The European Committee for Medicinal Products for Human Use (CHMP) has granted a positive opinion for LIVMARLI (maralixibat) oral solution as a treatment for Progressive Familial Intrahepatic Cholestasis (PFIC) in patients aged three months and above.
The CHMP concluded that LIVMARLI provides substantial clinical benefit compared to existing PFIC treatments based on improved efficacy and major contributions to patient care. This positive opinion serves as the scientific recommendation for marketing authorization in Europe by the European Commission.
The Committee for Orphan Medicinal Products (COMP) has also recommended maintaining Orphan Drug Designation for LIVMARLI in PFIC. The European Commission is anticipated to endorse the CHMP and COMP opinions in the third quarter of 2024.
Chris Peetz, CEO of Mirum, indicated that these positive opinions from the CHMP and COMP highlight the potential of LIVMARLI for treating PFIC patients in Europe. If approved by the European Commission, the PFIC community will gain access to a treatment that has demonstrated significant improvements in liver health parameters.
The CHMP and COMP opinions stem from data from Phase 3 of the MARCH study, which included 93 patients with various PFIC genetic types. LIVMARLI has already been approved in Europe for treating cholestatic pruritus in Alagille syndrome (ALGS) patients aged two months and older. In the US, LIVMARLI is approved for ALGS patients three months and older and PFIC patients five years and older.
Mirum has filed a supplemental new drug application to introduce a higher LIVMARLI concentration formulation later this year, enabling label expansion for younger PFIC patients in the United States.
Jon Napitupulu is Director of Media Relations at The Clinical Trial Vanguard. Jon, a computer data scientist, focuses on the latest clinical trial industry news and trends.
