特殊审评加速批准 (美国)、孤儿药 (美国)、罕见儿科疾病 (美国)、孤儿药 (欧盟)、优先药物（PRIME） (欧盟)、孤儿药 (日本)、孤儿药 (韩国)、孤儿药 (澳大利亚)、优先审评 (澳大利亚)、优先审评 (美国)、突破性疗法 (中国)、附条件批准 (中国)、优先审评 (中国)、突破性疗法 (美国)

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结构/序列

分子式C25H33ClN2O5

InChIKeyJUWBBUFSAGEROP-VVJLZRNGSA-N

CAS号2447007-60-3

关联

项与盐酸伊普可泮相关的临床试验

NCT07347990

/ RecruitingN/AIIT

A Prospective, Multicenter, Single-Arm Study: Safety and Efficacy of Iptacopan in the Treatment of High-Risk Hematopoietic Stem Cell Transplantation-Associated Thrombotic Microangiopathy (TA-TMA)

The goal of this clinical trial is to evaluate the efficacy and safety of Iptacopan as a second-line treatment for high-risk hematopoietic stem cell transplantation-associated thrombotic microangiopathy (TA-TMA). Iptacopan is a selective oral small-molecule complement factor B inhibitor. It acts by inhibiting factor B, blocking the formation of C3 convertase, reducing C3b deposition, thereby suppressing C5 convertase (C3bBbC3b) and ultimately decreasing the formation of the membrane attack complex (MAC), which is expected to mitigate endothelial damage in TA-TMA pathology. The main questions this study aims to answer are:
* Does Iptacopan improve 6-month overall survival in high-risk TA-TMA patients?
* What adverse events do participants experience while taking Iptacopan?
* Does Iptacopan provide hematological response and organ function recovery in TA-TMA patients? In this prospective, multicenter, open-label, single-arm Phase II study, all participants will receive Iptacopan treatment. The primary endpoint of this study is the 6-month overall survival rate from TA-TMA diagnosis. Secondary endpoints include safety evaluation, hematological response, and organ function recovery.
During the study, participants will:
* Receive Iptacopan treatment according to protocol
* Undergo regular assessments for safety and efficacy monitoring
* Be followed for up to 24 months post-treatment initiation

开始日期2026-08-25

申办/合作机构

浙江大学医学院附属第一医院（浙江省第一医院）

[+9]

NCT07331259

/ Not yet recruitingN/A

C3 Glomerulopathy Patient Characteristics and Treatment Response to Iptacopan in Routine Care: Analysis of Medical Charts (CHART-C3G)

This is a non-interventional chart abstraction cohort study with longitudinal follow up. Patients with C3G treated with iptacopan will be enrolled and characterized (i.e., systematically describe and summarize) regarding their medical history and iptacopan use and evaluated for clinical events, outcomes, and laboratory measurements upon and after iptacopan treatment initiation. Medical charts will be used to obtain secondary pseudonymized patient-level data with reference to 2 time anchors: at index date (date of iptacopan treatment initiation) with baseline covering 12 months prior to index date, and at 12-month follow-up (twelve months after the index date).The observation period includes baseline plus follow-up.
Iptacopan will be used as prescribed by the clinician in accordance with the terms of the marketing authorization. This Novartis-sponsored study, mainly executed by a contract research organization (CRO), will use secondary data from EHR obtained through reference centers/ centers of excellence in glomerular diseases in Germany.
The primary objective of this study is to characterize the demographic and clinical profiles of adult patients diagnosed with C3G upon iptacopan treatment initiation.

开始日期2026-01-01

申办/合作机构

Novartis Pharmaceuticals Canada, Inc.

NCT06994845

/ Recruiting临床3期

A Single-arm, Multicenter, Phase III Study to Assess Efficacy, Pharmacokinetics, Safety and Tolerability of Iptacopan in Pediatric Patients of 2 to <18 Years of Age With Primary Immunoglobulin A Nephropathy (IgAN)

The study is an open-label, single arm, multicenter, Phase III study to determine proteinuria reduction, pharmacokinetics (PK), safety and tolerability (including CV surveillance) of iptacopan in primary immunoglobulin A nephropathy (IgAN) pediatric patients aged 2 to <18 years.

开始日期2025-11-27

申办/合作机构-

100 项与盐酸伊普可泮相关的临床结果

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100 项与盐酸伊普可泮相关的转化医学

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100 项与盐酸伊普可泮相关的专利（医药）

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121

项与盐酸伊普可泮相关的文献（医药）

2026-12-31·Hematology

Improvement in anemia and symptoms after switching from crovalimab to iptacopan in paroxysmal nocturnal hemoglobinuria

Article

作者: Onizuka, Makoto ; Ogawa, Yoshiaki ; Nasukawa, Miina ; Ogiya, Daisuke ; Koyama, Seina ; Machida, Shinichiro ; Shiraiwa, Sawako ; Kawada, Hiroshi ; Tomita, Shunsuke ; Endo, Motoki ; Toyosaki, Masako

OBJECTIVES:

Paroxysmal nocturnal hemoglobinuria (PNH) may develop breakthrough hemolysis (BTH) despite C5 inhibition. Although iptacopan, an oral factor B inhibitor, has demonstrated efficacy in phase 3 trials, switching from crovalimab has not been reported. We describe the first clinical case of this switch in a real-world clinical setting.

METHODS:

A 72-year-old man with long-standing PNH received eculizumab followed by crovalimab. Infectious enteritis triggered BTH and worsening anemia during crovalimab therapy. Because anemia persisted after recovery, iptacopan 200 mg twice daily was initiated three weeks after the final crovalimab administration. Clinical symptoms and laboratory parameters were followed for 12 months. This case report complied with the Declaration of Helsinki.

RESULTS:

Hemoglobin increased from 9.6 to 11.6 g/dL within two weeks and remained ≥12 g/dL thereafter. Dyspnea resolved within one week, vitality improved by two weeks, and no BTH or treatment-related adverse events occurred during follow-up.

DISCUSSION:

This case demonstrates the feasibility of switching from crovalimab to iptacopan despite the absence of an established method, and pharmacokinetic considerations guided the timing.

CONCLUSION:

Switching to iptacopan led to rapid and durable improvement in anemia and symptoms, supporting proximal complement inhibition as a valuable option for PNH patients inadequately controlled with C5 inhibitors.

2026-12-31·JOURNAL OF MEDICAL ECONOMICS

Cost per responder analysis of iptacopan versus eculizumab and ravulizumab in treatment of paroxysmal nocturnal hemoglobinuria: implications for decision-making

Article

作者: Shafrin, Jason ; Muthukrishnan, Sanjana ; Kuypers, Nicholas ; Bilano, Ver ; Than, Kyi-Sin ; Paulose, Jincy

OBJECTIVE:

Paroxysmal nocturnal hemoglobinuria (PNH) is a rare and debilitating hematological disease with significant economic burden. Despite the availability of multiple therapies, there is a lack of consensus on optimal treatment strategies among physicians and payers in the United States. This study aimed to evaluate the economic value of iptacopan, a novel oral treatment option, compared to terminal complement inhibitors (specifically, complement component C5 inhibitor or C5i)-including eculizumab and ravulizumab-among patients with PNH who are either (i) C5i-experienced or (ii) complement-inhibitor-naïve.

METHODS:

A cost per responder analysis was conducted based on treatment efficacy from clinical trials comparing iptacopan with C5i treatments. Treatment response was defined as the proportion of patients achieving red blood cell transfusion independence. Treatment costs were estimated as pharmaceutical wholesale acquisition cost and treatment administration costs, accounting for discontinuation. Outcomes evaluated included the number needed to treat to achieve a response and the cost per responder over the treatment duration of 24 weeks.

RESULTS:

Over 24 weeks, the number needed to treat to achieve an additional response was lower for iptacopan than all C5i comparators (C5i-experienced: 1.05 with iptacopan vs. 3.86 with C5is; complement-inhibitor-naïve: 1.02 with iptacopan vs. 1.69 with C5is). Cost per responder was lower for iptacopan than C5i comparators for both C5i-experienced ($264,337 for iptacopan vs. $975,298 for ravulizumab, $1,060,511 for eculizumab, and $744,561-$955,194 for eculizumab biosimilar with 10%-30% discount from eculizumab cost) and complement-inhibitor-naïve patients ($256,754, vs. $428,139 for ravulizumab, $465,546 for eculizumab, and $326,849-$419,314 for eculizumab biosimilar).

CONCLUSION:

Among both C5i-experienced and complement-inhibitor-naïve patients, treatment with iptacopan resulted in higher response rates and lower cost per responder compared to C5is.

2026-12-31·Hematology

Switching patients with PNH from pegcetacoplan to iptacopan: a case series

Article

作者: Dingli, David ; Gurnari, Carmelo ; Maciejewski, Jaroslaw ; Sanikommu, Srinivasa ; Orland, Mark

OBJECTIVES:

Limited published data exist on switching therapy from pegcetacoplan to iptacopan in patients with paroxysmal nocturnal hemoglobinuria (PNH).

METHODS:

Three patient cases were collected from the Mayo Clinic, Cleveland Clinic, and Levine Cancer Institute.

RESULTS:

Patient 1 is a 57-year-old woman with PNH. She experienced extravascular hemolysis (EVH) while on C5 inhibitors and breakthrough hemolysis (BTH) after switching to pegcetacoplan. Patient 2 is a 37-year-old woman seeking care for PNH, who was initiated on C5 inhibitor therapy and switched to pegcetacoplan. EVH and BTH were observed while she was on C5 inhibitor therapy, and transfusions were required with both C5 inhibitor and pegcetacoplan therapy. Patient 3 is a 58-year-old man with aplastic anemia and PNH who was initiated on C5 inhibitor therapy, then switched to pegcetacoplan. He experienced both EVH and BTH.

DISCUSSION:

All three patients switched from pegcetacoplan to iptacopan, and none experienced EVH or BTH while on iptacopan. After switching to iptacopan, all three patients improved hemoglobin levels and abating transfusion requirements.

CONCLUSION:

In this case series, three patients with PNH were successfully transitioned from pegcetacoplan to iptacopan monotherapy. No patients exhibited laboratory evidence of BTH, EVH, or intravascular hemolysis after switching to iptacopan.

1,395

项与盐酸伊普可泮相关的新闻（医药）

2026-09-23

·Firstwordpharma

Roche's sefaxersen hits Phase III goal in IgA nephropathy

Roche on Wednesday said its investigational Factor B-directed antisense oligonucleotide (ASO) sefaxersen — which is partnered with Ionis Pharmaceuticals — met the primary endpoint of a Phase III study in patients with primary IgA nephropathy (IgAN) at high risk of progression."Sefaxersen may…offer a new treatment option to help slow disease progression and potentially reduce the long-term need for dialysis or kidney transplantation," said Levi Garraway, Roche's chief medical officer.The IMAgINATION trial randomised 459 participants with IgAN to receive once-monthly subcutaneous sefaxersen or placebo over a 105-week period. The study's main goal was reduction in proteinuria — a key surrogate marker of renal function — measured by change from baseline in 24-hour urine protein-to-creatinine ratio (UPCR) at week 37.Top-line findings from a prespecified interim analysis showed that sefaxersen achieved statistically significant and clinically meaningful proteinuria reductions versus placebo at 37 weeks. The company also noted sefaxersen's safety and tolerability profile aligned with previously reported data, with no new signals identified. Detailed interim data will be presented at an upcoming conference and submitted to regulatory agencies.Meanwhile, IMAgINATION will continue to evaluate the change in kidney function over two years, as measured by estimated glomerular filtration rate (eGFR) at week 105.Earlier this year, Morgan Stanley analysts estimated peak sales of up to CHF 1 billion ($1.2 billion) for sefaxersen — which is intended for self-administration in people with IgAN — despite "several competitors with similar efficacy data." For more, read — Spotlight On: Four things to watch at Roche.The IgAN treatment landscape has strongly flourished in recent times. While Travere Therapeutics' Filspari (sparsentan), Otsuka Pharmaceutical's Voyxact (sibeprenlimab), Novartis' Fabhalta (iptacopan) and Vera Therapeutics' Trutakna (atacicept) have already gained approvals for the indication, Vertex Pharmaceuticals' povetacicept is awaiting an FDA decision in November (for more, see – Spotlight On: Vertex's renal pipeline approaches an inflection point).

临床3期临床结果上市批准寡核苷酸

2026-09-22

·肾开讲

肾小球滤过率下降、肌酐高，治疗不止老方案！这些新靶点药物正在路上

几十年如一日，如今的肾病治疗方法，正在更新迭代，正在从清毒护肾，进入到降压减流量以精准靶向治疗的时代。药物方案可以更直接的直奔肾衰损伤因素入手，解决损伤机制，而不再兜圈子，绕弯子的仅仅只是改善指标或者缓解症状，能够更深入去解决肾脏深层次的损伤问题，这对无论属于早期还是肾衰竭中晚期的患者来说，都是一个非常好的消息。过去二十年，都是以控指标或调理纪委驻。如今肾病治疗的核心逻辑先是“血流动力学”，比如联合沙坦降压力，达格列净减流量，非奈利酮抗纤维化。这三个药确实撑起了护肾的“基本盘”。如今又深入到靶向治疗，这些靶向治疗与改善肾脏血流动力不同，它们更侧重于在“免疫攻击”或“补体激活”或“内皮素通路”等方面的控制。针对这些“非血流动力学”靶点，近几年有一批新药正在进入临床或已经获批。它们不是“替代”沙坦和达格列净，而是补位者，补上老方案覆盖不到的漏洞。今天把几个最有前景的新靶点讲清楚。新靶点一：IgA肾病的新武器——靶向内皮素A受体内皮素-1是一种强效缩血管物质。在IgA肾病患者中，内皮素通路被过度激活，导致肾小球内压升高、系膜细胞增殖、足细胞损伤。沙坦/普利主要作用于肾素-血管紧张素系统，对 endothelin 通路覆盖不够。代表药物目前有阿曲生坦，是一类选择性内皮素A受体拮抗剂。研究显示，阿曲生坦在IgA肾病患者中显著降低蛋白尿，且耐受性良好。目前正在等待FDA审批。适合IgA肾病、蛋白尿持续不降、沙坦/普利+达格列净效果不理想的患者。新靶点二：针对“补体介导”的肾病——靶向补体通路补体系统是免疫系统的一部分。在某些肾病中，补体被异常激活，直接攻击肾小球。比如C3肾病、膜增生性肾小球肾炎、非典型溶血性尿毒症综合征。这些病的根源不是“压力”或“流量”，是“补体在攻击”。代表药物目前有依库珠单抗，是一类C5补体抑制剂，用于非典型溶血性尿毒症综合征。Ravulizumab则食欲长效C5抑制剂，每8周一次。Iptacopan是一类口服B因子抑制剂，用于C3肾病和IgA肾病。研究显示，iptacopan在IgA肾病中显著降低蛋白尿。是补体介导的肾病比如C3肾病、膜增生性肾小球肾炎等，或IgA肾病中补体激活明显的患者的曙光。新靶点三：B细胞/抗体通路，精准清除“致病抗体” 很多肾病的根源是B细胞产生了攻击肾脏的异常抗体。比如膜性肾病（抗PLA2R抗体）、狼疮性肾炎（抗dsDNA抗体）、ANCA相关性肾炎。传统免疫抑制剂是“地毯式轰炸”，B细胞靶向药物是“精准打击”。代表药物：包括利妥昔单抗是典型的抗CD20单抗，主要核心点在于清除B细胞。在膜性肾病中，利妥昔单抗已成为一线治疗选择。泰它西普与利妥昔单抗不同，则是一类双靶点（BLyS/APRIL）生物制剂，用于狼疮性肾炎等多类肾病。贝利尤单抗是抗BLyS单抗，用于活动性狼疮性肾炎。对于膜性肾病、狼疮性肾炎、ANCA相关性肾炎，常规免疫抑制效果不理想或不能耐受的患者可以考虑尝试。新靶点四：FcRn可以加速清除致病抗体 FcRn是一种受体，负责把IgG抗体从溶酶体“救回来”，让它们重新进入血液循环。抑制FcRn，就能加速IgG抗体的降解包括那些攻击肾脏的致病抗体。目前的药物主要是艾加莫德，这类FcRn抑制剂，已获批用于重症肌无力。目前在膜性肾病、狼疮性肾炎中开展临床研究。对于抗体介导的肾病（膜性肾病、狼疮性肾炎、ANCA相关性肾炎），尤其是抗体滴度高、常规治疗不理想的患者更合适。静脉或皮下注射的给药方式。新靶点五：TGF-β通路，直接阻断“造瘢痕”信号 TGF-β是启动纤维化的核心信号。代表药物非奈利酮通过阻断盐皮质激素受体间接抑制TGF-β，但直接抑制TGF-β的药物还在研发中。目前还有多款TGF-β通路抑制剂在临床研究中，部分在糖尿病肾病和CKD中显示出延缓纤维化的信号。但需注意TGF-β也参与正常免疫调节，完全阻断可能带来副作用。目前尚无获批药物，仍在临床试验阶段。这些新药，要知道首先它们不是老方案或者一些“老药”的替代品，是“补位”。沙坦/普利+达格列净+非奈利酮仍然是基础。新靶点药物是在这个基础上“加码”，针对老方案覆盖不到的漏洞。此外这些新药有明确的适应人群，主要是多发性肾病IgA肾病、膜性肾病、狼疮性肾炎。不是所有CKD患者都适合。它们能进一步降低蛋白尿、延缓肾功能下降，让药物方案更为完善。所以未来针对肾小球滤过率下降、肌酐高等各方面治疗不止这些老方案。更多新靶点药物正在路上。大家也可以抱有期待，在等待新药到来之前最需要做的就是先稳定住当下的病情，积极清毒护肾，让肾衰竭的速度再慢一点，而不是坐以待毙。更多肾病问题，可长按识别留言了解更多肾科知识，关注肾事说，更多问题解答戳阅~读~原~文

2026-09-21

·甚是重要

IgA肾病在研新药盘点：下一批即将上市的药物，可能改变什么？

标签： #IgA肾病 #新药研发 #临床试验 #肾脏健康 #慢性肾脏病摘要：上一篇文章讲了5款已上市的新药。但IgA肾病的药物研发并没有停步——还有一批药物正在III期临床试验中，部分已经公布了令人振奋的数据。这篇文章盘点最有可能在未来1-3年内获批的在研药物，讲清楚它们的机制、临床数据和适用人群。拨开科普迷雾，只为少走弯路。为什么还有这么多在研药物？ IgA肾病的核心问题，是致病性IgA的产生。谁能在更上游、更精准的地方把它拦住，谁就能改变疾病的进程。已上市的5款药物，分别从肠道黏膜（布地奈德）、内皮素通路（司帕生坦、阿曲生坦）、补体旁路（伊普可泮）和APRIL信号（斯贝利单抗）切入。但在研的药物，还在往更上游走——有的直接抑制B细胞和浆细胞的存活信号，有的用反义寡核苷酸在基因层面抑制补体，有的用单克隆抗体清除产生致病性IgA的浆细胞。下面按作用机制分类，把最值得关注的几个在研药物讲清楚。一、APRIL/BAFF通路抑制剂：从源头掐断致病性IgA APRIL和BAFF是B细胞和浆细胞存活、分化、产生抗体的关键信号分子。抑制这两个信号，就能从源头上减少致病性IgA的产生。这条路径是目前在研药物最集中的方向。Atacicept：双重BAFF/APRIL抑制剂，III期数据已公布研发方： Vera Therapeutics机制： Atacicept是一种TACI-Fc融合蛋白，同时抑制BAFF和APRIL两个信号分子。 III期数据。 ORIGIN 3试验的中期分析结果于2025年11月发表在《新英格兰医学杂志》上。203名患者随机接受Atacicept 150mg每周一次皮下注射或安慰剂。第36周时，Atacicept组的尿蛋白/肌酐比值较基线降低45.7%，安慰剂组仅降低6.8%，组间差异41.8个百分点，具有高度统计学显著性。给药方式。 150mg，皮下注射，每周一次，患者可在家中自行注射。注意事项。最常见的不良事件为轻至中度，Atacicept组不良事件发生率59.3%，安慰剂组50.0%。需要监测感染风险。 Telitacicept：国产双重BAFF/APRIL抑制剂研发方：荣昌生物机制：与Atacicept类似，同时抑制BAFF和APRIL，但Telitacicept是一种TACI-Fc融合蛋白的优化版本，在B细胞和浆细胞的异常活化抑制方面有独特设计。 II期数据。在中国患者中开展的II期试验显示，240mg剂量组蛋白尿平均降低49%，160mg组降低25%。Gd-IgA1在240mg组降低50.4%，IgG-IgA免疫复合物降低42.7%。 III期进展。 III期临床试验正在进行中，目前主要在中国开展。儿科数据。一项回顾性研究显示，Telitacicept在儿童IgA肾病和IgA血管炎相关肾炎中，能显著减少蛋白尿和血尿，5名患儿在治疗后停用了糖皮质激素。适用病理表现。同属BAFF/APRIL通路抑制剂，从B细胞/浆细胞源头干预，理论上对免疫活动活跃的病理类型（M1、E1、C1/C2）更为对因。对已形成的慢性硬化（S1、T2）逆转作用有限。 Povetacicept：新一代BAFF/APRIL双抑制剂研发方： Alpine Immune Sciences（Vertex旗下）机制：同样靶向BAFF和APRIL，但设计上做了进一步优化，采用工程化TACI结构域，增强了对APRIL的结合亲和力。 II期数据。在12名IgA肾病患者中，80mg每4周一次皮下注射，24周时尿蛋白/肌酐比值降低53.5%，Gd-IgA1降低约60%。 III期进展。 80mg每4周一次的III期试验正在进行中，与安慰剂对照。给药方式。 80mg，皮下注射，每4周一次，相比Atacicept的每周一次，患者依从性可能更好。二、补体抑制剂：从下游控制炎症放大Sefaxersen：补体B因子反义寡核苷酸研发方：罗氏 / Ionis Pharmaceuticals研发代号： RO7434656 / IONIS-FB-LRx 机制。 Sefaxersen是一种GalNAc偶联的反义寡核苷酸，通过皮下注射靶向肝脏，抑制补体B因子的合成。补体B因子是补体旁路途径的关键成分，抑制它就能从源头阻断补体旁路的激活。 II期数据。在23名IgA肾病患者中，70mg每月一次皮下注射，第29周时24小时尿蛋白排泄较基线降低43%，eGFR保持稳定。蛋白尿的降低在参与延长治疗的7名患者中持续维持，其中4名患者治疗超过12个月。安全性方面。有3名患者出现短暂的丙氨酸氨基转移酶升高（正常上限3-5倍），但胆红素未变化，患者继续用药并完成了治疗。没有出现与药物相关的严重不良事件。 III期进展。 III期IMAGINATION研究已启动，主要评估Sefaxersen在具有高进展风险的原发性IgA肾病患者中的疗效和安全性。适用病理表现。补体旁路途径的抑制，对C3沉积明显或新月体形成（C1/C2）提示补体激活参与损伤的患者，可能更有针对性。三、细胞清除剂：直接清除产生致病性IgA的浆细胞Mezagitamab：抗CD38单克隆抗体研发方：武田制药研发代号： TAK-079 机制。 Mezagitamab是一种靶向CD38的单克隆抗体。CD38在浆细胞表面高表达，而浆细胞正是产生致病性IgA的“工厂”。通过清除这些浆细胞，Mezagitamab可以从最源头减少Gd-IgA1的产生。 Ib期数据。 17名IgA肾病患者在标准治疗基础上加用Mezagitamab皮下注射。第96周（末次给药后18个月）的数据显示：eGFR保持稳定（较基线平均变化+2.5），蛋白尿持续降低55.2%，Gd-IgA1降低50.1%，60%的患者血尿完全缓解。没有报告严重不良事件、机会性感染或3级以上感染。 III期进展。武田已启动Mezagitamab在原发性IgA肾病中的III期临床试验，首批患者已入组。给药方式。皮下注射，具体剂量方案在III期试验中确定。适用病理表现。从浆细胞层面清除致病性IgA的来源，对免疫活动活跃的病理类型更为对因。对于新月体性IgA肾病，如果病情进展迅速，可能需要联合糖皮质激素治疗。Zigakibart：APRIL抑制剂，皮下注射每2周一次研发方：诺华研发代号： BION-1301 机制。 Zigakibart是一种人源化单克隆抗体，通过阻断APRIL信号来减少致病性IgA的产生。 I/II期数据。 100周随访数据显示，Zigakibart治疗使eGFR持续稳定，无论疾病严重程度如何，蛋白尿和Gd-IgA1均持续降低。给药方式。 600mg，皮下注射，每2周一次。 III期进展。 III期BEYOND研究正在进行中，同时正在开展开放标签延长研究，评估长期安全性和耐受性，计划入组223名患者，预计2031年完成。四、病理分层用药：在研药物怎么选？在研药物的病理适用性，可以参考已上市药物的逻辑框架。活动性病变为主（M1、E1、C1/C2）。免疫活动还在，治疗重点是“控火”。优先考虑从B细胞/浆细胞源头干预的药物：Atacicept、Telitacicept、Mezagitamab、Zigakibart、Povetacicept。这些药物从上游减少致病性IgA的产生，对活动性病变的病理基础更匹配。补体激活明显（C3沉积、新月体）。如果病理提示补体旁路途径参与损伤，可以优先考虑Sefaxersen。慢性化病变为主（S1、T2）。肾单位损失不可逆，免疫抑制获益下降。在研药物中，目前没有专门针对慢性化病变的药物。这类患者仍应以已上市的非免疫性治疗为主（司帕生坦、阿曲生坦、RAS抑制剂、SGLT2抑制剂）。活动性和慢性化并存。先控活动（Atacicept、Mezagitamab等），再长期护肾（司帕生坦、阿曲生坦）。总结在研的IgA肾病新药，正在从三个方向推进：更上游：Mezagitamab直接清除浆细胞，Zigakibart、Atacicept、Telitacicept、Povetacicept抑制APRIL/BAFF信号。更精准：Sefaxersen用反义寡核苷酸在肝脏层面抑制补体B因子。更方便：Povetacicept每4周一次，Zigakibart每2周一次，相比每周一次的药物，患者依从性更好。其中，Atacicept的III期数据已经发表在《新英格兰医学杂志》上，离获批最近。Mezagitamab的Ib期数据显示停药后18个月肾功能仍稳定，这种“一次性治疗、长期获益”的模式，如果得到III期验证，将是一个非常值得期待的方向。当然，这些药物都还在研究阶段。具体选择哪一种，需要肾内科医生根据患者的病理类型、肾功能水平、蛋白尿程度和合并症情况来综合判断。对于患者来说，最重要的是把目前的治疗做好——控制血压、减少蛋白尿、定期复查。新药在路上，但基础治疗不能停。参考文献 Lafayette R, Barbour SJ, Brenner RM, et al. A Phase 3 Trial of Atacicept in Patients with IgA Nephropathy. N Engl J Med. 2026;394(7):647-657. DOI: 10.1056/NEJMoa2510198. Takeda. Mezagitamab Sustained Effect on Kidney Function 18 Months After Treatment in Primary IgA Nephropathy. News release, November 7, 2025. Barbour SJ, Hladunewich MA, McCaleb ML, et al. A single-arm phase 2 trial of an investigational RNA therapeutic to complement factor B sefaxersen for treatment of IgA nephropathy. Kidney Int. 2026;109(3):592-601. DOI: 10.1016/j.kint.2025.11.017. Reily C, Novak J, Rizk DV. The Roles of A Proliferation-Inducing Ligand (APRIL) and B-Cell Activating Factor (BAFF) in IgA Nephropathy. Am J Kidney Dis. 2026;87(5):709-715. Novartis. Open-label Extension Study of Zigakibart in Adults With IgA Nephropathy. ClinicalTrials.gov Identifier: NCT06858319. Jiang Y, et al. Efficacy evaluation of Telitacicept in IgA nephropathy: a real-world study. Nephrol Dial Transplant. 2025;40(Supplement_3):gfaf116.1106. DOI: 10.1093/ndt/gfaf116.1106. 如果你觉得这篇文章有帮助，欢迎转发给身边关心肾脏健康的朋友。关于IgA肾病的新药研发，你还有什么想了解的？欢迎在评论区聊聊。

100 项与盐酸伊普可泮相关的药物交易

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外链

KEGG	Wiki	ATC	Drug Bank
D12251 D12252	盐酸伊普可泮	-	DB16200

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
肾小球肾炎慢性补体成分 3 肾小球病	加拿大	Novartis AG	2026-05-04
补体因子H缺乏症	澳大利亚	Novartis Pharmaceuticals Australia Pty Ltd.	2025-12-24
C3肾小球病	美国	Novartis Pharmaceuticals Corp.	2025-03-20
免疫球蛋白a肾病	美国	Novartis Pharmaceuticals Corp.	2024-08-07
阵发性睡眠性血红蛋白尿症	美国	Novartis Pharmaceuticals Corp.	2023-12-05

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
重症肌无力	临床3期	美国	Novartis Pharma AG	2024-07-31
重症肌无力	临床3期	美国	Novartis Pharmaceuticals Canada, Inc.	2024-07-31
重症肌无力	临床3期	中国	Novartis Pharmaceuticals Canada, Inc.	2024-07-31
重症肌无力	临床3期	日本	Novartis Pharma AG	2024-07-31
重症肌无力	临床3期	日本	Novartis Pharmaceuticals Canada, Inc.	2024-07-31
重症肌无力	临床3期	阿根廷	Novartis Pharmaceuticals Canada, Inc.	2024-07-31
重症肌无力	临床3期	澳大利亚	Novartis Pharmaceuticals Canada, Inc.	2024-07-31
重症肌无力	临床3期	巴西	Novartis Pharmaceuticals Canada, Inc.	2024-07-31
重症肌无力	临床3期	丹麦	Novartis Pharmaceuticals Canada, Inc.	2024-07-31
重症肌无力	临床3期	丹麦	Novartis Pharma AG	2024-07-31

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临床结果

适应症

分期

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研究	分期	人群特征	评价人数	分组	结果	评价	发布日期


NCT04578834 (APPLAUSE-IgAN, Pubmed \| N Engl J Med)	临床3期	免疫球蛋白a肾病	477	Iptacopan 200 mg	鏇繭範顧鬱餘範獵顧鹽(艱築艱醖構選選壓襯觸) = 蓋壓餘夢淵選願艱憲膚淵製築憲艱廠淵鏇糧夢 (構糧選窪鏇網餘鹽鏇願 ) 更多	积极	2026-07-30
				Placebo	鏇繭範顧鬱餘範獵顧鹽(艱築艱醖構選選壓襯觸) = 糧壓構艱鏇構顧餘簾鹹淵製築憲艱廠淵鏇糧夢 (構糧選窪鏇網餘鹽鏇願 ) 更多
ISTH2026	N/A	阵发性睡眠性血红蛋白尿症	240	Proximal complement inhibitors	餘鏇鑰衊簾艱襯膚獵鏇(顧廠襯簾鬱積襯築鏇淵) = 淵壓醖窪獵糧繭鑰願網鹽鑰鏇繭蓋簾齋夢範襯 (壓淵淵蓋鏇醖築鬱齋憲 ) 更多	积极	2026-07-13
				C5 inhibitors	-
EHA2026	N/A	阵发性睡眠性血红蛋白尿症	51	Iptacopan	淵顧廠餘獵夢餘網齋壓(遞顧鏇築窪鏇鬱繭廠鑰) = 醖襯築積鹽襯繭鹽膚廠簾選醖網糧鑰淵顧築願 (餘築範製願壓繭顧顧淵, 1.53) 更多	积极	2026-05-12
EHA2026	N/A	阵发性睡眠性血红蛋白尿症	38	Eculizumab	餘膚夢衊繭醖膚範鬱夢(齋觸範鬱積觸糧構顧夢) = 齋顧鑰鏇鑰鹹構鑰簾醖鏇窪衊遞壓鹽醖鹽製鹽 (憲壓淵網衊選構鹽觸壓 ) 更多	积极	2026-05-12
				Iptacopan	餘膚夢衊繭醖膚範鬱夢(齋觸範鬱積觸糧構顧夢) = 觸窪鬱網觸獵蓋鏇鏇襯鏇窪衊遞壓鹽醖鹽製鹽 (憲壓淵網衊選構鹽觸壓 ) 更多
EHA2026	N/A	阵发性睡眠性血红蛋白尿症	40	Iptacopan	網遞蓋網鑰願襯醖淵衊(網積選鏇網餘獵廠夢構) = 膚襯夢選鑰範鹹繭膚選願襯鬱顧鏇糧鏇觸鹹構 (繭襯鬱衊觸簾網鬱積蓋 ) 更多	积极	2026-05-12
EHA2026	N/A	阵发性睡眠性血红蛋白尿症	124	Iptacopan	蓋蓋獵壓蓋襯網繭願衊(膚鏇齋壓廠願積糧鬱範) = 製鬱艱選觸窪鏇餘壓鏇蓋製構壓鹹觸壓繭觸夢 (襯鏇膚簾廠遞繭鑰觸膚, 41.20 ~ 54.14) 更多	积极	2026-05-12
				Eculizumab	蓋蓋獵壓蓋襯網繭願衊(膚鏇齋壓廠願積糧鬱範) = 獵鏇廠醖範淵膚壓遞繭蓋製構壓鹹觸壓繭觸夢 (襯鏇膚簾廠遞繭鑰觸膚, 28.93 ~ 41.87) 更多
NCT03439839 (EHA2026)	临床2期	阵发性睡眠性血红蛋白尿症	26	Iptacopan 200 mg BID	廠淵顧獵襯鹹願齋齋衊(繭齋憲觸餘製簾醖鹹鏇) = 鑰製顧襯遞鹹鹹顧鹽艱築選鬱簾艱構網繭範膚 (構襯膚蓋醖壓範餘遞觸 ) 更多	积极	2026-05-12
EHA2026	N/A	阵发性睡眠性血红蛋白尿症	20	Iptacopan 200 mg	襯顧構積憲廠範積鏇糧(繭鬱廠構糧範顧構獵遞) = 鬱餘淵鬱衊鹽鏇艱選糧淵膚廠淵顧鏇醖簾鬱膚 (網觸衊構繭蓋遞鬱憲齋 ) 更多	积极	2026-05-12
NCT03439839 (EHA2026)	临床2/3期	阵发性睡眠性血红蛋白尿症	223	Iptacopan 200 mg twice daily	繭窪醖醖遞簾遞選鏇窪(築構範衊壓壓積鬱鹽糧) = HDL cholesterol remained stable with mean (SD) change from baseline of 0.0 (0.3) and 0.1 (0.3) mmol/L at years 3 and 4, respectively 憲鑰窪齋鬱蓋餘鏇壓鹽 (遞鑰構淵鑰觸醖網廠襯 ) 更多	积极	2026-05-12
NCT03955445 (APPEAR-C3G, ISN WCN2026)	临床3期	C3肾小球病	74	Iptacopan	網窪獵遞壓範艱艱願衊(蓋窪廠範蓋糧觸廠襯淵) = 顧憲鹽簾獵獵選積壓壓鑰築積遞鬱鑰獵糧築壓 (繭鹹鑰艱艱齋顧鬱簾築 ) 更多	积极	2026-04-01
				Iptacopaniptacopan (Previously received placebo and switched to iptacopan)	網窪獵遞壓範艱艱願衊(蓋窪廠範蓋糧觸廠襯淵) = 築鑰窪壓憲壓築範餘築鑰築積遞鬱鑰獵糧築壓 (繭鹹鑰艱艱齋顧鬱簾築 ) 更多