for Exon 51 Duchenne muscular dystrophy
Sponsor announced submission . FDA filing review (~60 days) in progress; PDUFA date estimated as acceptance + 10 months Standard Review. Card updates with confirmed dates when FDA acceptance announcement lands.
Duchenne muscular dystrophy (DMD) is a severe muscle-wasting disease that starts weakening boys' legs by age four, often leaving them unable to walk by their early teens as the damage spreads to their arms and spine. Z-Rostudirsen is a targeted therapy designed to skip over exon 51 in the dystrophin gene, potentially allowing muscle cells to produce a shorter but functional version of this critical structural protein. By restoring some dystrophin production, the drug may help slow the muscle degeneration that robs patients of mobility.
The purpose of the study is to assess the efficacy, safety, and tolerability of zeleciment rostudirsen (DYNE-251) administered intravenously (IV) every 4 weeks to ambulatory Duchenne muscular dystrophy (DMD) participants, 4 to 18 years of age, with dystrophin mutations amenable to exon 51 skipping.
Source: ClinicalTrials.gov