The European Commission has granted marketing authorization for Livmarli* (maralixibat) oral solution for the treatment of progressive familial intrahepatic cholestasis (PFIC), a rare liver disease, in patients aged 3 months and older. The approval – explains Mirum Pharmaceutical in a note – follows the positive opinion of the Committee for Medicinal Products for Human Use (CHMP) of the European Agency (EMA), according to which the drug in PFIC provides a significant clinical benefit in terms of improved efficacy and an important contribution to patient care, compared to the existing approved treatment. The COMP, the EMA committee responsible for ‘orphan drug designation’, also recommended the maintenance of this designation.
The approval of Livmarli, an ileal bile acid transporter (IBAT) inhibitor that is administered orally, once daily, is based on data from the Phase 3 March study, the largest randomized study of Pfic, which included 93 patients with different genetic types of Pfic (including Pfic1, 2, 3, 4, and 6) and without an identified genetic mutation. The data demonstrated a statistically significant reduction in pruritus (p<0.0001) and serum bile acid levels (p<0.0001) for treated patients compared to placebo in the All-Pfic cohort (n=64). Other benefits observed were a significant improvement in total bilirubin concentrations and growth compared to placebo. The most common treatment-emergent adverse event was diarrhea, which was mostly mild and transient, with no serious cases.
“We are thrilled that the European Commission has granted marketing authorization for Livmarli in Pfic,” said Chris Peetz, CEO of Mirum, “recognizing the robust data collected and the important therapeutic opportunity it offers patients living with this rare liver disease. Our hope is that it will help improve key measures of liver health and give young patients diagnosed with Pfic in Europe the chance to live a better life. We are grateful to the researchers, patients and families who made this approval possible.”
The EC approval “brings to market a treatment that is supported by years of clinical research and significant data,” adds Richard Thompson of King’s College London, “demonstrating a reduction in cholestatic pruritus and prognostic markers of improved liver health, including a reduction in serum bile acids, all important signals of positive long-term outcomes. It is encouraging to know that physicians in Europe will have a new option that has the potential to improve liver health and the quality of life of patients and their families.”
“The patient community in Europe will also benefit greatly from the approval of Livmarli,” said Emily Ventura, executive director of the Pfic Network, “supported by years of impressive data showing improvements in the most burdensome aspects of the disease. Pfic can be life-changing and have a devastating impact on patients. It is encouraging to see that young patients will have a new medicine available and we hope,” she concluded, “they can live a life less burdened by cholestasis.”
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