Golodirsen

Overview

Golodirsen is an antisense oligonucleotide used for the treatment of Duchenne muscular dystrophy (DMD) in patients with confirmed mutation of the DMD gene that is amenable to exon 53 skipping. It works by binding to exon 53 of dystrophin pre-mRNA, resulting in the exclusion of this exon during mRNA processing.

Patent & exclusivity (US)

Per the FDA Orange Book (1 approved US product), the latest listed US patent on Golodirsen expires 27 Jun 2028 and exclusivity 12 Dec 2026. Generic API sourcing typically opens up once these lapse.

Therapeutic areas