Maralixibat shows efficacy in reducing pediatric cholestatic pruritus

Maralixibat, an oral ileal bile acid transporter (IBAT) inhibitor, has demonstrated significant efficacy in reducing cholestatic pruritus in the phase 3 EXPAND trial. In the pediatric primary analysis population, the treatment achieved a placebo-adjusted 0.98-point improvement in itch severity, while serum bile acids decreased by 69% relative to placebo across the overall study population.

Jennifer Vittorio, MD, of NYU Langone Health, noted that cholestatic pruritus is a debilitating symptom that affects sleep, mood, and energy levels. In infants, the condition often manifests as irritability or disturbed sleep, which can be misdiagnosed as colic or reflux. In school-aged children, it may present as fatigue or difficulty concentrating, while adolescents may experience social isolation and depression due to the distress caused by scratching.

The mechanism of maralixibat involves reversibly inhibiting IBAT to reduce intestinal bile acid reabsorption, thereby increasing fecal bile acid excretion and lowering circulating bile acid concentrations. This approach offers a potential management strategy for conditions such as Alagille syndrome, progressive familial intrahepatic cholestasis (PFIC), and biliary atresia.