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2 clusters · 3 sources · 5 days · First seen · Last updated
Maralixibat treatment for cholestatic pruritus
Overview
Clinical trials for maralixibat, an oral ileal bile acid transporter (IBAT) inhibitor, have demonstrated efficacy in treating cholestatic pruritus, a severe itching condition associated with Alagille syndrome, biliary atresia, and progressive familial intrahepatic cholestasis (PFIC).
Data from the Phase 3 EXPAND trial showed that the treatment achieved a 69% placebo-adjusted reduction in serum bile acids across the study population. In pediatric patients, the drug provided a 0.98-point improvement in average worst daily itch severity. Medical experts note that the condition can cause significant distress, affecting sleep, mood, and social interaction in children and adolescents.
Following these clinical findings, the National Institute for Health and Care Excellence (NICE) has recommended maralixibat for use within the NHS for patients aged two months and older. This recommendation follows years of campaigning by advocacy groups such as Liver UK to secure better treatment options for families affected by these rare liver diseases.
Entities
NYU Langone Health · maralixibat · Jennifer Vittorio · Liver UK · NICE
Timeline
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[HEALTH] 2 sourcesMaralixibat shows efficacy in reducing pediatric cholestatic pruritus
The phase 3 EXPAND trial shows that maralixibat significantly reduces cholestatic pruritus in pediatric patients by inhibiting bile acid reabsorption, improving quality of life for affected families.
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[HEALTH] 2 sourcesNICE recommends maralixibat for Alagille syndrome treatment
NICE has recommended maralixibat for treating severe cholestatic pruritus in Alagille syndrome patients aged two months and older within the NHS, following successful Phase 3 clinical trial results.
Sources
britishlivertrust.org.uk · consultantlive.com · mdmag.com