VIDEO: Livmarli reduces pruritus in progressive familial intrahepatic cholestasis subtypes
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WASHINGTON — In a Healio video exclusive, Alexander G. Miethke, MD, noted that Livmarli demonstrated significant reductions in pruritus and serum bile acids across multiple subtypes of progressive familial intrahepatic cholestasis.
Topline results from the phase 3 MARCH study were presented during a late-breaking oral abstract session at The Liver Meeting.
In their study, Miethke, associate professor of pediatrics from the PFIC Research Center at Cincinnati Children’s Medical Center, and colleagues assessed the safety and efficacy of maralixibat (Livmarli, Mirum Pharmaceuticals) 570 µg/kg BID in 93 patients with PFIC, between the ages of one and 17 years old. He noted the importance of examining safety in the clinical trial since it dealt with children.
“Safety is a very important tool,” he said. “It cannot be emphasized enough how important it was to do the study in a placebo-controlled fashion, and it was found that it was overall a safe study; treatment-emergent adverse events were common, about 50% developed diarrhea, but it was mostly mild and transient, and only led to discontinuation of the drug in one patient. There were no serious or severe adverse events. It was a well-tolerated intervention.”
He added: “We were surprised and encouraged to see that [maralixibat] not only improved itching and bile acid levels in a particular subtype of PFIC, which is a BSEP deficiency, but it was also effective and safe in other types of PFIC, including PFIC1 and PFIC4 which were thought to be refractory to even that intervention.”
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Thompson RJ, et al. Abstract 5001: Efficacy and safety of maralixibat in patients with progressive familial intrahepatic cholestasis (MARCH-PFIC): a randomized placebo-controlled study. Presented at: The Liver Meeting; Nov. 4-8, 2022; Washington (hybrid).
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