Garetosmab reduces new heterotopic bone lesions in FOP trial

The phase 3 OPTIMA trial randomised 63 adults with fibrodysplasia ossificans progressiva to monthly placebo or garetosmab (3 mg/kg or 10 mg/kg) and assessed new heterotopic bone lesions at week 56. The trial reported significantly fewer new lesions with garetosmab versus placebo (19 lesions with placebo versus one with 3 mg/kg and two with 10 mg/kg), with treatment-related adverse events of special interest occurring in 0% (3 mg/kg), 5% (placebo), and 17% (10 mg/kg) of participants and no deaths. The authors state this provides evidence of garetosmab's potential as a disease-modifying therapy for FOP.

Most relevant for: Adults with fibrodysplasia ossificans progressiva, an ultrarare genetic disorder causing progressive heterotopic ossification.

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