Suvodirsen

Suvodirsen is a stereopure antisense oligonucleotide developed by Wave Life Sciences for the treatment of Duchenne muscular dystrophy (DMD) in patients amenable to exon 51 skipping[1][2][3][5]. It was designed to bind specifically to exon 51 of the dystrophin pre-mRNA, promoting exon skipping during mRNA processing. This mechanism aims to restore the reading frame of DMD mRNA and enable production of a truncated but functional dystrophin protein in patients with certain mutations[1][2][3]. Suvodirsen was granted orphan drug designation and rare pediatric disease designation for DMD by regulatory agencies. Clinical development included phase 1 and phase 2/3 trials; however, all clinical development was discontinued after December 2019 due to lack of efficacy[3][4].

Brand names

suvodirsen

Other names

suvodirsen sodium