尼波卡利单抗（Nipocalimab）是首个也是唯一一个获得美国FDA突破性疗法认定的用于治疗中重度干燥综合征成人患者的在研药物，现已获得快速通道资格-动脉网

Nipocalimab, the first and only investigational treatment to be granted U.S. FDA Breakthrough Therapy designation for the treatment of adults with moderate-to-severe Sjögren's disease, has now received Fast Track designation

CISION 等信源发布 2025-03-18 20:03



可切换为仅中文







Sjögren's disease (SjD)

干燥综合征 (SjD)

is a

是

prevalent, debilitating autoantibody disease with no FDA-approved advanced treatments

普遍的、使人衰弱的自身抗体疾病，尚无FDA批准的先进治疗方法

The Company is actively enrolling patients in the Phase 3 DAFFODIL study

公司正在积极招募第三阶段DAFFODIL研究的患者。

This marks the fourth nipocalimab FDA Fast Track designation

这标志着尼波卡利单抗获得FDA快速通道指定的第四次认定

SPRING HOUSE, Pa.

宾夕法尼亚州斯普林豪斯

，

March 18, 2025

2025年3月18日

/PRNewswire/ -- Johnson & Johnson (NYSE:

/PRNewswire/ -- 强生公司（纽约证券交易所：

JNJ

强生公司

) today announced that the U.S. Food and Drug Administration (FDA) has granted investigational nipocalimab Fast Track designation (FTD) for the treatment of adult patients with moderate-to-severe Sjögren's disease (SjD), having previously been granted

）今天宣布，美国食品药品监督管理局（FDA）已授予nipocalimab快速通道资格（FTD），用于治疗中至重度干燥综合征（SjD）成年患者，此前该药已获得

Breakthrough

突破

Therapy designation (BTD) for the investigational therapy late last year. Currently, no advanced therapies are approved to treat this disease.

治疗指定（BTD）用于去年年底的研究性治疗。目前，尚无先进的疗法被批准用于治疗该疾病。

Building upon the BTD, which nipocalimab is the first and only therapeutic to receive for SjD, the U.S. FDA's FTD is also designed to accelerate the delivery of new therapeutics to patients by facilitating the development and expediting the review of drugs that demonstrate the potential to treat serious conditions and help address unmet needs for serious or life-threatening conditions..

基于BTD，nipocalimab是首个也是唯一一个获得SjD治疗资格的药物，美国FDA的FTD也旨在通过促进药物开发和加速审批那些展示出治疗严重疾病潜力、并有助于满足严重或危及生命疾病未满足需求的药物，从而加快新疗法向患者的交付。

'This marks an additional important step forward in our efforts to bring meaningful advancements to people living with Sjögren's disease, a serious and debilitating condition. We look forward to continuing to work closely with the FDA to advance the clinical development of nipocalimab and potentially provide a much-needed treatment option for this community,' said Katie Abouzahr, M.D., Vice President, Autoantibody Portfolio and Maternal Fetal Disease Area Leader, Johnson & Johnson Innovative Medicine..

“这标志着我们在为干燥综合症患者带来有意义的治疗进展方面又迈出了重要的一步，这种疾病是一种严重且使人衰弱的病症。我们期待继续与 FDA 紧密合作，推进尼波卡利单抗的临床开发，有望为这一群体提供一种迫切需要的治疗选择。”强生创新医药公司自体抗体产品组合及母婴疾病领域负责人、副总裁凯蒂·阿布扎赫尔医学博士表示。

There are no FDA-approved treatments that directly address the underlying causes of this complex disease, associated with serious health consequences including chronic dryness of moisture producing glands that may lead to systemic complications such as joint pain, fatigue and inflammation in multiple organ systems..

目前尚无FDA批准的疗法可以直接解决这种复杂疾病的根本原因，该疾病与严重的健康后果相关，包括产生水分的腺体长期干燥，可能导致全身并发症，如关节疼痛、疲劳和多器官系统炎症。

1,2,3

These systemic complications can lead to an increased risk of mortality and associated health conditions, including a 20 times greater risk of developing B-cell lymphomas when compared to the general population.

这些系统性并发症可能导致死亡风险增加，并引发相关健康问题，包括比普通人群高出20倍的B细胞淋巴瘤发病风险。

1,4

1，4

The Phase 2 DAHLIAS study, the

第二阶段的DAHLIAS研究，

results of which were presented last year

去年展示了其结果

, represented the first-ever positive results of an investigational FcRn blocker as a potential targeted therapy in SjD.

，代表了研究性FcRn阻断剂作为SjD潜在靶向治疗的首次取得积极结果。

The study

研究

achieved the primary endpoint in the 15 mg/kg Q2W nipocalimab group, showing a greater than 70% relative average improvement in systemic disease activity at Week 24 compared to placebo and IgG reductions of more than 77%.

在15 mg/kg Q2W nipocalimab组中达到了主要终点，与安慰剂相比，第24周系统性疾病活动相对平均改善超过70%，IgG降低超过77%。

Trends of improvement were similarly observed across multiple secondary endpoints.

在多个次要终点观察到类似的改善趋势。

Safety and tolerability were consistent with other nipocalimab clinical studies.

安全性与耐受性与其他nipocalimab临床研究一致。

ABOUT SJ

关于SJ

ÖGREN'S DISEASE

奥格伦病

Sjögren's disease (SjD) is one of the most prevalent autoantibody-driven diseases for which no therapies are currently approved that treat the underlying and systemic nature of the disease.

干燥综合征（SjD）是最常见的由自身抗体驱动的疾病之一，目前尚无获批疗法能够针对该病的根本性和系统性特征进行治疗。

It is a chronic autoimmune disease that is estimated to impact approximately four million people worldwide and is nine times more common in women than men.

这是一种慢性自身免疫疾病，据估计全球约有四百万人受到影响，女性患者数量是男性的九倍。

6,7

SjD is characterized by autoantibody production, chronic inflammation, and lymphocytic infiltration of exocrine glands. Most patients are affected by mucosal dryness (eyes, mouth, vagina), joint pain and fatigue.

SjD 的特征是自身抗体的产生、慢性炎症以及外分泌腺的淋巴细胞浸润。大多数患者会出现黏膜干燥（眼睛、口腔、阴道）、关节疼痛和疲劳等症状。

More than 50% of SjD patients have a moderate to severe form of the condition, and disease burden can be as high as that of rheumatoid arthritis or systemic lupus erythematosus. It is usually associated with impaired quality of life and functional capacity.

超过50%的SjD患者具有中度至重度的疾病形式，疾病负担可能与类风湿性关节炎或系统性红斑狼疮一样高。它通常与生活质量下降和功能能力受损相关。

6.8,9

ABOUT DAHLIAS

关于大丽花

DAHLIAS (

大丽花 (

NCT04968912

) is a Phase 2 multicenter, randomized, placebo-controlled double-blind study to evaluate the effects of nipocalimab in participants with primary Sjögren's disease. DAHLIAS is a Phase 2 dose-ranging study for adults with moderately-to-severely active primary SjD who were seropositive for anti-Ro60 and/or anti-Ro52 IgG antibodies.

）是一项二期多中心、随机、安慰剂对照双盲研究，旨在评估nipocalimab对原发性干燥综合征（Sjögren's disease）患者的效果。DAHLIAS是一项针对中度至重度活动性原发性干燥综合征（SjD）成人患者的二期剂量探索研究，这些患者对抗Ro60和/或抗Ro52 IgG抗体呈血清阳性。

163 adults aged 18-75 were randomized 1:1:1 to receive intravenous nipocalimab at 5 or 15 mg/kg or placebo every 2 weeks through Week 22 and received protocol-permitted background standard of care. Safety assessments were conducted through Week 30. The primary endpoint was change in baseline in the ClinESSDAI (Clinical European League Against Rheumatism Sjögren's Syndrome Disease Activity Index) Score at Week 24.

163名18-75岁的成人以1:1:1的比例随机分配，每2周通过第22周接受5或15 mg/kg的静脉注射nipocalimab或安慰剂，并接受方案允许的背景标准治疗。安全性评估持续至第30周。主要终点是第24周时ClinESSDAI（临床欧洲抗风湿病联盟干燥综合征疾病活动指数）评分相对于基线的变化。

ClinESSDAI is a systemic diseases activity index designed to measure disease activity in patients with primary SjD based on 11 domains including: constitutional, lymphadenopathy, glandular, articular, cutaneous, respiratory, renal, muscular, peripheral nervous system, central nervous system, and hematological. .

ClinESSDAI 是一种系统性疾病活动指数，旨在基于 11 个领域评估原发性 SjD 患者的疾病活动度，这些领域包括：全身症状、淋巴结病、腺体、关节、皮肤、呼吸系统、肾脏、肌肉、周围神经系统、中枢神经系统和血液系统。

ABOUT NIPOCALIMAB

关于尼泊卡利单抗

Nipocalimab is an investigational monoclonal antibody, designed to bind with high affinity to block FcRn and reduce levels of circulating immunoglobulin G (IgG) antibodies potentially without impact on other immune functions. This includes autoantibodies and alloantibodies that underlie multiple conditions across three key segments in the autoantibody space including Rare Autoantibody diseases, Maternal Fetal diseases mediated by maternal alloantibodies and Rheumatic diseases..

Nipocalimab是一种在研的单克隆抗体，旨在通过高亲和力结合来阻断FcRn，从而减少循环免疫球蛋白G（IgG）抗体的水平，可能不会影响其他免疫功能。这包括自身抗体和同种抗体，这些抗体是多种病症的基础，涵盖自体抗体领域的三个关键部分，包括罕见自体抗体疾病、由母体同种抗体介导的母胎疾病以及风湿性疾病。

5,10,11,12,13,14,15,16.17

5、10、11、12、13、14、15、16、17

Blockade of IgG binding to FcRn in the placenta is also believed to limit transplacental transfer of maternal alloantibodies to the fetus.

阻断IgG与胎盘中FcRn的结合也被认为可限制母体同种抗体向胎儿的跨胎盘转移。

,19

，19

The U.S. Food and Drug Administration (FDA) and European Medicines Agency (EMA) have granted several key designations to nipocalimab including:

美国食品药品监督管理局 (FDA) 和欧洲药品管理局 (EMA) 已授予尼泊珠单抗多项关键认定，包括：

U.S. FDA Fast Track designation in hemolytic disease of the fetus and newborn (HDFN) and warm autoimmune hemolytic anemia (wAIHA) in

美国FDA快速通道指定，用于胎儿和新生儿溶血性疾病（HDFN）及温抗体型自身免疫性溶血性贫血（wAIHA）。

July 2019

2019年7月

, gMG in

，gMG在

December 2021

2021年12月

and fetal neonatal alloimmune thrombocytopenia (FNAIT) in

和胎儿新生儿同种免疫性血小板减少症 (FNAIT) 在

March 2024

2024年3月

and Sjögren’s disease (SjD) in

和干燥综合征（SjD）在

March 2025

2025年3月

U.S. FDA Orphan drug status for wAIHA in

美国FDA授予wAIHA孤儿药资格

December 2019

2019年12月

, HDFN in

，HDFN 在

June 2020

2020年6月

, gMG in

，gMG在

February 2021

2021年2月

, chronic inflammatory demyelinating polyneuropathy (CIDP) in

慢性炎性脱髓鞘性多发性神经病（CIDP）在

October 2021

2021年10月

and FNAIT in

和FNAIT在

December 2023

2023年12月

U.S. FDA Breakthrough Therapy designation for HDFN in

美国FDA授予HDFN突破性疗法认定

February 2024

2024年2月

and for Sjögren's disease in

以及在干燥综合症中

November 2024

2024年11月

U.S. FDA granted Priority Review in gMG in Q4 2024

美国食品药品监督管理局（FDA）在2024年第四季度授予gMG优先审查。

EU EMA Orphan medicinal product designation for HDFN in

欧盟EMA授予HDFN孤儿药资格认定

October 2019

2019年10月

ABOUT JOHNSON & JOHNSON

关于强生公司

At Johnson & Johnson, we believe health is everything. Our strength in healthcare innovation empowers us to build a world where complex diseases are prevented, treated, and cured, where treatments are smarter and less invasive, and solutions are personal. Through our expertise in Innovative Medicine and MedTech, we are uniquely positioned to innovate across the full spectrum of healthcare solutions today to deliver the breakthroughs of tomorrow and profoundly impact health for humanity. .

在强生，我们相信健康就是一切。我们在医疗保健创新方面的实力使我们能够构建一个世界，在这个世界中，复杂疾病得以预防、治疗和治愈，治疗方法更智能、更少侵入性，并且解决方案是个性化的。凭借我们在创新药物和医疗技术方面的专业知识，我们有能力在当今整个医疗保健解决方案领域进行创新，以提供明天的突破性成果，并对人类健康产生深远影响。

Learn more at

了解更多请访问

https://www.jnj.com/

or at

或者在

https://innovativemedicine.jnj.com/

关注我们

@JNJInnovMed.

@JNJInnovMed

Janssen Research & Development, LLC and Janssen Biotech, Inc. are Johnson & Johnson companies.

扬森研发有限责任公司和扬森生物技术公司是强生公司的子公司。

CAUTIONS CONCERNING FORWARD-LOOKING STATEMENTS

关于前瞻性陈述的注意事项

This press release contains 'forward-looking statements' as defined in the Private Securities Litigation Reform Act of 1995 regarding product development and the potential benefits and treatment impact of nipocalimab. The reader is cautioned not to rely on these forward-looking statements. These statements are based on current expectations of future events.

本新闻稿包含《1995年私人证券诉讼改革法》中定义的关于产品开发以及nipocalimab潜在益处和治疗影响的“前瞻性陈述”。读者被警告不要依赖这些前瞻性陈述。这些陈述是基于对未来事件的当前预期。

If underlying assumptions prove inaccurate or known or unknown risks or uncertainties materialize, actual results could vary materially from the expectations and projections of Janssen Research & Development, LLC, Janssen Biotech, Inc. and/or Johnson & Johnson. Risks and uncertainties include, but are not limited to: challenges and uncertainties inherent in product research and development, including the uncertainty of clinical success and of obtaining regulatory approvals; uncertainty of commercial success; manufacturing difficulties and delays; competition, including technological advances, new products and patents attained by competitors; challenges to patents; product efficacy or safety concerns resulting in product recalls or regulatory action; changes in behavior and spending patterns of purchasers of health care products and services; changes to applicable laws and regulations, including global health care reforms; and trends toward health care cost containment.

如果基本假设被证明不准确，或已知或未知的风险或不确定性成为现实，实际结果可能与杨森研发有限责任公司、杨森生物科技公司和/或强生公司的预期和预测大相径庭。风险和不确定性包括但不限于：产品研发和开发过程中固有的挑战和不确定性，包括临床成功的不确定性和获得监管批准的不确定性；商业成功的不确定性；生产困难和延误；竞争，包括技术进步、竞争对手推出的新产品和获得的专利；对专利的挑战；因产品功效或安全问题导致的产品召回或监管行动；医疗保健产品和服务购买者的行为和支出模式的变化；适用法律法规（包括全球医疗改革）的变化；以及控制医疗成本的趋势。

A further list and descriptions of these risks, uncertainties and other factors can be found in Johnson & Johnson's most recent Annual Report on Form 10-K, including in the sections captioned 'Cautionary Note Regarding Forward-Looking Statements' and 'Item 1A. Risk Factors,' and in Johnson & Johnson's subsequent Quarterly Reports on Form 10-Q and other filings with the Securities and Exchange Commission.

这些风险、不确定性和其他因素的更多列表和描述，请参见强生公司最近的年度报告 Form 10-K，包括标题为“关于前瞻性陈述的警示声明”和“项目1A. 风险因素”的部分，以及强生公司随后的季度报告 Form 10-Q 和其他提交给证券交易委员会的文件。

Copies of these filings are available online at .

这些文件的副本可在线获取，网址为。

www.sec.gov

, www.jnj.com or on request from Johnson & Johnson. None of Janssen Research & Development, LLC, Janssen Biotech, Inc. nor Johnson & Johnson undertakes to update any forward-looking statement as a result of new information or future events or developments.

，www.jnj.com 或者可向 Johnson & Johnson 索取。Janssen Research & Development, LLC、Janssen Biotech, Inc. 以及 Johnson & Johnson 均不承担因新信息、未来事件或发展而更新任何前瞻性声明的义务。

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Roy S, Nanovskaya T, Patrikeeva S, et al. M281, an anti-FcRn antibody, inhibits IgG transfer in a human ex vivo placental perfusion model. Am J Obstet Gynecol. 2019;220(5):498 e491-498 e499.

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