肥厚型心肌病治疗新药:mavacamten

A new drug to treat hypertrophic cardiomyopathy:mavacamten

肥厚型心肌病为遗传性疾病,以心肌非对称性肥厚和左室流出道梗阻为主要特点,易引起恶性心律失常及猝死,临床治疗方法受限,目前药物治疗可不同程度地缓解病患症状,但不能从分子机制层面解决疾病。mavacamten是一种新型的心肌肌球蛋白ATP酶变构抑制剂,可减少横桥周期,降低心肌收缩力,减少心肌能量消耗。最近Ⅱ、Ⅲ期临床试验结果表明,mavacamten可改善肥厚型心肌病患者的运动能力、左室流出道(LVOT)峰值梯度压差、NYHA心功能分级等终点指标,还可显著降低左心室质量指数、左心室壁厚度、左心房容积,且安全性及耐受性良好。

Hypertrophic cardiomyopathy(HCM)is a common heritable cardiac muscle disease that characterized by asymmetric thickening of the left ventricular wall and blockage of the left ventricular outflow tract,which is associated with sudden mortality and dangerous cardiac arrhythmias.The myosin modulator mavacamten is a new ATPase-modifying inhibitor that can reduce the cross-bridge cycle,myocardial contractility and myocardial energy consumption.The results of recent Ⅱ and Ⅲ clinical trials showed that mavacamten improved exercise capacity,left ventricular outflow tract(LVOT)gradient,NYHA functional class in patients with HCM,and associated with significant reductions in left ventricular mass index,left ventricular wall thickness and left atrial volume index,with good safety and tolerability.