Summary

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• Maralixibat (Livmarli) is approved for the treatment of cholestatic pruritus in individuals 3 months of age and older with Alagille syndrome (ALGS) and in those 5 years of age and older with progressive familial intrahepatic cholestasis (PFIC).
• The information was derived from one systematic review/meta-analysis, which focused on maralixibat for treating ALGS.
• Maralixibat has been evaluated in four trials involving a total of 94 patients. Despite the small sample size due to the rarity of ALGS, substantial data regarding its efficacy and safety profiles is available.
• In terms of effectiveness, maralixibat showed significant reductions in ItchRO scores with an average decrease rate of 1.8 points, indicating alleviation in severity; serum bile acids decreased by approximately 75.8 µmol/L. Improvements were also observed in Multidimensional Fatigue Scale scores by about 11.4 points, with Pediatric Quality of Life scale scores increasing by up to 8.3 points.
• Regarding safety, an increase in alanine aminotransferase levels by approximately 40 U/L was observed among treated individuals, suggesting potential liver stress or damage that requires further monitoring. This highlights that while maralixibat is effective, careful optimization of the dosing regimen and overall safety profile is necessary for broader use.
• Direct comparisons with drugs outside the IBAT inhibitor class were not provided, but maralixibat shows strong efficacy in reducing symptoms related to cholestatic pruritus and improving quality of life indicators among the ALGS patient population. Despite adverse effects such as ALT elevation, indicating possible concerns, ongoing research is imperative for fine-tuning, especially concerning liver health aspects.