Dyne submits Biologics License Application (BLA) to the FDA for Exon 51-skipping z-rostudirsen

CureDuchenne was an early funder of Dyne Therapeutics, which today announced they have submitted a BLA to the FDA for z-rostudirsen, for the treatment of individuals with Duchenne amenable to skipping exon 51.  This application is for Accelerated Approval based on dystrophin as a surrogate endpoint, and includes positive data from Dyne’s Phase 1/2 DELIVER clinical trial, which showed significant increase in dystrophin production with functional improvement across multiple clinical endpoints.  If approved under FDA Priority Review, z-rostudirsen could be available in Q1 2027. 

In parallel, Dyne has launched a Phase 3 confirmatory trial of z-rostudirsen, and is advancing 4 additional exon-skipping programs for exons 53, 45, 44 and 55.     

Link to press release:  https://www.globenewswire.com/news-release/2026/05/26/3300969/0/en/dyne-therapeutics-announces-submission-of-biologics-license-application-bla-to-u-s-fda-for-z-rostudirsen-in-exon-51-duchenne-muscular-dystrophy-dmd.html

Community letter:  https://www.dyne-tx.com/forzetto-trial-initiated/