FDA Expands LIVMARLI Label for PFIC Patients

The FDA has authorised a label expansion for LIVMARLI® (maralixibat) oral solution, which is intended to treat cholestatic pruritus in patients with progressive familial intrahepatic cholestasis (PFIC). This announcement was made by Mirum Pharmaceuticals today. The higher concentration formulation of LIVMARLI, which was assessed in the MARCH Phase 3 research, and usage in PFIC patients aged 12 months and above are included in the enlarged label.

According to Chris Peetz, CEO of Mirum, "the launch of LIVMARLI in PFIC is going well, and we are thrilled that it will now be available for patients 12 months and older." Children with PFIC are typically identified while they are young, and starting therapy as soon as possible can assist to guarantee that they experience fewer days of pruritus related to this condition.

The U.S. Food and Drug Administration has authorised LIVMARLI® (maralixibat) oral solution, a once-daily ileal bile acid transporter (IBAT) inhibitor, for the treatment of two paediatric cholestatic liver disorders. In the United States and Europe, it is authorised for the treatment of cholestatic pruritus in individuals with Alagille syndrome (ALGS) who are three months of age or older. Additionally, it is licensed in Europe for the treatment of progressive familial intrahepatic cholestasis (PFIC) in patients three months of age and older, as well as in the United States for the treatment of cholestatic pruritus in patients 12 months of age and older.