Dyne gets priority review for DMD therapy

• The FDA accepted Dyne Therapeutics’ BLA for z-rostudirsen to treat Duchenne muscular dystrophy patients amenable to exon 51 skipping.
• Dyne Therapeutics shares were up 3.1% at $16.02 following the regulatory update.
• The company said it is preparing for a potential U.S. launch in Q1 2027 if the therapy receives approval.

Dyne Therapeutics (NASDAQ:DYN) received FDA acceptance of its Biologics License Application for z-rostudirsen, an investigational treatment for Duchenne muscular dystrophy amenable to exon 51 skipping.

The FDA granted Priority Review and set a Prescription Drug User Fee Act target action date of January 21, 2027, under the Accelerated Approval pathway using dystrophin as a surrogate endpoint.

“Dystrophin production increased in a robust and statistically significant manner following treatment with z-rostudirsen,” said Dyne Therapeutics President and Chief Executive Officer John Cox.

The application is supported by data from the registrational expansion cohort of the DELIVER trial, where dosing every four weeks was associated with increased dystrophin levels, functional measures and a favorable safety profile.

Following the announcement, Dyne Therapeutics' share price was up 3.1% at $16.02.

Dyne said z-rostudirsen is designed to restore dystrophin production by enabling exon 51 skipping in eligible DMD patients.

The company is also developing four additional DMD candidates targeting exon skipping for exons 53, 45, 44 and 55.