GtoPdb Ligand ID: 7534

Synonyms: AVI-4658 | Exondys 51®
eteplirsen is an approved drug (FDA (2016))
Compound class: Synthetic organic
Comment: Eteplirsen is a novel oligonucleotide drug for the treatment of Duchenne muscular dystrophy (DMD), caused by specific reading frame mutations in the dystrophin gene [2]. Structurally it is a 30 base morpholino antisense oligomer in which a six-membered morpholine ring replaces the ribose sugars, and a phosphorodiamidate group replaces the normal anionic phosphodiester links between bases. This type of nucleotide is sometimes referred to as a phosphorodiamidate morpholino oligo, or PMO. While the size of this causes molecular indexing and rendering problems two images of the chemical structure were found. The first from the NIH US National Library of Medicine and they provide the InChIKey KZOVCSBYYXUVDW-UHFFFAOYSA-N. The second is ChemSpider 34983391 with a different InChIKey GHBMWAGCLOEKQK-BMBJILFMSA-N
2D Structure
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No information available.
Summary of Clinical Use
In September 2016, the US FDA approved eteplirsen for DMD patients carrying mutations amenable to exon 51 skipping.
Mechanism Of Action and Pharmacodynamic Effects
The lack of dystrophin protein which gives rise to the fatal X-linked disease, Duchenne muscular dystrophy (DMD) is caused by mutations that disrupt the gene's reading frame and prevent full protein translation. Eteplirsen is designed to correct a specific exon 51 mutation, by annealing to a splicing element in the dystrophin mRNA which promotes skipping of exon 51 [1]. The aim is to restore the open reading frame for improved protein synthesis [2].