Avidity Biosciences has announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to AOC 1044 for the treatment of Duchenne muscular dystrophy (DMD) in people with mutations amenable to exon 44 skipping (DMD44).
AOC 1044 is being assessed in the Phase 1/2 EXPLORE44™ clinical trial for people living with DMD44 and is the first of multiple Antibody Oligonucleotide Conjugates (AOCs) the company is developing for DMD.
Fast Track designation enables more frequent interactions with the FDA to expedite the development and review process for drugs intended to treat serious or life-threatening conditions and that demonstrate the potential to address unmet medical needs. Currently, there are no therapies approved targeting exon 44.
“It is very encouraging to receive FDA Fast Track designation as it further validates the potential of AOC 1044 to target the underlying cause of DMD44 and the importance of bringing people living with this devastating disease an effective treatment option. This recognition also means that now all three of our clinical-stage programs have Fast Track status, further reinforcing our efforts to make a profound difference in people’s lives.”Steve Hughes, M.D., Chief Medical Officer at Avidity
EXPLORE44 is expected to enroll approximately 40 healthy volunteers and 24 participants with DMD44, ages seven to 27 years old, in the United States. Defeat Duchenne Canada is working with Avidity to understand plans for clinical trial enrollment in Canada.