STAT+: Regeneron drug for disease that causes dangerous bone growth earns FDA approval
A Regeneron drug that treats an ultra-rare disease that causes bone to form where it's not meant to be has won approval.
The US FDA has approved Regeneron's treatment for fibrodysplasia ossificans progressiva (FOP), a rare disease that causes muscle and connective tissue to be replaced with bone. The approved treatment is called garetosmab, also referred to as Pasatru by STAT News.
According to STAT News, people with FOP typically rely on wheelchairs by age 25 as bone accumulation locks up their limbs, and only some live into their 50s. The hope is that garetosmab can help patients maintain their mobility and live longer.
The approval was based on a pivotal trial led by Richard Keen of London’s Royal National Orthopaedic Hospital. Keen stated that the treatment "almost completely stops the new [bone] forming, and therefore their condition will not deteriorate."
Brief written by urgent.news from Endpoints News, STAT News — 2 reports on this story. Machine-written — may contain errors; check the original before relying on it.
Also reported by 2 other outlets
- FDA approves Regeneron’s garetosmab for disease that turns tissue to bone endpoints.news
- Regeneron to challenge Ipsen as FDA clears bone disease drug biopharmadive.com
