By Chris Wack
Avidity Biosciences said the U.S. Food and Drug Administration has granted a rare pediatric disease designation to AOC 1044, an investigational therapy for the treatment of Duchenne muscular dystrophy.
The biopharmaceutical company said AOC 1044 is being assessed in a Phase 1/2 trial for people living with Duchenne muscular dystrophy with mutations amenable to exon 44 skipping and is the first of multiple AOCs the company is developing for DMD.
In addition to receiving the rare pediatric disease designation, AOC 1044 has been granted an orphan drug designation by the FDA and the European Medicines Agency, and a fast track designation by the FDA.
In December, Avidity reported positive AOC 1044 data in healthy volunteers from the Phase1/2 trial.
Write to Chris Wack at [email protected]
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