替拉替考：首次批准。

IF 13 1区医学 Q1 PHARMACOLOGY & PHARMACY

Drugs Pub Date : 2025-06-23 DOI:10.1007/s40265-025-02197-7

Yvette N Lamb

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引用次数: 0

摘要

替拉ricol （Emcitate®）是Egetis Therapeutics公司开发的一种口服生物可利用小分子，用于治疗单羧酸转运蛋白8 （MCT8）缺乏症。替拉ricol是甲状腺激素三碘甲状腺原氨酸（T3）的类似物和代谢物，具有拟甲状腺作用，但与T3不同的是，它可以独立于MCT8进入细胞。tirricol于2025年2月12日在欧盟首次获得批准，用于治疗出生时MCT8缺乏症（Allan-Herndon-Dudley综合征）患者的周围性甲状腺毒症。替拉替考将以350µg分散片的形式提供。在包括美国在内的其他几个国家，替拉替康目前正在进行治疗MCT8缺乏症的临床开发。本文总结了替拉ricol发展的里程碑，导致首次批准用于MCT8缺乏症。

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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Tiratricol: First Approval.

Tiratricol (Emcitate^®) is an orally bioavailable small molecule being developed by Egetis Therapeutics for the treatment of monocarboxylate transporter 8 (MCT8) deficiency. Tiratricol, an analogue and metabolite of the thyroid hormone triiodothyronine (T3), has thyromimetic effects but differs from T3 in that it can enter cells independent of MCT8. Tiratricol received its first approval on 12 February 2025 in the European Union, for the treatment of peripheral thyrotoxicosis in patients with MCT8 deficiency (Allan-Herndon-Dudley Syndrome), from birth. Tiratricol will be available as 350 µg dispersible tablets. Tiratricol is currently undergoing clinical development for MCT8 deficiency in several other countries including the USA. This article summarizes the milestones in the development of tiratricol leading to this first approval for MCT8 deficiency.

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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.