Maralixibat Medicine 07 December 2021 Extended indication Treatment of cholestatic liver disease in patients with Alagille syndrome (ALGS) 1 year of age and older. Therapeutic value No estimate possible yet Total cost € 83,040,000.00 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 Treatment of cholestatic liver disease in patients with Alagille syndrome (ALGS) 1 year of age and older. Current proprietary name Already available biosimilars / generics Proprietary name Manufacturer Mirum Pharmaceuticals Portfolio holder Mechanism of action Unknown Route of administration Oral Therapeutical formulation Solution Budgetting framework Extramural (GVS) Centre of expertise Additional remarks Sodium-bile acid cotransporter. Registration Registration route Centralised (EMA) Type of trajectory Normal trajectory Particularity Unknown ATMP Unknown Submission date September 2021 Expected Registration October 2022 Orphan drug No 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 < 240 Market share is generally not included unless otherwise stated. Maximum patient volume for sluice References Orphanet Additional remarks De prevalentie van Alagille syndroom wordt geschat op 1:70.000. Voor Nederland zou dit ongeveer 240 patiënten betekenen. Hoeveel patiënten daadwerkelijk in aanmerking komen is nog onduidelijk. Expected cost per patient per year Cost € < 346,000.00 This amount gives an indication of the total cost. It is the result of the average expected patient volume times the average cost per patient. both per year. References San Francisco Business Times, sep 29, 2021 Additional remarks In de Verenigde Staten kost maralixibat voor Alagille syndroom $391.000 per jaar (€346.000). Mogelijk zal de prijs in de Europa lager uitvallen. Potential total cost per year Total cost € 83,040,000.00 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