Maralixibat

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
Registration phase	Registration application pending

Product

Active substance	Maralixibat
Domain	Metabolism and Endocrinology
Reason of inclusion	Indication extension
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
Proprietary name	Livmarli
Manufacturer	Mirum Pharmaceuticals
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 route	Centralised (EMA)
Type of trajectory	Normal trajectory
ATMP	No
Submission date	April 2023
Expected Registration	February 2024
Registration phase	Registration application pending

Therapeutic value	No estimate possible yet

There is currently nothing known about the expected patient volume.

There is currently nothing known about the expected cost.

There is currently nothing known about the possible total cost.

There is currently nothing known about off label use.

There is currently nothing known about indication extensions.

There is currently no futher information available.