Mavorixafor：首次批准。

IF 13 1区医学 Q1 PHARMACOLOGY & PHARMACY Drugs Pub Date : 2024-08-01 Epub Date: 2024-07-15 DOI:10.1007/s40265-024-02063-y

Sheridan M Hoy

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引用次数: 0

摘要

Mavorixafor（XOLREMDI™）是X4制药公司开发的一种口服选择性C-X-C趋化因子受体4（CXCR4）拮抗剂，可阻断C-X-C趋化因子配体12（又称基质衍生因子-1）与CXCR4的结合。2024 年 4 月，它在美国成为首个获准用于 WHIM 综合征（WHIM 综合征是根据其观察到的特征--尖锐湿疣、低丙种球蛋白血症、感染和骨髓性贫血--的首字母缩写而命名）的疗法，适用于年龄≥ 12 岁的 WHIM 综合征患者，以增加循环中的成熟中性粒细胞和淋巴细胞的数量。mavorixafor用于慢性中性粒细胞减少症的临床开发正在进行中。本文总结了mavorixafor开发过程中的里程碑式事件，这也是mavorixafor首次获批用于年龄≥12岁的WHIM综合征患者，以增加循环中的成熟中性粒细胞和淋巴细胞的数量。

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Mavorixafor: First Approval.

Mavorixafor (XOLREMDI™) is an oral, selective C-X-C chemokine receptor 4 (CXCR4) antagonist developed by X4 Pharmaceuticals that blocks the binding of C-X-C chemokine ligand 12 (also known as stromal derived factor-1) to CXCR4. In April 2024, it became the first therapy to be approved for WHIM syndrome (named by an acronym for its observed characteristics of Warts, Hypogammaglobulinaemia, Infections and Myelokathexis) in the USA, where it is indicated for use in patients aged ≥ 12 years with WHIM syndrome to increase the number of circulating mature neutrophils and lymphocytes. Clinical development of mavorixafor is ongoing for chronic neutropenic disorders. This article summarizes the milestones in the development of mavorixafor leading to this first approval for use in patients aged ≥ 12 years with WHIM syndrome to increase the number of circulating mature neutrophils and lymphocytes.

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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.