IntraBio Inc., a leader in the discovery and development of innovative drugs for rare neurodegenerative diseases,  announced that the FDA  has approved Aqneursa (levacetylleucine) for the treatment of neurological manifestations of Niemann-Pick disease type C (NPC) in adults and pediatric patients weighing  greater than 15 kg.

IntraBio Inc.是发现和开发罕见神经退行性疾病创新药物的领导者，宣布FDA已批准Aqneursa（左旋乙酰亮氨酸）用于治疗成人和体重超过15公斤的儿科患者的C型尼曼匹克病（NPC）神经系统表现。

Aqneursa is the only FDA-approved stand-alone therapy indicated for the treatment of NPC..

AQNEUSA是FDA批准的唯一用于治疗NPC的独立疗法。。

NPC is a rare, inherited lysosomal disease that occurs in about 1 in 100,000 live births. Patients with NPC typically experience systemic, neurological and psychiatric symptoms that can be debilitating and significantly impact functional abilities. Until now, current treatment approaches have not addressed the debilitating effects of NPC on patients’ daily lives..

NPC是一种罕见的遗传性溶酶体疾病，约每100000例活产中就有1例发生。NPC患者通常会出现全身，神经和精神症状，这些症状可能会使人衰弱并严重影响功能。到目前为止，目前的治疗方法尚未解决NPC对患者日常生活的衰弱影响。。

The FDA approval is based on data from the IB1001-301 multinational, randomized, double-blind, placebo-controlled, pivotal clinical trial (NCT05163288), which evaluated the impact of Aqneursa on neurological symptoms and functioning in pediatric (aged 4 years and older) and adult patients (n=60) with a confirmed diagnosis of NPC..

FDA的批准基于IB1001-301多国随机双盲安慰剂对照关键临床试验（NCT05163288）的数据，该试验评估了AQNEUSA对确诊为NPC的儿科（4岁及以上）和成年患者（n=60）神经症状和功能的影响。。

The trial met the primary efficacy endpoint and all secondary endpoints across all cohorts receiving Aqneursa. Results from the study showed Aqneursa significantly improved neurological signs and symptoms and demonstrated functional benefits important to everyday life that were evident within 12 weeks.

该试验符合接受AQNEUSA的所有队列的主要疗效终点和所有次要终点。研究结果显示，AQNEUSA可显着改善神经系统症状和体征，并在12周内显示出对日常生活重要的功能益处。