Keam Susan J
Springer Nature, Private Bag 65901, Mairangi Bay, Auckland, 0754, New Zealand.
Drugs. 2025 Jan;85(1):111-116. doi: 10.1007/s40265-024-02129-x. Epub 2024 Dec 24.
Arimoclomol (MIPLYFFA™), an oral small molecule that crosses the blood brain barrier and is thought to upregulate CLEAR (Coordinated Lysosomal Expression and Regulation) network genes and improve lysosomal function, is being developed by Zevra Therapeutics Inc., for the treatment of neurological manifestations of Niemann-Pick disease type C (NPC). In September 2024, arimoclomol was approved for use in combination with miglustat for the treatment of neurological manifestations of NPC in adult and pediatric patients 2 years of age and older in the USA. This article summarizes the milestones in the development of arimoclomol leading to this first approval for use in combination with miglustat for the treatment of neurological manifestations of NPC in adult and pediatric patients aged ≥ 2 years.
阿利莫克莫（MIPLYFFA™）是一种口服小分子药物，可穿越血脑屏障，被认为能上调CLEAR（溶酶体协调表达与调控）网络基因并改善溶酶体功能。Zevra Therapeutics公司正在研发该药物，用于治疗尼曼-匹克病C型（NPC）的神经表现。2024年9月，阿利莫克莫在美国被批准与米格鲁司他联合使用，用于治疗2岁及以上成人和儿童患者的NPC神经表现。本文总结了阿利莫克莫研发过程中的里程碑事件，这些事件促成了其首次获批与米格鲁司他联合用于治疗≥2岁成人和儿童患者的NPC神经表现。
