WALTHAM, MASSACHUSETTS
Dyne's z-rostudirsen BLA accepted with Priority Review for exon 51 Duchenne muscular dystrophy
- Sponsor
- Dyne Therapeutics $DYN
- Drug
- z-rostudirsen
- Indication
- Duchenne muscular dystrophy amenable to exon 51 skipping
FDA accepted Dyne Therapeutics's Biologics License Application for z-rostudirsen (DYNE-251) with Priority Review, seeking Accelerated Approval for adults and children with Duchenne muscular dystrophy amenable to exon 51 skipping. PDUFA target action date is January 21, 2027.
What this means
Z-rostudirsen is an experimental drug designed to treat a specific genetic form of Duchenne muscular dystrophy by helping muscle cells produce a needed protein. The FDA has accepted Dyne's application for review and will give it a faster, six-month evaluation due to its potential importance. This could provide a new treatment option for patients with this rare and serious muscle-wasting disease.