Regeneron's Pasatru Wins FDA Approval for Rare Bone Disease
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- Regeneron received FDA approval for Pasatru, a new treatment for fibrodysplasia ossificans progressiva (FOP), on Wednesday, marking the culmination of a three-decade research effort
- Pasatru is designed to halt new bone formation in FOP patients, who typically lose mobility by age 25 and face reduced life expectancy due to progressive skeletal immobilization
- Richard Keen of London’s Royal National Orthopaedic Hospital, primary investigator of the pivotal trial, stated that the treatment appears to nearly stop new bone formation, preventing disease progression
- FOP patients may maintain mobility longer and potentially live past their 50s with Pasatru, shifting what has historically been a relentlessly degenerative trajectory
Why it matters: People with FOP, a condition affecting about 1 in 2 million, have had no approved treatments until now. By halting abnormal bone growth, Pasatru could delay wheelchair dependence and extend lifespans, transforming care for this ultra-rare disease with immediate clinical relevance.
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