following a second resubmission assessed under the orphan medicine process:
maralixibat (Livmarli®) is accepted for restricted use for use within NHSScotland.
Indication under review: treatment of cholestatic pruritus in patients with Alagille syndrome (ALGS) 2 months of age and older.
SMC restriction: for use in patients whose condition has not responded to standard of care medicines.
In a double-blind, randomised withdrawal, phase IIb study, maralixibat maintained reduced serum bile acid levels, compared with a significant increase with placebo, in children (age 1 to 18 years) with ALGS.
This advice applies only in the context of an approved NHSScotland Patient Access Scheme (PAS) arrangement delivering the cost-effectiveness results upon which the decision was based, or a PAS/ list price that is equivalent or lower.
This advice takes account of the views from a Patient and Clinician Engagement (PACE) meeting.
Medicine details
- Medicine name:
- maralixibat (Livmarli)
- SMC ID:
- SMC2806
- Indication:
Indicated for the treatment of cholestatic pruritus in patients with Alagille syndrome (ALGS) 2 months of age and older.
- Pharmaceutical company
- Mirum Pharmaceuticals
- BNF chapter
- Nutrition and blood
- Submission type
- Resubmission
- Status
- Restricted
- Date advice published
- 08 December 2025