The European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) has recommended Mirum Pharmaceuticals’ LIVMARLI (maralixibat) oral solution for the treatment of progressive familial intrahepatic cholestasis (PFIC) in patients aged three months and older.
The CHMP’s endorsement, a pivotal step towards marketing authorisation in Europe, is based on data from the Phase III MARCH study. It signifies a major advancement in the treatment options available for PFIC, a rare genetic liver disorder.
The randomised trial involved 93 patients across a range of genetic PFIC types, including PFIC1, PFIC2, PFIC3, PFIC4, PFIC6 and unidentified mutational status.
It demonstrated a highly statistically significant (p<0.0001) reduction in pruritus severity when compared to placebo across all PFIC types studied.
CHMP’s positive opinion is complemented by the Committee for Orphan Medicinal Products’ (COMP) recommendation to maintain orphan drug designation for LIVMARLI in PFIC.
Mirum CEO Chris Peetz stated: “The positive and favourable opinions by both CHMP and COMP recognise the potential of LIVMARLI in treating patients with PFIC in Europe, underscoring the compelling long-term data collected in the largest PFIC study across a broad range of genetic types.
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“Further, we are thrilled that, if approved by the European Commission, the PFIC community will have a treatment option that has been shown to significantly improve key liver health parameters starting from three months of age.”
After the CHMP and COMP opinions, the European Commission’s final decision on marketing authorisation is expected in the third quarter of 2024.
LIVMARLI is already approved in Europe for treating cholestatic pruritus in Alagille syndrome (ALGS) patients aged two months and older.
It is also approved in the US for ALGS patients three months and older and for PFIC patients five years and older.
Mirum is seeking to expand its label for younger PFIC patients in the US in 2024.
In March, the US Food and Drug Administration (FDA) approved Mirum’s LIVMARLI (maralixibat) oral solution to treat cholestatic pruritus.
