米达美替尼：首次批准。

IF 13 1区医学 Q1 PHARMACOLOGY & PHARMACY

Drugs Pub Date : 2025-05-27 DOI:10.1007/s40265-025-02190-0

Sheridan M Hoy

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引用次数: 0

摘要

Mirdametinib （gomez - litm）是一种口服小分子丝裂原活化蛋白激酶1和2 （MEK1/2）抑制剂，由SpringWorks Therapeutics公司开发，用于治疗1型神经纤维瘤病（NF1）相关丛状神经纤维瘤（PN）。这是第一个在美国被批准用于治疗成人和儿童NF1相关PN的疗法，在美国，它适用于成人和2岁及以上患有NF1且症状性PN无法完全切除的儿童患者。mirdametinib在NF1-PN中的上市许可申请目前正在欧盟审查中。米达替尼治疗小儿低级别胶质瘤的临床研究正在进行中。本文总结了米达美替尼首次获批的发展里程碑。

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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Mirdametinib: First Approval.

Mirdametinib (GOMEKLI^TM) is an oral small molecule inhibitor of mitogen-activated protein kinase kinases 1 and 2 (MEK1/2) developed by SpringWorks Therapeutics for the treatment of neurofibromatosis type 1 (NF1)-associated plexiform neurofibromas (PN). It is the first therapy to be approved for the treatment of both adults and children with NF1-associated PN in the USA, where it is indicated for use in adult and paediatric patients 2 years of age and older with NF1 who have symptomatic PN not amenable to complete resection. A marketing authorisation application for mirdametinib in NF1-PN is currently under review in the EU. Clinical studies of mirdametinib for the treatment of paediatric low-grade glioma are ongoing. This article summarizes the milestones in the development of mirdametinib leading to this first approval.

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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.