Avidity Biosciences announced this week that it had received FDA Orphan Drug Designation for its experimental FSHD treatment, called AOC 1020. This does NOT mean the drug is now available for patients to take. But it’s great news, nonetheless. 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 exemption of FDA application fees and tax credits for qualified clinical trials. It also grants “market exclusivity,” which means AOCD 1020, if approved, will not have to compete with generic imitations for a period of time. All of this makes the drug a more attractive to investors.
Before Avidity can apply to the FDA for approval of AOC 1020, it will have to conduct a series of clinical trials to demonstrate the drug’s safety and effectiveness in treating FSHD. The drug will be evaluated in the Phase 1/2 FORTITUDE™ clinical trial in adults with FSHD. The trial has not yet begun but is expected to launch soon. 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.
To learn more about the science behind AOC 1020 and the design of the trial, watch our FSHD University webinar from February 14, 2023.