Mirum Pharmaceuticals has just disclosed topline data from its pivotal phase 3 MARCH study showing that maralixibat (Livmarli®), an ileal bile acid transporter inhibitor approved by the US Food and Drug Administration to treat patients with Alagille syndrome (ALGS), is also effective in improving pruritus in patients with progressive familial intrahepatic cholestasis (PFIC).

“The Livmarli data observed in the MARCH study showcase an unprecedented reduction in pruritus and serum bile acids. These data confirm our thesis that higher doses can result in improved efficacy and better outcomes for these patients,” said Chris Peetz, president and chief executive officer at Mirum. “We are grateful to the patients, caregivers, and healthcare providers who participated in this study and who helped make these groundbreaking results possible.”

Maralixibat treatment met the primary endpoint of improvement in pruritus severity in patients with PFIC2. Moreover, it showed statistically significant effects in all PFIC subtypes, delivering a 1.8-point improvement over placebo as evaluated by the observer-reported version of the Itch Reported Outcome. In addition, no new safety concerns emerged during the trial.

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“Knowing of the impact of itching on the quality of life for children with PFIC, the sustained and clinically significant reduction in itching scores in the LIVMARLI treatment arm is an important topline result,” said Alexander G. Miethke, MD, associate professor of pediatrics from the PFIC Research Center at Cincinnati Children’s Medical Center.

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Mirum’s announcement comes 2 weeks after Albireo reports positive topline results from its phase 3 ASSERT study evaluating the safety and efficacy of odevixibat (Bylvay®) in patients with ALGS.

The phase 3 MARCH trial enrolled 93 patients (age range, 1-17 years) with a broad range of PFIC subtypes.


Positive topline data announced from Mirum’s Livmarli phase 3 March study in progressive familial intrahepatic cholestasis (PFIC). News release. Mirum Pharmaceuticals, Inc.; October 24, 2022.