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FDA Approves Regeneron's Pasatru for Heterotopic Ossification

The U.S. Food and Drug Administration has approved Pasatru to reduce new heterotopic ossification and reduce clinician-assessed disease flare-ups in adults with fibrodysplasia ossificans progressiva. Pasatru is the second drug approved for patients with this very rare disease, the FDA said. The recommended starting dosage of Pasatru is 10 mg/kg infused intravenously over 60 minutes, once every four weeks. The dosage may be decreased to 3 mg/kg administered intravenously once every four weeks if 10 mg/kg is not tolerated. The effectiveness and safety of Pasatru were evaluated in a randomized, double-blind, placebo-controlled clinical study of 63 adults with FOP. Patients received Pasatru 3 mg/kg, Pasatru 10 mg/kg, or placebo, each given by IV infusion every 4 weeks for 56 weeks. After the initial 56 weeks, 61 patients continued in an extended follow-up phase on the same treatment. The primary efficacy endpoint was the number of new heterotopic ossification lesions that formed by Week 56, detected using full-body low-dose CT scans. The study also evaluated the number of disease flare-ups assessed by the clinician. Both doses of Pasatru significantly reduced new bone growth compared to placebo. Pasatru received Breakthrough Therapy, Fast Track, Orphan Drug and Priority Review designations for this indication. FDA granted this approval to Regeneron Pharmaceuticals.