Iptacopan：首次批准。

IF 13 1区医学 Q1 PHARMACOLOGY & PHARMACY Drugs Pub Date : 2024-05-01 Epub Date: 2024-03-22 DOI:10.1007/s40265-024-02009-4

Yahiya Y Syed

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引用次数: 0

摘要

Iptacopan（FABHALTA®）是诺华制药公司开发的一种口服补体B因子抑制剂，用于治疗补体介导的疾病。iptacopan作用于替代途径中补体5的上游，抑制末端补体介导的血管内溶血和补体3介导的血管外溶血。2023 年 12 月 5 日，iptacopan 在美国获批用于治疗成人阵发性夜间血红蛋白尿症（PNH）。本文总结了iptacopan 开发过程中的里程碑事件，这些事件促成了iptacopan 首次获批用于治疗 PNH。

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Iptacopan: First Approval.

Iptacopan (FABHALTA^®) is an oral complement Factor B inhibitor developed by Novartis Pharmaceuticals for the treatment of complement-mediated diseases. Acting upstream of complement 5 in the alternative pathway, iptacopan inhibits both terminal complement-mediated intravascular haemolysis and complement 3-mediated extravascular haemolysis. On 5 December 2023, iptacopan received approval in the USA for the treatment of adults with paroxysmal nocturnal haemoglobinuria (PNH). This article summarizes the milestones in the development of iptacopan leading to this first approval for PNH.

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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.

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