Pharmaceutical News Update
Although FOP is an ultra-rare genetic condition, we are fortunate to have a number of teams 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.
| Sponsor | Drug | Name / Known As | Mechanism | Status |
|---|---|---|---|---|
| ashibio | andecaliximab | MMP-9 Patient N=1 | MMP-9 Enzyme inhibitor | Phase 2/3 Active. Not recruiting |
| Incyte | zilurgisertib | PROGRESS trial | ALK2 (ACVR1) kinase inhibitor | Phase 2 Active. Recruiting |
| Ipsen | fidrisertib | FALKCON trial | ALK2 (ACVR1) kinase inhibitor | Terminated December 2025 |
| Ipsen | palovarotene | PIVOINE | Retinoic Acid Receptor Gamma Agonist | Study ended November 2024. Drug is now FDA-approved as Sohonos. It did not receive EMA approval so is not available in the UK. PIVOINE trial was a roll-over study. As of 2026, the study is in the “data cleaning and reporting” phase. |
| Regeneron | garetosmab | OPTIMA trial | Anti-Activin A Antibody | FDA Filing February 2026 EMA and other global health authority submissions underway or planned for first half of 2026 OPTIMA 2 Trial: for adolecents and children later in 2026 |
| StopFOP | saracatinib | StopFOP trial | ALK2 (ACVR1) kinase inhibitor | Phase 2a Active. Not recruiting Study completion expected May 2026 Awaiting results from Phase 2 study, 2026 |
