Clinical Report: FDA approves garetosmab for adults with FOP

Overview

The FDA has approved garetosmab for adults with fibrodysplasia ossificans progressiva (FOP) to reduce new heterotopic ossification lesions and flare-ups. The approval is based on findings from the phase 3 OPTIMA trial.

Background

Fibrodysplasia ossificans progressiva (FOP) is an ultra-rare genetic disorder characterized by the abnormal formation of bone in soft tissues. The condition affects approximately 900 patients worldwide.

Data Highlights

Key Findings

• Garetosmab 10 mg/kg resulted in 2 new HO lesions compared to 19 in the placebo group, representing a 90% reduction.
• Garetosmab 3 mg/kg resulted in 1 new HO lesion, representing a 94% reduction compared to placebo.
• Clinician-assessed flare-ups were 9 in the 10 mg/kg group, 53 in the 3 mg/kg group, and 66 in the placebo group.
• Serious treatment-emergent adverse events occurred in 2 patients receiving 10 mg/kg and 1 patient receiving 3 mg/kg.
• Adverse reactions in garetosmab-treated patients included abscess, acne, and oral ulcers.

Clinical Implications

Clinicians should be aware of the dosing regimen and the potential for serious adverse effects, particularly in pregnant patients.

Conclusion

Garetosmab's approval provides a new treatment option for adults with FOP.

Related Resources & Content

1. Regeneron Pharmaceuticals Inc., Press Release, 2026 -- FDA approves garetosmab for adults with FOP
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6. ICCFOP Treatment Guidelines
7. ENDO 2026 Presentation
8. Pasatru™ (garetosmab-grts) First and Only FDA-approved Treatment Demonstrating Reduction in New Heterotopic Ossification (HO) Lesions and Clinician-Assessed Flare-ups in a Placebo-controlled Trial in Adults with Fibrodysplasia Ossificans Progressiva (FOP) | Herald-Whig