FDA快车道AnnJi实验药物AJ201用于稀有肌肉疾病SBMA,目的是在20多年内进行首次治疗。 FDA fast-tracks AnnJi’s experimental drug AJ201 for rare muscle disease SBMA, aiming for first treatment in over 20 years.

林业发展局授予AJ201快车道称号,这是安吉制药公司为脊椎和灯泡肌肉萎缩症(SBMA)进行的实验治疗,这是一种罕见的遗传性神经肌肉紊乱症,影响4万分之一的男性。 The FDA has granted Fast Track Designation to AJ201, an experimental therapy by AnnJi Pharmaceutical for spinal and bulbar muscular atrophy (SBMA), a rare genetic neuromuscular disorder affecting about 1 in 40,000 males. SBMA没有经批准的治疗办法。 SBMA has no approved treatments. AJ201,一种一流的小分子,旨在减少与该疾病有关的有毒蛋白质,并改进运动功能。 AJ201, a first-in-class small molecule, aims to reduce toxic proteins linked to the disease and improve motor function. 该指定以及先前的 " 孤儿药品标识 " ,旨在加快发展和审查。 The designation, along with prior Orphan Drug Designations, is intended to speed development and review. AnnJi计划将AJ201改为第3阶段审判,目的是在20年多的时间里首次提供经核准的SBMA治疗。 AnnJi plans to move AJ201 into Phase 3 trials, aiming to deliver the first approved SBMA treatment in over 20 years.