Therapeutic effectiveness and safety of crovalimab, SKY 59, in patients with Paroxysmal Nocturnal Hemoglobinuria: a prospective multi-institutional observational study in Japan - ANSHAR study

开始日期2024-07-01

申办/合作机构

Chugai Pharmaceutical Co., Ltd.

NCT05494619

/ Withdrawn临床3期

A Phase III Randomized, Double-Blind, Placebo-Controlled, Multi-Center Study to Evaluate the Efficacy, Safety, Pharmacokinetics and Pharmacodynamics of Crovalimab in Patients With Guillain-Barré Syndrome

The purpose of this study is to evaluate the efficacy, safety, pharmacokinetics, and pharmacodynamics of crovalimab compared with placebo as an add-on therapy to intravenous immunoglobulin (IVIg) in participants with severe GBS.

开始日期2022-11-30

申办/合作机构

Hoffmann-La Roche, Inc.

[+1]

ISRCTN12809537

/ Recruiting临床1期

A phase I, multicenter, single-arm study to evaluate the pharmacokinetics, pharmacodynamics, and safety of crovalimab in patients with lupus nephritis

开始日期2022-03-31

申办/合作机构-

100 项与可伐利单抗相关的临床结果

登录后查看更多信息

100 项与可伐利单抗相关的转化医学

登录后查看更多信息

100 项与可伐利单抗相关的专利（医药）

登录后查看更多信息

项与可伐利单抗相关的文献（医药）

2025-08-12·Blood Advances

Advancements in complement inhibition for PNH and primary complement–mediated thrombotic microangiopathy

Review

作者: DeLoughery, Thomas G. ; King, Hannah ; Martens, Kylee ; Shatzel, Joseph J. ; Malhotra, Aditya ; Padilla Kelley, Thalia

Abstract:

Paroxysmal nocturnal hemoglobinuria (PNH) and primary complement–mediated thrombotic microangiopathy, also known as atypical hemolytic uremic syndrome (aHUS), are hematologic disorders characterized by dysregulation of the complement system leading to hemolysis and other systemic and potentially lethal complications. The advent of C5 inhibition with agents such as eculizumab and ravulizumab has revolutionized the management of patients with these disorders, although some may still experience clinically significant breakthrough extravascular hemolysis. Over the past several years, novel therapies targeting upstream pathways of complement inhibition have been approved for the treatment of patients with PNH experiencing breakthrough hemolysis despite C5 inhibition. These agents currently include pegcetacoplan, a C3 inhibitor; iptacopan, an oral factor B inhibitor; danicopan, an oral factor D inhibitor; and crovalimab, an anti-C5 monoclonal antibody. This review highlights recent advances in complement-targeted therapies for PNH, including their indications and efficacy and safety data from key randomized trials. In addition, we review current data supporting the use of these novel agents for aHUS, for which only the terminal complement inhibitors eculizumab and ravulizumab are currently approved. Future research is crucial to establish the long-term efficacy and safety profiles of these novel therapies, ensuring the best treatment strategies for patients with PNH and aHUS.

2025-07-24·BLOOD

Breakthrough hemolysis in paroxysmal nocturnal hemoglobinuria throughout clinical trials: from definition to clinical practice

Review

作者: Barcellini, Wilma ; Fattizzo, Bruno ; Versino, Francesco

Abstract:

Breakthrough hemolysis (BTH) is the hemolytic exacerbation occurring in a patient with paroxysmal nocturnal hemoglobinuria (PNH) on treatment with anticomplement therapies. In this review article, we analyzed the definition, frequency, and severity of BTH events across phase 3 clinical trials with terminal (anti-C5 ravulizumab and crovalimab) and complement inhibitors upstream C5 (anti-C3 pegcetacoplan, alternative-pathway inhibitors iptacopan, and danicopan), as well as from real-world reports. Furthermore, we reviewed the impact of the various compounds on quality of life and patient-reported outcomes. In particular, BTH may occur with all complement inhibitors, with a frequency of 10% to 15% over 6 months with eculizumab, crovalimab, and pegcetacoplan, and <5% with ravulizumab, iptacopan, and danicopan plus anti-C5. By prolonging the follow-up, the frequency of BTH appeared increased in patients treated with pegcetacoplan (nearly 24% at 1 year) as compared with both anti-C5, iptacopan, and double inhibition with danicopan plus anti-C5. BTH risk appears associated with patients’ features, particularly suboptimal response/failure of previous complement inhibitor. Transfusions were required in approximately half of cases and modifications of anticomplement therapy included anticipation of the next anti-C5 dose and addition of eculizumab in patients on proximal inhibitors. Breakthrough thromboses were rare, although anticoagulant prophylaxis should be considered during severe episodes. Complement amplifying conditions were observed in approximately half of cases and were more frequently infections. Treatment adherence, optimization of the administration schedule, anticoagulant prophylaxis, as well as education of patients and physicians remain important factors to prevent BTH and its complications.

2025-07-01·Health Science Reports

Crovalimab: A Novel Approach in the Management of Paroxysmal Nocturnal Hemoglobinuria

Article

作者: Jalal, Laiba ; Ahmed, Marium ; Khalid, Anum

ABSTRACT:

Background and Aims:

Paroxysmal Nocturnal Hemoglobinuria (PNH) is a rare acquired clonal blood disorder caused by mutations in the PIGA gene, leading to complement‐mediated hemolysis. Currently available terminal complement inhibitors, such as Eculizumab and Ravulizumab, pose several challenges, including the need for frequent intravenous infusions and the potential for resistance due to C5 polymorphisms. This study highlights the clinical significance of Crovalimab, a novel C5 inhibitor developed using SMART‐antibody technology, as a promising alternative.

Methods:

An extensive literature review was conducted using PubMed to evaluate the pharmacological properties, mechanism of action, and clinical trial data of Crovalimab. Phase 3 trials—COMMODORE 1, 2, and 3—were analyzed to assess Crovalimab's safety, efficacy, and potential benefits in both C5‐inhibitor naïve and previously treated patients.

Results:

Crovalimab demonstrated high bioavailability, an extended half‐life, and subcutaneous administration every 4 weeks, offering a better alternative to intravenous therapies. Unlike existing treatments, Crovalimab targets the C5 β‐chain, making it effective even in patients with the R885H polymorphism. The COMMODORE trials reported favorable outcomes, including effective hemolysis control, reduced transfusion dependence, and a manageable safety profile. Adverse events were mostly mild, with rare occurrences of transient immune complex reactions.

Conclusion:

Crovalimab represents a significant advancement in the management of PNH, with the potential to reduce treatment burden while maintaining efficacy. However, further research is required to evaluate its long‐term safety and effectiveness across diverse populations.

195

项与可伐利单抗相关的新闻（医药）

2025-06-23

·PipelineReview

Early data suggest Roche’s NXT007 may have the potential to provide haemostatic normalisation in people with haemophilia A

BASEL, Switzerland I June 23, 2025 I Roche (SIX: RO, ROG; OTCQX: RHHBY) announced today positive phase I/II data on NXT007 in people with haemophilia A, supporting its progression into phase III clinical development. NXT007 is a next-generation investigational bispecific antibody, engineered by Chugai, a member of the Roche Group. Early data from the NXTAGE study suggest that NXT007 may have the potential to provide haemostatic normalisation in people with haemophilia A (without factor VIII inhibitors). NXT007 showed a tolerable safety profile with no thromboembolic events reported so far. 1 These results were featured as an oral presentation at the 2025 International Society on Thrombosis and Haemostasis (ISTH) Congress, 21-25 June, Washington D.C., United States. 1 “These NXT007 data are promising for people with haemophilia A and underscore our ongoing commitment to advancing care and addressing the real-world challenges faced by this community,” said Levi Garraway, MD, PhD, Roche’s Chief Medical Officer and Head of Global Product Development. “Hemlibra established a new standard of care, and our focus is to continue to deliver breakthrough innovation that might ultimately help people with haemophilia to live their lives in a manner unaffected by this condition.” NXT007 leverages the Roche Group’s expertise in haemophilia A and bispecific antibody development. Our goal is to bring a next generation prophylactic to our portfolio, offering greater therapeutic choice, sustained, elevated bleed protection and reduced treatment burden with factor independence – to allow patients to experience freedom from constant vigilance and have confidence in bleed protection. NXT007 will be further evaluated in a robust clinical development programme, including ongoing phase I/II clinical trials, with additional phase II data expected later this year. There are also three phase III studies currently planned for 2026, including a phase III head-to-head study with Roche’s Hemlibra, the first available prophylactic treatment that can be administered subcutaneously and with flexible dosing options (every week, two weeks or four weeks). 4,5 Part B of the phase I/II NXTAGE study, conducted by Chugai, in Japan, Taiwan and South Korea, is evaluating the safety, pharmacokinetics, pharmacodynamics and efficacy of prophylaxis with NXT007 in people with haemophilia A without factor VIII inhibitors who had not been previously treated with Hemlibra® (emicizumab). 1 Thirty participants (from 12 to 65 years of age) were enrolled in four cohorts (B-1 to B-4) to receive ascending doses of subcutaneous NXT007 every two-to-four weeks during the maintenance period (following four-to-six weeks of loading doses). In presented data from the primary analysis, no treated bleeds were observed with NXT007 in the highest dose cohorts (B-3 and B-4). NXT007 was well tolerated, with no thromboembolic events observed so far. 1 About NXT007 NXT007 is a next-generation investigational bispecific antibody, being investigated as a prophylactic (preventive) treatment option for people with haemophilia A. 1,2,3 NXT007 was engineered by Chugai – a member of the Roche Group – built on Hemlibra® (emicizumab)’s framework, with the aim of optimising factor VIII-mimetic activity and half-life, to further enhance potency, efficacy, dosing and administration convenience. NXT007 brings together factor IXa and factor X, proteins required to activate the natural coagulation cascade. 1,2,3 NXT007 is being studied in a robust clinical development programme exploring its potential to achieve sustained elevated bleed protection equivalent to people who do not have haemophilia A (sustained haemostatic normalisation), and reduced treatment burden with factor independence, offering people living with haemophilia A greater therapeutic choice. 1,2,3 About haemophilia A Haemophilia A is an inherited, serious disorder in which a person’s blood does not clot properly, leading to uncontrolled and often spontaneous bleeding. Haemophilia A affects around 900,000 people worldwide. 6,7 People with haemophilia A either lack or do not have enough of a clotting protein called factor VIII. In a healthy person, when a bleed occurs, factor VIII brings together the clotting factors IXa- and X, which is a critical step in the formation of a blood clot to help stop bleeding. Depending on the severity of their symptoms, people with haemophilia A can bleed frequently, especially into their joints or muscles. 8 These bleeds can present a significant health concern as they often cause pain and can lead to chronic swelling, deformity, reduced mobility and long-term joint damage. 9 A serious complication of treatment is the development of inhibitors to factor VIII replacement therapies. Inhibitors are antibodies developed by the body’s immune system that bind to and block the efficacy of replacement factor VIII, making it difficult, if not impossible, to obtain a level of factor VIII sufficient to control bleeding. 6 About Roche in haematology Roche has been developing medicines for people with malignant and non-malignant blood diseases for more than 25 years; our experience and knowledge in this therapeutic area runs deep. Today, we are investing more than ever in our effort to bring innovative treatment options to patients across a wide range of haematologic diseases. Our approved medicines include MabThera®/Rituxan® (rituximab), Gazyva®/Gazyvaro® (obinutuzumab), Polivy® (polatuzumab vedotin), Venclexta®/Venclyxto® (venetoclax) in collaboration with AbbVie, Hemlibra® (emicizumab), PiaSky® (crovalimab), Lunsumio® (mosunetuzumab) and Columvi® (glofitamab). Our pipeline of investigational haematology medicines includes T-cell engaging bispecific antibody cevostamab, targeting both FcRH5 and CD3 and Tecentriq® (atezolizumab). Our scientific expertise, combined with the breadth of our portfolio and pipeline, also provides a unique opportunity to develop combination regimens that aim to improve the lives of patients even further. About Roche Founded in 1896 in Basel, Switzerland, as one of the first industrial manufacturers of branded medicines, Roche has grown into the world’s largest biotechnology company and the global leader in in-vitro diagnostics. The company pursues scientific excellence to discover and develop medicines and diagnostics for improving and saving the lives of people around the world. We are a pioneer in personalised healthcare and want to further transform how healthcare is delivered to have an even greater impact. To provide the best care for each person we partner with many stakeholders and combine our strengths in Diagnostics and Pharma with data insights from the clinical practice. For over 125 years, sustainability has been an integral part of Roche’s business. As a science-driven company, our greatest contribution to society is developing innovative medicines and diagnostics that help people live healthier lives. Roche is committed to the Science Based Targets initiative and the Sustainable Markets Initiative to achieve net zero by 2045. Genentech, in the United States, is a wholly owned member of the Roche Group. Roche is the majority shareholder in Chugai Pharmaceutical, Japan. For more information, please visit www.roche.com . All trademarks used or mentioned in this release are protected by law. References [1] 1. Shima M, et al. NXT007 Prophylaxis in Emicizumab-Naive Persons with Hemophilia A without Inhibitor: Phase I/II Study (NXTAGE) Presented at International Society on Thrombosis and Haemostasis (ISTH) congress; 2025 June. Abstract OC.20.3. [2] Shima M, et al. Safety, Pharmacokinetics, and Pharmacodynamics of Single Subcutaneous Injection of NXT007, an Emicizumab-Based Next-Generation Bispecific Antibody, in Healthy Volunteers (NXTAGE Study). Presented at: International Society on Thrombosis and Haemostasis (ISTH) Congress; 2023 July 28. Abstract OC 69.4. [3] Teranishi-Ikawa Y., et al. A bispecific antibody NXT007 exerts a hemostatic activity in hemophilia A monkeys enough to keep a non-hemophiliac state. Journal of Thrombosis and Haemostasis. 2023; doi: 10.1016/j.jtha.2023.09.034 [4] Hemlibra SmPC [Internet; cited 2025 June] Available from: https://www.medicines.org.uk/emc/product/9043/smpc [5] FDA Prescribing Information [Internet; cited 2025 June]. Available from: https://www.accessdata.fda.gov/drugsatfda_docs/label/2018/761083s002s004lbl.pdf . [6] Srivastava A, et al. WFH guidelines for the management of hemophilia, 3rd edition. Haemophilia. 2020;26 (Suppl 6): 1-158. [7] Iorio A, et al. Establishing the Prevalence and Prevalence at Birth of Hemophilia in Males. Ann Intern Med. 2019;171(8):540-546. [8] NHS. Symptoms of haemophilia [Internet; cited 2025 June]. Available from: https://www.nhs.uk/conditions/haemophilia/symptoms/ . [9] Franchini M, et al. Haemophilia A in the third millennium. Blood Rev. 2013; 179-84. SOURCE: Roche

临床结果临床3期

2025-06-09

·EINPresswire

Atypical Hemolytic Uremic Syndrome (aHUS): Breaking the Boundaries of Traditional Treatment | Competitive Intelligence

Next-gen aHUS treatments are shifting from IV infusions to oral and subcutaneous options-offering new hope in safety, efficacy, and patient ease. The aHUS therapy landscape is on the cusp of disruption-with emerging oral drugs and biosimilars reshaping market dynamics and patient access.” — DataM Intelligence AUSTIN, TX, UNITED STATES, June 9, 2025 / EINPresswire.com / -- Atypical hemolytic uremic syndrome (aHUS) is a rare, life-threatening condition that has challenged clinicians for decades due to its complex pathophysiology and unpredictable relapses. Primarily caused by uncontrolled activation of the complement system-a vital part of immune defense-this disorder results in the body mistakenly attacking its own endothelial cells. This autoimmune-like attack leads to blood clots in small vessels, hemolytic anemia, low platelets, and acute kidney injury. The cascade of events often unfolds rapidly, leaving minimal room for therapeutic delay. Book Your Free CI Consultation Call: https://www.datamintelligence.com/strategic-insights/ci/atypical-hemolytic-uremic-syndrome-ahus Historically, the clinical landscape for aHUS treatment was limited and reactive. However, the emergence of complement inhibitors such as Soliris (eculizumab) and Ultomiris (ravulizumab) has dramatically transformed patient outcomes. These targeted therapies block the complement component C5, thereby halting the terminal pathway responsible for endothelial injury. Despite their efficacy, these treatments bring challenges-namely, high costs, intravenous dosing burdens, and long-term immunosuppression. Epidemiology and Disease Burden Though rare, aHUS is now recognized more frequently due to improved diagnostic capabilities and awareness. Incidence estimates suggest approximately 1 to 2 cases per million population annually, with a higher prevalence in pediatric patients and young adults. Despite being uncommon, the economic and clinical burden is significant. Frequent hospitalizations, renal replacement therapies, and lifelong medication usage make it a resource-intensive condition for both patients and health systems. Looking ahead, as diagnosis rates continue to rise and access to therapy improves globally, the treated patient population is expected to expand steadily over the next decade. Market Leaders: Soliris and Ultomiris Soliris, the first-in-class anti-C5 monoclonal antibody, has long been the backbone of aHUS management. Delivered via intravenous infusion every two weeks, it provides rapid disease control and kidney function stabilization. Ultomiris, a long-acting version of Soliris, offers dosing every eight weeks and has quickly gained traction due to its extended half-life and comparable efficacy. While both drugs offer life-altering benefits, their IV delivery model and high cost present logistical and economic hurdles-particularly in resource-limited settings. Furthermore, patients on chronic complement inhibition face elevated risks of infections, especially from encapsulated organisms like Neisseria meningitidis, requiring mandatory vaccination and close monitoring. Biosimilars Enter the Arena: Expanding Access To address cost constraints, biosimilars such as Bkemv and EPYSQLI have recently entered the market. Both mirror the efficacy and safety profile of Soliris but at reduced prices. This has significant implications for healthcare systems seeking cost-effective treatment options and for patients in emerging markets where branded biologics were previously out of reach. The entry of biosimilars is expected to trigger pricing pressure on originator brands and encourage broader adoption of complement blockade therapy in previously underserved regions. Emerging Innovations: Convenience Redefined The next wave of innovation in aHUS centers around patient-centric improvements-most notably the shift from intravenous to oral or subcutaneous delivery. Fabhalta (Iptacopan), an oral complement factor B inhibitor, represents a potentially game-changing option. By targeting the alternative pathway upstream of C5, Fabhalta may offer broader complement control. Its oral daily dosing also addresses the burden of frequent infusions, enhancing quality of life and adherence. Another frontrunner is Crovalimab (Piasky), a subcutaneous anti-C5 monoclonal antibody recently approved in several regions. Its monthly SC administration empowers patients with greater autonomy and reduces hospital visits-especially appealing for younger, working patients or those in remote areas. These innovations are not merely incremental-they have the potential to disrupt the current therapeutic paradigm, offering alternatives that align more closely with patient preferences. Pipeline Outlook: What’s Next? The aHUS pipeline includes promising assets in various stages of development, including novel monoclonal antibodies, small molecules, and even RNA interference (RNAi) therapies. These next-gen modalities aim to deliver differentiated value-whether through new mechanisms of action, improved safety profiles, or enhanced pharmacokinetics. Key targets include: - Complement factor B and C3, which offer upstream inhibition for more comprehensive control. - Gene therapies under preclinical evaluation, aiming for long-term or potentially curative outcomes. - Oral agents and self-administered injectables, reducing the logistical barriers of hospital-based care. While Soliris and Ultomiris set a high bar for efficacy and safety, pipeline therapies must offer clear advantages-either in administration, tolerability, or cost-effectiveness-to capture significant market share. Target Opportunity Profile (TOP): Where the Gaps Lie To secure a strong foothold in the evolving aHUS market, developers must address the following unmet needs: - Convenience: Intravenous dosing every two to eight weeks limits patient freedom. Oral or subcutaneous routes are increasingly preferred. - Affordability: The high cost of branded therapies restricts global access. Cost-effective alternatives or pricing models will be key. - Safety: While current therapies are generally well tolerated, they carry infection risks. Novel agents must aim to reduce immunosuppression-related complications. - Innovation in Mechanism: Moving beyond C5 to upstream targets may offer added efficacy in difficult or resistant cases. - Real-World Outcomes: Long-term data on kidney preservation, survival, and quality of life will influence payer decisions and treatment guidelines. Competitive Dynamics: A Market in Transition The market for aHUS treatment is no longer defined solely by biologics delivered via infusion centers. With oral small molecules like Fabhalta and subcutaneous agents like Crovalimab gaining traction, the competitive landscape is undergoing rapid transformation. Manufacturers must now compete not only on efficacy but also on convenience, safety, price, and patient experience. Biosimilars are squeezing margins while expanding access. Meanwhile, pipeline entrants promise to reset expectations entirely-with oral, gene-based, and potentially curative approaches in development. Download Free Sample Report: https://www.datamintelligence.com/strategic-insights/sample/atypical-hemolytic-uremic-syndrome-ahus Conclusion The treatment of atypical hemolytic uremic syndrome has come a long way-from managing life-threatening relapses with supportive care to providing targeted, disease-modifying therapy. But the next frontier is patient-centric: offering safe, effective, and affordable treatment that fits seamlessly into patients’ lives. Oral drugs like Fabhalta and injectables like Crovalimab mark a pivotal shift, challenging the status quo and opening the door to more personalized and accessible care. As biosimilars democratize access and innovation drives differentiation, the aHUS market is poised for significant evolution in the years ahead. Read Related CI Reports: 1. Eosinophilic Esophagitis | Competitive Intelligence 2. Warm Autoimmune Hemolytic Anemia | CI Insights Sai Kumar DataM Intelligence 4market Research LLP +1 877-441-4866 email us here Visit us on social media: LinkedIn X Legal Disclaimer: EIN Presswire provides this news content "as is" without warranty of any kind. We do not accept any responsibility or liability for the accuracy, content, images, videos, licenses, completeness, legality, or reliability of the information contained in this article. If you have any complaints or copyright issues related to this article, kindly contact the author above.

上市批准

2025-05-27

·DIA资讯

2025 DIA中国年会丨中国创新药十年飞跃：从跟跑到领跑，全球新药实现“中国首发”格局加速成型

点击“DIA资讯” 关注我们2025 DIA CHINA ANNUAL MEETING上海，2025年5月24日——在2025年DIA中国年会上，来自全球三十多个地区的卫生监管机构和医药行业的千余名专家与领袖齐聚一堂，共同见证中国创新药发展的“黄金十年”。自2015年以来，中国通过系统性政策改革，实现了从“仿制跟跑”到“创新领跑”的历史性跨越，现已成为全球医药创新的核心枢纽。统计显示，截至2024年底，中国企业研发的活跃创新药数量已达3575个，超越美国，成为全球第一。全球新药如百济神州的替雷利珠单抗、罗氏的可伐利单抗和勃林格殷格翰的佩索利单抗等在中国实现了首发上市，标志着本土创新的崛起和产业生态的重构。政策改革十年：构建全球领先的创新生态过去十年，中国药品审评审批制度经过一系列改革，打通了创新的堵点，促成了中国医药产业从“赶潮人”、“弄潮人“到“领潮人”的转变。王海南国家药监局药品注册司副司长国家药监局药品注册司王海南副司长在演讲中回顾了过去取得的卓有成效的改革举措，包括优化药品上市审批机制，审评、核查、检验、通用名核准并联进行；注册检验可前置；基于风险的核查工作机制。临床试验申请默示许可制度以后，2024年又开展IND试点工作，试点将创新药审评时限缩短至30个工作日，临床试验启动用时缩短至12周。建立药品加快上市制度。完善变更管理制度，优化补充申请审评审批程序。鼓励放射性药品、儿童用药、罕见病药物研发。建立专利纠纷早期解决机制等。王司长特别提到，2024年国务院发布的53号文件（《关于全面深化药品医疗器械监管改革促进医药产业高质量发展的意见》）为行业注入了新动力，加大药品创新支持力度，坚持“提前介入、一企一策、全程指导、药审联动”；加快临床急需药品审批上市，优化临床试验审评审批机制、优化药品补充申请审评审批，加快罕见病用药品审评审批；支持医药产业扩大对外开放合作，探索生物制品分段生产；构建适合产业发展和安全需要的监管体系。秦晓岺国家药监局科技和国际合作司司长国家药监局科技和国际合作司秦晓岺司长强调，NMPA全面参与国际监管规则制定，不断提升药品监管的国际化水平和国际影响力，ICH指导原则已经全部在国内转化实施；持续扩大对外开放合作，2024年国家药监局发布《关于优化已在境内上市的境外生产药品转移至境内生产的药品上市注册申请相关事宜的公告》，商务部国家卫健委会同药监局发布关于在医疗领域开展扩大开放试点工作的通知。秦晓岺司长强调，NMPA不断深化与世界卫生组织（WHO）在药品监管等领域开展交流合作；积极参与国际人用药品注册技术协调会（ICH）、国际药品监管机构联盟（ICMRA）活动，全面参与国际监管规则制定并转化实施，不断提升中国药品监管的国际化水平和国际影响力。李璐国家药监局政策法规司法规处处长国家药监局政策法规司法规处李璐处长：药品全生命周期管理法律法规体系的“四梁八柱”基本建立。后续加快推动重点立法项目包括《药品管理法实施条例》等。全球创新格局的重塑：中国力量的崛起与2015年相比，中国已成为全球研发的重要策源地。2024年中国创新药海外授权交易总额达482亿美元，是2015年（23亿美元）的21倍。截至 2024 年底，全球共有923款创新药首次获批，其中美国首发的占49%，中国接近 38%，这一比例较2015年的4%提升了近10倍。谢松梅药品审评中心（CDE）化药临床二部部长药品审评中心（CDE）化药临床二部谢松梅部长介绍了改革成效及新进展。2024年国家药监局共批准48个新药上市。重大慢性病防治领域，仑卡奈单抗为我国首批用于阿尔茨海默症的1类创新药。自身免疫性皮肤疾病领域，我国自主研发的新药本维莫德乳膏为国内新机制、新靶点，2019年全球首批，2024年11月我国优先程序全球率先批准新适应症特异性皮炎，随后美、日同品种上市；2款口服小分子JAK抑制剂（乌帕提尼缓释片、阿布昔替尼片）全球同步研发、同步上市。通过加快路径批准上市了多款新药极大满足了我国患者临床迫切需求。最后分享了临床试验改革试点、临床急需优化、罕见病等监管改革新进展。2025 DIA CHINA ANNUAL MEETING刘艳玮武田大中华区注册事务副总裁武田大中华区注册事务副总裁刘艳玮女士回顾了十年行业发展变化。从药品上市迟滞角度，从2015-2024年，中美药品上市时间差从8年缩短至2年以内。NDA同步递交，2024年同步递交占比达42.3%，67%与全球首递时间差小于3个月。这些成绩离不开鼓励药物创新的一系列新举措。2025 DIA CHINA ANNUAL MEETING何静医学博士阿斯利康全球高级副总裁、全球研发中国负责人2024年全球授权大型交易中，1/3来自中国本土创新，中国创新正在成为全球解决方案。阿斯利康全球高级副总裁、全球研发中国负责人何静博士表示，这是多年积累水到渠成的结果。阿斯利康在北京建立全球第六个战略研发中心，中国已成为与美国并列的创新策源地。中国研发生态蓬勃发展得益于药监改革释放创新活力。阿斯利康已将同步研发作为默认策略，深化临床研究合作，探索Discovery research新道路，结合AI重塑研发过程，加强跨国企业与本土生态圈合作。2025 DIA CHINA ANNUAL MEETING张鹏康方生物联合创始人康方生物联合创始人张鹏则以PD-1产品派安普利单抗成功出海为例，接受FDA全面检查，中国的监管体系已与国际接轨。此次经历锻炼了公司团队，为未来的双抗出海积累经验。2025 DIA CHINA ANNUAL MEETING闻增玉泰格医药科技股份有限公司联席总裁杭州泰格医药科技股份有限公司联席总裁闻增玉先生表示泰格医药作为创新型CRO公司，过去几年，服务了超过60%的中国一类创新药研发，为助力中国创新药全球同步开发，完成了全球37个国家一体化布局，采用“global team with local activity”策略。2025 DIA CHINA ANNUAL MEETING董艳平罗氏中国临床注册事务负责人谈到罗氏中国临床注册事务负责人董艳平女士在分享可伐利单抗中国首发的经验时介绍，基于突阵发性睡眠性血红蛋白尿（PNH）在中国未满足的临床需求，监管机构以临床价值为导向，给予极大灵活性。执行过程中对于批准前境外核查，附条件批准关门等问题，监管机构通过“一企一策，前置指导“等，起到很好的示范作用。当前及未来的热点话题：1. 优化创新药临床试验审评审批试点目前，已有21项IND参与试点，业界期待能总结出有效的试点经验。未来，如何构建与国际接轨的IND审评审批长效工作机制是业内关注的焦点。国家药监局药品注册司王海南副司长表示，今年7月试点工作结束后，将开辟新的IND审评通道，工业界对此充满期待。2. 分段生产康方生物联合创始人张鹏先生谈到，药监局从委托生产推到分段生产是一个重要进步。分段生产的优势在于优化国内资源配置，促进CDMO产业发展，最大化生产企业的效能和优势领域。同时也应重视企业质量参差不齐的问题。3. 药品试验数据保护实施办法罗氏中国临床注册事务负责人董艳平女士谈到，以前IP在中国的生命周期较短，随着专利补偿与专利链接等政策出台，行业多年的呼吁得到固化；中国把数据保护与申报分类挂钩，释放了行动信号，对总部是兴奋剂，有利于促进企业把同步和加快落到实处，让专利悬崖推迟。4. AI赋能与未来挑战杭州泰格医药科技股份有限公司联席总裁闻增玉先生谈到，在降本增效背景下，AI的使用潜力巨大。AI的应用场景包括靶点选择、药物设计、临床试验设计优化、医学撰写等领域。未来的挑战包括：合规、数据安全、算法透明度，以及监管的要求。AI使用场景的落地、合规保证、效率、质量和监管还有一段路要走，应抱有开放态度接受，勇敢面对AI的挑战。展望中国医药创新的下一个十年，目标是成为全球规则制定者。全球新药“中国首发“，不仅是时间概念，更是创新话语权的体现。DIA中国近期会议/培训Jun13-14● 2025 DIA新药研发和全生命周期管理课程► 点击阅读Jun20-21● 2025 Rare to Aware: DIA罕见病基础培训► 点击阅读Jun27-28● 2025 DIA全球化药品开发与监管策略实战线下研讨班► 点击阅读Jul4-5● 2025 DIA稽查精英特训营：深入临床一线，掌握核心技能► 点击阅读Jul11-12● 2025 DIA 放射性药物研修班：从基础理论到临床应用的全面进阶► 点击阅读Aug15-16● 2025 DIA 细胞治疗CMC研究生产质量管理和临床实践► 点击阅读Aug22-23● 2025 DIA CNS创新论坛暨CCTN-P年会► 点击阅读点击了解详情关于DIADIA是一个全球化、跨学科的国际性学术平台，在中立的环境中，融合医药研发领域全行业的意见领袖，探讨当前研发的技术问题，提升专业能力，以及催化行业共识，在全球医药研发领域享有很高的声誉。扫码关注我们微信号｜DIA资讯长按识别左侧二维码即可关注我们努力为你提供有内容的内容。

临床申请放射疗法

100 项与可伐利单抗相关的药物交易

登录后查看更多信息

外链

KEGG	Wiki	ATC	Drug Bank
D11696	-	-	DB16128

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
非典型溶血性尿毒症综合征	加拿大	Hoffmann-La Roche Ltd.	2025-06-01
阵发性睡眠性血红蛋白尿症	中国	Roche Pharma (Schweiz) AG	2024-02-06

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
格林-巴利综合征	临床3期	-	Hoffmann-La Roche, Inc.	2022-11-30
格林-巴利综合征	临床3期	-	Chugai Pharmaceutical Co., Ltd.	2022-11-30
镰状细胞血症	临床2期	美国	Hoffmann-La Roche, Inc.	2022-03-09
镰状细胞血症	临床2期	巴西	Hoffmann-La Roche, Inc.	2022-03-09
镰状细胞血症	临床2期	法国	Hoffmann-La Roche, Inc.	2022-03-09
镰状细胞血症	临床2期	意大利	Hoffmann-La Roche, Inc.	2022-03-09
镰状细胞血症	临床2期	肯尼亚	Hoffmann-La Roche, Inc.	2022-03-09
镰状细胞血症	临床2期	黎巴嫩	Hoffmann-La Roche, Inc.	2022-03-09
镰状细胞血症	临床2期	荷兰	Hoffmann-La Roche, Inc.	2022-03-09
镰状细胞血症	临床2期	南非	Hoffmann-La Roche, Inc.	2022-03-09

登录后查看更多信息

临床结果

适应症

分期

评价

查看全部结果

研究	分期	人群特征	评价人数	分组	结果	评价	发布日期


NCT04654468 \| NCT04432584 \| NCT04434092 (COMMODORE, Pubmed \| Eur J Haematol)	临床3期	阵发性睡眠性血红蛋白尿症 C5 inhibitor (C5i)‐naive \| C5i‐switched	393	Crovalimab	製醖淵窪糧廠顧醖鹽鏇(鑰鏇鑰製選廠糧鏇鹹廠) = 鏇顧艱憲襯簾壓鹽餘選蓋鑰願衊憲顧醖衊艱鏇 (積遞醖壓艱範積憲願繭 ) 更多	积极	2024-11-13
				Eculizumab	製醖淵窪糧廠顧醖鹽鏇(鑰鏇鑰製選廠糧鏇鹹廠) = 築製憲鹽選鏇蓋衊製蓋蓋鑰願衊憲顧醖衊艱鏇 (積遞醖壓艱範積憲願繭 ) 更多
NCT04861259 \| NCT04958265 (COMMODORE, ISN WCN2024) 人工标引	临床3期	阵发性睡眠性血红蛋白尿症	488	Crovalimab	膚夢醖醖遞築遞願製顧(壓廠構選繭製選願艱積) = Type 3 hypersensitivity reactions, associated with complexes, occurred in 18% (33/185; 21 grade 1-2, 12 grade 3) of switch pts. 餘鏇遞鏇襯齋鑰壓選餘 (願繭鬱範積鏇齋繭築範 )	积极	2024-04-01
				Eculizumab
NCT04434092 (COMMODORE 2, ASH2023)	临床3期	阵发性睡眠性血红蛋白尿症	204	Crovalimab	艱襯窪窪醖繭遞膚範願(築壓選構願鑰製簾淵艱) = 襯網願衊觸壓窪構壓繭醖壓獵製鹽憲艱窪範淵 (選觸醖齋壓獵憲網鏇廠 )	-	2023-12-11
				Eculizumab	艱襯窪窪醖繭遞膚範願(築壓選構願鑰製簾淵艱) = 壓餘襯鑰鏇鏇繭鹽衊繭醖壓獵製鹽憲艱窪範淵 (選觸醖齋壓獵憲網鏇廠 )
NCT04654468 \| NCT04432584 \| NCT04434092 (COMMODORE 1 \| COMMODORE 2 \| COMMODORE 3, ASH2023)	临床3期	阵发性睡眠性血红蛋白尿症	488	Crovalimab	積獵淵糧顧顧窪壓醖淵(壓選構構簾醖艱鬱鬱鑰) = 蓋簾鬱膚餘獵構構繭獵鹽衊鏇範顧襯糧構選艱 (膚鏇遞壓廠窪鹽願憲壓 ) 更多	-	2023-12-10
				Eculizumab	積獵淵糧顧顧窪壓醖淵(壓選構構簾醖艱鬱鬱鑰) = 選鹹夢鹽積網襯齋鏇鏇鹽衊鏇範顧襯糧構選艱 (膚鏇遞壓廠窪鹽願憲壓 ) 更多
NCT04654468 (COMMODORE 3, Pubmed)	临床3期	阵发性睡眠性血红蛋白尿症	51	Crovalimab	製醖衊簾網積獵顧鏇繭(鹽繭壓製壓範選積廠夢) = 襯鑰顧廠築觸鑰憲夢積構繭憲鏇範鏇壓餘餘蓋 (襯鬱觸遞壓衊積遞觸鹽, 67.8 ~ 86.6) 更多	-	2023-07-08
NCT04432584 (COMMODORE 1, EHA2023)	临床3期	阵发性睡眠性血红蛋白尿症	89	Crovalimab	齋膚蓋獵蓋構衊網顧範(餘遞夢鹹膚艱憲觸繭齋) = 艱鹽選鏇鬱夢窪淵範膚繭築窪積膚蓋顧廠範繭 (壓齋憲憲糧鏇範製簾壓 ) 更多	-	2023-06-08
				Eculizumab	齋膚蓋獵蓋構衊網顧範(餘遞夢鹹膚艱憲觸繭齋) = 願願築壓網積觸膚鬱齋繭築窪積膚蓋顧廠範繭 (壓齋憲憲糧鏇範製簾壓 ) 更多
NCT04654468 (COMMODORE 3, EHA2023)	临床3期	阵发性睡眠性血红蛋白尿症	51	Crovalimab	鹹獵襯夢鑰選鏇構鹹網(壓構鹽範遞製窪糧膚鹽) = 鑰襯襯襯壓糧鹽齋鬱鑰簾遞製繭衊憲網遞膚鑰 (憲築積遞憲憲願願蓋構, 57.7 ~ 91.4) 更多	-	2023-06-08
NCT04654468 (COMMODORE 3, ASH 12022 \| ASH 22022) 人工标引	临床3期	阵发性睡眠性血红蛋白尿症	51	Crovalimab	積築壓夢夢憲鏇餘獵餘(願窪觸齋膚憲廠構醖簾) = 網鏇範網鏇選糧淵鹽積鬱醖衊構選鬱選選襯壓 (衊築淵繭壓膚壓鑰繭願, 67.8 ~ 86.6) 达到更多	积极	2022-11-15
NCT04654468 (COMMODORE 3, NEWS) 人工标引	临床3期	阵发性睡眠性血红蛋白尿症	500	Crovalimab	鏇遞夢餘夢糧鬱觸鹹鏇(網廠觸艱鹽積襯衊糧膚) = 达到共同主要终点範鬱鬱網觸齋蓋膚鏇蓋 (糧蓋襯築鬱蓋觸網艱鬱 ) 更多	积极	2022-05-23
NCT03157635 (COMPOSER, EHA2020)	临床1/2期	阵发性睡眠性血红蛋白尿症	44	Crovalimab	糧淵糧簾鹹艱範獵襯鏇(鹽觸衊蓋網憲蓋壓顧鏇) = Three serious adverse events (SAEs) were reported, none related to study treatment. One patient had two SAEs: bile duct stone and cholelithiasis. A second patient had an SAE of upper respiratory tract infection with admission to the hospital, which occurred after 20 months and was resolved while on treatment. 構艱網艱願鏇遞餘選餘 (顧憲鬱憲簾鏇製範選願 )	积极	2020-05-14