On August 12, 2020, the Food and Drug Administration (FDA) granted accelerated approval to Viltepso (viltolarsen; NS Pharma Inc.) injection for the treatment of Duchenne muscular dystrophy (DMD).
Viltepso is an antisense oligonucleotide indicated for the treatment of DMD in patients who have a confirmed mutation of the DMD gene that is amenable to exon 53 skipping. This indication is approved under accelerated approval based on an increase in dystrophin production in skeletal muscle observed in patients treated with Viltepso. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial.
A Hayes Emerging Technology Report on Viltepso is available here.
Food and Drug Administration (FDA). FDA Approves Targeted Treatment for Rare Duchenne Muscular Dystrophy Mutation [news release]. August 12, 2020. Available at: click here. Accessed August 13, 2020.