SPRING HOUSE, Pa., Aug. 29, 2024 /PRNewswire/ -- Johnson & Johnson (NYSE: JNJ) today announced the submission of a Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) seeking the first approval of nipocalimab globally for the treatment of people living with generalized myasthenia gravis (gMG). .

宾夕法尼亚州斯普林豪斯，2024年8月29日/PRNewswire/--强生公司（纽约证券交易所：JNJ）今天宣布向美国食品和药物管理局（FDA）提交生物制剂许可证申请（BLA），寻求全球首次批准nipocalimab用于治疗全身性重症肌无力（gMG）患者。

The application included data from the Phase 3 Vivacity-MG3 study which showed that outcomes for a broad population of antibody positive participants who received nipocalimab plus standard of care (SOC) were superior compared to those who received placebo plus SOC. The primary endpoint of the study measured improvement in the MG-ADLa score from baseline over 24 weeks and study participants included anti-AChR+, anti-MuSK+, and anti-LRP4+b antibody positive adults, which account for approximately 95 percent of the gMG patient population, making Vivacity-MG3 the first-and-only study to demonstrate sustained disease control in these subtypes.1,2 Safety and tolerability were consistent with other nipocalimab studies.3,4,5.

该应用包括来自第3阶段Vivacity-MG3研究的数据，该研究表明，接受nipocalimab加标准护理（SOC）的抗体阳性参与者的广泛人群的结果优于接受安慰剂加SOC的人群。该研究的主要终点测量了24周内MG ADLa评分从基线的改善，研究参与者包括抗AChR+，抗MuSK+和抗LRP4+b抗体阳性的成年人，这些成年人约占gMG患者人群的95%，这使得Vivacity-MG3成为第一个也是唯一一个证明这些亚型持续疾病控制的研究。1,2安全性和耐受性与其他nipocalimab研究一致。3,4,5。

'We are encouraged by the potential of nipocalimab to provide sustained disease control for people living with generalized myasthenia gravis, a chronic, life-long disease,' said Bill Martin, Ph.D., Global Therapeutic Area Head, Neuroscience, Johnson & Johnson Innovative Medicine. 'The filing for approval of nipocalimab represents an important step forward as Johnson & Johnson continues to push the boundaries of research to develop innovative solutions to treat autoantibody-driven diseases, building on decades of expertise in neuroscience and immunology.

强生创新医学神经科学全球治疗领域负责人比尔·马丁（BillMartin）博士说：“我们对尼泊卡利单抗为全身性重症肌无力（一种慢性终身疾病）患者提供持续疾病控制的潜力感到鼓舞。”申请批准nipocalimab是向前迈出的重要一步，因为强生公司在神经科学和免疫学领域拥有数十年的专业知识，继续推动研究领域的发展，以开发治疗自身抗体驱动疾病的创新解决方案。

We look forward to working with the FDA in their review of the data supporting the submission.'   .

我们期待着与FDA合作，审查支持提交的数据。”

Nipocalimab is the first-and-only FcRn blocker to demonstrate sustained disease control measured by improvement in MG-ADL when added to background SOC compared with placebo plus SOC over a period of six months of consistent dosing (every other week)c, which is the longest period of controlled safety and efficacy assessment of an FcRn blocker in gMG..

Nipocalimab是第一个也是唯一一个通过在6个月的持续给药（每隔一周）c期间加入背景SOC与安慰剂加SOC相比，通过改善MG-ADL来衡量持续疾病控制的FcRn阻滞剂，这是gMG中FcRn阻滞剂控制安全性和有效性评估的最长时期。。

Earlier this year at the American Academy of Neurology Annual Meeting, Johnson & Johnson presented data focused on the molecular properties of nipocalimab. Characteristics such as its high binding affinity and specificity to the immunoglobulin G (IgG) binding site of FcRn have the potential to differentiate nipocalimab in the FcRn blocker class of treatments.6 These properties, along with the dosing regimen chosen for the study, are thought to lower IgG, including IgG autoantibodies in diseases such as gMG and other autoantibody-driven diseases.7.

今年早些时候，强生公司在美国神经病学学会年会上提交了有关尼波卡利玛分子特性的数据。其对FcRn的免疫球蛋白G（IgG）结合位点的高结合亲和力和特异性等特征有可能在FcRn阻断剂类治疗中区分尼波卡利单抗[6]。这些特性以及为研究选择的给药方案被认为可以降低IgG，包括gMG和其他自身抗体驱动疾病中的IgG自身抗体。

About Generalized Myasthenia Gravis (gMG)

关于全身性重症肌无力（gMG）

Myasthenia gravis (MG) is an autoantibody disease in which autoantibodies target proteins at the neuromuscular junction, disrupt neuromuscular signaling, and impair or prevent muscle contraction.8 In MG, the immune system mistakenly attacks proteins at the neuromuscular junction by producing antibodies (e.g., anti-acetylcholine receptor [AChR], anti-muscle-specific tyrosine kinase [MuSK] or anti-low density lipoprotein-related protein 4 [LRP4]) that can block or disrupt normal functioning, preventing signals from transferring from nerves to muscles.9 The disease impacts an estimated 700,000 people worldwide.8 The disease affects both men and women and occurs across all ages, racial and ethnic groups, but most frequently impacts young women and older men.10 Roughly 50 percent of individuals diagnosed with MG are women, and about one in five of those women are of child-bearing potential.11,12,13.

重症肌无力（MG）是一种自身抗体疾病，其中自身抗体靶向神经肌肉接头处的蛋白质，破坏神经肌肉信号传导，损害或阻止肌肉收缩。8在MG中，免疫系统通过产生抗体（例如抗乙酰胆碱受体[AChR]，抗肌肉特异性酪氨酸激酶[MuSK]或抗低密度脂蛋白相关蛋白4[LRP4]）错误地攻击神经肌肉接头处的蛋白质，这些抗体可以阻断或破坏正常功能，阻止信号从神经转移到肌肉。9该疾病影响全世界约70万人。8该疾病影响男性和女性，发生在所有年龄段，种族和族裔群体中，但最常影响年轻女性和老年男性.10大约50%被诊断患有MG的人是女性，其中约五分之一的女性具有生育潜力[11,12,13]。

Initial disease manifestations are usually ocular but in 85 percent or more14,15 cases, the disease generalizes (gMG), which is characterized by fluctuating weakness of the skeletal muscles leading to symptoms like limb weakness, drooping eyelids, double vision and difficulties with chewing, swallowing, speech, and breathing.8,16,17 Approximately 100,000 individuals in the U.S.

最初的疾病表现通常是眼部的，但在85%或更多的病例中，该疾病是全身性的（gMG），其特征是骨骼肌的波动性无力，导致肢体无力，眼睑下垂，复视以及咀嚼，吞咽，言语和呼吸困难等症状。8,16,17美国约有100000人。

are living with gMG.18 Although gMG may be managed with current conventional therapies, new therapies are needed for those who may not respond well enough to or tolerate these options..

虽然gMG可能采用目前的常规疗法进行治疗，但对于那些对这些选择反应不佳或无法忍受的人来说，需要新的疗法。

About the Phase 3 Vivacity-MG3 Study

关于第三阶段Vivacity-MG3研究

The Phase 3 Vivacity-MG3 study was specifically designed to measure sustained efficacy and safety with consistent dosing in this unpredictable chronic disease where unmet need remains high. Antibody positive or negative adult gMG patients with insufficient response (MG-ADL ≥6) to ongoing SOC therapy were identified and 199 patients, 153 of which were antibody positive, enrolled in the 24-week double-blind placebo-controlled trial.

第3阶段Vivacity-MG3研究专门设计用于测量这种不可预测的慢性疾病的持续疗效和安全性，其中未满足的需求仍然很高。确定了对正在进行的SOC治疗反应不足（MG-ADL≥6）的抗体阳性或阴性成人gMG患者，并将199名患者（其中153名为抗体阳性）纳入了为期24周的双盲安慰剂对照试验。

Randomization was 1:1, nipocalimab plus current SOC (30 mg/kg IV loading dose followed by 15 mg/kg every two weeks) or placebo plus current SOC. Baseline demographics were balanced across arms (77 nipocalimab, 76 placebo). The primary endpoint of the study was mean change in MG-ADLa score from baseline over Weeks 22, 23 and 24 in antibody positive patients.

随机化为1:1，nipocalimab加上当前SOC（30 mg/kg IV负荷剂量，然后每两周15 mg/kg）或安慰剂加上当前SOC。基线人口统计学在各组之间平衡（77 nipocalimab，76安慰剂）。该研究的主要终点是抗体阳性患者在第22,23和24周的MG ADLa评分与基线的平均变化。

A key secondary endpoint included change in Quantitative Myasthenia Gravis (QMG) score, which is a 13-item assessment by a clinician that quantifies MG disease severity. Long-term safety and efficacy were further assessed in an ongoing OLE phase.19.

一个关键的次要终点包括定量重症肌无力（QMG）评分的变化，这是临床医生对MG疾病严重程度进行的13项评估。在正在进行的OLE阶段进一步评估了长期安全性和有效性。

About Nipocalimab

关于Nipocalimab

Nipocalimab is an investigational monoclonal antibody, purposefully designed to bind with high affinity to block FcRn and reduce levels of circulating immunoglobulin G (IgG) antibodies, while preserving immune function without causing broad immunosuppression. 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 Prevalent Rheumatology.19,20,21,22,23,24,25,26,27 Blockade of IgG binding to FcRn in the placenta is also believed to prevent transplacental transfer of maternal alloantibodies to the fetus.28,29.

Nipocalimab是一种研究性单克隆抗体，旨在以高亲和力结合以阻断FcRn并降低循环免疫球蛋白G（IgG）抗体的水平，同时保留免疫功能而不引起广泛的免疫抑制。这包括自身抗体和同种抗体，它们是自身抗体空间中三个关键部分的多种条件的基础，包括罕见的自身抗体疾病，由母体同种抗体介导的母胎疾病和流行的风湿病[19,20,21,22,23,24,25,26,27]。阻断IgG与胎盘中FcRn的结合也被认为可以防止母体同种抗体经胎盘转移到胎儿[28,29]。

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

美国食品药品管理局 (FDA) 和欧洲药品管理局 (EMA) 已授予 nipocalimab 几项重要称号，包括：

U.S. FDA Fast Track designation in hemolytic disease of the fetus and newborn (HDFN) and warm autoimmune hemolytic anemia (wAIHA) in July 2019, gMG in December 2021 and fetal neonatal alloimmune thrombocytopenia (FNAIT) in March 2024 by the FDAU.S. FDA Orphan drug status for wAIHA in December 2019, HDFN in June 2020, gMG in February 2021, chronic inflammatory demyelinating polyneuropathy (CIDP) in October 2021 and FNAIT in December 2023 by the FDAU.S.

美国 FDA 于 2019 年 7 月授予胎儿和新生儿溶血病 (HDFN) 和温抗体型自身免疫性溶血性贫血 (wAIHA) 快速通道资格，2021 年 12 月授予 gMG 快速通道资格，2024 年 3 月授予胎儿新生儿同种免疫性血小板减少症 (FNAIT) 快速通道资格。美国 FDA 于 2019 年 12 月授予 wAIHA 孤儿药资格，2020 年 6 月授予 HDFN 孤儿药资格，2021 年 2 月授予 gMG 快速通道资格，2021 年 10 月授予慢性炎症性脱髓鞘性多发性神经病 (CIDP) 孤儿药资格，2023 年 12 月授予 FNAIT 孤儿药资格

FDA Breakthrough Therapy designation for HDFN in February 2024 by the FDAEU EMA Orphan medicinal product designation for HDFN in October 2019 by the EMAAbout Johnson & Johnson .

2024年2月FDA对HDFN的突破性治疗指定由FDAEU EMA于2019年10月对HDFN的孤儿药品指定由Emabout Johnson＆Johnson于2019年10月指定。

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.  .

在强生公司，我们相信健康就是一切。我们在医疗保健创新方面的优势使我们能够建立一个预防、治疗和治愈复杂疾病的世界，在这个世界上，治疗更加智能，侵入性更小，解决方案更加个性化。通过我们在创新医学和医学技术方面的专业知识，我们拥有独特的优势，可以在今天的所有医疗保健解决方案中进行创新，以实现明天的突破，并深刻影响人类的健康。。