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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 (trade name XERMELO) is an oral tryptophan hydroxylase inhibitor developed by Lescomx. February 2017, the United States FDA approved its use for the treatment of carcinoid syndrome diarrhea. As an orphan drug, XERMELO is given fast track and preemptive review qualifications, encouraging the development of drugs for rare diseases and taking a major step forward in improving the quality of life of patients. The author of XERMELO’s basic properties, mechanism of action, pharmacokinetics, pharmacodynamics, clinical trials and adverse reactions and other information to make a summary, hoping to provide clinical guidance and help to the hospital.