> ## Content Index
> Fetch the complete content index at: https://www.fdaweb.com/llms.txt
> Use this file to discover other available public pages before exploring further.

# Nippon Shinyaku Completes Rolling NDA for Duchenne Drug
- URL: https://www.fdaweb.com/nippon-shinyaku-completes-rolling-nda-for-duchenne-drug/
- Published: 2019-10-03T12:00:00.000Z
- Updated: 2026-09-15T01:54:06.000Z
- Author: David McFarland
- Tags: Drugs, #legacy-id-D5145161

Nippon Shinyaku says it has completed submission of a rolling NDA for its viltolarsen that is under development to treat Duchenne muscular dystrophy amenable to exon 53 skipping. The company says the drug is expected to generate functional dystrophin proteins and be effective for Duchenne muscular dystrophy amenable to dystrophin exon 53 skipping.

FDA granted the drug rare pediatric disease, orphan drug, and fast track designations.

If approved, the drug could compete with Sarepta’s golodirsen injection that received an August FDA complete response letter that Sarepta [said](https://www.fdaweb.com/sarepta-golodirsen-complete-response-letter/) generally raised two concerns: the risk of infections related to intravenous infusion ports and renal toxicity seen in pre-clinical models of the drug and observed following administration of other antisense oligonucleotides.