Pasatru™ (garetosmab-grts) First and Only FDA-approved Treatment Demonstrating Reduction in New Heterotopic Ossification (HO) Lesions and Clinician-Assessed Flare-ups in a Placebo-controlled Trial in Adults with Fibrodysplasia Ossificans Progressiva (FOP)
Key Highlights
- ➤FDA approved Pasatru (REGN) for adults with FOP, reducing new HO lesions and clinician-assessed flare-ups.
- ➤OPTIMA 10 mg/kg cut new HO lesions 90% at 56 weeks: 2 lesions versus 19 placebo.
- ➤OPTIMA 3 mg/kg cut new HO lesions 94% at 56 weeks: 1 lesion versus 19 placebo.
- ➤Clinician-assessed flare-ups fell 88% with 10 mg/kg and 15% with 3 mg/kg versus placebo.
- ➤Pasatru dosing starts at 10 mg/kg intravenously monthly; may decrease to 3 mg/kg if not tolerated.
- ➤Serious treatment-emergent adverse events affected 2 patients on 10 mg/kg, 1 on 3 mg/kg, and 2 placebo.
- ➤EU regulatory submission remains under review; OPTIMA 2 pediatric Phase 3 trial planned later this year.
Expert Statements
Kathryn Dahir, Professor, Department of Internal Medicine, Division of Endocrinology, Diabetes, and Metabolism at Vanderbilt University; primary investigator for OPTIMA
“For people living with FOP, every irregular new bone formation is a step toward disability and potential loss of mobility”
Michelle Davis, Executive Director of the International FOP Association
“FOP is a relentless, restrictive condition that severely impacts the lives of those diagnosed and their families”
George D. Yancopoulos, Board co-Chair, President and Chief Scientific Officer at Regeneron
“The approval of Pasatru is the culmination of decades of pioneering research that Regeneron has pursued alongside the FOP community, rooted in our discovery of the role that Activin A plays in driving this disease”
FOP is an ultra-rare genetic disorder characterized by rogue bone formation that infiltrates muscles, tendons, ligaments and other connective tissues, resulting in significant disability
Approval based on results from the OPTIMA trial demonstrating a 90% or greater reduction in new HO lesions at 56 weeks with a dramatic reduction in clinician-assessed flare-ups in adults with FOP
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