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.
So if all goes well, maybe the general FSHD population will have access to this therapeutic by the mid 2030s? The process of moving therapeutics from the beginning of trials to approval for the general FSHD population is simply many times far too slow. I recently read an article from FDA’s CBER talking about getting to an Operation Warp Speed process for biologic genetic based therapeutics and in particular those for slowly progressing diseases. Operation Warp Speed was the process to get covid vaccines to the population in months rather than the decade or more that the normal approach with trials would have taken. It’s great that the FDA sees this as an issue, especially for slowly progressing diseases, but the FDA will probably still be just talking about this a decade or two from now.
hello i am FSHD muscle patient i am 24 years old i live in turkey i want to be treated i would be glad if you help me
Hello, my aunt suffers from FSHD and would like to participate in trial.