Rozanolixizumab：用于AChR或MuSK自身抗体阳性全身型重症肌无力的综述。

文献信息

中文摘要

Rozanolixizumab（rozanolixizumab-noli；RYSTIGGO®）是一种人源化免疫球蛋白G4P单克隆抗体，是一种皮下给药的新生儿Fc受体（FcRn）抑制剂，获批用于治疗成人乙酰胆碱受体（AChR）和肌肉特异性激酶（MuSK）自身抗体阳性（Ab+）全身型重症肌无力（gMG）。关键性、随机、安慰剂对照的III期MycarinG试验表明，与安慰剂相比，每周一次rozanolixizumab约7 mg/kg或约10 mg/kg的6周周期在第43天时在所有评估的重症肌无力特异性量表上产生了统计学显著且具有临床意义的改善。治疗获益迅速、持久，并且在重复的症状驱动rozanolixizumab周期中保持一致。在一项随机、开放标签的III期交叉试验中，自我给药的rozanolixizumab改善了MG症状，无论使用手动推注还是输注泵方法。Rozanolixizumab总体耐受性良好，具有可接受的安全性特征；最常见的不良事件是头痛、腹泻、发热和恶心。因此，rozanolixizumab是治疗AChR或MuSK Ab+ gMG成人患者的一种有效且总体耐受良好的疗法，为这些患者代表了一种便捷且有价值的额外治疗选择。

英文摘要

Rozanolixizumab (rozanolixizumab-noli; RYSTIGGO®), a humanised immunoglobulin G4P monoclonal antibody, is a subcutaneously administered neonatal Fc receptor (FcRn) inhibitor approved for the treatment of both acetylcholine receptor (AChR) and muscle-specific kinase (MuSK) autoantibody-positive (Ab+) generalised myasthenia gravis (gMG) in adults. The pivotal, randomised, placebo-controlled phase III MycarinG trial demonstrated that a 6-week cycle of once-weekly rozanolixizumab ≈ 7 mg/kg or ≈ 10 mg/kg resulted in statistically significant and clinically meaningful improvements across all myasthenia gravis-specific scales assessed at day 43 versus placebo. Treatment benefits were rapid, sustained and consistent across repeated symptom-driven rozanolixizumab cycles. In a randomised, open-label, phase III crossover trial, self-administered rozanolixizumab improved MG symptoms regardless of whether manual push or infusion pump method was used. Rozanolixizumab was generally well tolerated with an acceptable safety profile; the most common adverse events were headache, diarrhoea, pyrexia and nausea. Thus, rozanolixizumab is an efficacious and generally well tolerated therapy for adults with AChR or MuSK Ab+ gMG, representing a convenient and valuable additional treatment option for these patients.