Dyne Submits BLA for 20 mg/kg Z-Rostudirsen, Seeks Six-Month Approval and Q1 2027 Launch
DYN•Dyne Therapeutics has submitted a BLA to the FDA for z-rostudirsen, its exon 51 DMD therapy dosed at 20 mg/kg every four weeks, after Phase 1/2 data showed significant dystrophin increases and gains. The company requested Priority Review to shorten approval to six months, targeting U.S. launch in Q1 2027.
1. BLA Submission and Regulatory Plan
Dyne Therapeutics filed a Biologics License Application with the FDA for z-rostudirsen, its exon 51 skipping therapy dosed at 20 mg/kg every four weeks. The company has requested Priority Review, which would shorten the standard 10-month review to six months following the FDA’s 60-day filing review, and is targeting a U.S. launch in the first quarter of 2027.
2. Phase 1/2 DELIVER Trial Results
In the registrational expansion cohort of the global Phase 1/2 DELIVER trial, treatment with z-rostudirsen produced a robust and statistically significant increase in dystrophin production. Patients showed functional improvements across multiple clinical endpoints and z-rostudirsen demonstrated a favorable safety profile.
3. Broader DMD Pipeline
Beyond z-rostudirsen, Dyne is advancing four additional exon-skipping candidates: DYNE-253, DYNE-245, DYNE-244 and DYNE-255, targeting exons 53, 45, 44 and 55 respectively, to expand its Duchenne muscular dystrophy franchise.
