FDA Approves Rare Bone Disease Drug as Part of Week-Long Regulatory Sprint

The FDA approved Regeneron's garetosmab (Pasatru) on Wednesday, the first-ever treatment for fibrodysplasia ossificans progressiva (FOP) — an ultra-rare disease where muscle and soft tissue progressively turn into bone. Typically, patients are wheelchair-bound by age 25, and only some live into their 50s. The monoclonal antibody targets activin A signaling and, in its pivotal trial, essentially stopped new bone from forming. "You're almost completely stopping the new...