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 | Andecal MMP-9 Patient N=1 | MMP-9 Enzyme inhibitor | Part 1: Phase 2/3 study is currently recruiting participants (USA only) Part 2: This is planned to open after completion of Part 1 and is expected to include sites outside of the USA. |
| Mirum (Licensed from Incyte in May, 2026) | zilurgisertib | PROGRESS trial | ALK2 (ACVR1) kinase inhibitor | Cohort 1: Ages 12+ completed. Data filed with the FDA with decision on 12+ US approval expected September 2026.
Cohort 2: Ages 6 to <12 active, not 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. |
| Regeneron | garetosmab | OPTIMA trial | Anti-Activin A Antibody | Filed to FDA and EMA. Approval decisions are expected autumn 2026. |
| StopFOP Academic Consortium | saracatinib | StopFOP trial | ALK2 (ACVR1) kinase inhibitor | Phase 2a Completed. Awaiting results from Phase 2 study, expected summer 2026. |
