高罗特西普：首个获批

Golodirsen: First Approval.

机构信息

Springer Nature, Private Bag 65901, Mairangi Bay, Auckland, 0754, New Zealand.

出版信息

Drugs. 2020 Feb;80(3):329-333. doi: 10.1007/s40265-020-01267-2.

PMID:32026421

Abstract

Golodirsen (Vyondys 53), an antisense oligonucleotide of the phophorodiamidate morpholino oligomer (PMO) subclass designed to induce exon 53 skipping, has been developed by Sarepta Therapeutics for the treatment of Duchenne muscular dystrophy (DMD). In December 2019, intravenous golodirsen received its first global approval in the USA for the treatment of DMD in patients with a confirmed mutation of the DMD gene that is amenable to exon 53 skipping, based on positive results from a phase I/II clinical trial. Golodirsen is in phase III clinical development for the treatment of DMD worldwide. This article summarizes the milestones in the development of golodirsen leading to this first approval for DMD.

摘要

戈洛迪森（Vyondys 53）是一种反义寡核苷酸，属于磷二酰胺吗啉寡聚物（PMO）亚类，旨在诱导外显子 53 跳跃，由 Sarepta Therapeutics 开发用于治疗杜氏肌营养不良症（DMD）。2019 年 12 月，静脉注射戈洛迪森在美国获得首次全球批准，用于治疗 DMD 患者，这些患者的 DMD 基因突变可通过外显子 53 跳跃得到改善，这是基于一项 I/II 期临床试验的积极结果。戈洛迪森正在全球范围内进行 III 期临床试验，以治疗 DMD。本文总结了戈洛迪森开发过程中的重要里程碑，这些里程碑促成了该药在 DMD 方面的首次批准。