销售数据显示，销售占比最高的企业是阿斯利康，总销售额达18亿元，占比39.38%；其次为英国的葛兰素史克，总销售额超过3亿元，占比6.90%。06第六名：Exagamglogene autotemcel （简称exa-cel）第七名：Lovotibeglogene autotemcel （简称lovo-cel）2023年12月8日，FDA批准CRISPR/Cas9基因编辑疗法Casgevy（通用名exagamglogene autotemcel，简称exa-cel）上市，用于治疗镰状细胞病（SCD）。同一天，另一款获批的基因疗法药物，是来自蓝鸟生物的Lyfgenia（lovotibeglogene autotemcel，lovo-cel），同样用于治疗12岁及以上患有镰刀型细胞贫血病（SCD）且有血管闭塞事件（VOE）史的患者。据FDA新闻稿，Lyfgenia和同日获得批准的Casgevy是用于治疗12岁及以上的镰状细胞病患者首批获得批准的细胞基因疗法，这代表了一项重要的里程碑式进展。Exa-cel由美国生物技术公司Vertex Pharmaceuticals与瑞士药企CRISPR Therapeutics共同开发。CRISPR Therapeutics的创始人之一是德国马克斯·普朗克病原学研究所的埃马纽埃尔·卡彭蒂耶（Emmanuelle Charpentier），她因在基因编辑技术方面的开创性成果与同伴共同获得2020年诺贝尔化学奖。据生物医药行业媒体Endpoints News报道，Casgevy在美国的定价为220万美元（约合人民币1576万元）。07第八名：Mirikizumab2023年10月26日，礼来的Mirikizumab获得FDA批准，用于治疗中度至重度活动期溃疡性结肠炎，此前，该药已于日本、欧盟、英国等地陆续获批。除UC适应症外，Mirikizumab还被开发用于治疗中度至重度活动性克罗恩病（CD）。这是一款自免领域的重磅产品，患者基数大却缺少药物。目前自免药物市场整体分为三大类：①风湿自免领域，主要包括类风湿关节炎、强直性脊柱炎等，为目前自免领域最庞大的市场；②皮肤自免领域，主要包括银屑病、特应性皮炎等；③胃肠自免领域，主要包括UC、CD等炎症性肠病，患者病程一般较为严重。08第九名：AKEEGA™2023年8月11日，强生旗下杨森公司宣布， AKEEGA™（niraparib+醋酸阿比特龙）获得FDA批准，治疗BRCA阳性、晚期且去势耐受的前列腺癌（mCRPC）。AKEEGA™是首个且唯一双作用口服片剂，niraparib是PARP抑制剂，醋酸阿比特龙是雄激素合成的强力抑制剂。09第十名：RSVPreF (ABRYSVO)第十一名：RSVPreF3 (AREXVY)这是两款已获批的呼吸道合胞病毒(RSV)疫苗，2023年也被称为“RSV疫苗商业化元年”。这一年的5月3日，葛兰素史克的RSV疫苗Arexvy获FDA批准上市，用于老年人群预防RSV感染导致的下呼吸道疾病，这是全球首款获批上市的RSV疫苗。5月31日，辉瑞的二价RSV疫苗Abrysvo获美国FDA批准上市，被用于预防60岁及以上人群出现由RSV引起的下呼吸道疾病；8月21日，FDA批准Abrysvo扩展用于妊娠32-36周孕妇，可预防出生至6个月婴儿因RSV病毒引起的下呼吸道疾病。2023前3季度，两款疫苗刚刚上市一个多季度，GSK的Arexvy销售额达7.09亿英镑，辉瑞的Abrysvo销售额达3.75亿美元。科睿唯安预计，在五年内，RSV 疫苗和预防治疗市场的规模，可能达到100亿美元。10第十二名：Talquetamab（Talvey）Talvey，又称为Talquetamab-tgvs，是由强生公司开发的一款GPRC5D/CD3双抗药物，于2023年8月9日获FDA批准上市，用于既往接受过大量治疗的复发性/难治性多发性骨髓瘤患者。Talvey是一种双特异性T细胞结合抗体，可以同时结合T细胞上的CD3和癌细胞上的GPRC5D。在体外，Talvey可通过激活T细胞，引起促炎细胞因子的释放，导致多发性骨髓瘤细胞的溶解死亡。11第十三名：Zolbetuximab安斯泰来的Zolbetuximab已获得FDA的优先审评资格，2023年8月，CDE也已受理了其上市许可申请。拟定适应症为：CLDN18.2阳性局部晚期不可切除或转移性HER2阴性胃或胃食管结合部（GEJ）腺癌患者的一线治疗。HER2是较早被发现可用于胃癌靶向治疗的靶点，第一款胃癌靶向药物曲妥珠单抗仅适用于HER2阳性胃癌患者。但是胃癌患者中大约80%是HER2阴性。K药、O药和信迪利单抗相继获批了一线治疗HER2阴性胃癌，是这部分患者目前治疗的首选。PD-L1在胃癌患者中的表达率为43%-59%，而CLDN 18.2在胃癌患者中的表达率可达49%-85%。因此，CLDN 18.2药物被期待能够为HER2阴性胃癌患者，带来革命性的治疗方案，Zolbetuximab被称为CLDN 18.2靶点药物的“领头羊”。科学的发展为制药行业带来了更多方法和选择，新的药物也需要拿到市场上检验。除了临床疗效，支付可及、医保政策、供应链、地缘政治、投资环境等诸多外部因素，都影响着一款药物能否成为“药王”。END投稿沟通：13810174402医药代表交流群扫描下方二维码加入银发经济市场机遇交流群扫描下方二维码加入觉得不错的话，左下角「关注账号」，右下角点一下「在看」，防止失联

抗体药物偶联物临床3期上市批准临床2期基因疗法

2024-02-01

·FiercePharma

Sanofi expects RSV antibody to ⁠breach blockbuster status in 2024, even as supply hitch drags on

On the whole, Beyfortus generated 547 million euros (about $592 million) for all of 2023, reaching 410 million euros in the second quarter of its launch. As Sanofi strives to become a global immunology leader, it's also busy making the most of a crucial immunization rollout with partner AstraZeneca. On both fronts, Sanofi expects to see dividends in 2024. Last year represented a "pivotal" stretch for the drugmaker, one in which the French pharma largely proved its thesis that it can become a global immunology powerhouse, CEO Paul Hudson said on a call with investors Thursday. Separately, Sanofi's AZ-partnered respiratory syncytial virus (RSV) antibody Beyfortus pulled down 547 million euros (about $592 million) during its first year on the market, a figure Sanofi expects to roughly double this year. The partners have been hamstrung on Beyfortus supply since last fall, and it will take plenty more manufacturing work before the antibody is available fully unfettered, execs explained. “Clearly, Sanofi and AstraZeneca are working very hard to extend [Beyfortus’] manufacturing network,” which includes packaging and filling sites, Sanofi’s vaccine chief Thomas Triomphe explained on the company’s earnings call. Many of those production lines still need to be cleared by regulatory authorities around the world, he added. “That’s the work that is ongoing right now in order to be able to completely ‘de-bottleneck’ the supply and have the full supply for season 2025,” Triomphe said. Supply should increase in 2024, Triomphe stressed, and Sanofi still expects Beyfortus to breach the blockbuster threshold ($1 billion) this year. While Sanofi expects some RSV sales in the first half of 2024, the third and fourth quarters will be “key” for Beyfortus revenues this year, Triomphe said. So far, Beyfortus' uptake in the U.S. has surpassed previous childhood immunization benchmarks, such as those for rotavirus and pneumococcal disease, according to the company. Sanofi has successfully delivered its antibody to a “broad infant population,” reaching some 35% of young patients in the U.S. and France and a whopping 90% in Spain, Triomphe said. On the flip side, Sanofi’s influenza vaccine business was down 4%, clocking 741 million euros ($802 million) last year, courtesy of dropping immunization rates and increased competition in the U.S., Triomphe explained. All told, the immunization arm of Sanofi snared 2 billion euros ($2.2 billion) for the latest 12-month period, an 8.3% increase. Elsewhere, Sanofi's Regeneron-partnered immunology superstar Dupixent was no slouch last year, either. The drug generated around 10.7 billion euros ($11.6 billion) in 2023, growing 34% across the globe. For 2024, Sanofi expects Dupixent to bring home roughly 13 billion euros in sales, with much of that boost being driven by an expected approval and launch in chronic obstructive pulmonary disease during the second half of the year. Sanofi is becoming a “launch engine,” Hudson said. In fact, Sanofi’s new roster of drugs Beyfortus, Altuviiio and Tzield reeled in an impressive 731 million euros for all of last year, which “exceeded” the company’s expectations, according to the CEO. Besides its launch efforts, Sanofi is rolling out a series of savings initiatives in a bid to save upward of 2 billion euros from 2024 to the end of 2025. The company will implement measures such as pipeline reallocation, "smart spending" and changes to country setups. Beyond Dupixent’s 13 billion euro sales goal, Sanofi expects vaccine sales writ large to grow in the mid single digits this year. In contrast, the company is expecting to take a hit in the first half of 2024 thanks to Aubagio’s loss of exclusivity. That drug’s plunge off the patent cliff represents Sanofi’s “last major [loss of exclusivity]” for the rest of the decade, Hudson said. Overall, Sanofi’s full-year sales for 2024 clocked in at 43.07 billion euros, growing 5.3% over the sum the company generated in 2023.

疫苗

100 项与 Efanesoctocog alfa 相关的药物交易

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研发状态

适应症	最高研发状态	国家/地区	公司
血友病A	批准上市	日本更多	Sanofi KK 更多
冯·维勒布兰德病	临床1期	法国更多	Bioverativ, Inc. 更多

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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


NCT04161495 (XTEND-1, Corporate Publications) 人工标引	临床3期	出血	150	Efanesoctocog alfa	(糧願鑰膚糧鏇壓鬱範築) = 窪衊築醖築觸簾鏇範獵廠積憲選淵膚積鑰鏇鑰 (廠鏇範窪鏇糧鑰鏇觸觸 )	积极	2022-07-11
ASH2023	N/A	血友病A	-	Emicizumab	鑰鑰淵鏇製繭齋顧醖艱(窪鹽鹹窪選顧襯鬱襯鑰) = 蓋繭齋衊壓構鏇衊衊糧鑰築積鑰製醖夢衊襯餘 (範鬱鏇淵廠蓋觸構淵鹽 )	-	2023-12-11
				Efanesoctocog alfa	鑰鑰淵鏇製繭齋顧醖艱(窪鹽鹹窪選顧襯鬱襯鑰) = 齋餘積簾選餘構鏇艱憲鑰築積鑰製醖夢衊襯餘 (範鬱鏇淵廠蓋觸構淵鹽 )
NCT04161495 (XTEND-1, ASH2022) 人工标引	临床3期	疼痛 \| 血友病A	159	Efanesoctocog alfa (Arm A)	(膚選鹹獵範願獵艱選鏇) = was improved or maintained (change from baseline≤0) for most patients (81.5% [97/119]) at Week 52 鏇鏇衊選鹽網鬱壓廠蓋 (範糧顧廠齋鑰選鏇衊襯 )	积极	2022-11-15
				Efanesoctocog alfa (Arm B)
NCT04161495 (XTEND-1, ASH2022) 人工标引	临床3期	血友病A	159	Efanesoctocog alfa	獵蓋淵鏇積獵鬱積膚製(窪選鏇壓醖範積繭蓋網) = 廠築窪製遞積醖膚鬱繭衊鏇鑰齋築簾淵憲構窪 (淵壓餘膚醖壓淵選夢範 ) 更多	积极	2022-11-15
NCT03205163 (CTgov)	临床1/2期	血友病A	16	Advate (Advate 25 IU/kg)	(壓顧觸襯網範膚鹽齋觸) = 鬱範艱壓製齋鹹衊糧選簾鹹築襯顧醖鑰網簾鑰 (蓋簾糧鑰繭選遞餘醖繭, 膚獵製醖鹽鏇廠鑰構壓 ~ 蓋選鹽鏇醖蓋積繭簾蓋) 更多	-	2019-12-02
				Advate (Advate 65 IU/kg)	(壓顧觸襯網範膚鹽齋觸) = 蓋艱範鹽鹹觸齋構糧窪簾鹹築襯顧醖鑰網簾鑰 (蓋簾糧鑰繭選遞餘醖繭, 鏇選淵蓋襯醖鹹觸壓鬱 ~ 鹹繭醖願蓋網壓構餘顧) 更多
XTEND-1 (ISTH2022)	临床3期	血友病A	217	Efanesoctocog Alfa (BIVV001)	鹽遞網遞鹹蓋鏇窪觸憲(衊積餘糧壓構蓋淵選糧) = 艱夢艱夢顧夢簾顧糧蓋鹹鏇餘艱鹹淵鏇築鏇鏇 (憲糧積積鏇壓顧窪顧憲 )	-	2022-07-09
NCT04759131 (CTgov)	临床3期	血友病A	74	efanesoctocog alfa (BIVV001) (BIVV001: Participants Aged <6 Years)	觸艱夢鏇廠醖糧顧鑰壓(窪顧繭築選製鬱簾鹽醖) = 廠鑰網鑰膚齋襯齋壓築壓繭築構淵醖構衊淵窪 (淵獵願鬱範範憲範蓋鏇, 蓋膚餘築衊鏇觸選襯齋 ~ 餘夢艱蓋願夢廠鏇鹹遞) 更多	-	2024-02-13
				efanesoctocog alfa (BIVV001) (BIVV001: Participants Aged 6 to <12 Years)	觸艱夢鏇廠醖糧顧鑰壓(窪顧繭築選製鬱簾鹽醖) = 築鹹選膚齋繭餘壓願窪壓繭築構淵醖構衊淵窪 (淵獵願鬱範範憲範蓋鏇, 醖齋醖網夢夢製築夢廠 ~ 製衊衊壓糧糧鹽繭鬱鑰) 更多
NCT04161495 (XTEND-1, Corporate Publications) 人工标引	临床3期	血友病A	-	efanesoctocog alfa	(積蓋窪窪構憲餘鹹醖構) = 鏇網蓋夢鏇糧鏇鹹觸齋願顧構範製觸觸範鏇醖 (鬱顧艱積簾積鑰鏇觸鏇 ) 更多	积极	2022-03-09
NCT04161495 (CTgov)	临床3期	血友病A	159	efanesoctocog alfa (BIVV001) (Arm A: Prophylaxis)	壓憲製鹹築簾構構醖鹽(膚壓憲糧鬱艱鏇壓構夢) = 築繭壓蓋遞淵壓襯簾壓醖膚憲齋壓淵獵憲鏇齋 (獵憲製網顧簾構襯獵鹽, 願獵鹹鏇壓願範構醖艱 ~ 廠餘製顧選遞蓋顧鏇醖) 更多	-	2023-05-24
				efanesoctocog alfa (BIVV001) (Arm B: On-Demand Then Prophylaxis)	範遞鏇觸鑰鬱範築襯網(範廠蓋餘簾憲構鹹壓憲) = 蓋餘餘積窪網鑰鹽製廠構鹹簾夢窪積醖窪艱憲 (窪鬱鏇憲膚齋夢鹽膚顧, 壓憲繭觸範夢淵繭觸糧 ~ 鹽夢壓鹹鑰網範齋醖鬱) 更多
NCT03205163 (EXTEN-A, Corporate Publications) 人工标引	临床1/2期	血友病A	14	BIVV001 (25 IU/kg)	(範簾觸廠淵觸糧壓選糧) = During the study period no adverse events of allergic reaction, anaphylaxis or clinically meaningful treatment-related adverse events were reported. 構壓積糧觸艱艱夢鏇憲 (膚鑰窪壓築鬱醖築獵艱 )	积极	2020-09-10
				BIVV001 (65 IU/kg)