Eteplirsen

Overview

Eteplirsen is an antisense oligonucleotide used in the treatment of Duchenne Muscular Dystrophy (DMD) in patients with a confirmed mutation of the DMD gene that is amenable to exon 51 skipping. It works by binding to exon 51 of dystrophin pre-mRNA, resulting in the exclusion of this exon during mRNA processing which theoretically leads to production of a truncated but functional form of dystrophin.

Patent & exclusivity (US)

Per the FDA Orange Book (2 approved US products), the latest listed US patent on Eteplirsen expires 14 Mar 2034. Generic API sourcing typically opens up once these lapse.

Therapeutic areas