Novel Therapy for Duchenne Muscular Dystrophy Gets Fast Tracked

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There are currently no FDA-approved therapies targeting exon 44.

The 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).

Duchenne muscular dystrophy (DMD) is a rare genetic disorder caused by mutations in the DMD gene, which encodes the dystrophin protein. Lack of functional dystrophin results in the progressive loss of muscle function. In individuals with DMD44, AOC 1044 enables the production of dystrophin protein by delivering phosphorodiamidate morpholino oligomers to skeletal muscle and heart tissue to specifically skip exon 44 of the DMD gene.

The investigational treatment is being assessed in the phase 1/2 EXPLORE44 trial ( Identifier: NCT05670730). The study will evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamic effects of AOC144 administered as an intravenous infusion. Both healthy volunteers and DMD patients will be included; exon skipping and dystrophin protein levels will be assessed in DMD participants.

“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,” said Steve Hughes, MD, chief medical officer at Avidity. “We will continue to work closely with the FDA as we advance AOC 1044 and look forward to the anticipated data readout from the healthy volunteer portion of our phase 1/2 EXPLORE44 clinical trial later this year.”


Avidity Biosciences granted FDA Fast Track designation for AOC 1044 for treatment of Duchenne muscular dystrophy mutations amenable to exon 44 skipping. News release. Avidity Biosciences. April 24, 2023.