四盐酸三苯汀,从工作台到床边:叙述性综述。

IF 13 1区 医学 Q1 PHARMACOLOGY & PHARMACY Drugs Pub Date : 2024-10-17 DOI:10.1007/s40265-024-02099-0
C Omar F Kamlin, Timothy M Jenkins, Jamie L Heise, Naseem S Amin
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引用次数: 0

摘要

四盐酸三戊烯胺(TETA-4HCl,Cuvrior®)是一种活性分子为三乙烯四胺(三戊烯胺)的铜螯合剂,由 Orphalan 公司开发,以满足治疗威尔逊病的未满足需求。盐酸曲安奈德胶囊最初是为了满足对D-青霉胺(DPA)不耐受的患者的需求而开发的,其安全性已得到证实。盐酸三苯汀胶囊本身并不稳定,从生产、运输到患者在家中使用都需要严格的冷链储存条件。盐酸三苯汀四盐酸盐具有与众不同的、受专利保护的独特多晶型,可以大规模生产薄膜包衣片剂,其室温稳定性可达 36 个月。四盐酸三苯汀具有良好的药效学、药代动力学和新陈代谢特征,显示出可靠、可预测的剂量线性和剂量比例动力学。在一项前瞻性随机临床试验中,四盐酸三苯汀是唯一一种与 DPA 进行过比较的三苯汀制剂,在患有稳定型威尔逊病的成人患者中,其疗效不劣于 DPA。2022 年 4 月 28 日,美国食品和药物管理局批准 TETA-4HCl 用于对 DPA 无耐受性的威尔森氏病成人患者。全球多个国家的卫生部门已批准 TETA-4HCl 用于治疗对 DPA 不耐受的成人和 5 岁以上儿童,包括欧盟、英国、沙特阿拉伯、瑞士、哥伦比亚、澳大利亚、新西兰和中国。本文旨在全面回顾 TETA-4HCl 开发过程中的重要里程碑。
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Trientine Tetrahydrochloride, From Bench to Bedside: A Narrative Review.

Trientine tetrahydrochloride (TETA-4HCl, Cuvrior®) is a copper chelating agent with the active moiety triethylenetetramine (trientine), developed by Orphalan, Inc. to address the unmet needs in the treatment of Wilson disease. The journey from bench to bedside builds upon the documented safety profile of trientine hydrochloride capsules developed initially to meet the needs of individuals intolerant to D-penicillamine (DPA). Trientine hydrochloride capsules are inherently unstable requiring strict cold chain storage conditions from production, transportation, and use at home by the patient. Trientine tetrahydrochloride has a distinctive, patent-protected unique polymorphic form, which permits the production at scale of film-coated scored tablets deemed room temperature stable for 36 months. Trientine tetrahydrochloride is supported by a well-characterized pharmacodynamic, pharmacokinetic, and metabolic profile demonstrating reliable and predictable dose linearity and dose proportionality kinetics. Trientine tetrahydrochloride is the only trientine formulation that has been compared with DPA in a prospective randomized clinical trial, demonstrating non-inferiority to DPA in adults with stable Wilson disease. On 28 April, 2022, the US Food and Drug Administration approved TETA-4HCl for use in adult patients with Wilson disease who are de-coppered and tolerant to DPA. Health authorities in multiple countries worldwide have approved TETA-4HCl for the treatment of adults and children aged 5 years or more who are intolerant to DPA including the European Union, UK, Saudi Arabia, Switzerland, Colombia, Australia, New Zealand, and China. This article aims to provide a comprehensive narrative review of the key milestones in the development of TETA-4HCl.

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来源期刊
Drugs
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.
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