This study is a survey in Japan of recombinant ADAMTS13 used to treat or to prevent participants with congenital thrombotic thrombocytopenic purpura (cTTP). The study sponsor will not be involved in how the participants are treated but will provide instructions on how the clinics will record what happens during the study.
The main aim of the study is to check for side effects related from recombinant ADAMTS13 and to check if recombinant ADAMTS13 improves or prevents cTTP.
During the study, participants with cTTP will take recombinant ADAMTS13 intravenous injection according to their clinic's standard practice. The study doctors will check for side effects from recombinant ADAMTS13 for 18 months.

开始日期2024-05-30

申办/合作机构

Takeda Pharmaceutical Co., Ltd.

NCT05714969

/ Recruiting临床2期

A Phase 2b, Multicenter, Randomized, Double-blind Study of Safety and Efficacy of TAK-755 (rADAMTS13) With Minimal to No Plasma Exchange (PEX) in the Treatment of Immune-mediated Thrombotic Thrombocytopenic Purpura (iTTP)

This is a study of TAK-755 in adults with immune-mediated thrombotic thrombocytopenic purpura (iTTP). The main aim of this study is to determine the percentage of participants with a clinical (Part 1) or platelet (Part 2) response without plasma exchange during the study. Participants who have an acute attack of iTTP will receive TAK-755 and immunosuppressive therapy during their stay at the hospital until they achieve a clinical response in Part 1 or platelet response in Part 2. Participants will also be treated with TAK-755 for an additional time of up to 6 weeks after the acute phase. In total, participants will stay in the study for approximately 3 months.

开始日期2023-03-21

申办/合作机构

Takeda Pharmaceutical Co., Ltd.

[+1]

JPRN-jRCT2031220348

/ Not yet recruiting临床3期IIT

A Phase 3b, prospective, open-label, multicenter, single treatment arm, continuation study of the safety and efficacy of TAK-755 (rADAMTS-13, also known as BAX 930/SHP655) in the prophylactic and on-demand treatment of subjects with severe congenital thrombotic thrombocytopenic purpura (cTTP; Upshaw-Schulman Syndrome, or hereditary thrombotic thrombocytopenic purpura)

开始日期2021-09-29

申办/合作机构-

100 项与阿帕达酶α 相关的临床结果

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100 项与阿帕达酶α 相关的专利（医药）

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113

项与阿帕达酶α 相关的文献（医药）

2025-10-01·BIOCHIMICA ET BIOPHYSICA ACTA-MOLECULAR CELL RESEARCH

Recombinant human ADAMTS13 attenuates LPS-induced acute kidney injury and renal microangiopathy in murine advanced liver fibrosis by cleaving vWF

Article

作者: Yoshiji, Hitoshi ; Tsuji, Yuki ; Nishimura, Norihisa ; Masuda, Hiroyuki ; Namisaki, Tadashi ; Kitagawa, Koh ; Sato, Shinya ; Kaji, Kosuke ; Takaya, Hiroaki ; Takami, Masayoshi ; Shibamoto, Akihiko

Hepatorenal syndrome (HRS) has a poor prognosis among the complication of cirrhosis, yet treatment options are limited. Thrombotic microangiopathy with reduced ADAMTS13 activity and vWF accumulation has been reported to play a key role in the pathogenesis of acute kidney injury (AKI) in cirrhosis. This study investigated the effect of recombinant ADAMTS13 (rADAMTS13) on AKI with carbon tetrachloride (CCl₄)-induced advanced liver fibrosis in mice. AKI was induced by intraperitoneal administration of acute insult with double dose of CCl₄ and lipopolysaccharide (AKI-F mice), and mice were treated with rADAMTS13 (10 μg/body). AKI-F mice showed a marked liver dysfunction as well as renal dysfunction with elevated serum level of renal damage markers including kidney injury molecule-1, osteopontin, and neutrophil gelatinase-associated lipocalin. In AKI-F mice, reduced plasma ADAMTS13 activity and increased vWF antigen levels resulted in the decrease in hepatic and renal blood flow. Treatment with rADAMTS13 increased plasma ADAMTS13 activity, decreased vWF antigen levels leading to recovery of liver and kidney blood flow. Consequently, both hepatic and renal injuries showed serological and histopathologic improvements with decreased F4/80⁺ macrophage infiltration and 4-hydroxynonenal⁺ oxidative damage after rADAMTS13 treatment. The deposition of CD41a-positive microthrombi in kidney tissues observed in the AKI-F mice was significantly suppressed by treatment with rADAMTS13. Concomitantly, rADAMTS13 treatment promoted angiogenesis as well as inhibited vascular inflammation in the kidney of AKI-F mice. In conclusion, administration of rADAMTS13 may improve inflammation, oxidative stress, and reduced blood flow in liver and kidney tissues, thereby mitigating hepatorenal syndrome.

2025-10-01·CLINICAL PHARMACOLOGY & THERAPEUTICS

Use of PopPK and E‐R Analyses toward Explaining Causal Link Between ADAMTS13 in Recombinant vs. Plasma‐Based Therapies and Clinical Effects in cTTP

Article

作者: Barriere, Olivier ; Wang, Linda T. ; Xu, Huijuan ; Wang, Wenping ; Zhu, Andy Z. X. ; Diderichsen, Paul ; Mellgård, Björn ; Bhattacharya, Indranil ; Peyret, Thomas ; Patel, Munjal ; Marier, Jean François ; Patwari, Parth

Congenital thrombotic thrombocytopenic purpura (cTTP) is an ultra‐rare, potentially life‐threatening condition caused by a deficiency of the blood enzyme ADAMTS13. Until now, ADAMTS13 replacement has been achieved with infusions of plasma or plasma‐based therapies (PBT). However, the quantitative relationship between ADAMTS13 plasma activity and clinical manifestations is poorly understood. We therefore conducted integrated population pharmacokinetics (PopPK) analysis and exposure–response modeling based on three clinical trials of recombinant ADAMTS13 (rADAMTS13, Takeda Pharmaceuticals U.S.A., Inc.) in patients with cTTP. These aim to assess the clinical benefit of rADAMTS13, which at the proposed dose of 40 IU/kg provides ADAMTS13 peak levels of approximately 100% of normal levels. The PK model indicated that, besides body weight–based dosing, no further dose adjustment was required based on age or race. The only extrinsic covariates with a significant impact on ADAMTS13 plasma activity levels were dosing interval and treatment type (rADAMTS13 vs. PBT). The correlation between ADAMTS13 plasma activity levels and cTTP manifestations was investigated with three different exposure–response models. Increasing exposure to ADAMTS13, as measured by average activity over a one‐to‐two‐week period, predicted the probability of disease manifestations, primarily assessed as thrombocytopenia. Model simulations predicted that >90% of patients treated with 40 IU/kg rADAMTS13 achieve an average ADAMTS13 plasma activity >13% of normal, which was shown to be highly protective against thrombocytopenia (>70% lower hazard). Similar results were observed for protection against elevation of lactate dehydrogenase, a marker of microangiopathic hemolytic anemia. Overall, these results support the use of rADAMTS13 treatment for patients with cTTP.

2025-10-01·CPT-Pharmacometrics & Systems Pharmacology

Quantitative Systems Pharmacology Modeling of Platelet Responses to Recombinant ADAMTS13 in Patients With Congenital Thrombotic Thrombocytopenic Purpura

Article

作者: Vakilynejad, Majid ; Bhattacharya, Indranil ; Zhu, Andy Z. X. ; Zhang, Zhiwei ; Tolsma, John ; McBride, Cameron ; Patwari, Parth ; Jiang, Jiaolong ; Mellgård, Björn

ABSTRACT:

Congenital thrombotic thrombocytopenic purpura (cTTP) is an ultra‐rare, life‐threatening thrombotic microangiopathy caused by a severe inherited deficiency of ADAMTS13, a von Willebrand factor (VWF) cleaving enzyme. Inadequate clinical endpoint data often make it challenging to statistically power clinical trials in ultra‐rare diseases. Therefore, utilizing in vitro, adamts13‐knockout mouse, literature‐based, and clinical data, a quantitative systems pharmacology (QSP) model was developed to describe the mechanistic relationship between ADAMTS13, VWF, and platelet count, and to supplement evidence from clinical trials of recombinant ADAMTS13 (rADAMTS13) for the treatment of cTTP. The effect of long‐term prophylaxis with rADAMTS13 versus plasma‐based therapies (PBT) on platelet count in patients with cTTP was investigated. One‐year clinical trial simulations of thrombocytopenia occurrences in 1000 virtual patients, phenotype‐matched to a cTTP Phase 3 study population (NCT03393975), were produced. Simulations suggested that once‐weekly (Q1W) or once every 2 weeks (Q2W) rADAMTS13 administered over 1 year resulted in fewer patients experiencing thrombocytopenia versus patients treated with PBT (e.g., Q2W [rADAMTS13] relative to Q2W [PBT], HR = 0.47 [platelet count drop to < 150 × 109/L], HR = 0.41 [< 100 × 109/L]). These results provide confirmative evidence to support the use of rADAMTS13 in cTTP by integrating the current mechanistic understanding of interactions between ADAMTS13 and VWF multimers as its substrate, as well as key downstream parameters, primarily platelet count. Virtual patient clinical simulations from the QSP model supported the regulatory approval of rADAMTS13 in cTTP, highlighting the significant potential of QSP modeling to supplement clinical trial data in rare disease drug development.

100

项与阿帕达酶α 相关的新闻（医药）

2025-05-21

·蒲公英Ouryao

Paul's Insight｜解读国际法规药事(05.12-05.18)

◆ ◆ ◆ ◆Paul's Insight2025年5月12日-5月18日◆ ◆ ◆ ◆在过去一周中，FDA、EMA、USP、EDQM、MHRA、PDA和ISPE等多家监管机构和行业组织接连有重要更新。从疫苗安全预警到生产质量整顿，从标准更新到专家圆桌，从学术交流到创新表彰，一系列事件与热点既折射出全球药事监管的高压态势，也勾勒出行业协同创新的美好愿景。美国FDA安全审查与监管松绑并行1. Ixchiq疫苗安全警示5月12日，FDA与CDC联手建议暂停60岁及以上人群接种基孔肯雅活疫苗Ixchiq，并针对 серьieux不良反应报告展开紧急调查。截至5月7日，全球共报告17例严重不良事件，包括两例死亡病例，多数患者均有慢性基础疾病。FDA将开展最新的效益-风险评估，并与CDC一道持续监测，承诺在评估完成后及时向公众通报。图：Ixchiq疫苗安全警示https://www.fda.gov/safety/medical-product-safety-information/fda-and-cdc-recommend-pause-use-ixchiq-chikungunya-vaccine-live-individuals-60-years-age-and-older2. 中国药企遭警告信：海外飞行检查常态化5月13日，美国FDA官网公布了三封针对中国企业的警告信，三家企业分别位于宁波、汕头和台州。这些警告信指出，三家企业在药品及相关产品生产中存在严重的CGMP违规。例如，警告信开篇便明确指出：“本警告信总结了针对成品制剂在现行CGMP法规方面的重大违规”；并指出三家企业“生产、加工、包装或贮藏方法、设施或控制不符合CGMP”要求，其产品被认定为“掺假”。这意味着，上述企业未能满足FDA对于制造过程和质量控制的严格要求，所生产的产品在美国法律中被视为有重大问题，需要整改。2025年5月，美国政府发布行政令，要求FDA提高国内生产审查效率，同时加强对外国生产设施的检查。随后，FDA宣布，将扩大对向美国供应食品、药品和医疗器械的外国工厂实施无预告突击检查（飞行检查）的范围，确保对美国海外厂商和本土厂商实行同等监管。这一政策调整是FDA海外检查力度提升的标志性事件，意味着国外企业今后可能像美国本土工厂一样，随时面临FDA不通知的现场检查。图：宁波、汕头、台州三企业被警告，FDA海外监管新常态来临https://www.fda.gov/inspections-compliance-enforcement-and-criminal-investigations/warning-letters/taizhou-jingshang-cosmetics-technology-co-ltd-701945-04022025https://www.fda.gov/inspections-compliance-enforcement-and-criminal-investigations/warning-letters/shantou-sez-baojie-industry-co-ltd-703439-04152025https://www.fda.gov/inspections-compliance-enforcement-and-criminal-investigations/warning-letters/ningbo-riway-industrial-co-ltd-692870-043020253. 监管法规减负：10比1规则启动5月13日，美国FDA及其上级部门卫生与公众服务部（HHS）发布新闻稿，宣布启动针对“过时或不必要的监管法规”的公开征求意见，以识别并废除相关法规。官方声明指出，此举是美国联邦政府降低监管负担、提高透明度的整体行动的一部分，与特朗普总统颁布的行政命令《通过放松监管来实现繁荣》精神一致。图：FDA启动针对“过时或不必要的监管法规”的公开征求意见https://www.fda.gov/news-events/press-announcements/hhs-fda-issue-rfi-deregulatory-plan-lower-costs-and-empower-providers4. 滑石粉安全公开圆桌5月16日，FDA局长Martin A. Makary宣布将于5月20日在银泉市举办首场对公众开放的独立专家圆桌会议，聚焦滑石粉在食品、药品和化妆品中的安全评估。此次讨论将审查最新科学证据，并在媒体与公众见证下，决定未来监管路径。图：FDA 将举办首届有关滑石粉的独立科学专家小组会议，并向公众开放https://www.fda.gov/news-events/press-announcements/fda-host-inaugural-independent-scientific-expert-panel-open-public美国药典USP新一届董事会就位，宏观战略再出发5月16日，美国药典委员会USP宣布了2025-2030周期新当选董事会成员，涵盖来自学术、产业、监管、患者团体等450余家组织的代表。新任领导团队将引领USP在接下来的五年里，继续完善标准、提升供应链韧性，并与全球伙伴携手打造优质、安全的药品生态。图：USP宣布新一届董事会https://www.usp.org/news/usp-announces-board-of-trustees-for-the-2025-2030-cycle美国PDA校企合作，培养行业新血5月13日，美国注射剂协会PDA发布报道，俄亥俄州立大学学生应邀参访其校企合作项目STAQ Pharma，这家FDA注册的503B外包机构向学子们展示了从原料到包装的全流程，并通过专家讲座与小组讨论，为学生们打开了学术与产业对接的全新视野。图：PDA连接学术界和产业界https://www.pda.org/pda-letter-portal/home/full-article/bridging-academia-and-industry欧盟EMA疫苗成分与药品短缺1. 与病毒赛跑，疫苗成分再升级在欧洲，EMA紧急工作组（ETF）建议更新2025/2026年新冠疫苗，将目标由JN.1家族转向新兴LP.8.1变体。ETF综合世界卫生组织（WHO）及国际合作伙伴的数据，以及针对LP.8.1候选疫苗效果的动物研究，认为及时调整疫苗成分可维持对最新流行株的防护。在更新LP.8.1疫苗上市前，各上市许可持有人仍可考虑使用针对JN.1或KP.2毒株的疫苗，并应立即与EMA对接，讨论其授权疫苗成分的更新事宜。图：EMA建议更新新冠疫苗以针对新的LP.8.1变体https://www.ema.europa.eu/en/news/etf-recommends-updating-covid-19-vaccines-target-new-lp81-variant2. EMA药品短缺平台问答更新EMA在其欧洲短缺监测平台（ESMP）建议更新中，回答了六个与数据提交和报告要求相关的新问题：提交信息保密性：ESMP中提交的短缺数据不会在历史记录中公开显示；质量缺陷通知：涉及质量问题的短缺，无论批次是否已上市，都应提交质量缺陷通知；数据使用：EMA将利用平台数据进行短缺监测和风险分析，并在危机期间开放准备提交部分；根本原因报告：如标准选项无法覆盖短缺原因，应选择“其他”并提供自由文本说明；关键药物清单：目前与ESMP报告要求未直接关联，但两者可能存在内容重叠。图：EMA药品短缺平台问答更新https://www.ema.europa.eu/system/files/documents/other/202505_esmp-faqs_v31_for-publication-update_en.pdf欧洲EDQM新通则，支原体检测迈入新时代3月的第181届欧洲药典委员会会议上，EPC正式通过2.6.7章“支原体”通则及11个专论修订稿，纳入最新核酸扩增技术(NAT)与培养法联合检测策略，将于2025年10月发布并于2026年4月1日生效，为疫苗与生物制剂安全把关提供更灵活、风险导向的检测框架。修订后的通则规定，应联合使用培养法和指示细胞培养法（或NAT法），以确保检测出“可培养”和“不可培养”的支原体，除非专论另有规定或风险评估证明其合理性并经主管部门授权。此外，还明确指出，只要可能，样品应同时包含细胞和上清液。此外，现在规定，可以从拟议清单中选择合适的菌株，并可根据风险评估，结合产品类型和生产工艺，使用其他菌株。图：欧洲药典采用支原体通则和专论https://www.edqm.eu/en/-/epc-adopts-mycoplasmas-general-chapter-and-monographs-updated-to-incorporate-latest-analytical-developments英国MHRA英国多剂新药与新适应症获批在英国，监管机构也在稳步推进新药品的上市与适应症扩展。1. 首个专门针对先天性血栓性血小板减少性紫癜患者的治疗方案MHRA于5月12日采用国际认可程序（IRP）批准了rADAMTS13（商品名ADZYNMA），这标志着英国迎来了首个专门针对先天性血栓性血小板减少性紫癜（cTTP）患者的治疗方案。cTTP是一种罕见的遗传性血液病，患者往往因为ADAMTS13酶缺乏而发生血栓，导致多器官损伤。ADZYNMA的上市不仅填补了这一领域的治疗空白，也为各年龄段患者带来了真正意义上的个性化干预。图：先天性血栓性血小板减少性紫癜患者的治疗方案https://www.gov.uk/government/news/mhra-approves-first-uk-treatment-for-congenital-thrombotic-thrombocytopenic-purpura-cttp2. 批准了肺炎球菌疫苗Capvaxive紧接着，5月15日MHRA批准了肺炎球菌疫苗Capvaxive，可用于18岁及以上成年人。上市许可持有人为默沙东（Merck Sharp & Dohme）（英国）有限公司。这款疫苗旨在预防由肺炎链球菌引起的一系列严重感染，包括肺炎、脑膜炎和菌血症等。通过激发机体产生针对不同血清型的抗体，Capvaxive有望为高危人群提供更全面的保护，从而在秋冬流感季来临之际进一步降低呼吸系统疾病的负担。图：MHRA批准了肺炎球菌疫苗Capvaxivehttps://www.gov.uk/government/news/mhra-approves-vaccine-to-protect-against-pneumococcal-infections-such-as-pneumonia-and-meningitis3. MHRA 批准 Guselkumab 用于治疗克罗恩病和溃疡性结肠炎随后，在5月16日，MHRA又批准了Guselkumab（商品名Tremfya）在炎症性肠病领域的新适应症，将其应用范围从银屑病和银屑病关节炎扩展至克罗恩病和溃疡性结肠炎（UC）。上市许可持有人为杨森制药有限公司（Janssen-Cilag Limited）。多项大型III期临床试验数据显示，接受Guselkumab治疗的中重度活动性克罗恩病患者中，约一半能够在12周内达到临床缓解；在溃疡性结肠炎患者群体中，长期维持治疗的有效率也显著高于安慰剂组。这些结果表明，Guselkumab有望成为一线或二线治疗方案，为对现有疗法响应不佳的患者提供更多选择。图：MHRA 批准 Guselkumab 用于治疗克罗恩病和溃疡性结肠炎https://www.gov.uk/government/news/mhra-approves-guselkumab-for-crohns-disease-and-ulcerative-colitisISPE年度最佳设施奖，表彰制药创新先锋在5月12日至14日于伦敦举办的ISPE欧洲年会上，国际制药工程学会（ISPE）隆重宣布了2025年年度最佳设施奖（FOYA）的六位分类获奖者及两位荣誉提名者，以纪念该奖项成立20周年。来自英国的Touchlight公司凭借合成DNA项目斩获创新奖，强生创新医学分别赢得运营卓越与项目执行两项殊荣，澳大利亚CSL Behring的“奥罗拉”分馏设施荣获制药4.0奖，拜耳医疗公司在社会影响领域摘得“双料”大奖，而赛诺菲的多伦多废水回收厂则荣膺可持续性卓越奖；德国的Erbe Elektromedizin和美国United Therapeutics获得荣誉奖提名。ISPE副总裁Parag Sane点评称，这些先锋项目展现了行业对高质量生产、可持续发展和患者福祉的不懈追求，FOYA总冠军将在今年10月于北卡罗来纳州夏洛特举行的ISPE年会暨博览会上揭晓。图：ISPE 宣布 2025 年度最佳设施奖 (FOYA) 类别获奖者https://ispe.org/news/ispe-announces-2025-facility-year-awards-foya-category-winners-celebrating-foyas-20th结语过去一周，全球各大监管和行业组织频频发声，既有针对疫苗和新药品安全性的严格审查，也有法规简化和透明度提升的改革举措，更不乏前沿技术和产业合作的创新案例。END声明：本文仅代表作者个人观点，不代表任何组织及本公众号立场，如有不当之处，敬请指正。如需转载，请注明作者及来源：蒲公英Biopharma。活动推荐5月27日 | 线上 | 解锁2025版药典：药用原辅包标准升级与关键变化应对策略5月28日 | 线上 | 计算机化系统合规高效管理分享5月29日 | 线上 | 2025新药典升级：制药质控实验室技术及管理应对策略6月19日 | 线下 | 中国先进治疗药物先锋论坛6月25日 | 线下 | 2025 CPHI 生物药商业化生产与创新论坛——分论坛：抗体药物生产能力建设研讨会6月25日 | 线下 | 2025 CPHI 生命科学与技术创新发展论坛——论坛二：细胞药物质量控制法规与技术论坛

疫苗申请上市

2025-05-13

·PMLiVE

Takeda’s Adzynma approved by MHRA for rare blood clotting disorder cTTP

Takeda’s Adzynma (rADAMTS13) has been approved by the Medicines and Healthcare products Regulatory Agency (MHRA) to treat congenital thrombotic thrombocytopenic purpura (cTTP). The enzyme replacement therapy, which has been authorised to treat cTTP in patients of all ages, is now the first treatment approved in the UK for the ultra-rare blood clotting disorder. cTTP is an inherited condition caused by a deficiency in ADAMTS13, an enzyme critical to blood clotting. Patients can experience a variety of symptoms and severe complications, including thrombocytopenia, stroke, and renal and cardiovascular disease. Administered intravenously, Takeda’s Adzynma is a purified recombinant form of ADAMTS13 that is designed to provide a replacement for the low levels of the deficient enzyme. The MHRA’s decision on Adzynma occurred through the International Recognition Procedure, a fast-track approval process that takes into account prior authorisations from other regulatory partners, and follows the European Commission’s approval of the therapy in August. Among the evidence supporting the EU authorisation were results from a phase 3 trial of cTTP patients who were randomised to receive six months of Adzynma or plasma-based therapies and then crossed over to the other treatment for another six. All patients then received Takeda’s therapy for months 13 to 18. No patient experienced an acute thrombotic thrombocytopenic purpura (TTP) event while receiving Adzynma as a preventative treatment, while there was one acute TTP event in a patient receiving plasma-based therapies. Additionally, one subacute TTP event was seen in the Adzynma cohort during periods one and two of the study, versus seven subacute TTP events in six patients receiving plasma-based therapies. Efficacy results were consistent in the continuation period, the company said. “A century after the scientific discovery of cTTP, significant unmet needs remain for patients who continue to face life-threatening acute events and debilitating chronic symptoms with limited treatment options,” Ricardo Marek, president, Europe and Canada business unit, Takeda, said in August. Adzynma was also approved by the US Food and Drug Administration for CTTP in 2023.

临床3期上市批准临床结果快速通道

2025-05-09

·GBIHealth

再鼎、武田发布财报

企业动态No.1 / 再鼎医药2025Q1：收入增长22%至1.065亿美元2025年5月9日，再鼎医药（NASDAQ：ZLAB；HKEX：9688）公布了2025年第一季度（2025Q1）的财务业绩。公司报告期内总收入为1.065亿美元，同比增长22%；经营亏损显著改善，同比缩窄20%至5630万美元，有望稳步实现2025年第四季度盈利的目标。期内研发开支6070万美元，期末现金及现金等价物、短期投资和流动受限制现金总计为8.573亿美元。再鼎医药2025Q1产品收入净额为1.057亿美元，同比增长21%。这一增长主要由卫伟迦、则乐和纽再乐销售额增长所驱动。得益于销售放量与重症肌无力新适应证纳入新版国家医保目录（NRDL），卫伟迦（艾加莫德α静脉输注）及卫力迦（艾加莫德α皮下注射）期内录得1810万美元，市场覆盖率和渗透率持续提升。则乐（尼拉帕利）Q1收入净额为4950万美元，继续保持中国内地卵巢癌领域PARP抑制剂院内销售领军者地位。纽再乐（奥玛环素）录得1510万美元。在研管线中，ZL-1310（靶向 DLL3 的抗体药物偶联物）正在快速推进，近期将公布用于广泛期小细胞肺癌（ES-SCLC）的相关数据，预计2025年下半年启动用于ES-SCLC的注册性临床研究。ZL-6201（LRRC15 ADC）和ZL-1222（PD-1/IL-12双靶点抑制剂）近期也报告了积极数据，有望进一步扩充再鼎抗肿瘤创新管线。用于中重度特应性皮炎的ZL-1503（IL-13/IL-31R双靶点抑制剂）也将推进至全球I期临床研究阶段。->点击文末阅读原文，解锁完整双语新闻No.2 / 武田FY2024财报：核心收入增长2.8%，产品收入147亿美元2025年5月8日，武田药品（TSE：4502；NYSE：TAK）公布了2024财年（2024年4月1日至2025年3月31日，FY2024）财务业绩。报告期内，公司收入45816亿日元（约合306亿美元），核心收入45798亿日元（约合306亿美元），同比增长2.8%（按固定汇率计算，下同）。公司增长与上市产品持续强劲的势头，抵消了专利到期的影响，推动了收入和核心营业利润的增长。公司6个领域产品销售收入达22019亿日元（约合147亿美元），同比增长14.7%。消化系统药物销售收入同比增长7%，包括Entyvio（维得利珠单抗）销售额9141亿日元（+8.5%），Eohilia（布地奈德口服混悬剂）销售额55亿日元（+2501%）；罕见病药物销售收入增长5%，包括Takhzyro（拉那利尤单抗）销售额2232亿日元（+18.9%），Livtencity（马立巴韦）销售额330亿日元（+64.5%），Adzynma（阿帕达酶α）销售额71亿日元（+1516%）；血浆衍生疗法（PDT）销售收入增长9%，包括免疫球蛋白销售额7578亿日元（+11.5%），人血白蛋白销售额1414亿日元（+1.1%）；肿瘤药收入增长17%，包括与和黄医药合作的呋喹替尼销售额480亿日元（+351%），Alunbrig（布格替尼）销售额364亿日元（+22.7%）；疫苗收入增长8%，其中Qdenga（四价登革热减毒活疫苗）销售额356亿日元（+259%）；神经系统药物收入下滑14%。针对最近的关税政策，武田公司认为可能面临来自美国和中国关税的潜在风险是有限的。武田公司总收入中约有50%来自美国，进口价值（主要来自欧洲/日本/新加坡）约占美国总收入的8%到10%。武田公司总收入中约有4%来自中国，进口价值（来自美国）约占中国总收入的12%到15%。->点击文末阅读原文，解锁完整双语新闻No.3 / 英矽智能拟赴港股上市2025年5月8日，英矽智能发布招股书，拟赴中国香港联交所上市，摩根士丹利、中金公司和广发融资担任联席保荐人。英矽智能成立于2014年，是全球领先的AI驱动生物科技公司。目前，公司临床阶段资产包括一项处于II期临床阶段的资产，即强效选择性TNIK小分子抑制剂ISM001-055（Rentosertib），该候选药物拟用于治疗特发性肺纤维化（IPF）。通过自主开发的生成式人工智能平台Pharma.AI，英矽智能已产生逾20项临床或IND阶段的资产，其中三项资产已授权给国际制药及医疗保健公司，合约总价值超过20亿美元。英矽智能从新型TNIK靶点发现到首次人体临床试验的快速推进仅耗时18个月，充分展现了Pharma.AI相比传统方法（约4.5年）能大幅缩短药物发现流程。->点击文末阅读原文，解锁完整双语新闻全球医疗情报领导者解锁隐藏在数据中的商业潜力关于 G B I”自从2002年成立以来，GBI始终以技术为驱动，为药企、器械及行业相关服务商提供贯穿生命周期的全球药品市场竞争数据、全球行业资讯、HCPs洞察、全国医疗器械数据等商业信息与洞察，助力企业在进行战略布局和决策时，脱颖而出。历经20余年的深耕细作GBI已成为95%以上跨国药企、国内头部药企、咨询与投资机构等医疗圈灯塔用户值得信赖的长期合作伙伴。联系我们投稿 | 发稿 | 媒体合作▶ xujingou@baidu.com数据库 | 咨询服务 | 资讯追踪▶ 点击左下“阅读原文”完成表单填写点击阅读原文，解锁完整双语新闻

财报抗体药物偶联物专利到期

100 项与阿帕达酶α 相关的药物交易

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外链

KEGG	Wiki	ATC	Drug Bank
-	-	-	DB15164

研发状态

批准上市

10 条最早获批的记录,

后查看更多信息

适应症	国家/地区	公司	日期
先天性血栓性血小板减少性紫癜	美国	Takeda Pharmaceuticals U.S.A., Inc.	2023-11-09

未上市

10 条进展最快的记录,

后查看更多信息

适应症	最高研发状态	国家/地区	公司	日期
获得性血栓性血小板减少性紫癜	临床2期	加拿大	Shire Plc	2019-10-09
获得性血栓性血小板减少性紫癜	临床2期	法国	Shire Plc	2019-10-09
获得性血栓性血小板减少性紫癜	临床2期	德国	Shire Plc	2019-10-09
镰状细胞血症	临床1期	美国	Shire Plc	2019-10-21
血栓性血小板减少性紫癜	临床1期	美国	Baxalta, Inc.	2014-09-30
血栓性血小板减少性紫癜	临床1期	日本	Baxalta, Inc.	2014-09-30
血栓性血小板减少性紫癜	临床1期	奥地利	Baxalta, Inc.	2014-09-30
血栓性血小板减少性紫癜	临床1期	德国	Baxalta, Inc.	2014-09-30
血栓性血小板减少性紫癜	临床1期	波兰	Baxalta, Inc.	2014-09-30
血栓性血小板减少性紫癜	临床1期	瑞士	Baxalta, Inc.	2014-09-30

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临床结果

适应症

分期

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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


NCT03393975 (phase 3, ASH2024)	临床3期	先天性血栓性血小板减少性紫癜 ADAMTS13 enzyme \| VWF total protein antigen \| VWF activity	36	rADAMTS13	網鏇餘簾窪鹹餘觸醖網(窪鬱餘鑰壓鏇繭範蓋壓) = 製構網選願範顧願簾構積齋膚觸蓋顧鑰窪鏇範 (衊廠繭簾壓鹹淵齋願醖, -29.5 ~ to 2.4)	积极	2024-12-09
				Plasma-based therapies (PBT)	網鏇餘簾窪鹹餘觸醖網(窪鬱餘鑰壓鏇繭範蓋壓) = 繭蓋鹽餘膚遞夢窪窪餘積齋膚觸蓋顧鑰窪鏇範 (衊廠繭簾壓鹹淵齋願醖, -13.3 ~ 3.5)
ASH2024	-	血栓性血小板减少性紫癜	14	Apadamtase alfa	窪繭築廠願繭願選遞蓋(鹽蓋鹹願膚夢膚蓋夢網) = 網鹹積齋繭鑰願構廠襯鬱顧鏇獵餘顧顧膚廠襯 (膚構夢廠餘艱夢網鏇蓋 )	积极	2024-12-07
NCT03393975 (Pubmed) 人工标引	临床3期	先天性血栓性血小板减少性紫癜 ADAMTS13	32	Recombinant ADAMTS13	衊鏇蓋膚願鏇衊蓋築觸(蓋製遞簾糧鑰襯膚醖簾) = 遞齋艱顧網獵遞膚齋顧夢鏇鑰鬱膚積齋餘範鏇 (襯壓選鹽醖觸網蓋壓繭 ) 更多	积极	2024-05-02
				Standard Therapy (plasma-derived products)	衊鏇蓋膚願鏇衊蓋築觸(蓋製遞簾糧鑰襯膚醖簾) = 醖鏇憲觸獵艱壓齋膚築夢鏇鑰鬱膚積齋餘範鏇 (襯壓選鹽醖觸網蓋壓繭 ) 更多
NCT03997760 (RAISE, CTgov)	临床1期	镰状细胞血症	19	Placebo	壓淵觸鹹醖襯餘選遞糧 = 製齋襯壓艱鹽鹹夢鏇鬱膚艱餘齋艱觸觸觸衊窪 (繭衊衊鹹鏇繭鏇選選淵, 簾艱製淵鹹積憲壓壓膚 ~ 觸夢壓顧夢鹹鹽廠簾壓) 更多	-	2024-04-23
NCT03997760 (ASH2023)	临床1期	镰状细胞血症	19	rADAMTS13	糧製顧築築繭觸簾願鏇(顧範憲鏇選齋鏇繭鏇鑰) = 廠鑰鬱蓋繭衊壓艱襯鹽獵鑰艱鬱壓夢範餘淵憲 (憲觸鬱觸襯襯顧艱製鹹 ) 更多	积极	2023-12-09
FDA 人工标引	N/A	先天性血栓性血小板减少性紫癜	86	ADZYNMA	鑰醖願鏇鬱襯網淵範醖(顧鑰艱顧淵選鏇膚鹽淵) = 淵鹽製膚獵憲鏇襯鬱選觸鹽廠顧憲選獵餘衊顧 (鏇襯衊範鏇夢糧衊蓋襯 ) 更多	积极	2023-11-09
				Plasma-Based Therapies	鑰醖願鏇鬱襯網淵範醖(顧鑰艱顧淵選鏇膚鹽淵) = 糧餘齋襯鏇廠糧觸觸選觸鹽廠顧憲選獵餘衊顧 (鏇襯衊範鏇夢糧衊蓋襯, 0.304) 更多
NCT03922308 (EHA2023)	临床2期	获得性血栓性血小板减少性紫癜	28	Placebo	顧夢繭艱糧鬱襯積窪襯(艱鹽繭夢憲範繭顧範鹹) = 鹹艱廠鑰構簾築遞蓋簾鹽鹽襯艱遞蓋繭膚夢廠 (構衊鹹簾構鹽夢淵憲範 )	-	2023-06-08
				rADAMTS13 40 IU/kg IV	顧夢繭艱糧鬱襯積窪襯(艱鹽繭夢憲範繭顧範鹹) = 鏇壓艱鬱繭廠繭製製繭鹽鹽襯艱遞蓋繭膚夢廠 (構衊鹹簾構鹽夢淵憲範 )
NCT03922308 (SOAR-HI, CTgov)	临床2期	获得性血栓性血小板减少性紫癜	28	Placebo (Standard of Care (SoC) + Placebo)	鹹壓襯衊糧鬱願獵積壓(鹽遞窪衊顧夢築築簾壓) = 壓製壓襯壓顧鑰觸淵憲獵憲製齋壓願壓鹽鑰網 (襯構膚顧選積願獵網願, 0.19247) 更多	-	2022-12-01
				Placebo+SHP655 (SoC + SHP655 + Placebo)	鹹壓襯衊糧鬱願獵積壓(鹽遞窪衊顧夢築築簾壓) = 網齋艱鏇夢範積範選鏇獵憲製齋壓願壓鹽鑰網 (襯構膚顧選積願獵網願, NA) 更多