阿利莫洛尔：首次批准。

IF 13 1区医学 Q1 PHARMACOLOGY & PHARMACY

Drugs Pub Date : 2025-01-01 Epub Date: 2024-12-24 DOI:10.1007/s40265-024-02129-x

Susan J Keam

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引用次数: 0

摘要

阿利莫洛尔（MIPLYFFA™）是一种口服小分子，可穿过血脑屏障，被认为可上调CLEAR（协调溶酶体表达和调节）网络基因并改善溶酶体功能，由Zevra Therapeutics公司开发，用于治疗尼曼-匹克病C型（NPC）的神经学表现。2024年9月，美国批准阿利莫洛莫与米卢司他联合用于治疗2岁及以上成人和儿童鼻咽癌的神经系统症状。本文总结了阿利莫洛尔的发展历程，并首次批准与米卢司他联合用于治疗年龄≥2岁的成人和儿童鼻咽癌患者的神经系统症状。

本文章由计算机程序翻译，如有差异，请以英文原文为准。

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Arimoclomol: First Approval.

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.

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来源期刊

Drugs 医学-毒理学

CiteScore

22.70

自引率

0.90%

发文量

134

审稿时长

3-8 weeks

期刊介绍： Drugs is a journal that aims to enhance pharmacotherapy by publishing review and original research articles on key aspects of clinical pharmacology and therapeutics. The journal includes: Leading/current opinion articles providing an overview of contentious or emerging issues. Definitive reviews of drugs and drug classes, and their place in disease management. Therapy in Practice articles including recommendations for specific clinical situations. High-quality, well designed, original clinical research. Adis Drug Evaluations reviewing the properties and place in therapy of both newer and established drugs. AdisInsight Reports summarising development at first global approval. Moreover, the journal offers additional digital features such as animated abstracts, video abstracts, instructional videos, and podcasts to increase visibility and educational value. Plain language summaries accompany articles to assist readers with some knowledge of the field in understanding important medical advances.