Mirum Pharmaceuticals, Inc. has announced that the European Commission granted Marketing Authorization for LIVMARLI® oral solution, intended for treating progressive familial intrahepatic cholestasis (PFIC) in patients aged three months and older. This approval follows a positive CHMP opinion, highlighting LIVMARLI's significant clinical benefits over existing PFIC treatments based on improved efficacy.

The approval is based on data from the Phase 3 MARCH study, the largest randomized trial for PFIC involving 93 patients with various genetic types of PFIC. Results showed statistically significant reductions in pruritus (itching) and serum bile acids compared to placebo across all studied PFIC types. Additionally, improvements in total bilirubin levels and growth were observed. The most common treatment-related adverse event was mild diarrhea, with no severe cases reported.

Chris Peetz, Mirum's CEO, expressed delight at the approval, emphasizing LIVMARLI's potential to enhance liver health and quality of life for young PFIC patients in Europe. Professor Richard Thompson of King's College London underscored confidence in LIVMARLI's ability to improve liver health markers and patient outcomes. Emily Ventura, Executive Director of the PFIC Network, noted the approval's profound impact on patients' lives by addressing the debilitating symptoms of PFIC.

LIVMARLI, already approved by the FDA for treating cholestatic pruritus in PFIC patients aged five and older, has a supplemental NDA pending for a higher strength formulation used in the MARCH trial to expand its approval to younger PFIC patients.

LIVMARLI is a once-daily oral medication that inhibits the ileal bile acid transporter (IBAT). It is also approved in the U.S. for cholestatic pruritus in Alagille syndrome patients aged three months and older. LIVMARLI is the only IBAT inhibitor approved by the European Commission for treating cholestatic pruritus in Alagille syndrome patients from two months of age, and in Canada for Alagille syndrome patients with cholestatic pruritus.

 

Source: businesswire.com

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