Dyne Therapeutics, Inc. (DYN), a clinical-stage neuromuscular disease company, announced on Tuesday its submission of a Biologics License Application (BLA) to the U.S. Food and Drug Administration for Z-rostudirsen for the treatment of individuals with Duchenne muscular dystrophy (DMD) amenable to the skipping of exon 51.
Notably, Dyne Therapeutics has requested a priority review for Z-rostudirsen (Zeleciment rostudirsen, or DYNE-251), an investigational therapy for patients with DMD gene mutations amenable to exon 51 skipping.
DMD, a rare X-linked progressive neuromuscular disorder caused by mutations in the DMD gene. It results in a complete absence of dystrophin, a protein required to maintain muscle structure and function.
In the Phase 1/2 DELIVER clinical trial, Z- rostudirsen met its primary endpoint, and the data from the trial served as the basis for a potential BLA submission to the FDA.
In the registrational expansion cohort of the DELIVER trial, patients who were administered Z- rostudirsen showed a statistically significant increase in dystrophin production with functional improvement observed across multiple clinical endpoints and a favourable safety profile.
Also, the firm has proposed a dosage regimen of 20mg/kg administered intravenously once every 4 weeks.
The company continues to evaluate Z-rostudirsen in the long-term extension of the DELIVER trial and global Phase 3 FORZETTO clinical trial.
Dyne stated that if the FDA approves Z-rostudirsen as a potential drug for the treatment of DMD, it expects to launch in the U.S market in the first quarter of 2027.
The FDA has granted Fast Track and Rare Pediatric Disease designations for Z-rostudirsen. The European Medicines Agency (EMA) and the Ministry of Health, Labour and Welfare (MHLW) in Japan have granted orphan drug designation for the treatment of individuals with DMD amenable to exon 51 skipping.
In addition to Z-rostudirsen (DYNE-251), the company is advancing four potential candidates, DYNE-253, DYNE-245, DYNE-244, and DYNE-255, for the treatment of DMD amenable to skipping of exons 53, 45, 44, and 55.
According to Grand View Research, Duchenne muscular dystrophy (DMD) drugs market size was estimated at $3.47 billion in 2023 and is projected to reach $9.91 billion by 2030, growing at a Compound Annual Growth Rate or CAGR of 16.8% from 2024 to 2030.
DYN closed Tuesday's trading at $18.15, up 4.49%. In the overnight market, the stock is up 0.99% to $18.33.
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