Pasatru FDA approval gives adults with fibrodysplasia ossificans progressiva a second approved treatment designed to reduce new heterotopic ossification and clinician-assessed disease flare-ups.
What the Pasatru FDA Approval Means for Adults With FOP
Fibrodysplasia ossificans progressiva, or FOP, is a very rare condition in which bone forms in soft tissues. The U.S. Food and Drug Administration approved Regeneron’s Pasatru for adults, expanding the available treatment options for patients with the disease.
The recommended starting dose is 10 milligrams per kilogram, delivered by intravenous infusion over 60 minutes once every four weeks. The dose may be reduced to 3 milligrams per kilogram on the same schedule when the higher dose is not tolerated.
Regulators evaluated Pasatru in a randomized, double-blind and placebo-controlled study involving 63 adults with FOP. Participants received either a 3-milligram dose, a 10-milligram dose or placebo every four weeks for 56 weeks. Sixty-one patients continued into an extended follow-up phase on the same assigned treatment.
The primary endpoint measured the number of new heterotopic ossification lesions detected by full-body low-dose CT scans at Week 56. The study also assessed disease flare-ups reported by clinicians. Both Pasatru dose groups significantly reduced new bone growth compared with placebo.
Pasatru previously received Breakthrough Therapy, Fast Track, Orphan Drug and Priority Review designations for this indication. Those designations reflect the rarity and seriousness of FOP and the regulatory focus on treatments addressing unmet medical needs. The Pasatru FDA approval adds a new commercial and clinical milestone for Regeneron while extending the treatment landscape for affected adults.
