Extended indication Treatment of cholestatic liver disease in patients with Alagille syndrome (ALGS) 1 year of age a
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
Main indication Liver diseases
Extended indication Treatment of cholestatic liver disease in patients with Alagille syndrome (ALGS) 1 year of age and older.
Manufacturer Mirum Pharmaceuticals
Route of administration Oral
Therapeutical formulation Solution
Budgetting framework Extramural (GVS)
Additional comments Sodium-bile acid cotransporter.

Registration

Registration route Centralised (EMA)
Type of trajectory Normal trajectory
Submission date September 2021
Expected Registration October 2022
Orphan drug No
Registration phase Registration application pending

Therapeutic value

Therapeutic value No estimate possible yet

Expected patient volume per year

Patient volume

< 240

Market share is generally not included unless otherwise stated.

References Orphanet
Additional comments 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
References San Francisco Business Times, sep 29, 2021
Additional comments 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

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.

Off label use

There is currently nothing known about off label use.

Indication extension

There is currently nothing known about indication extensions.

Other information

There is currently no futher information available.