STAT+: Regeneron drug for disease that causes dangerous bone growth earns FDA approval

Regeneron Pharmaceuticals won FDA approval on Wednesday for Pasatru, a treatment for fibrodysplasia ossificans progressiva (FOP), an ultra-rare disease in which bone forms in inappropriate places. The approval caps a three-decade effort and offers hope of maintaining mobility and potentially extending lifespan for patients, who typically require wheelchairs by age 25 and only some live into their 50s.

Richard Keen of London's Royal National Orthopaedic Hospital, the pivotal trial's primary investigator, said the treatment 'almost completely stops the new [bone] forming' so that patients' condition will not deteriorate. The quote underscores the expectation that Pasatru can change the disease's trajectory.