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Avidity Biosciences Receives FDA Orphan Drug Designation for AOC 1044 for Treatment of Duchenne Muscular Dystrophy in People with Mutations Amenable to Exon 44 Skipping
SAN DIEGO, Aug. 15, 2023 /PRNewswire/ -- Avidity Biosciences, Inc. (Nasdaq: RNA), a biopharmaceutical company committed to delivering a new class of RNA
About this update from Atrium Therapeutics, Inc.
[{"type":"text","content":"SAN DIEGO, Aug. 15, 2023 /PRNewswire/ -- Avidity Biosciences, Inc. (Nasdaq: RNA), a biopharmaceutical company committed to delivering a new class of RNA therapeutics called Antibody Oligonucleotide Conjugates (AOCs™), today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug designation to AOC 1044, the company's investigational therapy in development for the treatment of Duchenne muscular dystrophy (DMD) in people with mutations amenable to exon 44 skipping (DMD44). 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.\n\n \n \n \n \n \n \n\n \nAOC 1044 is being assessed in the Phase 1/2 EXPLORE44™ clinical trial for people living with DMD44 and is the first of multiple AOCs in development at Avidity for the treatment of DMD. Avidity plans to share results from the healthy volunteer portion of the EXPLORE44 trial in the fourth quarter of 2023 and is now enrolling participants living with DMD44 into the study. In April 2023, AOC 1044 received FDA Fast Track designation for the treatment of DMD44.\n\"We are pleased that the FDA has granted both Orphan Drug and Fast Track designation to AOC 1044, highlighting the importance of advancing new treatments for people living with DMD,\" said Steve Hughes, M.D., chief medical officer at Avidity. \"There are currently no treatment options that target the underlying cause of DMD44. AOC 1044 is designed to specifically skip exon 44 of the dystrophin gene to enable the production of functional dystrophin protein. We look forward to advancing AOC 1044 in clinical development and bringing this very important treatment to patients as quickly and safely as possible.\"\nAvidity's proprietary AOCs are designed to combine the specificity of monoclonal antibodies (mAbs) with the precision of oligonucleotide therapies to target the root causes of diseases previously untreatable with RNA therapeutics. In the case of DMD, the disease is caused by a genetic mutation that prevents the body from producing the dystrophin protein, which protects muscle cells from injury during contraction. The lack of functional dystrophin leads to stress and tears of muscle cell membranes, resulting in muscle c...