Avidity Biosciences’ AOC 1044 granted FDA Orphan Drug designation in DMD

Avidity Biosciences announced that the FDA has granted Orphan Drug designation to AOC 1044, the company’s investigational therapy in development for the treatment of Duchenne muscular dystrophy, or DMD, in people with mutations amenable to exon 44 skipping. 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. AOC 1044 is being assessed in the Phase 1/2 EXPLORE44 clinical trial for people living with DMD44 and is the first of multiple AOCs in development at Avidity for the treatment of DMD. Avidity plans to share results from the healthy volunteer portion of the EXPLORE44 trial in Q4 and is now enrolling participants living with DMD44 into the study. In April 2023, AOC 1044 received FDA Fast Track designation for the treatment of DMD44.