NS Pharma's Viltepso now FDA-approved for the treatment of DMD

The US Food and Drug Administration has approved Viltepso (viltolarsen) from NS Pharma for patients with Duchenne muscular dystrophy (DMD) who are amenable to exon 53 skipping therapy.

NS Pharma is a wholly-owned subsidiary of Japan’s Nippon Shinyaku (TYO: 4516), whose shares closed up 6.8% at 8,780 yen following the news.

Viltepso, a rival to Sarepta Therapeutics’ already approved Vyondys 53 (golodirsen) in this group of patients, received an accelerated approval by the FDA based on an increase in dystrophin, a key protein for supporting muscle health. Since a lack of dystrophin is the underlying cause of DMD, increasing dystrophin as much and as early as possible is a key goal in the treatment of DMD. Viltepso is the first and only exon 53 skipping therapy to demonstrate an increase in dystrophin in children as young as four years old.