FDA approves new treatment for FOP

Total new heterotopic ossification volume decreased with treatment and increased with placebo at week 24.

By
Kathryn Wighton
September 28, 2026
2 min

MDSpire News

Objective:

To reduce total new heterotopic ossification volume in patients with fibrodysplasia ossificans progressiva.

Approach:

Study Design: A randomized, double-blind, placebo-controlled trial with 63 patients, followed by a 292-week open-label extension.
Efficacy Assessment: Changes in total new heterotopic ossification volume were measured using whole-body computed tomography scans.

Key Findings:

Mean total new heterotopic ossification volume declined by 3.2 cm³ in the zilurgisertib group.
Mean total new heterotopic ossification volume increased by 24.6 cm³ in the placebo group at week 24.

Interpretation:

Zilurgisertib is effective in reducing new heterotopic ossification in patients with fibrodysplasia ossificans progressiva.

Limitations:

The study included a limited number of participants (63 patients).
Long-term effects and safety beyond the study duration are not fully established.

Conclusion:

Zilurgisertib has been approved by the FDA for the treatment of fibrodysplasia ossificans progressiva, with specific recommendations regarding its use and potential risks.