首个用于治疗类癌综合征腹泻的口服药物——XERMELO

The first oral drug used in the treatment of carcinoid syndrome diarrhea——XERMELO

特罗司他乙酯(telotristatetiprate,商品名XERMELO)是由莱斯康公司研制的口服色氨酸羟化酶抑制剂。2017年2月,美国FDA批准其用于类癌综合征腹泻的治疗。XERMELO作为孤儿药物,获得快速通道和优先审评资格,这是对发展罕见疾病药物的鼓励,同时也迈出了改善患者生活质量的重要一步。笔者就XERMELO的基本性质、作用机制、药动学、药效学、临床试验及不良反应等信息作一概述,希望能对医院临床用药提供帮助和指导。

Telotristatetiprate（XERMELO） is an oral tryptophan hydroxylase inhibitor developed by Lexicon. In February 2017, the FDA approved its treatment for carcinoid syndrome diarrhea. As an orphan drug, XERMELO has been granted with fast track and priority review, which is an encouragement to develop medicines for rare diseases and an important step in improving the life quality of patients. The development process, mechanism, pharmacokinetics, pharmacodynamics, clinical trials and adverse reactions of XERMELO were reviewed in this artical in order to guide its clinical administration.