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  • Question on Document

    Has all of the relevant evidence been taken into account?

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    Are the summaries of clinical and cost effectiveness reasonable interpretations of the evidence?

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    Are the recommendations sound and a suitable basis for guidance to the NHS?

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    Are there any aspects of the recommendations that need particular consideration to ensure we avoid unlawful discrimination against any group of people on the grounds of race, gender, disability, religion or belief, sexual orientation, age, gender reassignment, pregnancy and maternity?
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Draft guidance consultation

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2 Information about maralixibat

Marketing authorisation indication

2.1

Maralixibat (Livmarli, Mirum Pharmaceuticals) is indicated for the 'treatment of cholestatic pruritus in patients with Alagille syndrome (ALGS) 2 months of age and older'.

Price

2.3

The list price for maralixibat is £43,970 per 30 ml bottle of 9.5 mg/ml maralixibat oral solution (company submission).

2.4

The company had proposed a commercial arrangement, which was considered by committee as an exception. It was considered to be 'at risk', that is, it had not been provisionally agreed with NHS England. A final agreement would be needed for this arrangement to be the basis for a final recommendation.