NICE recommends Livmarli (maralixibat) as add-on treatment for cholestatic pruritus in Alagille syndrome

Maralixibat can be used as an option to treat cholestatic pruritus in Alagille syndrome in people 2 months and over, only: i) as an add-on to standard first-line treatment (such as bile acid sequestrants, ursodeoxycholic acid, rifampicin and opioid receptor antagonists) if this has not worked well enough alone, and; ii) if the company provides it according to the commercial arrangement. Assess response to maralixibat after the first 12 weeks of treatment. Only continue treatment if there is clear evidence of a response, defined as a 50% decrease in serum bile acid levels from baseline. These recommendations are not intended to affect treatment with maralixibat that was started in the NHS before this guidance was published.

Why the committee made these recommendations:
Alagille syndrome is a rare genetic condition that often causes cholestatic pruritus (itching), which usually starts in early childhood. Cholestasis causes a build-up of bile acids in the liver and skin, which can lead to pruritus and liver cirrhosis. There are no licensed treatments for cholestatic pruritus in Alagille syndrome available in the NHS. Standard care includes treatments for pruritus that are not licensed for use in this syndrome. The company positioned maralixibat as an add-on treatment to standard care when these treatments have not worked well enough. This does not include everyone it is licensed for. Clinical trial evidence suggests that pruritus in Alagille syndrome in children is more likely to respond to maralixibat plus standard care than to placebo plus standard care. It is uncertain how effective maralixibat is at reducing pruritus in the longer term, and if it would help people live longer compared with standard care alone. There is no evidence on maralixibat use in adults. There are also uncertainties in the company’s economic model. But, with the commercial arrangement, the most likely cost-effectiveness estimate is within the range NICE considers an acceptable use of NHS resources.
So, maralixibat can be used.