Extended indication Treatment for activated phosphoinositide 3-kinase delta syndrome (APDS), a rare primary immunodefici
Therapeutic value No estimate possible yet
Registration phase Registration application pending

Product

Active substance Leniolisib
Domain Chronic immune diseases
Reason of inclusion New medicine (specialité)
Main indication Other chronic immune diseases
Extended indication Treatment for activated phosphoinositide 3-kinase delta syndrome (APDS), a rare primary immunodeficiency, in adolescents and adults 12 years or older.
Manufacturer Pharming
Portfolio holder Novartis
Route of administration Oral
Therapeutical formulation Capsule

Registration

Registration route Centralised (EMA)
Type of trajectory Accelerated assessment
ATMP No
Submission date November 2022
Expected Registration August 2023
Orphan drug Yes
Registration phase Registration application pending

Therapeutic value

Therapeutic value No estimate possible yet
Duration of treatment continuous
Frequency of administration 2 times a day
Dosage per administration 10-70 mg
References NCT02435173

Expected patient volume per year

There is currently nothing known about the expected patient volume.

Expected cost per patient per year

There is currently nothing known about the expected cost.

Potential total cost per year

There is currently nothing known about the possible total cost.

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.