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Sep 17, 2025 12:50 PM

Regeneron Touts Positive Data From Phase 3 Study In Patients With Ultra-Rare Genetic Disorder

Regeneron Pharmaceuticals Inc. (NASDAQ:REGN) reported positive results from its Phase 3 OPTIMA trial of garetosmab in adults with fibrodysplasia ossificans progressiva (FOP), meeting the study's primary endpoint.

FOP is a rare genetic disorder in which muscle, tendon, and ligament tissue progressively turn into bone, creating a "second skeleton" that limits mobility.

At 56 weeks, garetosmab at 3 mg/kg and 10 mg/kg reduced new bone lesions by 94% and 90%, respectively, compared with placebo.

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