Posted by Michael Wonder on 22 Feb 2024
Avidity Biosciences receives FDA rare paediatric disease designation for AOC 1044 for treatment of Duchenne muscular dystrophy in people with mutations amenable to exon 44 skipping
20 February 2024 - Avidity Biosciences today announced that the US FDA has granted rare paediatric disease designation to AOC 1044, the company's investigational therapy for the treatment of Duchenne muscular dystrophy in people living with mutations amenable to exon 44 skipping.
In addition to receiving rare paediatric disease designation, AOC 1044 has been granted orphan designation by the FDA and the EMA, and fast track designation by the FDA.
