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Avidity Biosciences: Receives FDA Orphan Drug Designation for AOC 1044

August 21, 2023

The U.S. Food and Drug Administration (FDA) recently granted orphan drug designation to AOC 1044, an experimental exon 44-skipping therapy for Duchenne muscular dystrophy (DMD) that’s being developed by Avidity Biosciences. EXPLORE44, the Phase 1/2 Trial of AOC 1044, is expected to enroll approximately 40 healthy volunteers and 24 participants in the United States with mutations amenable to exon 44 skipping (DMD44), ages seven to 27 years old.

Orphan drug designation is given to therapies with the potential to treat rare disorders, those that affect fewer than 200,000 people in the U.S. The designation confers certain incentives, including a guarantee of seven years of market exclusivity if AOC 1044 is ultimately approved.

The new designation comes just a few months after AOC 1044 received the FDA fast-track designation, designed to speed up the development of therapies with the potential to substantially improve care for serious or life-threatening conditions.

“We are pleased that the FDA has granted both Orphan Drug and Fast Track designation to AOC 1044. We look forward to advancing AOC 1044 in clinical development and bringing this very important treatment to patients as quickly and safely as possible.”

Steve Hughes, MD, Chief Medical Officer at Avidity Biosciences