Maralixibat Medicine 06 June 2023 Extended indication Extension of indication to include treatment of Progressive Familial Intrahepatic Cholestasis (PFIC) in patients 2 months of age and older Therapeutic value No estimate possible yet Total cost Registration phase Registration application pending Product Active substance Maralixibat Domain Metabolism and Endocrinology Reason of inclusion Indication extension IND Main indication Liver diseases Extended indication Extension of indication to include treatment of Progressive Familial Intrahepatic Cholestasis (PFIC) in patients 2 months of age and older Current proprietary name Already available biosimilars / generics Proprietary name Livmarli Manufacturer Mirum Pharmaceuticals Portfolio holder Mechanism of action Other, see general comments Route of administration Oral Therapeutical formulation Solution Budgetting framework Intermural (MSZ) Centre of expertise UMC Groningen Additional remarks Sodium-bile acid cotransporter inhibitor Registration Registration route Centralised (EMA) Type of trajectory Normal trajectory Particularity Unknown ATMP No Submission date April 2023 Expected Registration February 2024 Orphan drug Unknown Registration phase Registration application pending Reimbursement Medicine sluice Additional remarks Therapeutic value Current treatment options Therapeutic value No estimate possible yet This assessment does not indicate any potential inclusion in the package. Substantiation Duration of treatment Frequency of administration Dosage per administration References Additional remarks Expected patient volume per year Patient volume Market share is generally not included unless otherwise stated. Maximum patient volume for sluice References Additional remarks Expected cost per patient per year Cost References Additional remarks Potential total cost per year Total cost Total cost for sluice Additional remarks Off label use Off label use Unknown Indications off label use References Additional remarks Indication extension Indication extensions Unknown Indication extensions References Additional remarks Other information Additional remarks