A Comparative Clinical Effectiveness Trial of Rituximab Versus Ravulizumab, Inebilizumab, Satralizumab and Eculizumab To Prevent Relapses in Neuromyelitis Optica Spectrum Disorder

Neuromyelitis Optica Spectrum Disorder (NMOSD) is a rare autoimmune condition that mainly affects the eyes and spinal cord, causing serious symptoms such as vision loss, paralysis, and severe pain. This trial compares the effectiveness and safety of five medications commonly used to prevent NMOSD relapses: rituximab, ravulizumab, inebilizumab, satralizumab, and eculizumab.
In this study, 160 adults with NMOSD who test positive for a specific antibody (AQP4-IgG) will participate. They will be randomly assigned to receive either rituximab or one of the four other FDA-approved medications. The main goal is to find out which treatment best prevents relapses and has fewer serious side effects. The trial will also measure disability, patient satisfaction, quality of life, and biomarkers that help track disease activity.
Participants will have regular assessments, including medical exams, surveys, and tests for vision, walking ability, and brain function. They will report any side effects or health issues experienced during the study. The trial will last from one to four years for each participant.
This research aims to help patients and doctors make better-informed treatment decisions by providing clear evidence about the best available therapies for NMOSD.

开始日期2025-11-01

申办/合作机构

The Massachusetts General Hospital

[+1]

NCT07024563

/ Not yet recruiting临床3期

A Phase 3, Open-Label, Multicenter Study to Evaluate the Pharmacokinetics, Pharmacodynamics, Efficacy, and Safety of Ravulizumab in Pediatric Participants (2 to < 18 Years of Age) With Primary Immunoglobulin A Nephropathy (IgAN)

The primary objective of this study is to characterize the pharmacokinetics (PK) and pharmacodynamics (PD) of treatment with ravulizumab IV in pediatric participants to support the extrapolation of efficacy from the adult population.

开始日期2025-09-26

申办/合作机构

Alexion Pharmaceuticals, Inc.

NCT06333652

/ Recruiting临床2期IIT

Clinical Trial on the Use of Ravulizumab in Pregnancies Complicated by Severe Hypertensive Disorders

The researchers are testing a medication named ravulizumab for the treatment of severe preeclampsia and Hemolysis, Elevated Liver enzymes, Low Platelets (HELLP) syndrome.

开始日期2025-09-01

申办/合作机构

Mayo Clinic

100 项与瑞利珠单抗相关的临床结果

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100 项与瑞利珠单抗相关的转化医学

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100 项与瑞利珠单抗相关的专利（医药）

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282

项与瑞利珠单抗相关的文献（医药）

2025-06-01·JOURNAL OF NEUROLOGY

Ravulizumab for generalized Myasthenia Gravis: a multicenter real-life experience

Article

作者: Marini, Sofia ; Rini, Nicasio ; Habetswallner, Francesco ; Ricciardi, Dario ; Falso, Silvia ; Leonardi, Luca ; Marando, Demetrio ; Fionda, Laura ; Pennisi, Elena Maria ; Di Stefano, Vincenzo ; Tufano, Laura ; Maestri, Michelangelo ; Guida, Melania ; Di Martino, Giuseppe ; Iorio, Raffaele ; Vinciguerra, Claudia ; Antonini, Giovanni ; Morino, Stefania ; Garibaldi, Matteo ; Rossini, Elena

Abstract:

Introduction:

Ravulizumab, a monoclonal antibody targeting C5, was recently approved for the treatment of anti-AChR positive generalized myasthenia gravis (gMG) patients. The objective of this study is to present the Italian multicenter real-world experience evaluating the safety and efficacy of ravulizumab in gMG within the context of the Expanded Early Access Program (EAP).

Methods:

We conducted a retrospective study in 7 gMG referral centres in Italy. Demographic and clinical characteristics were recorded at baseline and during follow-up through clinical scale changes including Myasthenia Gravis-Activities of Daily Living (MG-ADL), Quantitative Myasthenia Gravis (QMG) and Myasthenia Gravis Composite (MGC). Frequency of minimal symptom expression (MSE) and changes in concomitant medications were also evaluated.

Results:

Twenty-four gMG patients (10/24 females) aged between 24 and 82 years (Median 60.5, IQR 52.5–67.5), were included. Fifteen patients had undergone thymectomy, and 14 had a thymoma. Median follow-up duration was 26 weeks (range 10–74, IQR 26–42). MG-ADL and QMG scores showed a significant decrease with respect to baseline (p < 0.001). MSE was achieved by 37.5% patients at the last available follow-up. Tapering of prednisone daily dosage was possible in 76% of patients. Thymoma was significantly associated with QMG score reduction and the frequency of QMG responders at week 2 (p = 0.03).Three patients discontinued treatment. One patient experienced a myasthenic exacerbation and needed rescue therapy. Infectious adverse events were reported in 5/24 patients, and a Stevens-Johnson syndrome in one patient.

Conclusions:

Real-world data confirm the effectiveness, safety, and prednisone-sparing effect of ravulizumab in patients with gMG, especially in those with thymoma.

2025-06-01·JOURNAL DE MYCOLOGIE MEDICALE

Disseminated infection with Candida dubliniensis after ravulizumab and treatment with rezafungin – a case report

Article

作者: Omic, Haris ; Aigner, Christof ; Vossen, Matthias G ; Muraközy, Gabriella ; Lötsch, Felix ; Scharitzer, Martina ; Eder, Michael ; Doberer, Konstantin ; Willinger, Birgit

2025-06-01·Lancet Haematology

Oral iptacopan monotherapy in paroxysmal nocturnal haemoglobinuria: final 48-week results from the open-label, randomised, phase 3 APPLY-PNH trial in anti-C5-treated patients and the open-label, single-arm, phase 3 APPOINT-PNH trial in patients previously untreated with complement inhibitors

Article

作者: Griffin, Morag ; Mauad, Vitor A Q ; Yang, Chen ; Di Bona, Eros ; Langemeijer, Saskia M C ; Uchiyama, Michihiro ; Maitra, Samopriyo ; Panse, Jens ; Röth, Alexander ; Pullarkat, Vinod ; Maciejewski, Jaroslaw P ; Barcellini, Wilma ; Marano, Luana ; Ferber, Philippe ; Gandhi, Shreyans ; Risitano, Antonio M ; Han, Bing ; Höchsmann, Britta ; Kitawaki, Toshio ; Solar-Yohay, Susan ; Lee, Lily Wong Lee ; Kutlar, Abdullah ; Ueda, Yasutaka ; Mahajan, Navin ; Tavitian, Suzanne ; Chew, Lee Ping ; Zhang, Li ; Jang, Jun Ho ; Frieri, Camilla ; Schafhausen, Philippe ; Li, Shujie ; Dahlke, Marion ; Kelly, Richard J ; Beggiato, Eloise ; de Latour, Régis Peffault ; Terriou, Louis ; Kulasekararaj, Austin G ; Schubert, Jörg ; Trikha, Roochi ; Fu, Rong ; Huang, Wei-Han ; Liu, Hui ; Gaya, Anna ; de Castro, Carlos M ; Yap, Eng-Soo ; Thorburn, Christine ; Verles, Aurelie ; de Fontbrune, Flore Sicre ; Wang, Zhixin ; Scheinberg, Phillip ; Schrezenmeier, Hubert ; Kerloëguen, Cécile ; Lawniczek, Tomasz ; Kumar, Rakesh ; Notaro, Rosario ; Alashkar, Ferras

BACKGROUND:

The factor B inhibitor iptacopan improved 24-week outcomes in adult patients with paroxysmal nocturnal haemoglobinuria in the phase 3 APPLY-PNH and APPOINT-PNH trials; the trial extension periods assessed clinical activity and safety up to 48 weeks. Here, we report the final 48-week data from APPLY-PNH and APPOINT-PNH.

METHODS:

In both APPLY-PNH and APPOINT-PNH trials, patients were aged 18 years or older, with paroxysmal nocturnal haemoglobinuria (red and white blood cell population sizes ≥10%) and without laboratory evidence of bone marrow failure. In APPLY-PNH (an open-label, randomised, phase 3 trial conducted in 39 centres [38 hospitals, one outpatient research clinic] from 12 countries or regions), patients with haemoglobin concentration lower than 10 g/dL on anti-C5 treatment (stable eculizumab or ravulizumab regimen for ≥6 months) were randomly assigned (8:5) via interactive response technology to either receive oral iptacopan 200 mg twice daily (iptacopan group) or to continue their individual intravenous eculizumab or ravulizumab regimen for 24 weeks (anti-C5 group). Randomisation was stratified by type of anti-C5 and receipt of red blood cell (RBC) transfusions in the preceding 6 months. In APPOINT-PNH (an open-label, single-arm, phase 3 trial conducted in 12 hospitals from eight countries), complement inhibitor-naive patients with paroxysmal nocturnal haemoglobinuria and with haemoglobin concentration lower than 10 g/dL and lactate dehydrogenase (LDH) concentration higher than 1·5 times the upper limit of normal received iptacopan 200 mg twice daily for 24 weeks. Both trials had 24-week extension periods in which all patients received iptacopan monotherapy. Primary endpoints were the proportion of patients with an increase from baseline in haemoglobin concentration of 2 g/dL or higher (APPLY-PNH and APPOINT-PNH) and haemoglobin concentration 12 g/dL or higher (APPLY-PNH) between weeks 18 and 24, all in the absence of RBC transfusions between weeks 2 and 24; results for these primary endpoints have been reported previously. We report final activity and safety data at the completion of both trials (week 48). Prespecified endpoints at week 48 included percentage of patients with a haemoglobin increase from baseline of 2 g/dL or higher or haemoglobin 12 g/dL or higher (including post-transfusion data). Efficacy data were analysed per the intention-to-treat principle, and safety was analysed according to the treatment that patients received. APPLY-PNH and APPOINT-PNH are registered with ClinicalTrials.gov, NCT04558918 and NCT04820530, respectively.

FINDINGS:

In APPLY-PNH, between Jan 25, 2021, and April 8, 2022, 62 patients (43 [69%] female, 19 [31%] male; 48 [77%] White, 12 [19%] Asian, two [3%] Black) were randomly assigned to the iptacopan group and 35 patients (24 [69%] female, 11 [31%] male; 26 [74%] White, seven [20%] Asian, two [6%] Black) to the anti-C5 group; 61 (98%) and 34 (97%), respectively, entered the extension period. At trial completion (March 6, 2023), the median duration of iptacopan treatment was 337 days (IQR 168-338). In APPOINT-PNH, 40 patients were enrolled between July 19, 2021, and May 17, 2022, and received iptacopan (17 [43%] female, 23 [58%] male; 12 [30%] White, 27 [68%] Asian, one [3%] Black); all entered the extension period. At trial completion (April 18, 2023), the median duration of iptacopan treatment was 337 days (IQR 337-344). At week 48, irrespective of RBC transfusions, the number of patients who had an increase in haemoglobin concentration of 2 g/dL or higher was 51 (86%) of 59 in the APPLY-PNH iptacopan group, 21 (72%) of 29 in the APPLY-PNH anti-C5-to-iptacopan group, and 38 (97%) of 39 in APPOINT-PNH. The number of patients who had haemoglobin concentration of 12 g/dL or higher at week 48 was 40 (68%) of 59 in the APPLY-PNH iptacopan group, 17 (59%) of 29 in the APPLY-PNH anti-C5-to-iptacopan group, and 31 (79%) of 39 in APPOINT-PNH. There were no treatment discontinuations because of treatment-emergent adverse events or deaths. Across the 48-week trials, clinical breakthrough haemolysis occurred in seven (7%) of 96 iptacopan-treated patients in APPLY-PNH (including both groups) and two (5%) of 40 in APPOINT-PNH, but it was generally mild or moderate with no iptacopan discontinuation. Three major adverse vascular events occurred in APPLY-PNH by trial completion; all were considered unrelated to iptacopan. The most common treatment-emergent adverse event was COVID-19 in APPLY-PNH (iptacopan: 18/62 patients [29%]; anti-C5-to-iptacopan: 8/34 [24%]) and headache in APPOINT-PNH (12/40 [30%]). Severe and serious treatment-emergent adverse events were experienced by six (10%) and nine (15%) of 62 patients in the APPLY-PNH iptacopan group, respectively; in APPOINT-PNH, these were experienced by four (10%) and eight (20%) of 40 patients, respectively. The most common serious treatment-emergent adverse event was COVID-19, occurring in one (2%) of 62 patients in the APPLY-PNH iptacopan group and two (5%) of 40 patients in APPOINT-PNH. No severe treatment-emergent adverse events occurred in more than one patient.

INTERPRETATION:

Long-term data indicate durable haemolysis control with iptacopan in paroxysmal nocturnal haemoglobinuria, maintained normal or near-normal haemoglobin, and no new safety concerns. We believe that these data support iptacopan as a potential therapy option, suggesting that we are in a new treatment era for paroxysmal nocturnal haemoglobinuria.

FUNDING:

Novartis.

475

项与瑞利珠单抗相关的新闻（医药）

2025-06-18

·药时代

中国药品又一个“专利延期王”诞生

正文共：3551字 10图预计阅读时间：8分钟近日，荣昌生物宣布收到国家知识产权局下发的《药品专利权期限补偿审批决定》，称其核心产品泰它西普注射液获1827天专利补偿，保护期从2027年6月15日延长至2032年6月15日。值得注意的是，这是我国2021年新《中华人民共和国专利法》（下称《专利法》）实施以来，药品行业又一个达成“顶格补偿”的案例。制度突破自2021年6月1日起，我国新《专利法》生效，其中增加了关于药品专利权期限补偿的条款。《专利法》第四十二条第三款规定：为补偿新药上市审批占用的时间，对在中国获得上市许可的新药相关发明专利，国务院专利行政部门应专利权人的请求给予专利权期限补偿。补偿期限不超过五年，新药批准上市后总有效专利权期限不超过十四年。新药从专利申请到上市平均耗时超十年，而普通专利保护期仅二十年，导致实际市场独占期大幅缩水。以贝达药业1类新药恩沙替尼为例，历经13年研发于2020年11月上市，其化合物专利2031年到期，上市后剩余有效专利期将近11年。如果该药能够被纳入专补期限补偿的适用范围之内，市场独占期将被延长。更早上市的西达苯胺（2015年1月获批），其化合物专利（ZL03139760.3）原定于2023年到期，上市后实际保护期仅8年，成为制度落地前的典型困境案例。药品专利权期限补偿制度直击行业痛点，核心价值在于破解药品研发周期长、审批流程复杂导致的专利保护期损耗这一特殊时间困境。2025年6月，荣昌生物泰它西普注射液的核心专利《优化的TACI-Fc融合蛋白》（ZL200710111162.2）获国家知识产权局1827天（近5年）专利补偿，保护期从2027年6月15日延长至2032年6月15日，覆盖系统性红斑狼疮、重症肌无力等关键适应症的市场独占期。此前，正大天晴/康方生物的派安普利单抗注射液用于肺癌治疗的专利（ZL201610705763.5）获140天补偿期，从2036年8月23日延至2037年1月10日。药品专利权期限补偿在保障创新药企研发动力的同时，也可能因延缓仿制药上市而增加患者用药负担。为此，相关专利法规明确提出，仅未获补偿的专利可申请延期，同一药品涉及多项专利时仅限选择一项延长，单一专利覆盖多种药品时仅能针对一种药品申请。这些规定既避免创新药企重复获取专利期限补偿，也防止保护期过度延长。专利保护期延长制度起源于美国1984年颁布的《药品价格竞争与专利期限补偿法案》，又称《Hatch-Waxman法案》。随后，日本、韩国、欧盟、加拿大等多个国家或地区也相继建立了类似制度，以补偿创新药审批过程占用的时间导致的专利保护期损失。各国之间的药品专利补偿期限的补偿期差异较大。在中国和美国，专利补偿期限不能超过5年，并且总专利期限不能超过14年。欧盟同样规定补偿期不能超过5年，但首次获批药品的总专利期限可延至15年。全球首个泰它西普为何能获得五年的药品专利权期限补偿？泰它西普是全球首个BLyS/APRIL双靶点融合蛋白创新药，采用人跨膜激活剂及钙调亲环素配体相互作用因子（TACI）受体胞外域与人免疫球蛋白G（IgG）的Fc段融合结构，通过同时抑制B淋巴细胞刺激因子（BLyS）和增殖诱导配体（APRIL）的过度表达，从源头阻断异常B细胞的分化成熟，从而降低自身免疫应答。这种“双管齐下”的作用机制，在提升治疗有效性的同时显著优化了药物安全性，为自身免疫性疾病领域带来突破性解决方案。2021年3月，泰它西普获国家药监局批准用于中重度系统性红斑狼疮（SLE）治疗，成为全球首款针对SLE的双靶点生物制剂。关键性临床数据显示，泰它西普高剂量组治疗48周的系统性红斑狼疮反应指数（SRI）达79.2%，较安慰剂组（32.0%）显著提升。泰它西普于2022年底纳入国家医保目录。2024年7月，泰它西普获批第二个适应症，用于类风湿关节炎治疗，进一步拓宽在自身免疫疾病领域的应用场景。2025年5月，泰它西普斩获其第三个适应症，获国家药监局批准，与常规治疗联合用于抗AChR抗体阳性的成人全身性重症肌无力（gMG）患者，成为全球首个治疗重症肌无力的BLyS/APRIL双靶点生物药。在2025年美国神经病学会年会（AAN）披露的3期临床数据显示，治疗24周后，98.1%患者的重症肌无力日常活动评分（MGADL）改善≥3分，87%患者的定量重症肌无力评分（QMG）改善≥5分，疗效优势显著。据弗若斯特沙利文数据，全球MG患者约120万人，中国占22万。中国重症肌无力市场规模预计从2020年的0.46亿美元增至2025年的2.5亿美元。目前国内已上市的治疗重症肌无力的创新药主要由跨国药企提供，例如阿斯利康的依库珠单抗和瑞利珠单抗、优时比的罗泽利昔珠单抗，以及再鼎医药从Argenx引进的艾加莫德。泰它西普获批全身性重症肌无力适应症，不仅填补了国产创新药在该领域的空白，其双靶点机制更有望改写治疗格局。此外，重症肌无力适应症已获国家药监局突破性治疗品种、优先审评资格，及美国FDA孤儿药资格与快速通道认定。荣昌生物正持续开发泰它西普的IgA肾病、视神经脊髓炎谱系疾病、原发性干燥综合征、多发性硬化等多个适应症，其中IgA肾病与干燥综合征适应症有望于2025年下半年申报上市。海外多中心关键3期临床研究同步推进。2024年泰它西普实现营收9.77亿元，同比增长94.87%，展现出强劲的市场增长动能。增强议价筹码荣昌生物2024年第一季度归母净利润-2.54亿元，对仍在亏损中奋战的荣昌生物而言，五年专利延期如同注入一剂强心针。泰它西普原专利2027年到期，可能导致仿制药在2028年上市，而将国内专利独占期延长至2032年，泰它西普将多享有五年无仿制竞争的黄金销售期。参考2024年泰它西普近10亿营收和94.87%的增速，未来五年独占期内泰它西普累计销售额可能突破百亿规模。这将显著改善公司现金流状况，支持其庞大的研发管线。荣昌生物2024年总营收17.17亿元，同比增长58.54%，主要由泰它西普和另一款新药维迪西妥单抗驱动。专利期限延长至2032年，不仅为泰它西普在国内市场构筑了更坚实的护城河，更通过强化知识产权价值，显著提升国际合作中的议价能力。目前，泰它西普治疗重症肌无力（gMG）适应症的美国III期临床试验正在推进，并且该适应症2022年获美国FDA孤儿药资格认定，在美国上市后可享受7年市场独占期。2024年3月，泰它西普治疗干燥综合征的适应症获美国FDA授予快速通道资格，目前该适应症全球尚无生物制剂获批上市。在欧洲及东南亚等地区，专利期限的延长为技术授权合作提供了更稳定的商业化周期预期。专利保护期延长至2032年可确保合作伙伴在更久的时间内获取投资回报，降低仿制药竞争风险，增强海外合作伙伴对产品生命周期的信心。荣昌生物透露，在第62届欧洲肾脏病学会年会（ERA）期间，多家跨国药企BD负责人主动接洽，围绕泰它西普的国际授权、技术合作等商业化路径展开深度磋商。结语截至2025年，泰它西普在中国已获批系统性红斑狼疮、类风湿关节炎和重症肌无力三大适应症，2024年销售额达9.77亿元，同比增长94.87%。专利保护延长至2032年，不仅为公司锁定百亿级市场空间，更使其在海外授权谈判中获得关键筹码。参考资料：1、各公司官网2、公开信息声明：本内容仅用作医药行业信息传播，为作者独立观点，不代表药智网立场。如需转载，请务必注明文章作者和来源。对本文有异议或投诉，请联系maxuelian@yaozh.com。图片来源：豆包AI重磅！创新药大利好！临床试验60日默示许可缩短为30日！2025-06-16GCG“燃脂”独特机制直击肥胖核心：脂肪过剩2025-06-15单国洪卸任武田中国总裁，加入百济神州2025-06-177.95亿美元被收购！Sage另觅他主，Biogen“抄底”失败2025-06-1713亿美元？传礼来计划收购Verve，后者股价盘后暴涨75%！2025-06-177.45亿美元！又一款ADC授权出海2025-06-17版权声明/免责声明本文为原创文章。本文仅作信息交流之目的，不提供任何商用、医用、投资用建议。文中图片、视频、字体、音乐等素材或为药时代购买的授权正版作品，或来自微信公共图片库，或取自公司官网/网络，部分素材根据CC0协议使用，版权归拥有者，药时代尽力注明来源。如有任何问题，请与我们联系。衷心感谢!药时代官方网站:www.drugtimes.cn联系方式:电话:13651980212微信:27674131邮箱:contact@drugtimes.cn点击这里，查看更多精彩！

专利到期专利侵权专利无效

2025-06-18

·PMLiVE

AstraZeneca’s Ultomiris shows promise in rare post-transplant complication

AstraZeneca (AZ) has announced promising results from a late-stage study of its C5 complement inhibitor Ultomiris (ravulizumab) in paediatric patients with a life-threatening post-transplant complication. The phase 3 ALXN1210-TMA-314 trial has been evaluating the drug in patients aged 28 days to less than 18 years with thrombotic microangiopathy (TMA) after haematopoietic stem cell transplantation (HSCT). TMAs are a group of rare, severe disorders that cause blood clots and damage to blood vessels, potentially resulting in organ injury. There are currently no approved treatment options for HSCT-TMA, which can occur following HSCT, a procedure to treat some types of cancers and other diseases. Initial results from ALXN1210-TMA-314, presented at this year’s European Hematology Association congress, showed that overall survival, a secondary endpoint, was 92.6% at day 100 and 87.2% at week 26. The primary endpoint of complete TMA response was achieved by 17.1% of patients during the 26-week treatment period, while 70.7% met at least one TMA response criterion and 53.7% reached one or two response criteria, with clinically meaningful improvements seen across the components of TMA response. ALXN1210-TMA-314 trial investigator, Franco Locatelli, said: “The impact of HSCT-TMA on patient outcomes is devastating, particularly among high-risk populations, such as children, for whom there are currently no targeted therapies approved to treat this rare, severe and potentially life-threatening condition. “The response rates and the overall survival observed with [Ultomiris] may be a practice-changing advancement in this field, providing a potentially effective option for these young patients and their families.” Administered intravenously, AZ’s Ultomiris already holds approvals to treat certain cases of paroxysmal nocturnal haemoglobinuria, atypical haemolytic uraemic syndrome, generalised myasthenia gravis and neuromyelitis optica spectrum disorder. Alongside the paediatric study, the drug is also being evaluated in the phase 3 ALXN1210-TMA-313 trial of adults and adolescents aged 12 years or older with HSCT-TMA. Gianluca Pirozzi, senior vice president, head of development, regulatory and safety at Alexion, AstraZeneca Rare Disease, said: “… we continue to evaluate Ultomiris in adults and adolescents suffering from this devastating, rare condition. “Together, these two trials represent the largest, global, phase 3 programme in patients with HSCT-TMA.”

临床3期临床结果

2025-06-17

·汇聚南药

中国药品“专利延期王”诞生

近日，荣昌生物宣布收到国家知识产权局下发的《药品专利权期限补偿审批决定》，称其核心产品泰它西普注射液获1827天专利补偿，保护期从2027年6月15日延长至2032年6月15日。值得注意的是，这可能是我国2021年新《中华人民共和国专利法》（下称《专利法》）实施以来，药品行业首个达成“顶格补偿”的案例。制度突破自2021年6月1日起，我国新《专利法》生效，其中增加了关于药品专利权期限补偿的条款。《专利法》第四十二条第三款规定：为补偿新药上市审批占用的时间，对在中国获得上市许可的新药相关发明专利，国务院专利行政部门应专利权人的请求给予专利权期限补偿。补偿期限不超过五年，新药批准上市后总有效专利权期限不超过十四年。新药从专利申请到上市平均耗时超十年，而普通专利保护期仅二十年，导致实际市场独占期大幅缩水。以贝达药业1类新药恩沙替尼为例，历经13年研发于2020年11月上市，其化合物专利2031年到期，上市后剩余有效专利期将近11年。如果该药能够被纳入专补期限补偿的适用范围之内，市场独占期将被延长。更早上市的西达苯胺（2015年1月获批），其化合物专利（ZL03139760.3）原定于2023年到期，上市后实际保护期仅8年，成为制度落地前的典型困境案例。药品专利权期限补偿制度直击行业痛点，核心价值在于破解药品研发周期长、审批流程复杂导致的专利保护期损耗这一特殊时间困境。2025年6月，荣昌生物泰它西普注射液的核心专利《优化的TACI-Fc融合蛋白》（ZL200710111162.2）获国家知识产权局1827天（近5年）专利补偿，保护期从2027年6月15日延长至2032年6月15日，覆盖系统性红斑狼疮、重症肌无力等关键适应症的市场独占期。此前，正大天晴/康方生物的派安普利单抗注射液用于肺癌治疗的专利（ZL201610705763.5）获140天补偿期，从2036年8月23日延至2037年1月10日。药品专利权期限补偿在保障创新药企研发动力的同时，也可能因延缓仿制药上市而增加患者用药负担。为此，相关专利法规明确提出，仅未获补偿的专利可申请延期，同一药品涉及多项专利时仅限选择一项延长，单一专利覆盖多种药品时仅能针对一种药品申请。这些规定既避免创新药企重复获取专利期限补偿，也防止保护期过度延长。专利保护期延长制度起源于美国1984年颁布的《药品价格竞争与专利期限补偿法案》，又称《Hatch-Waxman法案》。随后，日本、韩国、欧盟、加拿大等多个国家或地区也相继建立了类似制度，以补偿创新药审批过程占用的时间导致的专利保护期损失。各国之间的药品专利补偿期限的补偿期差异较大。在中国和美国，专利补偿期限不能超过5年，并且总专利期限不能超过14年。欧盟同样规定补偿期不能超过5年，但首次获批药品的总专利期限可延至15年。为何值得五年延期？泰它西普能获得中国药品专利权期限补偿制度实施以来最长的五年延期，源于其突破性的科学价值和巨大的临床需求。泰它西普是全球首个BLyS/APRIL双靶点融合蛋白创新药，采用人跨膜激活剂及钙调亲环素配体相互作用因子（TACI）受体胞外域与人免疫球蛋白G（IgG）的Fc段融合结构，通过同时抑制B淋巴细胞刺激因子（BLyS）和增殖诱导配体（APRIL）的过度表达，从源头阻断异常B细胞的分化成熟，从而降低自身免疫应答。这种“双管齐下”的作用机制，在提升治疗有效性的同时显著优化了药物安全性，为自身免疫性疾病领域带来突破性解决方案。2021年3月，泰它西普获国家药监局批准用于中重度系统性红斑狼疮（SLE）治疗，成为全球首款针对SLE的双靶点生物制剂。关键性临床数据显示，泰它西普高剂量组治疗48周的系统性红斑狼疮反应指数（SRI）达79.2%，较安慰剂组（32.0%）显著提升。泰它西普于2022年底纳入国家医保目录。2024年7月，泰它西普获批第二个适应症，用于类风湿关节炎治疗，进一步拓宽在自身免疫疾病领域的应用场景。2025年5月，泰它西普斩获其第三个适应症，获国家药监局批准，与常规治疗联合用于抗AChR抗体阳性的成人全身性重症肌无力（gMG）患者，成为全球首个治疗重症肌无力的BLyS/APRIL双靶点生物药。在2025年美国神经病学会年会（AAN）披露的3期临床数据显示，治疗24周后，98.1%患者的重症肌无力日常活动评分（MGADL）改善≥3分，87%患者的定量重症肌无力评分（QMG）改善≥5分，疗效优势显著。据弗若斯特沙利文数据，全球MG患者约120万人，中国占22万。中国重症肌无力市场规模预计从2020年的0.46亿美元增至2025年的2.5亿美元。目前国内已上市的治疗重症肌无力的创新药主要由跨国药企提供，例如阿斯利康的依库珠单抗和瑞利珠单抗、优时比的罗泽利昔珠单抗，以及再鼎医药从Argenx引进的艾加莫德。泰它西普获批全身性重症肌无力适应症，不仅填补了国产创新药在该领域的空白，其双靶点机制更有望改写治疗格局。此外，重症肌无力适应症已获国家药监局突破性治疗品种、优先审评资格，及美国FDA孤儿药资格与快速通道认定。荣昌生物正持续开发泰它西普的IgA肾病、视神经脊髓炎谱系疾病、原发性干燥综合征、多发性硬化等多个适应症，其中IgA肾病与干燥综合征适应症有望于2025年下半年申报上市。海外多中心关键3期临床研究同步推进。2024年泰它西普实现营收9.77亿元，同比增长94.87%，展现出强劲的市场增长动能。增强议价筹码荣昌生物2024年第一季度归母净利润-2.54亿元，对仍在亏损中奋战的荣昌生物而言，五年专利延期如同注入一剂强心针。泰它西普原专利2027年到期，可能导致仿制药在2028年上市，而将国内专利独占期延长至2032年，泰它西普将多享有五年无仿制竞争的黄金销售期。参考2024年泰它西普近10亿营收和94.87%的增速，未来五年独占期内泰它西普累计销售额可能突破百亿规模。这将显著改善公司现金流状况，支持其庞大的研发管线。荣昌生物2024年总营收17.17亿元，同比增长58.54%，主要由泰它西普和另一款新药维迪西妥单抗驱动。专利期限延长至2032年，不仅为泰它西普在国内市场构筑了更坚实的护城河，更通过强化知识产权价值，显著提升国际合作中的议价能力。目前，泰它西普治疗重症肌无力（gMG）适应症的美国III期临床试验正在推进，并且该适应症2022年获美国FDA孤儿药资格认定，在美国上市后可享受7年市场独占期。2024年3月，泰它西普治疗干燥综合征的适应症获美国FDA授予快速通道资格，目前该适应症全球尚无生物制剂获批上市。在欧洲及东南亚等地区，专利期限的延长为技术授权合作提供了更稳定的商业化周期预期。专利保护期延长至2032年可确保合作伙伴在更久的时间内获取投资回报，降低仿制药竞争风险，增强海外合作伙伴对产品生命周期的信心。荣昌生物透露，在第62届欧洲肾脏病学会年会（ERA）期间，多家跨国药企BD负责人主动接洽，围绕泰它西普的国际授权、技术合作等商业化路径展开深度磋商。结语截至2025年，泰它西普在中国已获批系统性红斑狼疮、类风湿关节炎和重症肌无力三大适应症，2024年销售额达9.77亿元，同比增长94.87%。专利保护延长至2032年，不仅为公司锁定百亿级市场空间，更使其在海外授权谈判中获得关键筹码。参考资料：1、各公司官网2、公开信息喜欢我们文章的朋友点个“在看”和“赞”吧，不然微信推送规则改变，有可能每天都会错过我们哦~免责声明“汇聚南药”公众号所转载文章来源于其他公众号平台，主要目的在于分享行业相关知识，传递当前最新资讯。图片、文章版权均属于原作者所有，如有侵权，请在留言栏及时告知，我们会在24小时内删除相关信息。信息来源：药智网往期推荐本平台不对转载文章的观点负责，文章所包含内容的准确性、可靠性或完整性提供任何明示暗示的保证。

专利到期专利无效专利侵权

100 项与瑞利珠单抗相关的药物交易

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外链

KEGG	Wiki	ATC	Drug Bank
-	瑞利珠单抗	L04AA43	DB11580

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
视神经脊髓炎	欧盟	Alexion Europe SAS	2023-07-12
视神经脊髓炎	冰岛	Alexion Europe SAS	2023-07-12
视神经脊髓炎	列支敦士登	Alexion Europe SAS	2023-07-12
视神经脊髓炎	挪威	Alexion Europe SAS	2023-07-12
血栓性微血管病	韩国	Astrazeneca Korea Ltd.	2020-05-21
水通道蛋白4抗体阳性视神经脊髓炎谱系疾病	澳大利亚	Alexion Pharmaceuticals Australasia Pty Ltd.	2019-10-17
重症肌无力	澳大利亚	Alexion Pharmaceuticals Australasia Pty Ltd.	2019-10-17
非典型溶血性尿毒症综合征	欧盟	Alexion Europe SAS	2019-07-02
非典型溶血性尿毒症综合征	冰岛	Alexion Europe SAS	2019-07-02
非典型溶血性尿毒症综合征	列支敦士登	Alexion Europe SAS	2019-07-02
非典型溶血性尿毒症综合征	挪威	Alexion Europe SAS	2019-07-02
阵发性睡眠性血红蛋白尿症	美国	Alexion Pharmaceuticals, Inc.	2018-12-21

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
Henoch-Schonlein紫癜性肾炎	临床3期	美国	Alexion Pharmaceuticals, Inc.	2025-09-26
Henoch-Schonlein紫癜性肾炎	临床3期	中国	Alexion Pharmaceuticals, Inc.	2025-09-26
Henoch-Schonlein紫癜性肾炎	临床3期	意大利	Alexion Pharmaceuticals, Inc.	2025-09-26
Henoch-Schonlein紫癜性肾炎	临床3期	韩国	Alexion Pharmaceuticals, Inc.	2025-09-26
Henoch-Schonlein紫癜性肾炎	临床3期	西班牙	Alexion Pharmaceuticals, Inc.	2025-09-26
Henoch-Schonlein紫癜性肾炎	临床3期	中国台湾	Alexion Pharmaceuticals, Inc.	2025-09-26
过敏性紫癜	临床3期	美国	Alexion Pharmaceuticals, Inc.	2025-09-26
过敏性紫癜	临床3期	中国	Alexion Pharmaceuticals, Inc.	2025-09-26
过敏性紫癜	临床3期	意大利	Alexion Pharmaceuticals, Inc.	2025-09-26
过敏性紫癜	临床3期	韩国	Alexion Pharmaceuticals, Inc.	2025-09-26

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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


PB2822 (EHA2025)	N/A	阵发性睡眠性血红蛋白尿症	127	Ravulizumab	糧衊築鏇鑰鬱遞憲築憲(範襯獵顧憲夢夢遞製顧) = 壓願夢醖餘網糧積鏇壓蓋鹽鬱夢構顧獵獵繭餘 (淵鏇糧願鏇選壓構鑰餘, 1.8)	积极	2025-05-14
				Ravulizumab (Ci-naive patients)	糧衊築鏇鑰鬱遞憲築憲(範襯獵顧憲夢夢遞製顧) = 製夢築廠範願襯壓積選蓋鹽鬱夢構顧獵獵繭餘 (淵鏇糧願鏇選壓構鑰餘, 1.5)
ALPHA (EHA2025)	临床3期	阵发性睡眠性血红蛋白尿症追加	-	Ravulizumab	齋糧夢衊築糧觸糧膚膚(齋鹹顧鹹鏇淵網鹹鹹網) = 製範網獵製窪願顧鏇製獵膚窪壓壓鹽築築醖餘 (製遞選糧製餘膚鹹衊廠, per 10 person ~ years) 更多	积极	2025-05-14
				Danicopan	遞淵淵衊艱鑰簾鑰艱衊(鏇鑰鑰築衊製製膚膚觸) = 憲蓋艱鬱願蓋簾顧糧網醖窪顧製積窪鬱遞膚鑰 (憲獵膚構築積選壓製積 )
NCT04557735 (ALXN1210-TMA-314, EHA2025)	临床3期	血栓性微血管病	40	Ravulizumab	淵膚廠窪夢鑰顧鹽選製(窪範夢製構淵鬱蓋積餘) = 製觸構選築製網範觸範壓積網鏇鹹餘繭製衊選 (醖壓糧觸廠鏇淵衊壓積 )	积极	2025-05-14
NCT04557735 (ALXN1210-TMA-314, EHA2025)	临床3期	血栓性微血管病	41	Ravulizumab	壓鹹餘齋艱遞夢製簾憲(壓窪膚廠衊願願鹽廠夢) = 簾願醖襯憲醖齋糧憲餘繭廠觸積鏇簾衊醖積遞 (製衊淵繭製夢衊窪淵廠 ) 更多	积极	2025-05-14
				Ravulizumab (Best Supportive Care)	構觸鏇夢膚繭範觸窪築(遞醖鑰艱獵網顧範網齋) = 醖鬱鹹淵築廠願蓋築觸鬱繭窪衊餘遞夢襯餘網 (蓋衊簾顧壓糧範廠鹹構 ) 更多
NCT03056040 (302 study, EHA2025)	临床3期	阵发性睡眠性血红蛋白尿症	195	Ravulizumab	顧繭積鏇醖構衊衊繭鑰(繭壓糧繭顧餘選遞夢願) = 淵餘糧鹹憲淵鬱艱憲淵醖齋夢鹹廠餘襯淵鏇鹹 (襯範糧窪襯鬱鏇窪願襯, 29.9)	积极	2025-05-14
				Eculizumab	顧繭積鏇醖構衊衊繭鑰(繭壓糧繭顧餘選遞夢願) = 鏇鹹獵鹽淵願襯醖艱齋醖齋夢鹹廠餘襯淵鏇鹹 (襯範糧窪襯鬱鏇窪願襯, 30.9)
P1-11.001 (AAN2025)	N/A	重症肌无力 anti-acetylcholine receptor antibody-positive (AChR-Ab+)	152	Ravulizumab	簾鹽鏇鹹蓋構願顧構蓋(醖觸網鹹鹽製膚廠遞繭) = 簾鬱糧構選遞範廠製夢製鬱選夢獵襯夢觸願衊 (簾蓋顧獵膚糧願遞醖艱 )	积极	2025-04-07
				Efgartigimod	鑰構鬱觸醖廠鏇糧鹹鑰(獵鹽獵顧淵積選製顧醖) = 餘選襯糧鏇齋築願鑰夢夢獵網簾齋淵蓋夢艱製 (窪遞齋餘願齋願襯鏇選 ) 更多
P7-11.023 (AAN2025)	N/A	重症肌无力	-	Ravulizumab	簾鏇衊遞積膚窪壓獵構(夢憲糧夢網醖壓鹽蓋鑰) = 繭顧鑰窪壓醖窪淵鬱鏇淵膚範艱範夢築夢願衊 (鑰積獵淵糧獵蓋餘選遞 )	积极	2025-04-07
NCT05274633 (REACTION, ASH2024) 人工标引	N/A	阵发性睡眠性血红蛋白尿症	80	Eculizumabravulizumab	鹹壓壓蓋襯壓鹽選鏇選(鬱積鏇構範憲壓齋願廠) = 觸鑰顧壓膚鏇醖鑰觸簾繭製艱積顧襯製觸蓋糧 (繭構衊淵遞鹽窪遞獵網 ) 更多	积极	2024-12-08
ASH2024	N/A	非典型溶血性尿毒症综合征	-	Eculizumab	構窪鹽顧糧醖範鹽襯積(齋糧鹽遞淵鹽襯網鬱鏇) = 鹹窪鑰願鑰壓遞築網廠網蓋顧繭繭艱製齋醖構 (鏇廠鏇顧選窪襯簾齋餘, 230.0 ~ 362.0) 更多	-	2024-12-08
				Ravulizumab	憲觸淵範鏇衊繭選鏇艱(衊齋築願築顧齋鹹鹹觸) = 鑰餘積衊鑰蓋繭衊艱遞齋構範鹽醖願齋範膚繭 (夢糧襯鏇廠觸鬱糧壓廠, 11.5% ~ 36.4%)