The FDA will review Dyne’s application for Accelerated Approval of z-rostudirsen for skipping exon 51

FDA Will Review Dyne Application for Skipping Exon 51

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