Regeneron's Pasatru approved for ultra-rare bone disease
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- Regeneron Pharmaceuticals won FDA approval on Wednesday for Pasatru, its treatment for fibrodysplasia ossificans progressiva (FOP), an ultra-rare disease that causes bone to grow where it shouldn't.
- The approval is the capstone of a three-decade research effort to address FOP, a condition that typically forces patients into wheelchairs by age 25 as accumulating bone locks up their joints.
- Richard Keen, primary investigator of the pivotal trial at London's Royal National Orthopaedic Hospital, said Pasatru almost completely stops new bone formation, preventing further deterioration.
- Keen expressed hope the treatment will change FOP's trajectory — preserving mobility and potentially extending lives, given that only some patients currently survive into their 50s.
Why it matters: FOP patients, typically wheelchair-bound by age 25 and rarely living past their 50s, may gain mobility and lifespan if Pasatru's pivotal trial results hold in real-world use — Keen reported the drug almost completely halts new bone formation, rather than merely addressing symptoms.
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