Signal FDA grants volixibat breakthrough therapy and orphan designations for PSC

Summary

The US FDA granted Mirum Pharmaceuticals' volixibat both Breakthrough Therapy and Orphan Drug designations. Both designations target treatment of cholestatic pruritus, an itching condition caused by primary sclerosing cholangitis. If ultimately approved, volixibat would become the first FDA-approved therapy specifically targeting this symptom. The condition has until now lacked a therapy specifically approved to target it. The dual designation signals accelerated regulatory attention to a rare-disease treatment gap.