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Maralixibat

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 remarks 
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 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
References 
San Francisco Business Times, sep 29, 2021
Additional remarks 
In de VS kost maralixibat voor Alagille syndroom $391.000 per jaar (€346.000). Mogelijk zal de prijs in de EU 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.