Avidity Biosciences Receives FDA Orphan Drug Designation for AOC 1020 for the Treatment of Facioscapulohumeral Muscular Dystrophy

Avidity Biosciences, a biopharmaceutical company committed to delivering a new class of RNA therapeutics called Antibody Oligonucleotide Conjugates (AOCs™), today announced that the US Food and Drug Administration (FDA) has granted Orphan Drug designation to AOC 1020 for the treatment of facioscapulohumeral muscular dystrophy (FSHD). FSHD is a serious, rare, hereditary muscle-weakening condition marked by life-long, progressive loss of muscle function that causes significant pain, fatigue, and disability.

AOC 1020 is being evaluated in the Phase 1/2 FORTITUDE™ clinical trial in adults with FSHD and is the company’s second muscle-targeting small interfering RNA (siRNA) AOC in clinical development. Avidity plans to share data from a preliminary assessment of AOC 1020 in approximately half of study participants from the FORTITUDE trial in the first half of 2024.

“We are pleased that the FDA has granted Orphan Drug designation to AOC 1020, reinforcing the importance of finding an effective treatment option for people living with FSHD. There are currently no treatment options to address this devastating and debilitating muscular dystrophy disorder,” said Steve Hughes, M.D., chief medical officer at Avidity. “AOC 1020 is designed to directly target the disease-causing gene, DUX4, with the goal of treating the underlying biological cause of FSHD. We look forward to advancing AOC 1020 and bringing this much-needed therapy to patients.”

The FDA’s Office of Orphan Drug Products grants orphan status to support the development of medicines for rare disorders that affect fewer than 200,000 people in the U.S. Orphan drug designation provides certain benefits, including market exclusivity upon regulatory approval, exemption of FDA application fees, and tax credits for qualified clinical trials.