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更新于：2025-06-25

Cemdisiran

更新于：2025-06-25

概要

概要

基本信息

药物类型

siRNA

别名

AD-62643、ALN-62643、ALN-CC5

靶点

作用方式

抑制剂

作用机制

C5抑制剂(补体C5抑制剂)

治疗领域

肿瘤免疫系统疾病神经系统疾病+ [6]

在研适应症

年龄相关性黄斑变性地图样萎缩阵发性睡眠性血红蛋白尿症+ [4]

非在研适应症

非典型溶血性尿毒症综合征

原研机构

Alnylam Pharmaceuticals, Inc.

在研机构

Regeneron Pharmaceuticals, Inc.

Curia, Inc

再鼎医药（上海）有限公司

+ [1]

非在研机构

Curiato, Inc.

权益机构

Regeneron Pharmaceuticals, Inc.

Alnylam Pharmaceuticals, Inc.

最高研发阶段临床3期

首次获批日期-

最高研发阶段(中国)临床3期

特殊审评孤儿药 (美国)、孤儿药 (欧盟)

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结构/序列

使用我们的RNA技术数据为新药研发加速。

或

查看完整样例数据

Sequence Code 32355856

来源: *****

Sequence Code 1103240105

来源: *****

关联

项与 Cemdisiran 相关的临床试验

NCT06541704

/ Recruiting临床3期

A Multicenter, Randomized, Double-Masked, Placebo-Controlled Phase 3 Study of the Efficacy, Safety, and Tolerability of Subcutaneously Administered Pozelimab in Combination With Cemdisiran or Cemdisiran Alone in Participants With Geographic Atrophy Secondary to Age-Related Macular Degeneration

This study is researching experimental (study) drugs called pozelimab and cemdisiran. The study is focused on participants who have geographic atrophy (GA) caused by age-related macular degeneration (AMD). Geographic atrophy is a medical term that refers to later-stage cases of AMD which is an eye condition affecting central vision (what one sees straight ahead).
The purpose of this study is to evaluate the progression rate of Geographic Atrophy in eyes of patients treated with cemdisiran alone or in combination with pozelimab compared to those treated with placebo.
The study is looking at several other research questions, including:
* What side effects may happen from taking the study drug(s)
* How much study drug(s) are in the blood at different times
* Whether the body makes antibodies against the study drug(s) (which could make the study drug(s) less effective or could lead to side effects)

开始日期2024-10-30

申办/合作机构

Regeneron Pharmaceuticals, Inc.

NCT06479863

/ Recruiting早期临床1期IIT

Efficacy and Safety of Pozelimab and Cemdisiran Combination Therapy in Patients With Sporadic Inclusion Body Myositis

To evaluate the efficacy of Pozelimab/Cemdisiran combination therapy in patients with sIBM

开始日期2024-08-08

申办/合作机构

Regeneron Pharmaceuticals, Inc.

NCT05131204

/ Terminated临床3期

A Randomized, Open-Label, Eculizumab and Ravulizumab Controlled Study to Evaluate the Efficacy and Safety of Pozelimab and Cemdisiran Combination Therapy in Patients With Paroxysmal Nocturnal Hemoglobinuria Who Are Currently Treated With Eculizumab or Ravulizumab

The primary objective of the study is:
To evaluate the effect of pozelimab and cemdisiran combination therapy on hemolysis, as assessed by lactate dehydrogenase (LDH), after 36 weeks of treatment, in patients with PNH who switch from eculizumab or ravulizumab therapy versus patients who continue their eculizumab or ravulizumab therapy
The secondary objectives of the study are to:
* Evaluate the effect of pozelimab and cemdisiran combination treatment versus anti-C5 standard-of-care treatment (eculizumab or ravulizumab) on the following:
* Transfusion requirements and transfusion parameters
* Measures of hemolysis: LDH control, breakthrough hemolysis, and inhibition of CH50
* Hemoglobin levels
* Fatigue as assessed by Clinical Outcome Assessments (COAs)
* Health-related quality of life (HRQoL) as assessed by COAs
* Safety and tolerability
* To assess the concentrations of total pozelimab and either total eculizumab or total ravulizumab in serum and total cemdisiran and total C5 protein in plasma
* To assess the immunogenicity of pozelimab and cemdisiran

开始日期2022-10-06

申办/合作机构

Regeneron Pharmaceuticals, Inc.

100 项与 Cemdisiran 相关的临床结果

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100 项与 Cemdisiran 相关的转化医学

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100 项与 Cemdisiran 相关的专利（医药）

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项与 Cemdisiran 相关的文献（医药）

2024-05-01·International journal of laboratory hematology

Complement inhibition in paroxysmal nocturnal hemoglobinuria: From biology to therapy

Review

作者: Fattizzo, Bruno ; Versino, Francesco

Abstract:

Complement inhibitors are the mainstay of paroxysmal nocturnal hemoglobinuria (PNH) treatment. The anti‐C5 monoclonal antibody eculizumab was the first treatment to improve hemolysis, thrombotic risk, and survival in PNH although at the price of a life‐long intravenous fortnightly drug. Additionally, suboptimal response may occur in up to 2/3 of patients with persistent anemia due to incomplete control of intravascular hemolysis, development of upstream C3‐mediated extravascular hemolysis (EVH), or concomitant bone marrow failure. Ravulizumab, a longer half‐life anti‐C5 developed from eculizumab, administered every 8 weeks, improved patient convenience, and reduced pharmacokinetic breakthrough hemolysis (BTH) by establishing more stable anti‐C5 concentrations. More recently, several other anti‐C5 compounds (crovalimab, pozelimab, tesidolumab, cemdisiran, zilucoplan, and coversin) are on study in clinical trials. Upstream inhibition of complement cascade was also explored with the anti‐C3 pegcetacoplan, and with the alternative pathway inhibitors iptacopan (anti‐factor B) and danicopan (anti‐factor D). These drugs efficiently target EVH and are able to improve anemia and transfusion need in suboptimal responders to anti‐C5. The route and schedule of administration (twice weekly subcutaneously for pegcetacoplan and twice or thrice oral daily dosing for iptacopan and danicopan, respectively) are very convenient but pose novel issues regarding adherence. Additionally, both anti‐C5 and upstream inhibitors do not resolve the unmet need of pharmacodynamic BTH events due to complement amplifying conditions such as infections, traumas, and surgery. In this review, we will recapitulate PNH physiopathology, clinical presentation, and diagnosis and describe available and developing drugs that will lead to a precision medicine approach for this rare though heterogenous disease.

2024-04-01·Clinical journal of the American Society of Nephrology : CJASN

Phase 2 Trial of Cemdisiran in Adult Patients with IgA Nephropathy: A Randomized Controlled Trial

Article

作者: Bhan, Ishir ; Wang, Dazhe ; Villanueva, Russell ; Fernström, Anders ; Badri, Prajakta ; Barbour, Sean J. ; Borodovsky, Anna ; Cattran, Daniel ; Sperati, C. John ; Liew, Adrian ; Wu, Ming-Ju ; Yureneva, Elena ; Barratt, Jonathan ; Yeo, See Cheng

Background:

IgA nephropathy is the most common primary GN. Clinical features of IgA nephropathy include proteinuria, which is the strongest known surrogate of progression to kidney failure. Complement pathway activation is a critical driver of inflammation and tissue injury in IgA nephropathy. Cemdisiran is an investigational RNA interference therapeutic that suppresses hepatic production of complement component 5 (C5), thereby potentially reducing proteinuria in IgA nephropathy. We evaluated the efficacy and safety of cemdisiran in adult patients with IgA nephropathy at high risk of kidney disease progression.

Methods:

In this phase 2, 36-week, double-blind study, adult patients with IgA nephropathy and urine protein ≥1 g/24 hours were randomized (2:1) to subcutaneous cemdisiran 600 mg or placebo every 4 weeks in combination with the standard of care. The primary end point was percentage change from baseline at week 32 in urine protein-to-creatinine ratio (UPCR) measured by 24-hour urine collection. Additional end points included change from baseline in UPCR measured by spot urine, serum C5 level, and safety assessments.

Results:

Thirty-one patients were randomized (cemdisiran, N=22; placebo, N=9). Cemdisiran-treated patients had a placebo-adjusted geometric mean change in 24-hour UPCR of –37.4% (cemdisiran-adjusted geometric mean ratio to baseline [SEM], 0.69 [0.10]) at week 32. Spot UPCR was consistent with 24-hour UPCR placebo-adjusted change of –45.8% (cemdisiran-adjusted geometric mean ratio to baseline [SEM], 0.73 [0.11]). Mean (SD) change in serum C5 level from baseline at week 32 was –98.7% (1.2) with cemdisiran and 25.2% (57.7) with placebo. Over 36 weeks, most adverse events were mild or moderate and transient; the most common adverse event after cemdisiran treatment was injection-site reaction (41%).

Conclusions:

These findings indicate that treatment with cemdisiran resulted in a reduction of proteinuria at week 32 and was well tolerated.

2023-12-04·Clinical kidney journal

Targeting complement in IgA nephropathy

Review

作者: Gutiérrez, Eduardo ; Caravaca-Fontán, Fernando ; Sevillano, Ángel M ; Praga, Manuel

ABSTRACT:

Immunoglobulin A nephropathy (IgAN) is the most common primary glomerulonephritis worldwide. Recent years have witnessed significant improvements in the understanding of the pathogenesis of IgAN and particularly, the pathogenic role of complement activation. The alternative complement pathway is the major complement cascade activator in IgAN, and glomerular C3 deposition has been shown to correlate with disease progression. In addition, several studies have provided insight into the pathogenic role of factor H–related proteins -1 and -5 in IgAN, as independent players in complement dysregulation. The lectin pathway has also been shown to be associated with the severity of IgAN. Glomerular deposition of C4d has been associated with increased histologic disease activity, faster decline in estimated glomerular filtration rate and higher risk of kidney failure. On the other hand, although overlooked in the Oxford classification, numerous studies have shown that the coexistence of thrombotic microangiopathy in IgAN is a significant indicator of a poorer prognosis. All the breakthroughs in the understanding of the contributing role of complement in IgAN have paved the way for the development of new complement-targeted therapies in this disease. Several ongoing trials are evaluating the efficacy of new agents against factor B (iptacopan, Ionis-FB-LRX), C3 (pegcetacoplan), factor D (vemircopan, pelecopan), C5 (ravulizumab, cemdisiran) and C5a receptor 1 (avacopan). In this study, we provide a comprehensive review of the role of complement in IgAN, including the emerging mechanisms of complement activation and the promising potential of complement inhibitors as a viable treatment option for IgAN.

项与 Cemdisiran 相关的新闻（医药）

2025-06-17

·医药观澜

寻找“治愈”曙光：7款新药已获批上市，下一代重症肌无力疗法有望迎来新突破

编者按：刚刚过去的“全球重症肌无力关爱日”再次提醒大家关注这一罕见疾病。重症肌无力（MG）是一种罕见的神经肌肉-自身免疫性疾病，会导致患者虚弱和可能危及生命的肌无力。目前该疾病尚无治愈方法。随着研究进展和产业持续投入，近十年来多款针对重症肌无力的创新药获监管机构批准上市，极大改善了患者的生活。与此同时，全球更多MG新药管线已进入临床阶段，涵盖抗体疗法、siRNA药物、小分子、CAR-T疗法等多种类型，有望在不远的未来迎来新突破。作为全球医药及生命科学行业创新的赋能者，药明康德也持续通过一体化、端到端的CRDMO平台，为全球合作伙伴提供赋能，助力重症肌无力等罕见肌肉疾病新药的开发，为患者带来更多突破性的治疗方案。回顾2025年上半年，重症肌无力疾病领域新药再次迎来多项新进展。5月，强生公司（Johnson & Johnson）宣布，美国FDA已批准其抗体疗法Imaavy（nipocalimab）上市，用于治疗抗AChR、抗MuSK抗体阳性的全身性重症肌无力（gMG）成人与12岁以上儿童患者。这是FDA批准的首个用于治疗该类患者群体的新生儿Fc受体（FcRn）阻断剂。这款疗法还被Evaluate列为今年有望上市的10大潜在重磅疗法之一。同月，翰森制药宣布其伊奈利珠单抗（inebilizumab）的新适应症上市申请获得中国NMPA受理，用于治疗全身性重症肌无力成人患者。这是一款靶向CD19 B细胞消耗性抗体，由翰森制药和安进（Amgen）共同开发。该药物的全球关键性3期试验MINT的结果于今年4月发表于《新英格兰医学杂志》。同样是5月，荣昌生物的BLyS/APRIL双靶点融合蛋白创新药物泰它西普在中国获批新适应症，用于治疗全身性重症肌无力成人患者。该产品可同时靶向B细胞激活因子（又称BLyS）和增殖诱导配体（APRIL），针对致病性抗体产生的源头——B细胞及浆细胞。此外，在CAR-T疗法领域，Cartesian Therapeutics于今年4月公布其在研mRNA细胞疗法Descartes-08用于治疗gMG的2b期研究的12个月数据。该研究早前已经取得积极结果，80%的可评估患者维持了临床显著应答达1年。Descartes-08是一款靶向BCMA的在研CAR-T细胞疗法，目前已启动3期临床研究，是MG领域目前研发进度较快的CAR-T细胞疗法，有望成为MG治疗领域首个细胞疗法。从这些进展可以看到，在重症肌无力治疗药物研发领域，2025年正在迎来多元化的新突破。图片来源：123RF过去十年间，至少7款重症肌无力新药获批上市重症肌无力是一种慢性的罕见自身免疫疾病，目前尚无治愈方法。该病的致病机制涉及患者体内的免疫球蛋白G（IgG）抗体，这些抗体会破坏神经与肌肉之间的突触传递，导致肌肉疲劳无力，进而导致极度疲劳和面部表情、言语、吞咽和活动困难，严重时甚至危及生命。大约85%的重症肌无力患者属于全身性重症肌无力（gMG）。根据2021年文献数据，gMG的患病率和发病率在全球范围内正在增加，全球患病率估计为每10万人中有2~36例。过去十年间，全球至少有7款重症肌无力新药获批上市，包括FcRn拮抗剂、补体抑制剂和B细胞耗竭剂等，为MG患者带来了更多治疗选择。C5补体抑制剂包括抗体和大环肽类药物，比如阿斯利康的ravulizumab（瑞利珠单抗）、eculizumab（依库珠单抗），以及优时比公司的大环肽类C5补体抑制剂zilucoplan（泽勒普肽）等，这类药物能够减少补体激活介导的神经肌肉接头损伤；FcRn抑制剂类药物以抗体为主，比如开头提到的强生公司的nipocalimab（尼卡利单抗），以及优时比公司的rozanolixizumab（罗泽利昔珠单抗）、Argenx和再鼎医药共同开发的efgartigimod（艾加莫德）等，它们可降低循环IgG抗体水平，从而减少致病抗体的循环，改善MG症状；B细胞耗竭剂类的代表药物为荣昌生物研发的BLyS/APRIL双靶点融合蛋白泰它西普，针对致病性抗体产生的源头——B细胞及浆细胞，减少致病性自身抗体的产生，发挥治疗作用。寻找“治愈”曙光，下一代重症肌无力疗法有望迎来新突破梳理公开资料可知，除了已获批的新药，全球范围内还有数十款新药正在临床阶段探索用于重症肌无力治疗的潜力，未来有望在多个方向取得新突破。在重症肌无力临床管线中，抗体类药物数量较多，包含单抗、双抗，以及一些抗体融合蛋白等，靶点涉及C5、FcRn、CD19/CD20等。例如阿斯利康在研的第三代C5补体抑制剂抗体gefurulimab已经进入3期临床阶段，其分子量小，具更好的渗透性，有望开发成为一款每周一次、由患者自行给药的皮下注射疗法。再如开头提到的抗CD19单抗伊奈利珠单抗，其翰森制药提交的针对重症肌无力的上市申请已获得中国NMPA受理，安进在两个月前也宣布了该产品治疗gMG成年患者的疗效与安全性的最新临床数据：每年两次给药的情况下，其在乙酰胆碱受体自身抗体阳性（AChR+）gMG患者中具有持久的疗效。CAR-T疗法也在临床研究中展现出“治愈”重症肌无力的潜力。CAR-T疗法近年来在自身免疫性疾病领域进展迅速，其可以使患者实现长期无药物缓解，被认为有望对重症肌无力的治疗产生潜在变革作用。在重症肌无力临床管线中，包括了十多款CAR-T疗法。这些在研管线以靶向B细胞或浆细胞为主，主要包括BCMA和CD19。在MG临床研发管线中，也有多款小分子药物。例如诺华在研的补体B因子口服抑制剂iptacopan（伊普可泮）和BTK抑制剂remibrutinib（瑞米布替尼），目前均已进入重症肌无力治疗研究3期临床阶段。再如NMD Pharma在研的氯离子通道（ClC-1）抑制剂NMD670正处于重症肌无力2b期临床阶段，该产品作用机制是针对骨骼肌的非免疫调节。多肽和核酸药物研发管线也有新突破。泽勒普肽（zilucoplan）作为首个获FDA批准用于治疗重症肌无力的每日一次经皮下给药的新型大环肽类C5补体抑制剂，是肽类药物在这一治疗领域的重要突破。除此之外，siRNA疗法cemdisiran联合抗体疗法pozelimab治疗重症肌无力的临床研究已处于3期阶段。前者由Alnylam公司和再生元（Regeneron）联合开发，作为一种靶向C5补体的RNAi疗法，cemdisiran在进入细胞后通过与靶标mRNA结合，从而使mRNA降解，避免功能性蛋白产生。Pozelimab是再生元开发的新一代C5补体抑制剂，此前已获FDA批准用于CD55缺陷型蛋白丢失性肠病。再生元正在开发这一C5抑制剂“抗体+siRNA”组合疗法以治疗包括重症肌无力在内的补体介导的疾病。其中，cemdisiran可以减少C5的产生，pozelimab可以阻断剩余C5的功能，二者共同抑制终端补体活性，有望在较低剂量下达到完全、持续的C5抑制，减少给药频率，并且皮下给药方便临床应用。值得一提的是，cemdisiran使用的GalNAc递送技术，是当下核酸疗法尤其是siRNA递送到肝细胞的主要策略。迄今FDA批准的7款siRNA疗法中，6款均使用了这项技术。也得益于此类递送技术的持续革新，纵览全球，siRNA疗法研发蓬勃发展，在研管线已超过350款。在这一产业新兴的趋势下，围绕GalNAc等复杂分子，药明康德旗下专注于寡核苷酸和多肽及相关化学偶联药物的WuXi TIDES平台已打造了涵盖定制合成、工艺开发及生产的一站式服务平台，更好地满足全球合作伙伴的寡核苷酸及各类化学偶联物的研发需求。公开信息显示，在定制化GalNAc方面，WuXi TIDES团队已经生产过涵盖不同类型、总数超过100款的GalNAc化合物，覆盖不同类型的GalNAc分子；通过优化GalNAc树脂负载工艺，将固相合成的收率提高50%。同时，借助流动化学平台，加速高纯度GalNAc的规模化生产。在疾病的征程上，科学与技术探索的步伐从未停歇。作为创新的赋能者、客户信赖的合作伙伴以及全球健康产业的贡献者，药明康德也将持续以独特的CRDMO业务模式，助力全球合作伙伴开发包括重症肌无力在内的更多疾病领域的创新疗法，持续造福病患！参考资料：[1]Binks, S.N.M., Morse, I.M., Ashraghi, M. et al. Myasthenia gravis in 2025: five new things and four hopes for the future. J Neurol 272, 226 (2025). https://doi-org.libproxy1.nus.edu.sg/10.1007/s00415-025-12922-7[2]Dresser L., Wlodarski, R., Rezania, K., & Soliven, B. (2021). Myasthenia Gravis: Epidemiology, Pathophysiology and Clinical Manifestations. J Clin Med, 10(11):2235.[3]Bubuioc, A. M., Kudebayeva, A., Turuspekova, S., Lisnic, V., & Leone, M. A. (2021). The epidemiology of myasthenia gravis. Journal of Medicine and Life, 14(1):7-16.版权说明：本文欢迎个人转发至朋友圈，谢绝媒体或机构未经授权以任何形式转载至其他平台。转载授权或其他合作需求，请联系wuxi_media@wuxiapptec.com。免责声明：本文仅作信息交流之目的，文中观点不代表药明康德立场，亦不代表药明康德支持或反对文中观点。本文也不是治疗方案推荐。如需获得治疗方案指导，请前往正规医院就诊。

细胞疗法上市批准免疫疗法临床3期信使RNA

2025-03-18

·PRNewswire

EMPAVELI's Market Success Underscores Growing Demand for Complement Inhibitor Therapies | DelveInsight

EMPAVELI's unique mechanism of targeting C3 broadens its applicability beyond PNH, including other complement-driven diseases like geographic atrophy and immune complex-mediated diseases. With a high unmet need in these areas and limited competition, EMPAVELI's market is poised for steady growth. LAS VEGAS, March 18, 2025 /PRNewswire/ -- DelveInsight's " EMPAVELI Market Size, Forecast, and Market Insight Report" highlights the details around EMPAVELI, a complement inhibitor indicated to treat adult patients with PNH. The report provides product descriptions, patent details, and competitor products (marketed and emerging therapies) of EMPAVELI. 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]. Apellis Pharmaceutical's EMPAVELI (pegcetacoplan) Overview EMPAVELI (pegcetacoplan) is a targeted inhibitor of C3 designed to control excessive complement activation—a key immune process that can contribute to the development and progression of serious diseases. Pegcetacoplan binds to complement protein C3 and its activation fragment C3b, helping to regulate the cleavage of C3 and the production of downstream effectors involved in complement activation. In PNH, extravascular hemolysis (EVH) is driven by C3b opsonization, while intravascular hemolysis (IVH) results from the formation of the membrane attack complex (MAC). By acting early in the complement cascade, pegcetacoplan controls both C3b-driven EVH and terminal complement-mediated IVH. EMPAVELI is approved for treating adult patients with paroxysmal nocturnal hemoglobinuria (PNH). The US FDA has granted pegcetacoplan Priority Review and Fast Track Designation (FTD) for the treatment of PNH. EMPAVELI reported US net product revenue of $23.4 million in the fourth quarter and $98.1 million for the full year of 2024. Currently, EMPAVELI is being evaluated in different phases of development for C3G & IC-MPGN, HSCT-TMA, FSGS, and DGF. Learn more about EMPAVELI projected market size for PNH @ EMPAVELI Market Potential Paroxysmal nocturnal hemoglobinuria (PNH) is a rare condition characterized by a triad of symptoms: intravascular hemolysis, thromboembolic events, and cytopenia. The disease presents differently among patients, making it difficult to classify based on typical clinical patterns due to its unpredictable nature. In 2023, there were approximately 12,000 diagnosed cases of PNH in the 7MM, with projections suggesting this number could rise to around 13,000 by 2034. Until 2007, PNH was a life-threatening disease with no effective treatment for hemolysis and thrombosis, which were the primary causes of death. However, the introduction of the anti-C5 agent eculizumab over the past decade marked a major breakthrough, significantly reducing hemolysis, decreasing the need for transfusions, and lowering the risk of thrombosis. The current standard of care for PNH involves complement inhibition therapy, with FDA-approved drugs like SOLIRIS, ULTOMIRIS, EMPAVELI, FABHALTA, VOYDEYA, and PIASKY being considered the gold standard treatments. According to DelveInsight, the total PNH market size in the 7MM was valued at USD 1.4 billion in 2023 and is expected to grow to about USD 2.5 billion by 2034. This growth is driven by advances in understanding disease mechanisms, leading to improved diagnostics and therapeutics, along with an increasing number of cases and the introduction of new treatments during the forecast period. Discover more about the paroxysmal nocturnal hemoglobinuria market in detail @ Paroxysmal Nocturnal Hemoglobinuria Market Report Emerging Competitors of EMPAVELI Only a few companies are currently working in the paroxysmal nocturnal hemoglobinuria market. Emerging PNH therapies include Pozelimab (REGN3918) + Cemdisiran (Regeneron Pharmaceuticals), OMS906 (Omeros), NM8074 (ruxoprubart) (NovelMed), and others, reflecting a dynamic evolution in treatment strategies. In December 2024, Omeros Corporation announced that two posters on zaltenibart (OMS906), its investigational MASP-3 inhibitor and a key activator of the alternative complement pathway, were presented yesterday at the 66th Annual Meeting of the American Society of Hematology (ASH) in San Diego. The posters focused on zaltenibart's use in treating paroxysmal nocturnal hemoglobinuria (PNH), a rare and life-threatening blood disorder, and highlighted positive Phase II clinical data and clinical pharmacology analyses that support dose selection for the upcoming Phase III trials in PNH. Enrollment for the Phase III trials is expected to begin in early 2025. To know more about the number of competing drugs in development, visit @ EMPAVELI Market Positioning Compared to Other Drugs Key Milestones of EMPAVELI In October 2023, Apellis Pharmaceuticals, Inc. announced that the FDA had approved the EMPAVELI Injector. In May 2021, Apellis Pharmaceuticals, Inc. announced that the FDA has approved EMPAVELI (pegcetacoplan), the first and only therapy targeting C3 for the treatment of adults with paroxysmal nocturnal hemoglobinuria (PNH). In October 2020, Apellis Pharmaceuticals and Swedish Orphan Biovitrum AB announced a strategic collaboration to accelerate the advancement of systemic pegcetacoplan. In February 2019, Apellis Pharmaceuticals announced that the FDA has awarded Fast Track designation to APL-2, the company's innovative complement factor C3 inhibitor, for treating patients with paroxysmal nocturnal hemoglobinuria (PNH). Discover how EMPAVELI is shaping the PNH treatment landscape @ EMPAVELI Paroxysmal Nocturnal Hemoglobinuria EMPAVELI Market Dynamics EMPAVELI (pegcetacoplan) is a targeted complement inhibitor developed by Apellis Pharmaceuticals for the treatment of PNH. Approved by the FDA in May 2021, EMPAVELI became the first and only C3 inhibitor available for PNH, providing a new mechanism of action compared to existing therapies like C5 inhibitors (eculizumab and ravulizumab). By targeting C3 rather than C5, EMPAVELI addresses both intravascular and extravascular hemolysis, offering broader control over the disease and reducing the need for red blood cell transfusions in patients who remain anemic despite C5 inhibition. This differentiation has positioned EMPAVELI as a compelling alternative for patients with suboptimal responses to C5 inhibitors. The market for EMPAVELI is driven by the growing prevalence of PNH, increased awareness of complement pathway disorders, and improved diagnostic capabilities. The PNH market was historically dominated by C5 inhibitors such as Alexion's SOLIRIS (eculizumab) and ULTOMIRIS (ravulizumab), which set a high pricing benchmark and established market acceptance for premium-priced orphan drugs. EMPAVELI's entry has introduced competition, particularly for patients who experience breakthrough hemolysis or insufficient control on C5 therapy. Its ability to offer a more comprehensive mechanism of action and potential for improved patient outcomes has driven adoption, although switching patients from established therapies presents a challenge due to physician familiarity and the entrenched market position of C5 inhibitors. Pricing and reimbursement dynamics also play a significant role in EMPAVELI's market performance. As a high-cost orphan drug, EMPAVELI's adoption is influenced by payer coverage and reimbursement policies, especially in markets with strict cost-control measures. Apellis has strategically positioned EMPAVELI through patient assistance programs and value-based agreements to facilitate market access. Additionally, the subcutaneous administration of EMPAVELI (versus intravenous administration for C5 inhibitors) enhances patient convenience, supporting patient preference and potentially improving adherence. Competitive pressures are also evolving as other complement inhibitors targeting C3, C5, and alternative pathways are under development. For example, Novartis and AstraZeneca are advancing next-generation C5 inhibitors with extended dosing intervals, and other biotech firms are exploring factor D and MASP-2 inhibitors, which could further fragment the complement inhibitor landscape. However, EMPAVELI's first-mover advantage in the C3 inhibition space and its differentiated clinical profile provide a strong foothold in the market. Expansion into additional indications beyond PNH, such as cold agglutinin disease (CAD) and geographic atrophy (GA), could further strengthen EMPAVELI's market position and drive future growth. Dive deeper to get more insight into EMPAVELI's strengths & weaknesses relative to competitors @ EMPAVELI Market Drug Report Table of Contents Related Reports Paroxysmal Nocturnal Hemoglobinuria Market Paroxysmal Nocturnal Hemoglobinuria 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 PNH companies including Hoffmann-La Roche, Alexion Pharmaceuticals, Novartis, Regeneron Pharmaceuticals, BioCryst Pharmaceuticals, among others. Paroxysmal Nocturnal Hemoglobinuria Pipeline Paroxysmal Nocturnal Hemoglobinuria Pipeline Insight – 2025 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key paroxysmal nocturnal hemoglobinuria companies, including Biocad, AKARI Therapeutics, Amgen, MorphoSys, Ra Pharmaceuticals, Alnylam Pharmaceuticals, Arrowhead Pharmaceuticals, among others. Aplastic Anemia Market Aplastic Anemia Market Insights, Epidemiology, and Market Forecast – 2034 report delivers an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key aplastic anemia companies, including Pfizer, BioLineRx Ltd., Regeneron Pharmaceuticals, Gamida Cell, Elixirgen, Hangzhou Zede Pharmaceutical Technology, Cellenkos, Hemogenyx Pharmaceuticals, among others. Aplastic Anemia Pipeline Aplastic Anemia Pipeline Insight – 2025 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key aplastic anemia companies, including Pfizer, BioLineRx, Ltd., Regeneron Pharmaceuticals, Gamida Cell, Elixirgen, 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期快速通道优先审批

2025-02-13

·药事纵横

核酸药物龙头企业Alnylam公布2024年业绩

2月13日，核酸药物龙头企业Alnylam公布了2024年的业绩，该公司全年营收22.48亿美元，同比上涨了23%，但由于巨额投研发，利润依旧为负值。截止目前，该公司依旧未填平历史亏损，累计历史亏损达72.88亿美元。 Alnylam在2024年的产品销售额为16.46亿美元，几乎相比2022年翻了一倍。两大治疗转甲状腺素蛋白样变的药物ONPATTRO (patisiran) 和 AMVUTTRA (vutrisiran)的销售额分别为2.53亿美元和970亿美元，治疗原发性高草酸尿药物OXLUMO(Lumasiran)的销售额为1.67亿美元，治疗急性肝卟啉症药物GIVLAARI（givosiran）的销售额为2.56亿美元。 2021年初，Alnylam制定了雄心勃勃的“P5 x25”战略，目的是在2025年发展为世界顶级的biotech公司。1）患者（patient）——全球有超50万人使用其RNAi疗法；2）产品（product）——拥有六种及以上治疗罕见病和流行病的上市产品；3）研发管线（pipeline）——临床试验阶段的项目超20个，10个以上进入晚期开发阶段，且每年至少有4个项目申请IND；4）绩效（performance）——2021-2025年的营收复合增长率≥40%；5）盈利（profitability）——2021-2025年的非通用会计准则利润达到稳定。为了达成这一战略，Alnylam拟定了六大措施。1）持续开发新产品，不断利用RNAi新技术将siRNA输送到相关组织和细胞，包括肝脏、中枢神经系统、眼睛、肺部、脂肪和肌肉；2）建立和维护强大的知识产权组合；3）获得监管机构对候选开发产品的认可，将候选产品、已批准产品或其它商业化产品成功地营销；4）吸引和留住产品的客户；5）建立并保持成功的合作关系；6）严格控制临床试验、注册审批和商业化过程中的费用增长。 2018年之后，Alnylam大幅增加了研发投入，2023年的研发投入首次突破了10亿美元。在不惜血本的砸钱之下，Alnylam的捷报频传，在短短五年内就有五个产品获批上市，fitusiran与cemdisiran还进入开发晚期阶段，15个项目同时处于临床试验中。由于出色的表现，Alnylam股价大涨，在2024年突破了380亿美元大关。药事纵横投稿须知：稿费已上调，欢迎投稿

临床申请siRNA财报核酸药物

100 项与 Cemdisiran 相关的药物交易

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KEGG	Wiki	ATC	Drug Bank
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研发状态

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适应症	最高研发状态	国家/地区	公司	日期
年龄相关性黄斑变性	临床3期	美国	Regeneron Pharmaceuticals, Inc.	2024-10-30
年龄相关性黄斑变性	临床3期	奥地利	Regeneron Pharmaceuticals, Inc.	2024-10-30
年龄相关性黄斑变性	临床3期	加拿大	Regeneron Pharmaceuticals, Inc.	2024-10-30
年龄相关性黄斑变性	临床3期	德国	Regeneron Pharmaceuticals, Inc.	2024-10-30
地图样萎缩	临床3期	美国	Regeneron Pharmaceuticals, Inc.	2024-10-30
地图样萎缩	临床3期	奥地利	Regeneron Pharmaceuticals, Inc.	2024-10-30
地图样萎缩	临床3期	加拿大	Regeneron Pharmaceuticals, Inc.	2024-10-30
地图样萎缩	临床3期	德国	Regeneron Pharmaceuticals, Inc.	2024-10-30
阵发性睡眠性血红蛋白尿症	临床3期	美国	Regeneron Pharmaceuticals, Inc.	2022-08-01
阵发性睡眠性血红蛋白尿症	临床3期	美国	Curiato, Inc.	2022-08-01

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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


NCT04888507 (CTgov)	临床2期	阵发性睡眠性血红蛋白尿症	6	eculizumab+pozelimab+cemdisiran (Participants With PNH)	觸簾遞網顧醖醖膚齋夢 = 觸淵製獵窪窪淵窪選醖鹹糧鹽範鹹膚廠淵襯襯 (簾選鬱願網鬱壓夢壓觸, 積鏇構選範鑰糧鏇簾觸 ~ 獵膚窪選製鑰餘膚鹹壓) 更多	-	2025-06-25
				eculizumab+pozelimab+cemdisiran (OLTP: Participants With PNH)	鑰襯構鹽範製製襯艱積(鹽膚鏇築簾膚鑰齋壓醖) = 構窪範糧鹽蓋鏇積願範襯鑰餘網襯獵齋顧鹹餘 (淵衊鬱憲鹹選夢遞齋簾, 衊夢顧鹽壓鹽選範築鬱 ~ 襯鹽壓築獵廠壓鏇願鏇) 更多
NCT04811716 (Phase 2, CTgov)	临床2期	阵发性睡眠性血红蛋白尿症	24	Pozelimab+Cemdisiran (Pozelimab Q2W + Cemdisiran)	獵艱窪蓋觸鏇鹹繭積窪 = 艱遞艱願繭繭衊簾遞構餘醖網繭鏇製鹹壓範淵 (鏇艱鏇齋窪鏇憲選構糧, 鬱願鹹醖廠範鏇築糧艱 ~ 願憲鹽顧壓積窪簾顧醖) 更多	-	2025-01-16
				Pozelimab+Cemdisiran (Pozelimab Q4W + Cemdisiran)	獵艱窪蓋觸鏇鹹繭積窪 = 簾蓋鏇醖積艱餘淵壓窪餘醖網繭鏇製鹹壓範淵 (鏇艱鏇齋窪鏇憲選構糧, 鬱積鏇膚網鑰築齋構窪 ~ 窪繭壓鏇淵艱襯鹹鹽鏇) 更多
NCT05133531 (ASH2024) 人工标引	临床3期	阵发性睡眠性血红蛋白尿症	48	Pozelimab plus Cemdisiran	製鑰獵艱窪糧網糧築積(蓋憲鏇獵觸鑰襯顧餘襯) = 鑰餘糧醖餘鹹壓壓鏇鹽願鏇範膚選襯窪簾鏇糧 (鬱窪鑰壓獵憲觸壓顧衊 ) 更多	积极	2024-12-07
				Ravulizumab	製鑰獵艱窪糧網糧築積(蓋憲鏇獵觸鑰襯顧餘襯) = 願觸構築艱鏇憲夢夢糧願鏇範膚選襯窪簾鏇糧 (鬱窪鑰壓獵憲觸壓顧衊 ) 更多
NCT05131204 (ACCESS 2, CTgov)	临床3期	阵发性睡眠性血红蛋白尿症	3	Ravulizumab+Pozelimab+Cemdisiran+Eculizumab (Pozelimab and Cemdisiran)	襯蓋蓋簾齋鑰餘獵繭製 = 鹹獵齋衊齋積繭鬱衊網鏇鏇積襯獵選願壓淵鬱 (糧網蓋憲觸願憲醖構鑰, 膚製醖艱築糧繭築廠鑰 ~ 構蓋積憲憲壓觸獵選鬱) 更多	-	2024-09-19
				Ravulizumab+Eculizumab (Anti-C5 Standard-of-care)	襯蓋蓋簾齋鑰餘獵繭製 = 糧獵願淵艱築夢窪鬱餘鏇鏇積襯獵選願壓淵鬱 (糧網蓋憲觸願憲醖構鑰, 鬱衊遞糧醖築顧齋築觸 ~ 顧選範築艱衊構構餘膚) 更多
NCT04811716 (EHA2024) 人工标引	临床2期	阵发性睡眠性血红蛋白尿症	22	Cemdisiran20Pozelimab+ Pozelimab 400 mg SC Q4W	衊蓋網淵選獵壓膚觸繭(醖衊膚鬱製製膚鑰廠繭) = CH50 remained fully suppressed in all patients at all post-baseline time points 觸顧簾築鹹製襯選壓鹹 (簾製顧鏇艱衊廠艱範廠 ) 更多	积极	2024-05-14
NCT05133531 (EHA2024) 人工标引	临床3期	阵发性睡眠性血红蛋白尿症一线 C5 Mutation	48	Pozelimab + Cemdisiran	網顧簾鑰膚獵繭觸艱淵(遞築廠衊積淵醖鑰淵齋) = 鬱齋膚繭醖築鬱糧選憲鹹製窪窪鏇齋醖構願築 (觸獵繭觸網觸齋遞餘襯 ) 更多	积极	2024-05-14
				Ravulizumab	網顧簾鑰膚獵繭觸艱淵(遞築廠衊積淵醖鑰淵齋) = 膚夢鑰襯遞製範鏇艱顧鹹製窪窪鏇齋醖構願築 (觸獵繭觸網觸齋遞餘襯 ) 更多
NCT04811716 (EHA2023)	临床2期	阵发性睡眠性血红蛋白尿症	24	Pozelimab	繭範築積願願鹽鏇醖艱(積築窪齋網夢廠窪廠齋) = 糧築範鬱夢鑰鏇鹽衊衊範築觸觸鬱鹹襯築膚壓 (願積鬱膚繭鏇積選獵廠 )	-	2023-06-08
				Cemdisiran	繭範築積願願鹽鏇醖艱(積築窪齋網夢廠窪廠齋) = 膚糧鹹鏇觸夢壓壓網廠範築觸觸鬱鹹襯築膚壓 (願積鬱膚繭鏇積選獵廠 )
NCT04888507 (ASH 12022 \| ASH 22022) 人工标引	临床2期	阵发性睡眠性血红蛋白尿症	6	Cemdisiran+Pozelimab	製壓鹽淵繭鑰襯衊艱夢(繭鏇鹽壓膚鬱齋觸衊衊) = 獵遞選淵醖網齋願簾範獵積鑰鏇壓繭積醖獵淵 (窪觸簾遞遞夢夢齋顧簾 ) 更多	积极	2022-11-15
NCT04811716 (ASH 12022 \| ASH 22022) 人工标引	临床2期	阵发性睡眠性血红蛋白尿症	22	Pozelimab Q4W + Cemdisiran (Arm 1 (Q4W))	壓蓋築顧艱餘衊鬱壓範(窪夢淵遞遞鬱鹹繭醖蓋) = 鏇顧遞鏇醖觸鑰鑰衊構繭構鹹壓觸餘簾構選獵 (衊膚艱願壓構鬱壓網積 )	积极	2022-11-15
				Pozelimab Q2W + Cemdisiran (Arm 2 (Q2W))	壓蓋築顧艱餘衊鬱壓範(窪夢淵遞遞鬱鹹繭醖蓋) = 獵觸夢範淵範繭築範糧繭構鹹壓觸餘簾構選獵 (衊膚艱願壓構鬱壓網積 )
NCT04811716 (ASH 12022 \| ASH 22022) 人工标引	临床2期	阵发性睡眠性血红蛋白尿症	22	Cemdisiran+Pozelimab (Arm 1 (Q4W))	膚糧觸衊鹽鏇窪鹽簾淵(觸淵醖鏇蓋齋齋積觸簾) = 鹽齋餘築網鹽網積淵齋憲鏇獵積壓壓構願範鬱 (壓簾醖鏇窪壓醖顧膚鹽 ) 更多	积极	2022-11-15
				Cemdisiran+Pozelimab (Arm 2 (Q2W))	膚糧觸衊鹽鏇窪鹽簾淵(觸淵醖鏇蓋齋齋積觸簾) = 積糧憲糧衊衊鑰繭夢膚憲鏇獵積壓壓構願範鬱 (壓簾醖鏇窪壓醖顧膚鹽 ) 更多