Lamb Yvette N
Springer Nature, Private Bag 65901, Mairangi Bay, Auckland 0754, New Zealand.
Drugs. 2025 Jun 23. doi: 10.1007/s40265-025-02197-7.
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.
替曲考尔（Emcitate）是一种口服生物可利用的小分子药物，由Egetis Therapeutics公司研发，用于治疗单羧酸转运蛋白8（MCT8）缺乏症。替曲考尔是甲状腺激素三碘甲状腺原氨酸（T3）的类似物和代谢产物，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。替曲考尔于2025年2月12日在欧盟首次获批，用于治疗从出生起就患有MCT8缺乏症（艾伦-赫恩登-达德利综合征）的患者的外周甲状腺毒症。替曲考尔将以350微克可分散片剂的形式提供。替曲考尔目前正在包括美国在内的其他几个国家进行针对MCT8缺乏症的临床开发。本文总结了替曲考尔开发过程中导致其首次获批用于治疗MCT8缺乏症的各个里程碑事件。
