FDA批准首个治疗 VII 型粘多糖贮积症的重组生物酶
今日，美国 FDA 宣布批准 Ultragenyx Pharmaceutical 的原研新药 Mepsevii（vestronidase alfa-vjbk）上市，用于治疗儿童及成人 VII 型粘多糖贮积症（MPS VII），也称为Sly综合征。在今天之前，患有这种罕见的遗传疾病的患者基本没有可靠的治疗方案。
MPS VII 是一种极为罕见的遗传病，全球影响了不到 150 名患者，而且每一名患者的症状都会有所不同。但绝大多数患者会有严重的骨骼异常，病情随着年龄增长不断加重。此外，患者还可能出现心脏瓣膜异常、肝脏脾脏增大、以及呼吸道狭窄等问题。根据病情严重程度的不同，一些患者会在婴儿期去世，而很多孩子将从婴幼儿消磨到青少年，灯枯油尽直至死亡。对于这些患者来说，他们急需一种有效的治疗手段来挽救生命。
这种疾病属于溶酶体贮积症的一种，病因是由于患者体内缺乏足够的β- 葡糖苷酸酶（β-glucuronidase）。因此，患者无法代谢特定的底物，造成有毒代谢产物在细胞中堆积，引起症状。今日获批的 Mepsevii 是一种酶替代疗法，能行使正常的酶活功能，帮助患者进行代谢，对症下药地治疗疾病。先前，这款创新疗法曾获得美国 FDA 颁发的孤儿药资格以及快速通道资格。
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