FDA OK NS Pharma’s viltolarsen for certain Duchenne patients

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.

https://seekingalpha.com/news/3604746-fda-ok-ns-pharmas-viltolarsen-for-certain-duchenne-patients