- Nippon Shinyaku Co., Ltd. (OTC:NPNKF) unit NS Pharma announces the accelerated FDA nod for Viltepso (viltolarsen) for the treatment of patients with Duchenne muscular dystrophy (DMD) amenable to exon 53 skipping, a population representing ~8% of total DMD cases.
- Viltolarsen works by masking exon 53 causing cells to "skip" the exon when synthesizing mRNA thereby restoring the reading frame of mRNA and allowing the cell to synthesize a shorter but working dystrophin protein.
- Viltepso will compete directly with Sarepta Therapeutics' (SRPT -0.0%) Exondys 53 (golodirsen), conditionally approved in the U.S. in December 2019.
- Update: According to reports, the price/vial will be $1,410.