Mirum Pharmaceuticals Gets Positive CHMP Opinion For LIVMARLI Oral Solution For The Treatment Of PFIC In Patients 3-Months Or Older

Author: Benzinga Newsdesk | May 31, 2024 08:39am

Positive opinion from CHMP based on Phase 3 MARCH study with highly statistically significant (p<0.0001) reduction in pruritus severity between LIVMARLI vs. placebo across all PFIC types studied.
CHMP assessment concluded that LIVMARLI in PFIC brings significant clinical benefit over the existing approved therapy.
Additionally, LIVMARLI received favorable COMP opinion recommending maintenance of Orphan Drug Designation in PFIC.
Following CHMP and COMP opinions, a decision by the European Commission is expected in the third quarter of 2024.

Mirum Pharmaceuticals, Inc. (NASDAQ:MIRM) today announced that the European Committee for Medicinal Products for Human Use (CHMP) has adopted a positive opinion of LIVMARLI® (maralixibat) oral solution for the treatment of Progressive Familial Intrahepatic Cholestasis (PFIC) in patients three months of age and older. The CHMP also concluded that LIVMARLI in PFIC brings significant clinical benefit based on improved efficacy and a major contribution to patient care compared to the existing approved treatment for PFIC. The CHMP positive opinion is the scientific recommendation to the European Commission for marketing authorization in Europe.

Posted In: MIRM