Pharmaceutical News Update
Although FOP is an ultra-rare genetic condition, we are fortunate to have a number of pharmaceutical companies who are actively researching into a treatment and a cure for FOP.
We are thankful to everyone who is working towards a treatment and a cure for those living with FOP.
OPTIMA Phase 3 Trial Update
Regeneron’s Phase 3 OPTIMA trial for garetosmab showed highly positive results, with up to a 99% reduction in abnormal bone formation in adults with FOP. Following these promising results, Regeneron plans to seek US marketing approval from the FDA by the end of 2025. Submissions for other regions, including the UK, and a trial for children will follow, but the full data is still undergoing regulatory review.
Trustee Dr Andrew Rankin has written a summary of the report here: OPTIMA Summary September 2025
For the full press release, visit: Regeneron
Andecaliximab by Āshibio
Āshibio is a clinical-stage biotechnology company focused on developing treatments for bone and connective-tissue disorders, including FOP. Their lead programme, andecaliximab, is a humanised monoclonal antibody that targets MMP‑9, an enzyme linked to flare-ups and abnormal bone formation in FOP. Research suggests that reduced or altered MMP‑9 activity may help protect against heterotopic ossification, supported by both human genetic studies and animal models.
Read more here: Andecaliximab
Visit the website: Āshibio
