FDA给Sanofi的实验基因疗法提供快车道, 治疗罕见的DM1病症。 FDA grants Fast Track to Sanofi’s experimental gene therapy for rare DM1 disorder.

美国食品和药物管理局授予SAR446268号快车道称号,这是一个实验性AAV基因疗法,由Sanofi为非遗传性青少年和成人1型心肌衰竭(DM1)提供,这是一种罕见的遗传疾病,造成累进性肌肉弱弱、鼻孔炎和多机体损伤。 The U.S. FDA has granted Fast Track designation to SAR446268, an experimental AAV gene therapy by Sanofi for non-congenital juvenile and adult myotonic dystrophy type 1 (DM1), a rare genetic disorder causing progressive muscle weakness, myotonia, and multi-organ damage. 由于没有经批准的治疗方法,446268沙特阿拉伯里亚尔是临床开发中唯一的DM1疗法,目前处于人类第一一至二阶段试验阶段,对安全、可耐性和有效性进行评估。 With no approved treatments available, SAR446268 is the only DM1 therapy in clinical development, currently in a first-in-human Phase 1-2 trial evaluating safety, tolerability, and effectiveness. 治疗旨在解决这一疾病的根本原因。 The therapy aims to address the root cause of the disease. Sanofi在美国和欧盟也收到了4462668沙特阿拉伯里亚尔的 " 孤儿药品指认 " ,强调了它治疗这一严重、罕见情况的潜力。 Sanofi also received Orphan Drug Designation for SAR446268 in the U.S. and EU, highlighting its potential to treat this serious, rare condition.