Mirum Pharmaceuticals announced that the U.S. FDA has granted Breakthrough Therapy Designation for maralixibat for the treatment of pruritus associated with Alagille syndrome (ALGS).
ALGS is a rare genetic disorder that ultimately leads to progressive liver disease.The Breakthrough Therapy Designation of Mirum's lead product candidate, maralixibat, was granted based on evidence from the ICONIC Phase 2b clinical trial in children with ALGS.
Mirum, which completed its initial public offering in July 2019, is a California-based clinical-stage biopharma company focused on the development and commercialization of a late-stage pipeline of novel therapies for debilitating liver diseases.
Read the details of the trial results
