Dyne Therapeutics receives early 2027 FDA decision date for Duchenne therapy, analysts anticipate smooth review

Dyne Therapeutics' investigational Duchenne muscular dystrophy therapy, zeleciment rostudirsen, has had its BLA accepted by the FDA with a target action date of Jan 21, 2027.

Analysts at Oppenheimer said they expect a smooth review and believe the drug could capture most of the exon-51 market because of stronger dystrophin expression and a more convenient dosing schedule.

The current standard of care, Sarepta Therapeutics’ Exondys 51, requires weekly dosing, whereas z-rostudirsen is designed for once-every-four-weeks administration, which analysts view as an advantage.

Early Phase 1/2 DELIVER trial data showed the therapy outperformed Exondys 51, leading Jefferies to describe the results as best-in-class.