Golodirsen: First Approval

• Published in: Drugs (New York, N.Y.) Vol. 80; no. 3; pp. 329 - 333
• Main Author: Heo, Young-A
• Format: Journal Article
• Language: English
• Published: Cham Springer International Publishing 01.02.2020; Springer Nature B.V
• Subjects: AdisInsight Report; Agreements; Antisense oligonucleotides; Drug Approval; Drug Development; Drug dosages; Duchenne's muscular dystrophy; Dystrophin; Dystrophy; FDA approval; Humans; Internal Medicine; Intravenous administration; Medicine; Medicine & Public Health; Muscular dystrophy; Muscular Dystrophy, Duchenne - drug therapy; Muscular Dystrophy, Duchenne - genetics; Musculoskeletal system; Mutation; Oligonucleotides - administration & dosage; Oligonucleotides - therapeutic use; Pharmacology/Toxicology; Pharmacotherapy; Proteins; United States > US
• ISSN: 0012-6667; 1179-1950
• DOI: 10.1007/s40265-020-01267-2

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.