Extended indication Autosomal Dominant Polycystic Kidney Disease (ADPKD).
Therapeutic value No estimate possible yet
Registration phase No registration expected

Product

Active substance Lixivaptan
Domain Metabolism and Endocrinology
Reason of inclusion New medicine (specialité)
Main indication Metabolic diseases
Extended indication Autosomal Dominant Polycystic Kidney Disease (ADPKD).
Mechanism of action Unknown
Route of administration Oral
Therapeutical formulation Capsule
Budgetting framework Extramural (GVS)

Registration

Registration route Centralised (EMA)
Type of trajectory Unknown
Particularity Unknown
ATMP No
Orphan drug Yes
Registration phase No registration expected
Additional remarks De fabrikant heeft in juli 2022 aangegeven in een persbericht dat de klinische studies gestopt zijn vanwege het ontstaan van leverenzymstoornissen.

Therapeutic value

Current treatment options Tolvaptan
Therapeutic value No estimate possible yet

This assessment does not indicate any potential inclusion in the package.

Substantiation Het is sterk de verwachting dat dit middel een gelijk effect heeft als de huidige standaard tolvaptan op het beloop van ADPKD.
Frequency of administration 2 times a day
References NCT04152837

Expected patient volume per year

Patient volume < 180

Market share is generally not included unless otherwise stated.

Expected cost per patient per year

Potential total cost per year

Off label use

Off label use Unknown

Indication extension

Indication extensions Unknown

Other information