Posted by Michael Wonder on 28 Oct 2019
Mirum Pharmaceuticals announces breakthrough therapy designation for maralixibat for the treatment of pruritus associated with Alagille syndrome
28 October 2019 - Mirum Pharmaceuticals today announced that the U.S. FDA has granted breakthrough therapy designation for maralixibat for the treatment of pruritus associated with Alagille syndrome in patients 1 year of age and older.
The Breakthrough Therapy Designation of maralixibat was granted based on evidence from the ICONIC Phase 2b clinical trial in children with Alagille syndrome. Results from the clinical trial were recently presented at the International Liver Congress.
