NICE recommends maralixibat for Alagille syndrome treatment

The National Institute for Health and Care Excellence (NICE) has recommended maralixibat for use within the NHS to treat cholestatic pruritus, a severe itching condition, in patients aged two months and older with Alagille syndrome.

Clinical data from the Phase 3 EXPAND trial indicates that maralixibat significantly reduces pruritus compared to a placebo. In the trial, which included patients with ultra-rare cholestatic liver diseases such as biliary atresia, participants saw a 69% placebo-adjusted drop in serum bile acids. Pediatric patients in the primary analysis showed a 0.98-point improvement in average worst daily itch severity during weeks 13 to 20.

Liver UK noted that the recommendation follows over five years of campaigning by their Children and Families team to provide better treatment options for families affected by the debilitating condition.