Hoy Sheridan M
Springer Nature, Private Bag 65901, Mairangi Bay, Auckland, 0754, New Zealand.
Drugs. 2024 Aug;84(8):969-975. doi: 10.1007/s40265-024-02063-y. Epub 2024 Jul 15.
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.
莫伏利沙福（XOLREMDI™）是由X4制药公司研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（也称为基质衍生因子-1）与CXCR4的结合。2024年4月，它成为美国首个获批用于治疗WHIM综合征（由疣、低丙种球蛋白血症、感染和粒细胞减少症的首字母缩写命名）的疗法，适用于年龄≥12岁的WHIM综合征患者，以增加循环中成熟中性粒细胞和淋巴细胞的数量。莫伏利沙福用于慢性中性粒细胞减少症的临床开发正在进行中。本文总结了莫伏利沙福的研发历程中的各个里程碑，这些里程碑促成了其首次获批用于年龄≥12岁的WHIM综合征患者，以增加循环中成熟中性粒细胞和淋巴细胞的数量。
