FDA Clears Next Step for Forazapadin in FSHD
The news in brief
Satellos Bioscience, a drug development company, announced that the U.S. Food and Drug Administration (FDA) has cleared its application to study a drug called forazapadin in people with FSHD. This clearance means the company is allowed to begin testing the drug in a phase 2 clinical trial.
Satellos also announced a partnership with the FSHD Canada Foundation, which has agreed to provide up to US$5 million to help fund the trial.
About the drug
Forazapadin is a pill taken by mouth. It is designed to help the body repair and regrow muscle, a natural process that is disrupted in FSHD patients. The drug works by blocking a protein called AAK1, which the company believes may help restore that muscle repair process.
Forazapadin is also being studied in Duchenne muscular dystrophy (DMD). Early results from adults with DMD showed a favorable safety profile, less fat in the muscle on MRI scans, and increased effort during physical tasks after six months. The company believes these findings may be consistent with muscle regeneration, but these are early results and need to be confirmed in larger studies.
The planned FSHD trial
- Start date: Expected in the fourth quarter of 2026
- Type: Phase 2 trial, which looks at safety and early signs of whether a drug works
- Design: Randomized, double-blind, and placebo-controlled. Participants are randomly assigned to receive either the drug or a placebo, and neither participants nor researchers know who is receiving which.
- Who can take part: Adults aged 18 and older living with FSHD
- Doses: Two dose levels, 60 mg and 120 mg
- What it will measure: Safety, how well the drug is tolerated, how the body processes it, and whether it may help
How the study is being funded
The FSHD Canada Foundation has agreed to provide up to US$5 million over the next five quarters to help pay for the study. In exchange, the Foundation will receive a capped share of any future revenue Satellos earns from the drug in FSHD. This is called non-dilutive financing, meaning Satellos does not have to sell more company shares to raise the money.
The FSHD Canada Foundation thanks Solve FSHD, the FSHD Society, and FSHD Global for their support in making this possible.
What this means for the FSHD community
Neil Camarta, co-founder of the FSHD Canada Foundation and someone living with FSHD, said the community wants treatments that stop muscles from getting weaker, and also hopes to get some muscle back. He described this as an important step, while noting that there is still a long road ahead. His message: “Time is muscle!”
Important to keep in mind
- FDA clearance to start a study is not approval of the drug. Forazapadin has not yet been shown to work in people with FSHD.
- There are currently no approved therapies for FSHD.
- Phase 2 results will take time, and early findings may not be repeated in later studies.