Viltolarsen: First Approval

被引：150

作者：

Dhillon, Sohita ^{[1
]}

机构：

[1] Springer Nat, Private Bag 65901, Auckland 0754, New Zealand

来源：

DRUGS | 2020年 / 80卷 / 10期

关键词：

DUCHENNE MUSCULAR-DYSTROPHY; TREATMENT PHASE-2; NS-065/NCNP-01; RESTORATION;

D O I：

10.1007/s40265-020-01339-3

中图分类号：

R9 [药学];

学科分类号：

1007 ;

摘要：

Viltolarsen (Viltepso(R) in Japan) is a phosphorodiamidate morpholino antisense oligonucleotide being developed by Nippon Shinyaku, in collaboration with the National Center of Neurology and Psychiatry (NCNP), for the treatment of Duchenne muscular dystrophy (DMD). Viltolarsen binds to exon 53 of the dystrophin mRNA precursor and restores the amino acid open-reading frame by skipping exon 53, resulting in the production of a shortened dystrophin protein that contains essential functional portions. In March 2020, intravenous viltolarsen received its first global approval in Japan for the treatment of DMD in patients with confirmed deletion of thedystrophingene that is amenable to exon 53 skipping. Viltolarsen is under regulatory review in the USA and clinical trials continue in the USA, Canada and globally. This article summarizes the milestones in the development of viltolarsen leading to the first approval for DMD.

引用

页码：1027 / 1031

页数：5

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