Introduction

Acetylcholine receptor antibody-positive generalized myasthenia gravis (gMG) is effectively treated with symptomatic and immunosuppressive drugs but a proportion of patients has a persistent disease and severe adverse events (AEs). The unmet medical needs are specific immunosuppression and AE lowering. Eculizumab blocks C5 protecting neuromuscular junction from the destructive autoantibody effects. Phase II (Study C08-001) and III (ECU-MG-301) studies, with the open-label extension (ECU-MG-302), demonstrated eculizumab efficacy and safety in refractory gMG patients.

Areas covered

We provide an overview of eculizumab biological features, clinical efficacy, and safety in gMG patients, highlighting our perspective on the drug positioning in the MG treatment algorithm.

Expert opinion

Eculizumab has the potential to significantly change the immunosuppressive approach in gMG offering the opportunity to avoid or delay corticosteroids’ use due to its speed and selective mechanism of action. Eculizumab prescription will depend on: 1. ability to modify the natural disease course; 2. sustainability in the clinical practice (cost/effectiveness ratio); 3. drug-induced AE reduction. At present we are missing a controlled study on its use as a first-line treatment. We think that immunosuppression in MG will change significantly in the next years by adopting more focused ‘Precision Medicine’ approaches, and Eculizumab seems to satisfy such a promise.

中文翻译：

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依库丽单抗治疗重症肌无力。

介绍

乙酰胆碱受体抗体阳性的广义重症肌无力（gMG）可通过对症和免疫抑制药物进行有效治疗，但一部分患者患有持续性疾病和严重不良事件（AEs）。未满足的医疗需求是特异性的免疫抑制和AE降低。Eculizumab阻止C5保护神经肌肉接头免受破坏性自身抗体的影响。II期（研究C08-001）和III期（ECU-MG-301）以及开放标签扩展（ECU-MG-302）研究证明了依库丽单抗在难治性gMG患者中的疗效和安全性。

覆盖区域

我们对gMG患者的依库丽单抗生物学特征，临床疗效和安全性进行了概述，突出了我们对MG治疗算法中药物定位的看法。

专家意见

Eculizumab有潜力显着改变gMG中的免疫抑制方法，由于其速度和选择性作用机制，它有可能避免或延迟使用皮质类固醇。Eculizumab处方将取决于：1.改变自然疾病进程的能力；2.临床实践中的可持续性（成本/效果比）；3.药物引起的AE减少。目前，我们缺少对其用作一线治疗的对照研究。我们认为，通过采用更具针对性的“精密医学”方法，MG的免疫抑制作用将在未来几年发生显着变化，依库丽单抗似乎可以满足这一希望。