Avidity Biosciences’ candidate for DMD designated Rare Pediatric Disease by FDA

Avidity Biosciences announced that the FDA has granted Rare Pediatric Disease designation to AOC 1044, the company’s investigational therapy for the treatment of Duchenne muscular dystrophy, or DMD, in people living with mutations amenable to exon 44 skipping – DMD44 -. AOC 1044 is being assessed in the Phase 1/2 EXPLORE44 trial for people living with DMD44 and is the first of multiple AOCs the company is developing for DMD. In addition to receiving Rare Pediatric Disease Designation, AOC 1044 has been granted Orphan Designation by the FDA and the EMA, and Fast Track Designation by the FDA. DMD is a rare genetic condition that is characterized by progressive muscle damage and weakness due to the loss of dystrophin protein that typically starts at a very young age. Currently, there are no therapies approved targeting exon 44.Avidity plans to provide a first look at AOC 1044 data in people living with DMD44 in 2H 2024.