Maralixibat Medicine 06 June 2023 Extended indication Treatment of cholestasis in Progressive Familial Intrahepatic Cholestasis. Therapeutic value No estimate possible yet Total cost € 5,370,000.00 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 cholestasis in Progressive Familial Intrahepatic Cholestasis. 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 Extramural (GVS) Centre of expertise UMC Groningen Additional remarks Apical sodium-bile acid cotransporter/ileal bile acid transporter inibitor. 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 Ursodeoxycholzuur (UDCA) therapie moet bij alle patiënten worden gestart om leverschade te voorkomen. Bij sommige PFIC1- of PFIC2-patiënten kan galafleiding ook de pruritus verlichten en de progressie van de ziekte vertragen. De meeste PFIC-patiënten zijn uiteindelijk echter kandidaten voor levertransplantatie. Monitoring van hepatocellulair carcinoom, vooral bij PFIC2-patiënten, moet vanaf het eerste levensjaar worden aangeboden. In deze leeftijd is odevixibat al geregistreerd. Therapeutic value No estimate possible yet This assessment does not indicate any potential inclusion in the package. Substantiation Duration of treatment Frequency of administration 2 times a day Dosage per administration tot maximaal 600 microgram per kilogram References NCT04185363 Additional remarks Expected patient volume per year Patient volume 10 - 20 Market share is generally not included unless otherwise stated. Maximum patient volume for sluice References www.orpha.net (1); CBS (2); Additional remarks Er worden per jaar 1/50.000 - 1/100.000 kinderen geboren met deze aandoening. In 2017 zijn er 169.836 kinderen geboren in Nederland (CBS), dit zou gaan om 1 tot 4 personen. Er wordt verwacht dat er maximaal 10 tot 20 patiënten in aanmerking zullen komen. Expected cost per patient per year Cost € < 358,000.00 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 Reuter Additional remarks De prijs in Nederland is nog niet bekend. In de Verenigde Staten is wel een prijs bekend. 'Mirum priced the drug, to be made available for distribution immediately under brand name Livmarli, at $1,550 per unit. It expects the per unit annual cost at about $391,000 for a 17-kilogram child, the average weight of patients in it's studies.' Dit komt omgerekend in Euro neer op een prijs van €358.754,23 per patiënt. De prijs in Nederland zal mogelijk lager liggen. Potential total cost per year Total cost € 5,370,000.00 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