Regeneron announces positive phase 3 trial in adults with ultra-rare genetic disorder fibrodysplasia

 

Demonstrating that Garetosmab Prevents Greater than 99% of Abnormal Bone Formation

Regeneron (NASDAQ: REGN) announced breakthrough results from its Phase 3 OPTIMA trial for garetosmab in treating fibrodysplasia ossificans progressiva (FOP), a rare genetic disorder. The trial met its primary endpoint, demonstrating that garetosmab reduced new bone lesions by 90-94% and achieved a greater than 99% reduction in total lesion volume compared to placebo.

The study involved 63 adult FOP patients who received either placebo or garetosmab (3 mg/kg or 10 mg/kg) every four weeks. Both dosing regimens showed high efficacy, with the 10 mg/kg dose reducing flare-ups by 89%. Based on these positive results, the Independent Data Monitoring Committee recommended transitioning placebo patients to garetosmab treatment. Regeneron plans to submit for U.S. regulatory approval by year-end 2025.

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