2023-04-24 10:26:48 ET
RNA therapeutics company Avidity Biosciences ( NASDAQ: RNA ) announced Monday that the FDA issued Fast Track designation for AOC 1044, its candidate for genetically driven muscular disorder Duchenne muscular dystrophy (DMD).
The agency has issued the designation for AOC 1044 targeting DMD patients with mutations amenable to exon 44 skipping (DMD44).
Avidity's ( RNA ) Phase 1/2 EXPLORE44 trial for AOC 1044 is currently underway, with results from its healthy volunteer portion expected in H2 2023.
The FDA offers the Fast Track designation to accelerate the development and review of drugs targeted at serious conditions with unmet medical needs. It allows developers to communicate frequently with the regulator on plans for clinical studies.
If certain criteria are met, such programs will also be able to win Accelerated Approval and Priority Review, enabling patients to access those treatments sooner.
Read more on FDA and DMD
Sarepta ( SRPT ) stock -20% on FDA advisory meeting for its muscular dystrophy treatment
Sarepta announces May AdCom for muscular dystrophy therapy
Sarepta sheds 11% as report hints at early FDA opposition to gene therapy
For further details see:
Avidity wins FDA fast track status for Duchenne candidate