Regeneron

January 2026: Garetosmab update

The Food and Drug Administration has accepted Regeneron’s application for priority review of garetosmab, a potential treatment for adults living with FOP.  This means regulators in the United States will review the data more quickly than usual, with a decision expected by August 2026.

While garetosmab is not yet approved and there are still uncertainties ahead, this is a hopeful milestone for our community and for everyone working towards safe and effective treatments for FOP.

Regeneron has not yet applied for approval in the UK, so we don’t yet know what this will mean for families here, but we will be watching developments closely.

You can read the full press release here: Regeneron Official Press Release

September 2025: 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