Johnson & Johnson announced that the FDA has approved Imaavy (nipocalimab-aahu), a human FcRn-blocking monoclonal antibody, for the treatment of generalized myasthenia gravis (gMG).

强生公司宣布，FDA已批准Imaavy（尼泊珠单抗-aahu），一种人源化的FcRn阻断单克隆抗体，用于治疗全身性重症肌无力（gMG）。

The approval, which follows FDA Priority Review designation, offers a new treatment option in a proven class with the potential for lasting disease control in the broadest population of people living with gMG (adults and pediatric patients 12 years of age and older who are anti-acetylcholine receptor [AChR] or anti-muscle-specific kinase [MuSK] antibody positive)..

FDA优先审查认定后的批准，在已验证的类别中提供了一种新的治疗选择，有可能为最广泛的gMG患者群体（12岁及以上的抗乙酰胆碱受体[AChR]或抗肌肉特异性激酶[MuSK]抗体阳性的成人和儿童患者）带来持久的疾病控制。

gMG is a chronic, debilitating autoantibody disease for which significant unmet patient need exists for additional efficacious therapies with demonstrated safety profiles that offer sustained disease control. Anti-AChR and anti-MuSK antibody positive individuals comprise ≥90% of the total antibody-positive gMG population.

gMG 是一种慢性、致残性的自身抗体疾病，患者对于额外的有效疗法有显著未满足的需求，这些疗法需具备已证明的安全性，并能提供持续的疾病控制。抗 AChR 和抗 MuSK 抗体阳性个体占总抗体阳性 gMG 人群的 ≥90%。

Imaavy is an immunoselective therapy designed to substantially reduce immunoglobulin G (IgG), including harmful IgG autoantibodies, without additional detectable effects on other adaptive and innate immune functions..

Imaavy是一种免疫选择性疗法，旨在大幅减少免疫球蛋白G（IgG），包括有害的IgG自身抗体，同时对其他适应性和先天性免疫功能没有额外可检测到的影响。

The approval is supported by data from the pivotal, ongoing Vivacity-MG3 study – the longest primary endpoint of a registrational trial of any FcRn blocker in adults with living with gMG. Highlights of the study include: i) · Imaavy plus standard of care (SOC) provided superior disease control throughout 24 weeks when compared to placebo plus SOC, as measured by improvement in the MG-ADL score.4 This translates into patients regaining essential daily functions, such as chewing, swallowing, speaking and breathing.

该批准得到了关键性、正在进行的 Vivacity-MG3 研究数据的支持，这是针对成年 gMG 患者的 FcRn 阻滞剂注册试验中持续时间最长的主要终点。研究亮点包括：i) Imaavy 加上标准治疗（SOC）在 24 周内相比安慰剂加 SOC 提供了更优的疾病控制，这通过 MG-ADL 评分的改善得以衡量。这意味着患者重新获得了诸如咀嚼、吞咽、说话和呼吸等基本日常功能。

ii) · Participants on Imaavy plus SOC maintained improvements out to 20 months of follow-up in the ongoing open-label extension (OLE) study in gMG. iii) · Imaavy demonstrated a rapid and sustained reduction in autoantibody levels by up to 75% from the first dose and throughout a 24-week period of monitoring..

ii) · 在持续开放标签扩展（OLE）研究中，接受Imaavy联合SOC治疗的gMG患者在长达20个月的随访期内维持了改善效果。iii) · Imaavy在首次给药后即显示出快速且持续的自身抗体水平降低，最大降幅达75%，并在24周的监测期内保持稳定。

“The clinical results we’ve seen with Imaavy represent a significant milestone in the treatment of gMG,” said Dr. Nicholas J. Silvestri, M.D., Professor of Neurology at University of Buffalo. “Patients experienced substantial symptom relief and lasting disease control that translated into better daily function and did not fade over 24 weeks in the pivotal Vivacity-MG3 study.

“我们在Imaavy的临床结果中看到的代表了gMG治疗的一个重要里程碑，”布法罗大学神经学教授尼古拉斯·J·西尔维斯特里博士表示。“患者在关键的Vivacity-MG3研究中，症状得到了显著缓解，并且疾病控制持久，转化为更好的日常功能，在24周内效果并未减退。”

Having a treatment that delivers this level of durable symptom stability is a meaningful step forward for managing a complex and unpredictable disease like gMG, and to have it in both AChR+ and MuSK+ adults and pediatric patients 12 years and older brings an additional FcRn treatment to a broader range of patients.”.

拥有一种能够提供这种持久症状稳定性的治疗方法，对于管理像gMG这样复杂且不可预测的疾病来说是一个有意义的进步，而该疗法适用于AChR+和MuSK+的成人以及12岁及以上的儿科患者，为更广泛的患者群体提供了额外的FcRn治疗选择。

Results from the ongoing Vibrance Phase II/III pediatric study in anti-AChR and anti-MuSK antibody positive adolescents aged 12-17 years showed that Imaavy plus SOC met its primary endpoint with a 69% reduction in total serum IgG over 24 weeks, and secondary endpoints of improvements in MG-ADL and QMG scales.

正在进行的 Vibrance II/III 期儿科研究的初步结果表明，针对 12-17 岁抗 AChR 和抗 MuSK 抗体阳性的青少年患者，Imaavy 联合标准治疗（SOC）达到了主要终点，在 24 周内总血清 IgG 减少了 69%，同时在 MG-ADL 和 QMG 量表的次要终点上也表现出改善。

Imaavy has demonstrated a consistent safety profile across both Vivacity-MG3 and the ongoing Vibrance-MG studies, with comparable tolerability in adult and pediatric populations..

伊玛维在Vivacity-MG3和正在进行的Vibrance-MG研究中展现了持续的安全性，成年和儿童群体的耐受性相当。

“Today’s FDA approval of Imaavy marks a historic milestone for the more than 240 million patients suffering with autoantibody diseases, many with few or no approved targeted treatments,” said David Lee, M.D., Ph.D., Global Immunology Therapeutic Area Head, Johnson & Johnson Innovative Medicine. “This approval is the result of years of scientific commitment, collaboration and determination for our nipocalimab program, and we’re proud to bring this new treatment option to patients living with anti-AChR or anti-MuSK antibody positive gMG.”.

“今天FDA批准Imaavy标志着超过2.4亿自身抗体疾病患者的历史性里程碑，其中许多患者几乎没有或根本没有获批的靶向治疗，”约翰逊约翰逊创新药物公司全球免疫治疗领域负责人David Lee博士表示。“这一批准是我们nipocalimab项目多年科学承诺、合作和决心的结果，我们很自豪能为抗AChR或抗MuSK抗体阳性的gMG患者带来这一新的治疗选择。”

Health authority submissions seeking approval for nipocalimab in the treatment of gMG are currently under review with numerous regulatory authorities worldwide.

目前，寻求批准nipocalimab用于治疗gMG的健康权威机构提交的文件正在接受全球众多监管机构的审查。

About the Phase III Vivacity-MG3 study - The Phase III Vivacity-MG3 study (NCT04951622) was specifically designed to measure sustained efficacy and safety with consistent dosing in this unpredictable chronic condition 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 whom were antibody positive, enrolled in the 24-week double-blind placebo-controlled trial.

关于第三阶段Vivacity-MG3研究 - 第三阶段Vivacity-MG3研究（NCT04951622）专门设计用于在这种不可预测的慢性疾病中通过持续剂量来评估持久的有效性和安全性，目前该病的未满足需求仍然很高。筛选出对现有标准治疗（SOC）反应不足（MG-ADL ≥6）的抗体阳性或阴性的成年gMG患者，并在24周的双盲安慰剂对照试验中招募了199名患者，其中153名为抗体阳性。

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.4 Baseline demographics were balanced across arms (77 nipocalimab, 76 placebo). The primary efficacy endpoint was the comparison of the mean change from baseline to Weeks 22, 23, and 24 between treatment groups in the MG-ADL total score.

随机分组为1:1，接受尼波卡利单抗加当前标准治疗（30 mg/kg 静脉负荷剂量，随后每两周 15 mg/kg）或安慰剂加当前标准治疗。基线人口统计学在各组之间均衡（77 名尼波卡利单抗，76 名安慰剂）。主要疗效终点是比较治疗组之间从基线到第 22、23 和 24 周的 MG-ADL 总分的平均变化。

A key secondary endpoint included change in QMG score. Long-term safety and efficacy were further assessed in an ongoing open-label extension (OLE) phase..

一个关键的次要终点包括QMG评分的变化。长期安全性和有效性在正在进行的开放标签扩展（OLE）阶段中得到了进一步评估。

About the Phase II/III Vibance-MG study - The Phase II/III Vibrance-MG study (NCT05265273) is an on-going open-label study to determine the effect of nipocalimab in pediatric participants with gMG. Seven participants aged 12-17 years with a diagnosis of gMG as reflected by a Myasthenia Gravis Foundation of America (MGFA) Class of II through IV at screening, and an insufficient clinical response to ongoing, stable SOC therapy, have been enrolled in the trial.

关于第二/三期Vibance-MG研究 - 第二/三期Vibrance-MG研究（NCT05265273）是一项正在进行的开放标签研究，旨在评估nipocalimab在患有gMG的儿科参与者中的效果。已有七名12-17岁、在美国重症肌无力基金会（MGFA）分级为II至IV级的gMG诊断患者参与试验，这些患者对当前稳定的常规治疗反应不佳。

Participants must have a positive blood test for either anti-AChR or anti-MUSK autoantibodies. The study consists of a screening period of up to four weeks, a 24-week open-label Active Treatment Phase during which participants receive nipocalimab intravenously every two weeks, and a Long-term Extension Phase; a safety follow-up assessment will be conducted at eight weeks after last dose.

参与者必须对抗AChR或抗MUSK自身抗体的血液检测呈阳性。研究包括最长四周的筛选期、为期24周的开放标签活性治疗阶段（在此期间参与者每两周接受一次静脉注射尼波卡利单抗）以及长期延长期；最后一次给药后八周将进行安全性随访评估。

The primary outcome of the study is the effect of nipocalimab on total serum IgG, safety and tolerability, and pharmacokinetics in pediatric participants with gMG at 24 weeks. Secondary endpoints include change in MG-ADL and QMG scores at 24 weeks..

研究的主要结果是nipocalimab对患有gMG的儿科参与者在24周时总血清IgG、安全性和耐受性以及药代动力学的影响。次要终点包括24周时MG-ADL和QMG评分的变化。

Condition:

条件：

Myasthenia Gravis

重症肌无力

Type:

类型：

drug

药物