CureDuchenne was an early funder of Dyne Therapeutics, and we are pleased to share that the FDA has accepted the BLA (Biologics License Application) for z-rostudirsen for individuals with Duchenne amenable to skipping exon 51. This submission is for Accelerated Approval based on dystrophin as a surrogate endpoint, and the FDA has granted the application Priority Review with the PDUFA target action date set for January 21, 2027. The BLA includes data from the positive Phase 1/2 DELIVER trial, and Dyne’s global confirmatory Phase 3 trial is already underway.
Link to press release: https://investors.dyne-tx.com/news-releases/news-release-details/dyne-therapeutics-announces-us-fda-acceptance-biologics-license
Link to community letter: https://www.dyne-tx.com/fda-accepts-bla-for-z-rostudirsen
Want to learn more? Join us for a live CureDuchenne Community Webinar with Dyne Therapeutics on August 19, 2026. Hear directly from the Dyne team and ask your questions during the live Q&A.
Register here: https://lnkd.in/gFBTAs5a
The post The FDA will review Dyne’s application for Accelerated Approval of z-rostudirsen for skipping exon 51 appeared first on CureDuchenne.