The purpose of this study is to determine if zigakibart is safe and effective for long-term use in patients with immunoglobulin A nephropathy (IgAN). This is an extension study for patients who have already completed an another zigakibart study.

开始日期2025-07-28

申办/合作机构

Novartis Pharmaceuticals Canada, Inc.

ChiCTR2500096892

/ Recruiting临床3期IIT

A Phase 3, Randomized, Double-blind, Placebo-controlled Study of BION-1301 in Adults with IgA Nephropathy(The BEYOND Study)

开始日期2024-11-11

申办/合作机构-

NCT05852938

/ Recruiting临床3期

A Phase 3, Randomized, Double-blind, Placebo-controlled Study of BION-1301 in Adults With IgA Nephropathy (The BEYOND Study)

Safety and Efficacy of BION-1301 in Adults with IgA Nephropathy

开始日期2023-07-06

申办/合作机构

Novartis Pharmaceuticals Canada, Inc.

100 项与 Zigakibart 相关的临床结果

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100 项与 Zigakibart 相关的转化医学

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100 项与 Zigakibart 相关的专利（医药）

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项与 Zigakibart 相关的文献（医药）

2023-12-04·Clinical kidney journal

The contribution of a proliferation-inducing ligand (APRIL) and other TNF superfamily members in pathogenesis and progression of IgA nephropathy

Review

作者: Barratt, Jonathan ; Yeo, See Cheng

ABSTRACT:

Advances in our understanding of the pathogenesis of immunoglobulin A nephropathy (IgAN) have led to the identification of novel therapeutic targets and potential disease-specific treatments. Specifically, a proliferation-inducing ligand (APRIL) has been implicated in the pathogenesis of IgAN, mediating B-cell dysregulation and overproduction of pathogenic galactose-deficient IgA1 (Gd-IgA1). Animal and clinical studies support the involvement of APRIL in the pathogenesis and progression of IgAN. An elevated level of APRIL is found in IgAN when compared with controls, which correlates with the level of Gd-IgA1 and associates with more severe disease presentation and worse outcomes. Conversely, anti-APRIL therapy reduces pathogenic Gd-IgA1 and IgA immune complex formation and ameliorates the severity of kidney inflammation and injury. Genome-wide association studies in IgAN have identified TNFSF13 and TNFRSF13B, a cytokine ligand-receptor gene pair encoding APRIL and its receptor, respectively, as risk susceptibility loci in IgAN, further supporting the causal role of the APRIL signalling pathway in IgAN. Several novel experimental agents targeting APRIL, including atacicept, telitacicept, zigakibart and sibeprenlimab, are currently under investigation as potential therapies in IgAN. Preliminary results suggest that these agents are well-tolerated, and reduce levels of Gd-IgA1, with corresponding improvement in proteinuria. Further studies are ongoing to confirm the safety and efficacy of anti-APRIL approaches as an effective therapeutic strategy in IgAN.

2023-12-01·Clinical rheumatology

A narrative review of potential drug treatments for nephritis in children with IgA vasculitis (HSP)

Review

作者: Lamond, Megan ; Chetwynd, Andrew J ; Marro, Julien ; Oni, Louise ; Williams, Chloe E C

Abstract:

Immunoglobulin A (IgA) vasculitis (IgAV, also known as Henoch-Schoenlein purpura, HSP) is the most common vasculitis of childhood. It usually presents with a simple, self-limiting disease course; however, a small subset of patients may develop kidney involvement (IgAV-N) which occurs 4–12 weeks after disease onset and is the biggest contributor to long-term morbidity. Treatment currently targets patients with established kidney involvement; however; there is a desire to work towards early prevention of inflammation during the window of opportunity between disease presentation and onset of significant nephritis. There are no clinical trials evaluating drugs which may prevent or halt the progression of nephritis in children with IgAV apart from the early use of corticosteroids which have no benefit. This article summarises the latest scientific evidence and clinical trials that support potential therapeutic targets for IgAV-N that are currently being developed based on the evolving understanding of the pathophysiology of IgAV-N. These span the mucosal immunity, B-cell and T-cell modulation, RAAS inhibition, and regulation of complement pathways, amongst others. Novel drugs that may be considered for use in early nephritis include TRF-budesonide; B-cell inhibiting agents including belimumab, telitacicept, blisibimod, VIS649, and BION-1301; B-cell depleting agents such as rituximab, ofatumumab, and bortezomib; sparsentan; angiotensin converting enzyme inhibitors (ACE-Is); and complement pathway inhibitors including avacopan, iptacopan, and narsoplimab. Further clinical trials, as well as pre-clinical scientific studies, are needed to identify mechanistic pathways as there may be an opportunity to prevent nephritis in this condition.Key Points• Kidney involvement is the main cause of long-term morbidity and mortality in IgA vasculitis despite the current treatment recommendations.• The evolving understanding of the pathophysiology of IgA vasculitis is allowing exploration of novel treatment options which target underlying immune pathways.• Novel treatments currently being trialled in IgA nephropathy may have benefit in IgA vasculitis due to the similarities in the underlying pathophysiology, such as TRF-budesonide, B-cell modulators, and complement inhibitors.• Further studies, including clinical trials of novel drugs, are urgently needed to improve the long-term outcomes for children with IgA vasculitis nephritis.

2021-09-02·Nature

APRIL limits atherosclerosis by binding to heparan sulfate proteoglycans

Article

作者: Smeets, Diede ; Frommlet, Florian ; Weißer, Juliane ; Mueller, André ; Jørgensen, Helle F ; Mallat, Ziad ; Simon, Tabassome ; Donzé, Olivier ; Obermayer, Georg ; Pasterkamp, Gerard ; Willen, Laure ; Lambert, Jordi ; Hendrikx, Tim ; Fogelstrand, Per ; März, Winfried ; Enders, Lennart ; Ozsvar-Kozma, Maria ; Hoke, Matthias ; Scharnagl, Hubert ; Clement, Marc ; Binder, Christoph J ; Porsch, Florentina ; Murphy, Jane E ; Eslami, Mahya ; Kiss, Máté G ; Borén, Jan ; Hess, Henry ; Schneider, Pascal ; Afonyushkin, Taras ; Mayer, Florian J ; Danchin, Nicolas ; Tsiantoulas, Dimitrios ; Kubicek, Stefan ; Göderle, Laura

Atherosclerotic cardiovascular disease causes heart attacks and strokes, which are the leading causes of mortality worldwide¹. The formation of atherosclerotic plaques is initiated when low-density lipoproteins bind to heparan-sulfate proteoglycans (HSPGs)² and become trapped in the subendothelial space of large and medium size arteries, which leads to chronic inflammation and remodelling of the artery wall². A proliferation-inducing ligand (APRIL) is a cytokine that binds to HSPGs³, but the physiology of this interaction is largely unknown. Here we show that genetic ablation or antibody-mediated depletion of APRIL aggravates atherosclerosis in mice. Mechanistically, we demonstrate that APRIL confers atheroprotection by binding to heparan sulfate chains of heparan-sulfate proteoglycan 2 (HSPG2), which limits the retention of low-density lipoproteins, accumulation of macrophages and formation of necrotic cores. Indeed, antibody-mediated depletion of APRIL in mice expressing heparan sulfate-deficient HSPG2 had no effect on the development of atherosclerosis. Treatment with a specific anti-APRIL antibody that promotes the binding of APRIL to HSPGs reduced experimental atherosclerosis. Furthermore, the serum levels of a form of human APRIL protein that binds to HSPGs, which we termed non-canonical APRIL (nc-APRIL), are associated independently of traditional risk factors with long-term cardiovascular mortality in patients with atherosclerosis. Our data reveal properties of APRIL that have broad pathophysiological implications for vascular homeostasis.

186

项与 Zigakibart 相关的新闻（医药）

2025-05-27

·PRNewswire

FILSPARI to Transform Kidney Disease Care with Its Revolutionary Treatment Approach | DelveInsight

FILSPARI holds strong market potential as the first FDA-approved non-immunosuppressive therapy for IgA nephropathy, a rare kidney disease with significant unmet needs. As more patients and providers seek disease-modifying options, FILSPARI is well-positioned to capture substantial market share, especially with favorable long-term data. LAS VEGAS, May 27, 2025 /PRNewswire/ -- DelveInsight's " FILSPARI Market Size, Forecast, and Market Insight Report" highlights the details around FILSPARI, the first and only oral, once-daily, non-immunosuppressive therapy approved in both the US and Europe for IgAN. The report provides product descriptions, patent details, and competitor products (marketed and emerging therapies) of FILSPARI. The report also highlights the historical and forecasted sales from 2020 to 2034 segmented into 7MM [the United States, the EU4 (Germany, France, Italy, and Spain), the United Kingdom, and Japan]. Travere Therapeutics' FILSPARI (sparsentan) Overview FILSPARI (sparsentan) is a once-daily oral treatment that uniquely targets two key pathways, endothelin-1 and angiotensin II (also known as DEARA), in the progression of IgA nephropathy. It is the first approved non-immunosuppressive therapy for this condition. Endothelin-1 and angiotensin II contribute to kidney function decline by promoting inflammation and fibrosis, altering podocyte structure, causing podocyte loss, and increasing the permeability of the glomerular filtration barrier. Additionally, both are vasoconstrictors that narrow blood vessels and raise pressure in the glomeruli. The drug received accelerated approval based on its ability to reduce proteinuria. Due to potential safety concerns, it is distributed under the FILSPARI Risk Evaluation and Mitigation Strategy (REMS) program. In Europe, it is marketed by CSL Vifor, and in Japan by Renalys Pharma. Results from the registration-enabling study are expected in the second half of 2025. Learn more about FILSPARI projected market size for IgAN @ FILSPARI IgA Nephropathy IgA nephropathy (IgAN) is an autoimmune disorder that disrupts kidney function by damaging the small blood vessels responsible for filtration. This damage is caused by an abnormal protein that harms the glomeruli, the kidneys' main filtering units. According to DelveInsight, there were approximately 415,000 diagnosed prevalent cases of IgAN across the seven major markets in 2024, and this number is projected to grow at a CAGR of 0.6% through 2034. Current standard care involves the use of ACE inhibitors and angiotensin II receptor blockers (ARBs), primarily to manage associated symptoms like hypertension. Currently, only a few drugs have been approved for IgAN treatment, including VANRAFIA (Atrasentan) and FABHALTA (Iptacopan) by Novartis, FILSPARI (Sparsentan) by Travere Therapeutics, and TARPEYO/KINPEYGO (budesonide) by Asahi Kasei (Calliditas Therapeutics), among others. The treatment landscape is expected to undergo major changes between 2024 and 2034, driven by the introduction of innovative therapies. DelveInsight estimates the IgAN market across the seven key regions was valued at around USD 730 million in 2024 and is projected to grow at a strong CAGR of 30.5% from 2025 to 2034. With targeted treatment options for IgAN only recently emerging and a pressing need for therapies that can delay progression to end-stage kidney disease (ESKD), significant advancements in this area are likely to profoundly reshape the market in the coming years. Discover more about the IgAN market in detail @ IgA Nephropathy Market Assessment Emerging Competitors of FILSPARI Key companies advancing therapies for IgA nephropathy include Novartis (Zigakibart/FUB523), F. Hoffmann-La Roche and Ionis Pharmaceuticals (Sefaxersen/RG6299/IONIS-FB-LRx), AstraZeneca's Alexion Pharmaceuticals (ULTOMIRIS), Vera Therapeutics (Atacicept), Vertex Pharmaceuticals (Povetacicept), Otsuka Pharmaceutical (Sibeprenlimab), Biogen (Felzartamab), Arrowhead Pharmaceuticals (ARO-C3), NovelMed (NM8074), Q32 Bio (ADX-097), Walden Biosciences (WAL0921), and Takeda Pharmaceutical (TAK-079), among others. In April 2025, Vera Therapeutics announced it had completed patient enrollment for its pivotal Phase III ORIGIN trial assessing atacicept in IgA nephropathy. The previous month, Otsuka Pharmaceutical submitted a Biologics License Application (BLA) to the FDA for sibeprenlimab, a monoclonal antibody aimed at inhibiting APRIL (A PRoliferation-Inducing Ligand) in adults with IgAN. Additionally, in July 2022, the European Commission granted Orphan Drug Designation (ODD) to BION-1301 for primary IgAN, offering regulatory benefits to support its European development. Strengthening its renal pipeline, Novartis acquired Chinook Therapeutics in August 2023 for up to $3.5 billion, adding zigakibart and other late-stage kidney-focused assets to its portfolio. To know how does FILSPARI compare to other treatments for IgA nephropathy, visit @ FILSPARI Approval Date Key Milestones of FILSPARI In November 2024, FILSPARI was approved by the MHRA for primary IgAN treatment, and sparsentan received approval in Germany for the same indication. In September 2024, the FDA granted full approval for FILSPARI to slow kidney function decline in IgAN patients, based on positive long-term results from the PROTECT Study. In April 2024, CSL Vifor and Travere Therapeutics received Conditional Marketing Authorization (CMA) from the European Commission for FILSPARI in the EU, targeting adults with IgAN and significant proteinuria. In February 2023, the US FDA granted accelerated approval to FILSPARI (sparsentan) to reduce proteinuria in adults with primary IgAN at risk of rapid progression, with priority review. Discover how FILSPARI is shaping the IgAN treatment landscape @ FILSPARI IgAN FILSPARI Market Dynamics FILSPARI, developed by Travere Therapeutics, represents a significant advancement in the treatment of IgA Nephropathy, a rare and chronic autoimmune kidney disease. Approved under the FDA's accelerated approval program in early 2023, FILSPARI is the first non-immunosuppressive therapy specifically targeting proteinuria in IgAN. It is a dual endothelin angiotensin receptor antagonist (DEARA), a novel mechanism that distinguishes it from conventional RAAS inhibitors. Its market entry has been notable for both its scientific innovation and potential to reshape the standard of care for IgAN, particularly in patients at high risk of progression to end-stage kidney disease (ESKD). The market dynamics are influenced by multiple factors, including the unmet need in IgAN, the orphan drug designation, and the limited competition in the space. Prior to FILSPARI's launch, treatment was largely supportive, focusing on blood pressure control and proteinuria reduction via ACE inhibitors or ARBs. FILSPARI's ability to deliver greater proteinuria reduction without the side effects associated with corticosteroids or immunosuppressants provides a compelling clinical and commercial value proposition. Competition is evolving, with several companies advancing novel IgAN therapeutics. Calliditas Therapeutics' TARPEYO (budesonide) was approved ahead of FILSPARI for a similar indication, but works through a corticosteroid pathway. Other players are also advancing programs, suggesting the market could become more segmented based on patient profile and mechanism of action. FILSPARI's differentiated profile, especially its dual-acting mechanism and oral administration, positions it favorably among nephrologists and specialists, but maintaining this edge will require strong real-world evidence and ongoing clinical data. From a market access and adoption standpoint, Travere has made early efforts in physician education, patient support programs, and collaboration with nephrology groups to accelerate uptake. However, broader adoption will depend on securing favorable formulary placements, long-term safety data, and sustained efficacy in delaying disease progression. As I gAN awareness grows and diagnosis rates improve through genetic and biomarker testing, the addressable patient population may expand, offering a significant growth opportunity for FILSPARI over the next 5–10 years. Dive deeper to get more insight into FILSPARI's strengths & weaknesses relative to competitors @ FILSPARI MoA Table of Contents Related Reports IgA Nephropathy Market IgA Nephropathy Market Insight, Epidemiology, and Market Forecast – 2034 report delivers an in-depth understanding of market trends, market drivers, market barriers, and key IgAN companies such as Calliditas Therapeutics AB, Travere Therapeutics, Inc., Omeros, Novartis Pharmaceuticals, Chinook Therapeutics, Inc., Vera Therapeutics, Inc., among others. IgA Nephropathy Pipeline IgA Nephropathy Pipeline Insight – 2025 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key IgA nephropathy companies, including Chinook Therapeutics, Inc., RemeGen Co., Ltd., Novartis, Jiangsu HengRui Medicine Co., Ltd., Ionis Pharmaceuticals, Inc., Vera Therapeutics, Inc., Eledon Pharmaceuticals, Guangdong Hengrui Pharmaceutical Co., Ltd, Omeros Corporation, Otsuka Pharmaceutical, Alnylam Pharmaceuticals, MorphoSys AG, Rohto Pharmaceutical, Alexion Pharmaceuticals, Apellis Pharmaceuticals, Inc., Arrowhead Pharmaceuticals, Takeda, Travere Therapeutics, BioCryst Pharmaceuticals, Transcenta Holding, Shanghai Alebund Pharmaceuticals, DiaMedica Therapeutics, SELECTA BIOSCIENCES, Kira Pharmaceuticals, Alpine Immune Sciences, among others. Chronic Kidney Disease Market Chronic Kidney Disease Market Insights, Epidemiology, and Market Forecast – 2034 report deliver an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key chronic kidney disease companies, including ProKidney, Reata Pharmaceuticals, Inc., Novo Nordisk A/S, Boehringer Ingelheim, Eli Lilly and Company, KBP Biosciences, Kibow Pharma, Cincor Pharma, AstraZeneca, Allena Pharmaceuticals, DiaMedica Therapeutics Inc, Lexicon Pharmaceuticals, Sanofi , among others. Chronic Kidney Disease Pipeline Chronic Kidney Disease Pipeline Insight – 2025 report provides comprehensive insights about the pipeline landscape, including clinical and non-clinical stage products and the key chronic kidney disease companies, including KBP Biosciences, Eli Lilly and Company, Novo Nordisk, Prokidney, Boryung Pharmaceutical, among others. About DelveInsight DelveInsight is a leading Business Consultant and Market Research firm focused exclusively on life sciences. It supports pharma companies by providing comprehensive end-to-end solutions to improve their performance. Get hassle-free access to all the healthcare and pharma market research reports through our subscription-based platform PharmDelve . Contact Us Shruti Thakur [email protected] +14699457679 Logo: SOURCE DelveInsight Business Research, LLP WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

上市批准临床3期并购孤儿药免疫疗法

2025-04-04

·CPHI制药在线

罗氏多发性硬化症重磅药奥瑞利珠单抗国内获批；2024年畅销药TOP100公布，K药294.82亿美元排第一|制药在线一周药闻复盘

点击蓝字关注我们本周，热点不少。首先看审评审批方面，非常值得关注的就是，罗氏多发性硬化症重磅药物奥瑞利珠单抗国内获批上市，成为国内第二款获批上市的FcRn单抗；其次是研发方面，值得一提的是，复宏汉霖贝伐珠单抗眼科Ⅲ期研究成功；再次是交易及投融资方面，和誉医药匹米替尼全球权益落地；最后是其他方面，2024年全球畅销药TOP100公布，K药以294.82亿美元排名第一。本周盘点包括审评审批、研发、交易及投融资以及其他四大板块，统计时间为2025.3.31-4.3，包含24条信息。审评审批NMPA上市批准1、3月31日，NMPA官网显示，罗氏的奥瑞利珠单抗（Ocrelizumab、商品名：OCREVUS）获批上市，每六个月静脉输注一次给药，用于治疗成人复发型多发性硬化症（RMS，包括临床孤立综合征、复发缓解型多发性硬化和活动性继发进展型多发性硬化）和成人原发进展型多发性硬化症（PPMS）。奥瑞利珠单抗是一种靶向CD20-阳性B细胞的人源化单克隆抗体。2、3月31日，NMPA官网显示，百时美施贵宝的纳武利尤单抗与伊匹木单抗联合疗法获批新适应症，用于不可切除或晚期肝细胞癌(HCC)成人患者的一线治疗，成为中国首个且目前唯一获批的肝细胞癌一线双免疫联合疗法。纳武利尤单抗是全球首个获批的PD-1抑制剂，伊匹木单抗是一种细胞毒性T淋巴细胞抗原-4（CTLA-4）抑制剂。3、3月31日，NMPA官网显示，优时比的罗泽利昔珠单抗获批上市，预测用于治疗重症肌无力（gMG）。该药是一款靶向FcRn的单抗，最早于2023年6月在美国获批，优时比还在开发罗泽利昔珠单抗对于纤维肌痛、MOG抗体相关疾病等适应症的应用潜力。国内已有一款FcRn单抗获批上市，即再鼎医药的艾加莫德α。石药基团的巴托利单抗已申报上市，正在审评中。4、3月31日，NMPA官网显示，参天公司5.1类化药他氟噻吗滴眼液获批上市，适应症为降低开角型青光眼或高眼压症患者的眼压，用于对局部单独使用β-受体阻滞剂或前列腺素衍生物疗效不佳而需要联合治疗，以及对使用无防腐剂滴眼液可能获益的患者。他氟噻吗滴眼液是一款含有青光眼一线治疗药物前列腺素（PG）衍生物，同时不含防腐剂的眼用复方制剂。5、3月31日，NMPA官网显示，恒瑞医药的硫酸艾玛昔替尼（SHR0302片）新适应症获批，用于治疗对一种或多种传统合成改善病情抗风湿药（csDMARDs）疗效不佳或不耐受的成人中重度活动性类风湿关节炎患者。艾玛昔替尼是恒瑞医药自主研发的高选择性的JAK1抑制剂，首次在中国获批上市，用于治疗活动性强直性脊柱炎成人患者。申请6、4月1日，CDE官网显示，永泰生物1类新药爱可仑赛注射液递交附条件新药上市申请，这是永泰生物在研的广谱性的细胞免疫治疗产品（EAL），于2025年3月正式被CDE纳入优先审评，拟定适应症为：原发性肝细胞癌根治术后高复发风险的部分人群［免疫特性人群：患者需满足以下标准中的至少两项：无微血管侵犯；术后中性粒细胞和淋巴细胞比值（NLR）＜3；术后血小板和淋巴细胞比值（PLR）＜110］术后预防复发治疗。7、4月1日，CDE官网显示，华东医药的司美格鲁肽注射液生物类似药（研发代码：HDG1901）申报上市，用于成人2型糖尿病患者的血糖控制。司美格鲁肽注射液是一种长效胰高血糖素样肽-1（GLP-1）受体激动剂，与人GLP-1有94%的序列同源性。原研产品由诺和诺德（Novo Nordisk）研发（商品名：诺和泰），已经在全球范围内获批用于2型糖尿病患者的血糖控制、肥胖或超重患者的长期体重管理、降低心血管事件风险等适应症。8、4月3日，CDE官网显示，第一三共的注射用德曲妥珠单抗新适应症申报上市。德曲妥珠单抗（商品名：Enhertu）是一款靶向HER2的抗体偶联药物（ADC），由阿斯利康和第一三共联合开发，此前已经在中国获批4项适应症。德曲妥珠单抗治疗HER2表达实体瘤适应症正在中国处于上市申报阶段，这是一类“不限癌种”的适应症。9、4月3日，CDE官网显示，默沙东的Clesrovimab注射液申报上市，预测适应症为：用于即将进入或出生在第一个呼吸道合胞病毒（RSV）流行季的新生儿和婴儿预防RSV所致的下呼吸道感染。Clesrovimab（MK-1654）是一种在研的半衰期延长的单克隆抗体，可作为被动免疫手段，用于预防RSV疾病。10、4月3日，CDE官网显示，阿斯利康的本瑞利珠单抗注射液新适应症申报上市。本瑞利珠单抗（Benralizumab）是阿斯利康开发的一款靶向IL-5受体的单克隆抗体，此前已经在中国获批用于成人和12岁及以上青少年重度嗜酸粒细胞性哮喘的维持治疗。在美国，该产品还已经获FDA批准用于治疗嗜酸性肉芽肿性多血管炎（EGPA）的成年患者。临床批准 11、3月31日，CDE官网显示，中美瑞康（Ractigen Therapeutics）的RAG-1C获批临床，用于治疗增殖性玻璃体视网膜病变（PVR）。RAG-1C是中美瑞康第二款进入临床的saRNA药物。此前，该公司的首款saRNA药物RAG-01已获得美国FDA的IND许可，并在澳大利亚开展的Ⅰ期临床试验中获得了积极的初步疗效和安全性数据。12、4月1日，CDE官网显示，恒瑞医药1类新药HRS-6719片获批临床，拟开发治疗甲硫腺苷磷酸化酶（MTAP）缺失的实体瘤。本次为该产品首次在中国获批临床。PRMT5是MTAP突变的“合成致死”靶点。全球范围内已有数十款PRMT5抑制剂进入临床开发阶段，进展较快的产品已经进入Ⅱ期临床阶段。13、4月1日，CDE官网显示，海博为药业1类新药HBW-012336胶囊获批临床，拟开发治疗携带KRAS G12D突变的局部晚期或转移性实体瘤。这是一款KRAS G12D抑制剂。本次为该产品首次在中国获批临床。除了对KRAS G12D 抑制剂的开发，海博为药业还布局了口服Pan-KRAS抑制剂研发管线，已筛选出具有显著开发潜力的候选化合物HBW-016-K。14、4月2日，CDE官网显示，长春高新1类新药GenSci128片获批临床，拟开发治疗携带TP53 Y220C突变的局部晚期或转移性实体瘤。这是一款是针对TP53 Y220C突变的选择性重激活剂。本次为该产品首次在中国获批IND。15、4月2日，CDE官网显示，阿斯利康1类新药AZD5492获批两项临床，拟用于治疗系统性红斑狼疮、特发性炎症性肌病。这是一款CD20×TCR×CD8三特异性抗体，是由CD8引导的T细胞衔接器（TCE）。该产品最早于2024年10月在中国获批临床，适应症为复发性或难治性B细胞恶性肿瘤。16、4月3日，CDE官网显示，三生国健1类新药SSGJ-627注射液获批临床，拟开发治疗溃疡性结肠炎（UC）。这是一款抗TL1A单抗。本次是该产品首次在中国获批临床。SSGJ-627是三生国健研发的重组抗TL1A人源化单克隆抗体。FDA上市批准17、4月3日，FDA官网显示，诺华的阿曲生坦（Atrasentan、商品名：Vanrafia）获加速批准，用于降低有疾病进展风险的原发性免疫球蛋白A肾病（IgAN）成人患者的蛋白尿。阿曲生坦成为了首个获批用于减少原发性IgA肾病蛋白尿的选择性ETA拮抗剂。2023年，诺华以35亿美元收购了Chinook，获得了atrasentan和APRIL单抗zigakibart两款IgA肾病药物。申请18、3月31日，FDA官网显示，大冢制药的Sibeprenlimab递交生物制品许可申请（BLA），用于治疗免疫球蛋白A肾病（IgAN）。Sibeprenlimab是Visterra开发的一款靶向抑制增殖诱导配体（APRIL）的单抗。目前，全球仅Nefecon（16mg、每日1次）、Sparsentan（片剂、200/400mg、每日1次）和伊普可泮（胶囊、200mg、每日2次）三款药物获批用于治疗IgAN。EMA上市批准19、4月1日，EMA官网显示，辉瑞的二价呼吸道合胞病毒（RSV）疫苗（商品名：Abrysvo）新适应症获批，扩展至预防18至59岁人群由RSV引起的下呼吸道疾病（LRTD），进一步扩大了原有针对60岁及以上人群的适应症，使Abrysvo成为欧盟目前覆盖范围最广的RSV疫苗，Abrysvo是一种无佐剂二价疫苗，旨在预防RSV-A和RSV-B亚群引起的下呼吸道疾病。研发临床状态20、3月31日，药物临床试验登记与信息公示平台显示，恒瑞医药启动了GLP-1受体激动剂HRS-7535片用于超重或肥胖受试者的Ⅲ期临床研究。2024年5月，恒瑞将包括HRS-7535在内的GLP-1产品组合以NewCo的形式授权出海，获得了首付款和近期里程碑总计1.1亿美元，累计不超过59.25亿美元的的临床开发、监管和销售里程碑款，Hercules19.9%的股权。临床数据21、4月2日，罗氏公布了奥瑞利珠单抗治疗复发性多发性硬化症（RMS）的Ⅲ期MUSETTE结果，显示未达到主要终点。该研究是一项多中心、随机、双盲、阳性药物对照临床试验（n=864），评估了高剂量奥瑞利珠单抗（1200mg或1800mg，静脉注射，每24周1次）对比已获批剂量奥瑞利珠单抗（600mg，静脉注射，每24周1次）治疗RMS成人患者的有效性、安全性和药代动力学（PK）。该药是罗氏与渤健合作开发的一款靶向CD20阳性B细胞的人源化单抗。22、4月2日，复宏汉霖公布了HLX04-O治疗湿性年龄相关性黄斑变性（wAMD）中国患者的Ⅲ期HLX04-O-wAMD-CN研究结果，已达到预设主要研究终点。该研究为一项多中心、随机、双盲、阳性对照的非劣效Ⅲ期临床试验（n=388），旨在评估玻璃体内注射（IVT）HLX04-O（1.25mg，每4周1次）或雷珠单抗（0.5mg，每4周1次）治疗wAMD患者的有效性和安全性。HLX04-O是复宏汉霖利用基因工程技术构建的一款重组抗血管内皮生长因子（VEGF）人源化单抗。交易及投融资23、4月1日，和誉医药宣布，与默克签订授权协议，行使匹米替尼全球商业化选择权，行权费用为8500万美元。2023年12月，和誉医药与默克就CSF-1R抑制剂匹米替尼订立协议。默克最初获得匹米替尼在中国内地、中国香港、中国澳门及中国台湾所有适应症进行商业化的独家授权，并拥有全球商业化权利的独家选择权。匹米替尼是由和誉医药独立研发的一种新型、口服、高选择性且高效的小分子CSF-1R抑制剂。其他24、2024年，2024年全球销售额超过10亿美元的药物约169款，TOP100药品的上榜门槛是17.66亿美元（折合人民币约125.77亿元），合计销售总收入为5049.17亿美元。2024年，K药以294.82亿美元排名第一，GLP-1巨头司美格鲁肽三款产品Wegovy/Ozempic/Rybelsus合计创收292.96亿美元排名第二。END2025金笔奖征文活动开启，来投稿吧！领取CPHI & PMEC China 2025展会门票智药研习社直播预告来源：CPHI制药在线声明：本文仅代表作者观点，并不代表制药在线立场。本网站内容仅出于传递更多信息之目的。如需转载，请务必注明文章来源和作者。投稿邮箱：Kelly.Xiao@imsinoexpo.com▼更多制药资讯，请关注CPHI制药在线▼点击阅读原文，进入智药研习社~

上市批准申请上市免疫疗法临床3期临床申请

2025-04-03

·FiercePharma

Vanrafia, 2nd drug in Novartis' IgAN troika, crosses FDA finish line in payday for Chinook investors

Novartis has positioned Vanrafia as a “foundational therapy” in IgAN because it can address a broad population of patients with persistent proteinuria, the company’s U.S. president, Victor Bulto, told Fierce Pharma. When Novartis bought kidney disease biotech Chinook Therapeutics in 2023 for $3.2 billion upfront, the deal included a conditional payment tied to the timely FDA approval of atrasentan without a specific safety restriction.Now, investors in Chinook can claim an additional $2-per-share payout, or about $160 million in total, as the milestone has been met.The FDA has granted an accelerated approval for atrasentan to reduce elevated protein in the urine in adults with primary immunoglobulin A nephropathy (IgAN) at risk of rapid progression, Novartis said Wednesday.Bearing the commercial moniker Vanrafia, the selective endothelin A receptor antagonist (ERA) does not have to be used under an FDA-mandated safety-related program called REMS. A sans-REMS FDA approval for the drug in IgAN before the end of 2025 was needed to trigger a $2-per-share contingent value right (CVR) payment under Novartis’ merger agreement with Chinook.The special CVR arrangement highlights how important having a non-REMS label is for Novartis.The lack of a REMS requirement sets Vanrafia apart from Travere Therapeutics’ Filspari— which acts as both an ERA and an angiotensin receptor blocker (ARB)—for now. Filspari got its IgAN nod in 2023 and is only available through a REMS program mainly because of a potential risk of liver toxicity, which is also reflected as a boxed warning item on the drug’s label.However, a step ahead of Novartis, Travere last fall turned Filspari’s accelerated approval into a full go-ahead based on evidence that it can preserve kidney function. So far, Travere said the REMS program does not appear to pose a barrier for Filspari patients. The drug’s 2024 sales also suggested a good launch progress, with its $132 million beating analysts’ consensus estimates. However, as Leerink Partners analysts pointed out in a March note, “the question is, how many more patients are out there that might be a candidate with a looser REMS?”Both Novartis and Travere are trying to level the playing field against each other.For Travere, the biotech has submitted an application to the FDA to potentially reduce the REMS liver monitoring requirement from monthly testing to quarterly in the first year. The FDA has set a decision date of Aug. 28 this year.For Novartis, the company is collecting longer-term data for Vanrafia in the hopes of also showing a kidney function benefit to support a traditional approval.Novartis has positioned Vanrafia as a “foundational therapy” in IgAN because it can address a broad population of patients with persistent proteinuria, the company’s U.S. president, Victor Bulto, said during a recent interview with Fierce Pharma.In the phase 3 Align trial, Vanrafia led to a 36% reduction in proteinuria as measured by the urine protein-to-creatinine ratio (UPCR) compared with placebo after 36 weeks of treatment. For Travere's Filspari, the result was a 35% reduction in UPCR at week 36 versus control, in its own IgAN trial called Protect.Because the two meds feature different mechanisms, their trials were designed differently. While Filspari was pitted directly against the ARB drug irbesartan, Vanrafia was tested against placebo in patients who were on maximally tolerated renin angiotensin system inhibitor, such as an ARB.As Filspari is a drug with dual mechanisms, it offers the convenience of a single drug, whereas Vanrafia is used on top of other agents. Novartis has argued that separate dosing allows doctors to adjust the dosage of each drug component, although Travere has contended that titration is more of an academic question than a real-world practice.Vanrafia is Novartis’ second IgAN drug to reach the market. Before that, the company’s complement inhibitor Fabhalta expanded into IgAN in August 2024. Another Chinook agent, the anti-APRIL antibody zigakibart, is also being developed for the rare kidney disease.With Fabhalta’s launch, Novartis is targeting patients with residual inflammation and activation of the complement pathway, Bulto said. This subgroup represents about 15% to 30% of at-risk patients with persistent proteinuria who are typically eligible for novel treatments.By comparison, Novartis believes Vanrafia can be used in all 45,000 U.S. IgAN patients potentially eligible for new therapies.“What we do fully expect is that this is going to be a combination market, given that heterogeneity [of disease manifestation], and that’s why we want to bring all the different tools that physicians may need to treat this disease,” Bulto said. In addition to IgAN, both Novartis and Travere are targeting other rare kidney diseases.Fabhalta a few days ago became the first FDA-approved agent for complement 3 glomerulopathy (C3G). Travere has recently filed Filspari for focal segmental glomeruloscelrosis (FSGS) despite missing the primary endpoint of a phase 3 trial.Novartis believes its commercial prowess and broad offerings may give it an edge.“By constructing the portfolio like this, like a multi-indication, multi-asset, it also creates compounding capabilities and expertise,” Bulto said.There are about 9,000 nephrologists in the U.S. who each treat about three IgAN patients and one C3G patient, so deploying a commercial force “for a single asset is a very difficult task,” the Novartis exec noted.“That’s why we just want to double down here in the space,” Bulto said. “I think that, in itself, will create a good competitive differentiation, which ultimately will help patients.”

上市批准临床结果临床3期并购免疫疗法

100 项与 Zigakibart 相关的药物交易

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适应症	最高研发状态	国家/地区	公司	日期
慢性肾病	临床3期	-	Novartis Pharmaceuticals Canada, Inc.	2025-07-28
肾小球疾病	临床3期	-	Novartis Pharmaceuticals Canada, Inc.	2025-07-28
免疫球蛋白沉积病	临床3期	-	Novartis Pharmaceuticals Canada, Inc.	2025-07-28
免疫球蛋白a肾病	临床3期	中国	Novartis Pharmaceuticals Canada, Inc.	2023-07-06
免疫球蛋白a肾病	临床3期	日本	Novartis Pharmaceuticals Canada, Inc.	2023-07-06
免疫球蛋白a肾病	临床3期	阿根廷	Novartis Pharmaceuticals Canada, Inc.	2023-07-06
免疫球蛋白a肾病	临床3期	澳大利亚	Novartis Pharmaceuticals Canada, Inc.	2023-07-06
免疫球蛋白a肾病	临床3期	比利时	Novartis Pharmaceuticals Canada, Inc.	2023-07-06
免疫球蛋白a肾病	临床3期	巴西	Novartis Pharmaceuticals Canada, Inc.	2023-07-06
免疫球蛋白a肾病	临床3期	加拿大	Novartis Pharmaceuticals Canada, Inc.	2023-07-06

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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


NCT03945318 (ADU-CL-19, ISN WCN2023)	临床1/2期	免疫球蛋白a肾病 APRIL \| IgA \| Gd-IgA1 ... 更多	-	BION-1301 450 mg IV	艱鑰築膚廠範鬱鑰艱顧(膚獵夢網觸獵衊鹽壓製) = 遞廠襯製繭構餘願鏇積襯鹽鑰鬱簾鏇選夢衊淵 (醖選襯餘構壓積範壓廠 )	积极	2023-03-01
NCT03945318 (ADU-CL-19, ISN WCN2023)	临床1/2期	免疫球蛋白a肾病 APRIL \| IgA \| Gd-IgA1 ... 更多	-	BION-1301 600 mg SC	艱鑰築膚廠範鬱鑰艱顧(膚獵夢網觸獵衊鹽壓製) = 鹽鏇夢鑰繭窪鑰窪選鬱襯鹽鑰鬱簾鏇選夢衊淵 (醖選襯餘構壓積範壓廠 )	积极	2023-03-01
NCT03945318 (Corporate Publications) 人工标引	临床1/2期	免疫球蛋白a肾病	40	BION-1301 (450 mg Q2W IV → 600 mg Q2W SC, up to 104 weeks ‖)	製製網襯顧築築範糧網(繭積範襯襯鏇築範觸鏇) = 網鹽繭遞範餘鏇範膚構願襯鑰壓遞醖蓋獵壓製 (窪膚製構壓願糧簾憲衊 ) 更多	积极	2022-11-01
NCT03945318 (Corporate Publications) 人工标引	临床1/2期	免疫球蛋白a肾病	40	BION-1301 (600 mg Q2W de novo SC, up to 104 weeks)	製製網襯顧築築範糧網(淵積糧餘鹽鬱廠遞鬱淵) = 壓衊顧鹽衊製醖鏇範鬱淵鏇顧選鹽齋鬱範遞憲 (積積鑰鏇願繭膚獵鬱獵 )	积极	2022-11-01
NCT03945318 (Corporate Publications) 人工标引	临床1/2期	免疫球蛋白a肾病	10	BION-1301	鑰餘積構選糧願艱鹹醖(願鏇選淵築膚醖顧衊衊) = 醖網顧願夢積醖醖齋鑰範蓋窪鹽醖鹽簾願衊鬱 (鑰顧齋鏇鬱觸齋網獵繭 ) 更多	积极	2021-11-04
NCT03340883 (CTgov)	临床1/2期	复发性多发性骨髓瘤 \| 难治性多发性骨髓瘤	21	BION-1301 (BION-1301 50 mg Q2W)	選選鹹積鏇蓋構獵艱繭 = 醖蓋鑰獵遞襯鬱鬱醖鑰鏇壓襯願顧獵蓋鹽製範 (獵蓋憲鏇衊網窪齋窪衊, 願遞廠網遞壓製簾壓遞 ~ 觸積鏇範鏇繭築壓鹹繭) 更多	-	2020-09-07
NCT03340883 (CTgov)	临床1/2期	复发性多发性骨髓瘤 \| 难治性多发性骨髓瘤	21	BION-1301 (BION-1301 150 mg Q2W)	選選鹹積鏇蓋構獵艱繭 = 壓築築淵鹹顧壓遞鹹齋鏇壓襯願顧獵蓋鹽製範 (獵蓋憲鏇衊網窪齋窪衊, 獵廠顧蓋夢蓋鏇築鏇蓋 ~ 齋糧鏇艱遞壓鏇願願糧) 更多	-	2020-09-07
NCT03340883 (ASCO 12019 \| ASCO 22019) 人工标引	临床1期	难治性多发性骨髓瘤	15	Zigakibart	醖選鹽廠遞艱蓋窪鏇膚(鹽願膚構壓艱壓鏇襯鹹) = none 選齋齋鏇獵餘選淵夢顧 (選齋淵選醖鹽夢鹹網鏇 ) 更多	不佳	2019-06-03
BION-1301 (ASN2018)	N/A	免疫球蛋白a肾病 APRIL \| BCMA \| TACI	-	BION-1301	膚蓋齋鬱淵餘窪壓蓋夢(蓋壓壓製顧鹹餘鹹鹽齋) = 鹹膚膚遞獵鹽積選夢糧網鹽顧餘鏇膚簾襯鏇襯 (鹽憲艱鹹糧鑰淵遞製醖, 0.89) 更多	积极	2018-10-23