An Open-label, Phase 2 Trial to Evaluate the Efficacy and Safety of a Single Intravenous Administration of GNT0003 (an Adeno-associated Viral (AAV) Vector Expressing the UGT1A1 Transgene) Following Imlifidase Pre-treatment in Adult Participants With Severe Crigler-Najjar Syndrome (CNS) Requiring Daily Phototherapy and Presenting Pre-existing Anti-AAV8 Antibodies

Clinical trial rationale:
CNS is an ultra-rare (<1/1 million newborns), autosomal recessive disorder of bilirubin conjugation caused by mutation in the gene coding for uridine 5'-diphosphate glucuronosyltransferase (UGT1A1), that causes the accumulation of neurotoxic unconjugated bilirubin (UCB).
Reduction of UCB is managed with phenobarbital in mild CNS, and daily phototherapy in severe CNS.
There is no authorized curative medical treatment for CNS. Liver transplantation is currently the only curative treatment for severe CNS.
GNT0003 is a genetically modified recombinant (r) viral vector composed of the AAV8 viral capsid carrying the UGT1A1 transgene which aims to correct the dysfunction of the mutated gene by achieving durable expression of a functional copy of the affected gene.
Imlifidase (IgG-degrading enzyme) has demonstrated its efficacy in highly sensitized adult kidney transplant patients.
To give participants with pre-existing anti-AAV8 antibodies access to gene therapy treatments, this trial aims to demonstrate the safety and efficacy of GNT0003 following imlifidase pre-treatment in adult participants with severe CNS requiring daily phototherapy and presenting with pre-existing anti-AAV8 antibodies.
Primary objective: to assess efficacy of a single intravenous administration of GNT0003 following imlifidase pre-treatment in participants with severe CNS requiring phototherapy and pre-existing AAV8 antibodies
Secondary objective: to collect data on safety and tolerability of GNT0003 and imlifidase, efficacy of imlifidase, pharmacokinetic and pharmacodynamic profile of GNT0003, and Quality of Life.
The trial will include 3 parts:
* A baseline period for at least 3 months
* A treatment period
* A follow-up period:
* Initial post-treatment follow-up over 48 weeks
* Long-term follow-up for 4 additional years
This trial will be conducted in accordance with the International Conference on Harmonization Guideline for Good Clinical Practice and the Declaration of Helsinki. Participants must be consented using the approved Informed Consent Form before any procedures specified in the protocol are performed.

开始日期2024-11-08

申办/合作机构

Genethon

[+1]

NCT06461546

/ Withdrawn临床2期IIT

Imlifidase in Living Donor Renal Transplantation Highly Sensitized Recipients (LIVEDES)

This is a Phase II, pilot, prospective, unicentric trial, to evaluate Imlifidase could improve the transplantability of the highly sensitized patients with good outcomes respect to survival and functionality of the graft.

开始日期2024-10-22

申办/合作机构-

NCT06241950

/ Terminated临床1期

An Open-Label, Systemic Gene Delivery Study to Evaluate the Safety, Tolerability and Expression of SRP-9001 in Association With Imlifidase in Subjects With Duchenne Muscular Dystrophy With Pre-existing Antibodies to rAAVrh74

This is a gene transfer therapy study evaluating the safety of delandistrogene moxeparvovec and delandistrogene moxeparvovec dystrophin expression in association with imlifidase, in participants with DMD with pre-existing antibodies to rAAVrh74 over a period of 104 weeks.

开始日期2024-01-29

申办/合作机构

Sarepta Therapeutics, Inc.

[+1]

100 项与 Imlifidase 相关的临床结果

登录后查看更多信息

100 项与 Imlifidase 相关的转化医学

登录后查看更多信息

100 项与 Imlifidase 相关的专利（医药）

登录后查看更多信息

108

项与 Imlifidase 相关的文献（医药）

2026-04-01·Transplantation Reviews

Molecular diagnostics in HLA-incompatible kidney transplantation

Review

作者: Guney, Miray ; Viklicky, Ondrej ; Hruba, Petra

Human leukocyte antigen-incompatible (HLA-i) kidney transplantation enables access to transplantation for highly sensitized patients with preformed donor-specific antibodies (DSA) but remains associated with frequent early DSA rebound and alloimmune endothelial injury, resulting in a high incidence of antibody-mediated rejection (AMR) despite current desensitization and induction protocols that include imlifidase. Kidney allograft biopsy remains the diagnostic gold standard for detecting overt and subclinical AMR, yet conventional histology does not always capture early or subclinical antibody-mediated injury, particularly in sensitized recipients. Tissue-based transcriptomics, Molecular Microscope Diagnostic system (MMDx) and Banff Human Organ Transplant gene panel (B-HOT), provide enhanced sensitivity for detecting molecular AMR phenotypes that often precede morphologic changes and may be particularly informative in the early post-transplant period. Plasma donor-derived cell-free DNA (dd-cfDNA) as non-invasive biomarker correlates with molecular rejection activity and may support longitudinal monitoring in sensitized high-risk patients, although interpretation is limited in the immediate post-transplant period. The integration of histology, DSA monitoring, transcriptomics, and emerging biomarkers enables a multimodal approach to AMR phenotyping, which is particularly critical in HLA-i transplantation to detect subclinical injury early, guide timely intervention, and prevent the development of chronic and largely untreatable transplant glomerulopathy.

2026-01-24·NEPHROLOGY DIALYSIS TRANSPLANTATION

Trends in Nephrology: From nihilism to targeted treatment of antibody-mediated rejection.

Article

作者: Oberbauer, Rainer ; Pickl, Josef Filip

Antibody-mediated rejection (AMR) is among the leading causes of kidney transplant attrition. Traditional AMR therapy consisted of interventional studies that were small and often uncontrolled. Although a blockade of the interleukin-6 pathway failed to prevent allograft loss in a phase 3 trial, anecdotal evidence had previously suggested potential benefit. Likewise, anti-CD20 antibodies have not been shown to improve AMR outcomes in terms of graft survival. Imlifidase, which cleaves all IgGs, and hence also all donor specific antibodies, showed a higher rate of graft loss in the experimental group of an AMR study. Terminal complement inhibitors showed inconsistent efficacy in underpowered trials; currently, C3 inhibitors are a promising focus for complement-targeted strategies against AMR. Anti-CD38 antibodies currently represent the most promising novel therapy option for AMR based on encouraging phase 2 study results. We furthermore discuss genetically engineered CD38 knock-out multitarget natural killer cells, expressing anti-B cell maturation antigen (BCMA) CAR, IL-15RF and hnCD16, administered in combination with daratumumab (anti-CD38) in this context. This approach represents a cheaper and safer alternative to chimeric HLA antigen receptor (CHAR) T cell therapy. CHAR T cells showed a high specificity in recognizing their respective target anti-HLA class I B cell receptors in-vitro. Quantitative results from in-vivo trials are pending. Likewise, we hypothesize the use of bi-specific T cell engagers such as CD19 blinatumomab or BCMA teclistamab for the treatment of AMR. In addition, clinical studies investigating conversion from calcineurin inhibitors to co-stimulation therapies such as anti-CD40L antibodies may reduce dnDSAs and AMR. In summary, anti-CD38 antibodies currently constitute the drug class with the strongest evidence for AMR treatment. To date, most CD38 antibodies have been shown to be safe, even after several years of administration in patients with multiple myeloma.

2026-01-02·Clinical Kidney Journal

Complement activation in anti-glomerular basement membrane disease before and after treatment with imlifidase

Article

作者: Hellmark, Thomas ; Sonesson, Elisabeth ; Segelmark, Mårten ; Blom, Anna M ; Tyrberg, Linnéa ; Uhlin, Fredrik ; Alam, Shanavaz

ABSTRACT:

Background:

The involvement of the complement system in anti-glomerular basement membrane (GBM) disease is well known but incompletely characterized. The ability of autoantibodies to trigger the classical pathway is evident, while the lectin and alternative pathways also seem to be of importance. We studied complement activation in patients treated with imlifidase, which leads to rapid IgG depletion, to elucidate the role of complement in anti-GBM disease.

Methods:

The GOOD-IDES-01 trial included 15 anti-GBM disease patients treated with one dose of imlifidase in addition to standard therapy with 6 months of follow-up. Plasma samples were analyzed for C3, C4 and complement activation products [C4d, C3bBbP and soluble terminal complement complexes (sTCC)]. Ratios of C4d/C4 and C3bBbP/C3 were calculated to correct for plasmapheresis. Serum samples were analyzed for anti-drug antibodies (ADA) directed against imlifidase.

Results:

The C4d/C4 ratio decreased rapidly from its pre-dose level, while sTCC decreased more slowly. sTCC and C3bBbP/C3 were above the reference level throughout the trial. We observed a transient increase in C4d/C4 and C3bBbP/C3, but not sTCC, immediately following treatment with imlifidase, which tended to be more pronounced in patients with more pre-existing ADA.

Conclusions:

Classical pathway activation decreased rapidly after autoantibody removal by imlifidase and increased again in most of those that experienced a rebound, but terminal complement activation remained elevated throughout the trial. However, due to the small sample size our results must be interpreted with caution.

107

项与 Imlifidase 相关的新闻（医药）

2026-02-24

·动脉网

汉莎生物制药的伊米非酶生物制品许可申请（BLA）获FDA受理 Hansa Biopharma’s Biologics License Application (BLA) for imlifidase accepted by the FDA

Hansa Biopharma AB, today announced that its Biologics License Application (BLA) for imlifidase has been accepted by the U.S. Food and Drug Administration (FDA). Hansa Biopharma AB今天宣布，其针对imlifidase的生物制品许可申请（BLA）已被美国食品药品监督管理局（FDA）接受。FDA's filing review was completed on day 60 which is meant to verify that the submission is substantially complete and meets the requirements for a full evaluation. FDA的文件审查在第60天完成，这是为了核实提交的文件基本完整并符合全面评估的要求。Renée Aguiar-Lucander, CEO, Hansa Biopharma said: “We now look forward to receiving the 74-Day Letter which will provide details regarding review plan, timelines and other pertinent information and start working with the FDA as they go through their review over the coming months.” 汉莎生物制药公司首席执行官雷内·阿吉亚尔-卢坎德表示：“我们期待收到74天函件，其中将提供有关审评计划、时间表和其他相关信息的细节，并在接下来的几个月中开始与FDA合作进行审评。”Imlifidase is a unique IgG‑cleaving enzyme that rapidly inactivates > 95% of donor‑specific antibodies within 2–6 hours of administration, providing a crucial window to enable HLA‑incompatible kidney transplantation. Imlifidase 是一种独特的 IgG 切割酶，可在给药后 2 至 6 小时内迅速使超过 95% 的供体特异性抗体失活，为实现 HLA 不相容的肾移植提供了关键窗口。The BLA submission for imlifidase is supported by the previously communicated highly statistically significant outcome of the pivotal U.S. Phase 3 ConfIdeS trial, which evaluated 12-month kidney function in highly sensitized adult kidney transplant patients (cPRA ≥99.9%) with a positive crossmatch against a deceased donor, compared to a control arm. imlifidase的BLA提交得到了此前公布的关键性美国III期ConfIdeS试验结果的支持，该试验评估了高致敏成人肾移植患者（cPRA≥99.9%）在与已故供体交叉配型呈阳性的情况下12个月的肾功能，对照组为对照臂。The trial successfully met its primary endpoint, demonstrating significantly improved kidney function in the imlifidase arm at 12 months as measured by mean estimated glomerular filtration rate (eGFR) (p < 0.0001). A key secondary endpoint—dialysis independence at 12 months—was also statistically significant in favor of imlifidase (p = 0.0007). 试验成功达到了主要终点，结果显示在 12 个月时，imlifidase 组的肾功能显著改善，以平均估算肾小球滤过率 (eGFR) 衡量 (p < 0.0001)。一个关键的次要终点——12 个月时无需透析——也显示出对 imlifidase 具有统计学意义的优势 (p = 0.0007)。Imlifidase was generally well tolerated, with a safety profile consistent with previous clinical trial experience. . Imlifidase 通常耐受性良好，其安全性与之前的临床试验经验一致。About ConfIdeS 关于ConfIdeSConfIdeS is a pivotal Phase 3 open label, randomized, controlled trial of imlifidase in kidney transplantation. The trial evaluated kidney function at 12 months in 64 highly sensitized (cPRA ≥99.9%) kidney transplant patients with positive crossmatch against a deceased donor, comparing desensitization using imlifidase with a control arm. ConfIdeS 是一项关键的 III 期开放标签、随机、对照试验，评估了 imlifidase 在肾移植中的应用。该试验在 64 名高度致敏（cPRA ≥99.9%）且针对 deceased donor 呈阳性交叉配型的肾移植患者中，比较了使用 imlifidase 进行脱敏与对照组的效果，并在 12 个月时评估了肾功能。A total of 25 U.S. sites participated in the trial, and the primary endpoint was kidney graft function at 12 months, measured by mean eGFR (estimated glomerular filtration rate). The total trial duration is five years, which includes a long-term follow-up agreed with the FDA as part of the accelerated approval pathway. 共有 25 个美国试验点参与了这项试验，主要终点是通过平均 eGFR（估算肾小球滤过率）测量的 12 个月时的肾脏移植物功能。试验总时长为五年，其中包括作为加速批准途径的一部分而与 FDA 达成共识的长期随访。About imlifidase 关于 imlifidaseImlifidase is conditionally approved in the European Union, Norway, Lichtenstein, Iceland and the UK under the tradename IDEFIRIX® for the desensitization treatment of highly sensitized adult kidney transplant patients with a positive crossmatch against an available deceased donor. IDEFIRIX® is also approved in Australia and Switzerland.. Imlifidase 在欧盟、挪威、列支敦士登、冰岛和英国获得附条件批准，商标名为 IDEFIRIX®，用于对高致敏成人肾移植患者进行脱敏治疗，这些患者对可用的已故供体呈阳性交叉配型。IDEFIRIX® 也已在澳大利亚和瑞士获得批准。Information about the trial is available at ClinicalTrials.gov: NCT04935177 有关该试验的信息可在 ClinicalTrials.gov 查阅：NCT04935177This is information that Hansa Biopharma AB (publ) is obliged to make public pursuant to the EU Market Abuse Regulation. The information was submitted for publication, through the agency of the contact person set out below, at 19:53 CET on February 18, 2026. 这是Hansa Biopharma AB（publ）根据欧盟市场滥用条例规定必须公开的信息。该信息通过以下列出的联系人提交发布，时间为2026年2月18日欧洲中部时间19:53。Contacts for more information: 更多信息请联系：Evan Ballantyne, Chief Financial Officer 埃文·巴拉ntyne，首席财务官IR@hansabiopharma.com IR@hansabiopharma.comKerstin Falck, VP Global Corporate Affairs 克尔斯廷·法尔克，全球企业事务副总裁 media@hansabiopharma.com 媒体@汉萨生物制药.comkerstin.falck@hansabiopharma.com kerstin.falck@hansabiopharma.comAbout IDEFIRIX® (imlifidase) 关于IDEFIRIX®（imlifidase）Imlifidase is an antibody-cleaving enzyme originating from Streptococcus pyogenes that specifically targets and cleaves immunoglobulin G (IgG) antibodies and inhibits IgG-mediated immune response.1 It has a rapid onset of action, cleaving IgG-antibodies and inhibiting their activity within hours after administration.. Imlifidase是一种源自化脓性链球菌的抗体切割酶，专门靶向并切割免疫球蛋白G（IgG）抗体，抑制IgG介导的免疫反应。它起效迅速，在给药后数小时内即可切割IgG抗体并抑制其活性。Imlifidase has conditional marketing approval in Europe and is marketed under the tradename IDEFIRIX for the desensitization treatment of highly sensitized adult kidney transplant patients with a positive crossmatch against an available deceased donor. The use of IDEFIRIX should be reserved for patients who are unlikely to be transplanted under the available kidney allocation system, including prioritization programs for highly sensitized patients.1 IDEFIRIX was reviewed as part of the European Medicines Agency’s (EMA) PRIority Medicines (PRIME) program, which supports medicines that may offer a major therapeutic advantage over existing treatments or benefit patients without treatment options.1. Imlifidase 在欧洲获得附条件上市许可，并以商品名 IDEFIRIX 上市，用于对具有阳性交叉配型的高度致敏成年肾移植患者进行脱敏治疗。IDEFIRIX 的使用应保留给那些在现有肾脏分配系统（包括针对高度致敏患者的优先计划）下不太可能接受移植的患者。IDEFIRIX 作为欧洲药品管理局 (EMA) 的 PRIority Medicines (PRIME) 计划的一部分接受了审查，该计划支持可能较现有治疗方法具有重大治疗优势或使无治疗选择的患者受益的药物。The efficacy and safety of imlifidase as a pre-transplant treatment to reduce donor-specific IgG was studied in four Phase 2 open-label, single-arm, six-month clinical trials.2,3-5 Hansa is collecting further clinical evidence and will submit additional efficacy and safety data based on one observational follow-up study and one post-approval efficacy study.. 在四项二期开放标签、单臂、六个月的临床试验中研究了 imlifidase 作为移植前治疗以减少供体特异性 IgG 的有效性和安全性。Hansa 正在收集更多的临床证据，并将基于一项观察性随访研究和一项批准后的有效性研究提交额外的有效性和安全性数据。Full EU product information can be accessed via the initial Summary of Product Characteristics found here. 完整的欧盟产品信息可以通过此处的初始产品特性摘要访问。About kidney failure 关于肾衰竭Kidney disease can progress to kidney failure or End-Stage Renal Disease (ESRD), identified when a patient’s kidney function is less than 15%.6 ESRD poses a significant health burden, affecting nearly 2.5 million patients worldwide.6 A kidney transplant is the treatment of choice for suitable patients with ESRD because it offers improved survival and quality of life benefits, and is cost savings compared to long-term dialysis. 肾病可进展为肾衰竭或终末期肾病 (ESRD)，当患者的肾功能低于 15% 时即可诊断为ESRD。ESRD 带来了显著的健康负担，影响着全球近 250 万名患者。对于适合进行移植的 ESRD 患者，肾移植是首选治疗方案，因为它可以提高生存率和生活质量，并且与长期透析相比节省了成本。There are approximately 170,000 kidney patients in the US and Europe waiting for a new kidney.7. 美国和欧洲大约有 17 万名肾病患者在等待新的肾源。About Hansa Biopharma 关于Hansa BiopharmaHansa Biopharma AB is a pioneering commercial-stage biopharmaceutical company developing and commercializing novel immunomodulatory therapies to transform care for patients with acute or complex immune disorders. Hansa’s proprietary IgG-cleaving enzyme technology platform to address serious unmet medical needs in transplantation, gene therapy and autoimmune diseases. Hansa Biopharma AB 是一家处于商业化阶段的先锋生物制药公司，致力于开发和推广新型免疫调节疗法，以改变急性或复杂免疫疾病患者的治疗方式。Hansa 专有的 IgG 切割酶技术平台旨在满足移植、基因治疗和自身免疫疾病领域中未被满足的重大医疗需求。The company’s portfolio includes imlifidase, a first-in-class immunoglobulin G (IgG) antibody-cleaving enzyme therapy, which has been shown to enable kidney transplantation in highly sensitized patients, and HNSA-5487, a next-generation IgG-cleaving molecule that will be developed for Guillain-Barré Syndrome (GBS). 公司的产品组合包括 imlifidase，这是一种首创的免疫球蛋白 G (IgG) 抗体切割酶疗法，已被证明能够使高度致敏患者接受肾移植；以及 HNSA-5487，这是一种下一代 IgG 切割分子，将用于开发治疗吉兰-巴雷综合征 (GBS)。Hansa Biopharma is based in Lund, Sweden, and has operations in Europe and the U.S. The company is listed on Nasdaq Stockholm under the ticker HNSA. Find out more at www.hansabiopharma.com and follow us on LinkedIn.. 汉莎生物制药公司总部位于瑞典隆德，在欧洲和美国开展业务。该公司在纳斯达克斯德哥尔摩上市，股票代码为HNSA。更多信息请访问www.hansabiopharma.com，并在LinkedIn上关注我们。©2026 Hansa Biopharma AB. Hansa Biopharma, the beacon logo, IDEFIRIX, and IDEFIRIX flower logo are trademarks of Hansa Biopharma AB, Lund, Sweden. All rights reserved. ©2026 汉莎生物制药公司。汉莎生物制药、灯塔标志、IDEFIRIX 和 IDEFIRIX 花形标志是瑞典隆德汉莎生物制药公司的商标。保留所有权利。Forward-Looking Statements 前瞻性声明This press release contains forward-looking statements relating to the business of Hansa, including, without limitation, statements regarding Hansa’s strategy, commercialization efforts, business plans, regulatory submissions, clinical development plans, revenue and product sales projections or forecasts and focus. 本新闻稿包含与汉莎业务相关的前瞻性声明，包括但不限于关于汉莎的战略、商业化努力、业务计划、监管提交、临床开发计划、收入和产品销售预测或展望的声明。The words “may,” “will,” “could,” “would,” “should,” “expect,” “plan,” “anticipate,” “intend,” “believe,” “estimate,” “predict,” “project,” “potential,” “continue,” “target,” and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. “可能”、“将”、“可能”、“会”、“应该”、“预期”、“计划”、“预期”、“打算”、“相信”、“估计”、“预测”、“项目”、“潜力”、“继续”、“目标”等类似表述旨在识别前瞻性陈述，尽管并非所有前瞻性陈述都包含这些识别词。Any forward-looking statements in this press release are based on management’s current expectations and beliefs and are subject to a number of risks, uncertainties, and important factors that may cause actual events or results to differ materially from those expressed or implied by any forward-looking statements contained in this press release, including, without limitation, any related to Hansa’s business and operations, the presumed mechanism of action of imlifidase, the safety and efficacy of imlifidase in the patient population above or other potential indications, market acceptance of imlifidase, competitive products, anticipated timelines and other factors that may cause actual results, performance or achievements to be materially different from any future results, performance or achievement expressed or implied by these forward-looking statements. 本新闻稿中的任何前瞻性陈述均基于管理层的当前预期和信念，并受到多种风险、不确定性和重要因素的影响，这些因素可能导致实际事件或结果与本新闻稿中包含的任何前瞻性陈述所表达或暗示的内容存在重大差异，包括但不限于与Hansa的业务和运营、imlifidase的假定作用机制、imlifidase在上述患者群体或其他潜在适应症中的安全性和有效性、imlifidase的市场接受度、竞争产品、预期时间表等相关因素，以及其他可能导致实际结果、表现或成就与这些前瞻性陈述所表达或暗示的未来结果、表现或成就存在重大不同的因素。Hansa cautions you not to place undue reliance on any forward-looking statements, which speak only as of the date they are made. Hansa disclaims any obligation to publicly update or revise any such statements to reflect any change in expectations or in events, conditions, or circumstanc. 汉莎航空提醒您，不要过分依赖任何前瞻性声明，这些声明仅在其发布之日有效。汉莎航空不承担任何义务公开更新或修改任何此类声明，以反映预期、事件、条件或情况的任何变化。References 参考文献Source: hansabiopharma.com 来源：hansabiopharma.com

临床结果

2026-02-19

·药明康德

产业新闻 | 默沙东长效单抗3期结果积极，有望扩展适应症；治疗“石化”疾病，FDA授予再生元单抗优先审评资格……

默沙东长效单抗3期结果积极，有望扩展适应症默沙东（MSD）今日公布其3期SMART研究（MK-1654-007）的最新结果。该研究在两个RSV流行季中开展，旨在评估Enflonsia（clesrovimab）这一长效预防性单克隆抗体在易发展为重症呼吸道合胞病毒（RSV）感染的儿童人群中的保护作用。最新数据显示，在年龄不足2岁且在第二个RSV流行季仍处于重症风险中的儿童中，于第二个流行季开始时接受Enflonsia治疗后，其整体安全性表现良好。该结果与第一流行季接受该疗法的婴儿人群总体一致。基于上述研究结果，默沙东计划将第二个RSV流行季的数据提交美国FDA及全球其它监管机构，用于支持该疗法在高风险儿童中拓展至第二个RSV流行季预防适应症的评估。Enflonsia是一种用于预防RSV感染的长效单克隆抗体，通过直接、快速且持久的作用机制，可提供约5个月的保护期，覆盖典型RSV流行季，并采用与婴儿体重无关的统一给药剂量设计，旨在为高风险儿童群体提供更便捷且持续的防护方案。治疗“石头人症”，FDA授予再生元单抗优先审评资格今日，再生元（Regeneron Pharmaceuticals）宣布，美国FDA已受理其单克隆抗体garetosmab的生物制品许可申请（BLA），并授予优先审评资格，用于治疗进行性骨化性纤维发育不良（FOP）成人患者。FDA预计将于2026年8月前作出审批决定。此次申报基于3期OPTIMA研究的积极疗效与安全性数据。研究结果显示，在56周时，与安慰剂相比，接受garetosmab治疗的两种剂量组（3 mg/kg与10 mg/kg）均显著减少新发异位骨化（HO）病灶的数量与总体积。在主要终点分析中，3 mg/kg剂量组新发病灶数量较安慰剂减少94%（1处vs 19处，p=0.0274），10 mg/kg剂量组减少90%（2处vs 19处，p=0.0260）。此外，事后分析显示，两种剂量均使新发HO病灶平均总体积较安慰剂降低超过99%（3 mg/kg：0.01 cm³ vs 10.45 cm³，p=0.0013；10 mg/kg：0.02 cm³ vs 10.45 cm³，p=0.0005）。 FOP的另一个俗称是“石头人症”，意指随着疾病的发展，人会像石头一样无法活动。这一罕见病的主要症状是人体中原本柔软、有弹性的肌肉和结缔组织会在轻微受伤后就出现炎症，并且会变成骨骼，将原本可以自由活动的关节锁死在一个位置上。再生元科学家发现，Activin A通过驱动HO这一FOP的核心病理过程，在疾病的发生与发展中发挥关键作用。Garetosmab是一种基于VelocImmune平台开发的全人源单克隆抗体，可结合并中和Activin A，从而干预FOP患者异位骨形成过程。此前，美国FDA已授予garetosmab孤儿药资格，并授予其用于预防FOP患者HO的快速通道资格。助力肾移植，FDA受理创新疗法上市申请 Hansa Biopharma近日宣布，其创新疗法imlifidase的BLA已获美国FDA受理。Imlifidase是一种独特的IgG裂解酶，可在给药后2至6小时内快速灭活超过95%的供者特异性抗体，从而为HLA不相容肾移植创造关键时间窗口。这一机制有望帮助高度致敏患者突破免疫屏障，使原本难以实施的肾移植成为可能。此次BLA申报基于关键性3期ConfIdeS研究的积极结果。该研究在高度致敏成人肾移植患者（校正群体反应性抗体[cPRA]≥99.9%）中评估imlifidase的疗效，并与对照组进行比较。结果显示，研究成功达到主要终点，在12个月时imlifidase治疗组的平均估算肾小球滤过率（eGFR）显著改善（p＜0.0001），显示出更优的肾功能恢复。同时，关键次要终点12个月时不再需要透析的比例，同样达到统计学显著性（p＝0.0007）。安全性方面，imlifidase总体耐受性良好，其安全性特征与既往临床研究结果一致。参考资料： [1] Hansa Biopharma's Biologics License Application (BLA) for imlifidase accepted by the FDA. Retrieved February 19, 2026 from https://www.prnewswire.com/news-releases/hansa-biopharmas-biologics-license-application-bla-for-imlifidase-accepted-by-the-fda-302691919.html [2] Merck Announces Positive New Data for ENFLONSIA™ (clesrovimab) for Infants and Children Under 2 Years of Age at Increased Risk for Severe Respiratory Syncytial Virus (RSV) Disease Over Two RSV Seasons. Retrieved February 19, 2026 from https://www.businesswire.com/news/home/20260219213035/en/Merck-Announces-Positive-New-Data-for-ENFLONSIA-clesrovimab-for-Infants-and-Children-Under-2-Years-of-Age-at-Increased-Risk-for-Severe-Respiratory-Syncytial-Virus-RSV-Disease-Over-Two-RSV-Seasons [3] Garetosmab Biologics License Application Accepted for FDA Priority Review for the Treatment of Fibrodysplasia Ossificans Progressiva (FOP). Retrieved February 19, 2026 from https://www.globenewswire.com/news-release/2026/02/19/3240927/0/en/Garetosmab-Biologics-License-Application-Accepted-for-FDA-Priority-Review-for-the-Treatment-of-Fibrodysplasia-Ossificans-Progressiva-FOP.html 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。版权说明：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。转载授权请在「药明康德」微信公众号回复“转载”，获取转载须知。分享，点赞，在看，聚焦全球生物医药健康创新

2026-02-18

·PRNewswire

Hansa Biopharma's Biologics License Application (BLA) for imlifidase accepted by the FDA

LUND, Sweden, Feb. 18, 2026 /PRNewswire/ -- Hansa Biopharma AB, ("Hansa" or "the Company"), (Nasdaq Stockholm: HNSA), today announced that its Biologics License Application (BLA) for imlifidase has been accepted by the U.S. Food and Drug Administration (FDA). FDA's filing review was completed on day 60 which is meant to verify that the submission is substantially complete and meets the requirements for a full evaluation. Renée Aguiar-Lucander, CEO, Hansa Biopharma said: "We now look forward to receiving the 74-Day Letter which will provide details regarding review plan, timelines and other pertinent information and start working with the FDA as they go through their review over the coming months." Imlifidase is a unique IgG-cleaving enzyme that rapidly inactivates > 95% of donor-specific antibodies within 2–6 hours of administration, providing a crucial window to enable HLA-incompatible kidney transplantation. The BLA submission for imlifidase is supported by the previously communicated highly statistically significant outcome of the pivotal U.S. Phase 3 ConfIdeS trial, which evaluated 12-month kidney function in highly sensitized adult kidney transplant patients (cPRA ≥99.9%) with a positive crossmatch against a deceased donor, compared to a control arm. The trial successfully met its primary endpoint, demonstrating significantly improved kidney function in the imlifidase arm at 12 months as measured by mean estimated glomerular filtration rate (eGFR) (p < 0.0001). A key secondary endpoint—dialysis independence at 12 months—was also statistically significant in favor of imlifidase (p = 0.0007). Imlifidase was generally well tolerated, with a safety profile consistent with previous clinical trial experience. About ConfIdeS ConfIdeS is a pivotal Phase 3 open label, randomized, controlled trial of imlifidase in kidney transplantation. The trial evaluated kidney function at 12 months in 64 highly sensitized (cPRA ≥99.9%) kidney transplant patients with positive crossmatch against a deceased donor, comparing desensitization using imlifidase with a control arm. A total of 25 U.S. sites participated in the trial, and the primary endpoint was kidney graft function at 12 months, measured by mean eGFR (estimated glomerular filtration rate). The total trial duration is five years, which includes a long-term follow-up agreed with the FDA as part of the accelerated approval pathway About imlifidase Imlifidase is conditionally approved in the European Union, Norway, Lichtenstein, Iceland and the UK under the tradename IDEFIRIX® for the desensitization treatment of highly sensitized adult kidney transplant patients with a positive crossmatch against an available deceased donor. IDEFIRIX® is also approved in Australia and Switzerland. Information about the trial is available at ClinicalTrials.gov: NCT04935177 This is information that Hansa Biopharma AB (publ) is obliged to make public pursuant to the EU Market Abuse Regulation. The information was submitted for publication, through the agency of the contact person set out below, at 19:53 CET on February 18, 2026. --- ENDS --- Contacts for more information: Evan Ballantyne, Chief Financial Officer [email protected] Kerstin Falck, VP Global Corporate Affairs [email protected] [email protected] Notes to editors About IDEFIRIX® (imlifidase) Imlifidase is an antibody-cleaving enzyme originating from Streptococcus pyogenes that specifically targets and cleaves immunoglobulin G (IgG) antibodies and inhibits IgG-mediated immune response.1 It has a rapid onset of action, cleaving IgG-antibodies and inhibiting their activity within hours after administration. Imlifidase has conditional marketing approval in Europe and is marketed under the tradename IDEFIRIX for the desensitization treatment of highly sensitized adult kidney transplant patients with a positive crossmatch against an available deceased donor. The use of IDEFIRIX should be reserved for patients who are unlikely to be transplanted under the available kidney allocation system, including prioritization programs for highly sensitized patients.1 IDEFIRIX was reviewed as part of the European Medicines Agency's (EMA) PRIority Medicines (PRIME) program, which supports medicines that may offer a major therapeutic advantage over existing treatments or benefit patients without treatment options.1 The efficacy and safety of imlifidase as a pre-transplant treatment to reduce donor-specific IgG was studied in four Phase 2 open-label, single-arm, six-month clinical trials.2,3-5 Hansa is collecting further clinical evidence and will submit additional efficacy and safety data based on one observational follow-up study and one post-approval efficacy study. Full EU product information can be accessed via the initial Summary of Product Characteristics found here. About kidney failure Kidney disease can progress to kidney failure or End-Stage Renal Disease (ESRD), identified when a patient's kidney function is less than 15%.6 ESRD poses a significant health burden, affecting nearly 2.5 million patients worldwide.6 A kidney transplant is the treatment of choice for suitable patients with ESRD because it offers improved survival and quality of life benefits, and is cost savings compared to long-term dialysis. There are approximately 170,000 kidney patients in the US and Europe waiting for a new kidney.7 About Hansa Biopharma Hansa Biopharma AB is a pioneering commercial-stage biopharmaceutical company developing and commercializing novel immunomodulatory therapies to transform care for patients with acute or complex immune disorders. Hansa's proprietary IgG-cleaving enzyme technology platform to address serious unmet medical needs in transplantation, gene therapy and autoimmune diseases. The company's portfolio includes imlifidase, a first-in-class immunoglobulin G (IgG) antibody-cleaving enzyme therapy, which has been shown to enable kidney transplantation in highly sensitized patients, and HNSA-5487, a next-generation IgG-cleaving molecule that will be developed for Guillain-Barré Syndrome (GBS). Hansa Biopharma is based in Lund, Sweden, and has operations in Europe and the U.S. The company is listed on Nasdaq Stockholm under the ticker HNSA. Find out more at and follow us on LinkedIn. ©2026 Hansa Biopharma AB. Hansa Biopharma, the beacon logo, IDEFIRIX, and IDEFIRIX flower logo are trademarks of Hansa Biopharma AB, Lund, Sweden. All rights reserved. Forward-Looking Statements This press release contains forward-looking statements relating to the business of Hansa, including, without limitation, statements regarding Hansa's strategy, commercialization efforts, business plans, regulatory submissions, clinical development plans, revenue and product sales projections or forecasts and focus. The words "may," "will," "could," "would," "should," "expect," "plan," "anticipate," "intend," "believe," "estimate," "predict," "project," "potential," "continue," "target," and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Any forward-looking statements in this press release are based on management's current expectations and beliefs and are subject to a number of risks, uncertainties, and important factors that may cause actual events or results to differ materially from those expressed or implied by any forward-looking statements contained in this press release, including, without limitation, any related to Hansa's business and operations, the presumed mechanism of action of imlifidase, the safety and efficacy of imlifidase in the patient population above or other potential indications, market acceptance of imlifidase, competitive products, anticipated timelines and other factors that may cause actual results, performance or achievements to be materially different from any future results, performance or achievement expressed or implied by these forward-looking statements. Hansa cautions you not to place undue reliance on any forward-looking statements, which speak only as of the date they are made. Hansa disclaims any obligation to publicly update or revise any such statements to reflect any change in expectations or in events, conditions, or circumstances on which any such statements may be based, or that may affect the likelihood that actual results will differ from those set forth in the forward-looking statements. Any forward-looking statements contained in this press release represent Hansa's views only as of the date hereof and should not be relied upon as representing its views as of any subsequent date. References 1. European Medicines Agency. Idefirix® summary of product characteristics. Available at: . 2. Heidt S, et al. Highly Sensitized Patients are Well Serves by Receiving a Compatible Organ Offer Based on Acceptable Mismatches. Front Immunol. 2021;12:687254. Available at: 3. Jordan SC, et al. IgG Endopeptidase in Highly Sensitized Patients Undergoing Transplantation. N Engl J Med. 2017 Aug 3;377(5):442-453. doi: 10.1056/NEJMoa1612567. Erratum in: N Engl J Med. 2017 Oct 26;377(17):1700. doi: 10.1056/NEJMx170015. 4. Winstedt L, et al. Complete Removal of Extracellular IgG Antibodies in a Randomized Dose-Escalation Phase I Study with the Bacterial Enzyme IdeS--A Novel Therapeutic Opportunity. PLoS One. 2015 Jul 15;10(7):e0132011. doi: 10.1371/journal.pone.0132011. PMID: 26177518; PMCID: PMC4503742. 5. Lorant T, et al. Safety, immunogenicity, pharmacokinetics, and efficacy of degradation of anti-HLA antibodies by IdeS (imlifidase) in chronic kidney disease patients. Am J Transplant. 2018 Nov;18(11):2752-2762. doi: 10.1111/ajt.14733. 6. NIH (2018). What is kidney failure? Available at: . 7. Newsletter Transplant 2022. International figures on donation and transplantation. Available at: Newsletter Transplant - latest edition I Freepub (edgm.eu) Accessed: May 2025 This information was brought to you by Cision The following files are available for download: 21% more press release views with Request a Demo

临床结果上市批准临床3期临床2期

100 项与 Imlifidase 相关的药物交易

登录后查看更多信息

外链

KEGG	Wiki	ATC	Drug Bank
D11470	Imlifidase	L04AA41	DB15258

研发状态

批准上市

10 条最早获批的记录,

后查看更多信息

适应症	国家/地区	公司	日期
肾移植排斥反应	欧盟	Hansa Biopharma AB	2020-08-25
肾移植排斥反应	冰岛	Hansa Biopharma AB	2020-08-25
肾移植排斥反应	列支敦士登	Hansa Biopharma AB	2020-08-25
肾移植排斥反应	挪威	Hansa Biopharma AB	2020-08-25

未上市

10 条进展最快的记录,

后查看更多信息

适应症	最高研发状态	国家/地区	公司	日期
抗肾小球基膜疾病	临床3期	美国	Hansa Biopharma AB	2022-12-22
抗肾小球基膜疾病	临床3期	奥地利	Hansa Biopharma AB	2022-12-22
抗肾小球基膜疾病	临床3期	比利时	Hansa Biopharma AB	2022-12-22
抗肾小球基膜疾病	临床3期	捷克	Hansa Biopharma AB	2022-12-22
抗肾小球基膜疾病	临床3期	丹麦	Hansa Biopharma AB	2022-12-22
抗肾小球基膜疾病	临床3期	法国	Hansa Biopharma AB	2022-12-22
抗肾小球基膜疾病	临床3期	德国	Hansa Biopharma AB	2022-12-22
抗肾小球基膜疾病	临床3期	爱尔兰	Hansa Biopharma AB	2022-12-22
抗肾小球基膜疾病	临床3期	意大利	Hansa Biopharma AB	2022-12-22
抗肾小球基膜疾病	临床3期	荷兰	Hansa Biopharma AB	2022-12-22

登录后查看更多信息

临床结果

适应症

分期

评价

查看全部结果

研究	分期	人群特征	评价人数	分组	结果	评价	发布日期


NCT03611621 (17-HMedIdeS-14, PRNewswire)	临床1/2期	肾移植排斥反应 --	39	Imlifidase	範糧築網簾廠壓獵壓獵(簾遞鬱觸鏇廠範網膚衊) = 鏇餘憲簾齋積壓遞醖鬱鑰餘糧醖簾網網艱築鹽 (鬱選糧窪餘糧齋夢範築 ) 更多	积极	2025-06-30
NCT03943589 (15-HMedIdeS-09, CTgov)	临床2期	格林-巴利综合征	30	Imlifidase	範蓋簾繭糧襯襯簾醖鑰(憲簾簾齋糧鹹齋壓鹹鏇) = 遞獵選糧壓窪膚餘製積齋顧築鏇範範夢網鬱襯 (製齋積膚廠蓋膚餘襯願, 顧網鹹鏇積壓獵顧襯夢 ~ 觸淵簾積壓糧網網鬱鹹) 更多	-	2025-04-09
NCT05049850 (CTgov)	临床2期	肾移植排斥反应	3	Imlifidase	願鑰範鏇繭觸積構願選 = 蓋繭夢膚製憲繭糧餘積網廠餘壓鹽鏇鹹鑰繭鹽 (餘網憲觸廠選醖鏇積膚, 淵醖糧構簾製憲鹽鑰願 ~ 顧築夢淵醖蓋窪鹹淵衊) 更多	-	2025-03-17
NCT03943589 (15-HMedIdeS-09, PRNewswire) 人工标引	临床2期	格林-巴利综合征	30	imlifidase	鏇鬱淵網製壓遞糧觸築(鹹鑰廠觸蓋遞鹹繭築繭) = 觸鏇蓋選繭壓鬱鬱築壓壓選範糧廠網淵齋構獵 (膚淵鑰襯築製艱網淵觸 ) 更多	积极	2024-12-17
NCT04711850 (EudraCT number: 2018-000022-66, 2020-004777-49, Pubmed \| Clin Transplant)	N/A	肾移植排斥反应 DSA	-	Imlifidase	艱夢廠觸顧觸獵夢鏇構(鑰構淵淵鬱繭襯製鬱淵) = 膚鏇淵餘壓鏇顧觸鹽構製齋鹽積夢衊製製憲獵 (鹹衊觸積窪壓鑰壓遞醖 ) 更多	不佳	2024-07-01
				Imlifidase (Plasmapheresis)	艱夢廠觸顧觸獵夢鏇構(鑰構淵淵鬱繭襯製鬱淵) = 繭衊膚鹹蓋壓糧鏇選醖製齋鹽積夢衊製製憲獵 (鹹衊觸積窪壓鑰壓遞醖 ) 更多
NCT03897205 (16-HMedIdes-12, CTgov)	临床2期	抗体介导的排斥反应 \| 肾移植排斥反应	30	Imlifidase (Imlifidase)	簾鏇衊醖製壓願蓋鹽繭(遞鏇窪鏇鹹選選醖範願) = 襯鏇遞觸鹽鹽鹹壓選範顧醖糧獵淵網醖鑰窪鏇 (艱選艱網簾願範壓網壓, 4) 更多	-	2024-02-28
				Plasma Exchange (Plasma Exchange)	簾鏇衊醖製壓願蓋鹽繭(遞鏇窪鏇鹹選選醖範願) = 鹹蓋網獵膚顧糧積鹹網顧醖糧獵淵網醖鑰窪鏇 (艱選艱網簾願範壓網壓, 26) 更多
NCT03157037 (GOOD-IDES-01, Pubmed \| Nephrol Dial Transplant)	临床2期	抗肾小球基膜疾病 anti-GBM antibodies \| EA and EB epitopes \| IgG subclass ... 更多	15	Imlifidase	願衊築鏇構夢襯構顧糧(壓蓋鹹簾淵廠繭襯選糧) = 積廠糧廠壓鑰鹽鏇遞衊遞觸鏇繭觸憲醖餘廠窪 (窪鏇壓範鹽餘鬱鑰築構 ) 更多	积极	2023-12-20
				imlifidase (Plasmapheresis)	繭遞襯遞鏇獵製構積簾(選醖蓋鏇積範醖繭選壓) = 獵糧廠憲製觸衊鹹衊觸網獵製遞積鹹觸觸衊窪 (憲窪範壓範襯艱膚簾簾 )
NCT03897205 (16-HMedIdes-12, PRNewswire) 人工标引	临床2期	AMR综合征	30	imlifidase	憲網鑰製願積製憲齋齋(製蓋獵鬱鹽積顧鏇襯鏇) = 襯壓範觸鬱獵製廠觸壓衊鹹壓網鬱網顧齋鹹壓 (獵蓋製顧簾膚糧遞繭艱 ) 更多	积极	2023-12-14
				standard of care	憲網鑰製願積製憲齋齋(製蓋獵鬱鹽積顧鏇襯鏇) = 遞糧淵範廠醖鏇範衊簾衊鹹壓網鬱網顧齋鹹壓 (獵蓋製顧簾膚糧遞繭艱 ) 更多
NCT03943589 (15-HMedIdeS-09, Biospace) 人工标引	临床2期	格林-巴利综合征	-	imlifidase+ intravenous immunoglobulin	製鏇製糧繭艱淵齋淵鬱(齋窪膚膚選蓋膚廠製選) = Imlifidase was safe and well tolerated 選鬱遞鏇餘淵蓋製鏇願 (襯窪憲淵壓窪鏇範構鹽 ) 更多	积极	2023-12-07
NCT03897205 (Biospace) 人工标引	临床2期	器官移植排斥	30	Imlifidase	顧繭選餘鹹壓繭齋遞膚(憲繭鬱窪蓋鑰壓鬱簾製) = With regard to safety, imlifidase was well tolerated, and no safety signals were encountered during the study 網構衊鑰齋積鹹網衊廠 (繭衊憲獵範鏇鹽蓋製簾 )	积极	2022-11-28
				plasma exchange