Regeneron wins FDA approval for Pasatru, targeting rare bone disease FOP

The U.S. Food and Drug Administration has approved Regeneron Pharmaceuticals' drug Pasatru for the treatment of fibrodysplasia ossificans progressiva (FOP), a very rare condition in which muscle and connective tissue turn into bone.

Phase 3 data released last September showed that Pasatru cut the formation of new abnormal bone by at least 90 percent compared with placebo. The development program had been paused after several trial participants died, but Regeneron concluded the deaths were unrelated and resumed testing.

Pasatru is priced at roughly $1.4 million per patient per year and is administered at either 3 or 10 milligrams per kilogram of body weight. The approval sets the stage for a commercial contest with Ipsen's existing therapy for FOP, which has struggled to generate strong sales.