Avidity Biosciences' AOC 1044 Gets FDA Rare Pediatric Disease Designation

(RTTNews) - Biopharmaceutical company Avidity Biosciences, Inc. (RNA), Tuesday announced that the U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease designation to AOC 1044, the company's investigational therapy for the treatment of Duchenne muscular dystrophy (DMD).

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 European Medicines Agency (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.

"We are pleased that the FDA has granted Rare Pediatric Disease designation to AOC 1044, adding to the Orphan Drug and Fast Track designations already granted. The effects of DMD44 are devastating, with symptoms often starting in childhood. These designations by the FDA underscore the urgent need for innovative treatments and validate the potential of AOC 1044 to address the unmet need of people living with Duchenne muscular dystrophy," said Steve Hughes, M.D., chief medical officer at Avidity.

"We recently shared healthy volunteer data of AOC 1044 from our Phase 1/2 EXPLORE44 trial demonstrating unprecedented delivery of therapeutic oligonucleotide in skeletal muscle and consistent exon skipping in healthy volunteers, and we look forward to sharing data from that study in people living with DMD44 later this year. We remain steadfast in our commitment to advancing science and improving the lives of people and their families affected by this devastating condition."