Leniolisib Medicine 07 December 2022 Extended indication Treatment for activated phosphoinositide 3-kinase delta syndrome (APDS), a rare primary immunodeficiency, in adolescents and adults 12 years or older. Therapeutic value No estimate possible yet Total cost 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. Current proprietary name Already available biosimilars / generics Proprietary name Manufacturer Pharming Portfolio holder Novartis Mechanism of action Unknown Route of administration Oral Therapeutical formulation Capsule Budgetting framework Unknown Centre of expertise Additional remarks Registration Registration route Centralised (EMA) Type of trajectory Accelerated assessment Particularity Unknown ATMP No Submission date November 2022 Expected Registration August 2023 Orphan drug Yes Registration phase Registration application pending Reimbursement Medicine sluice Additional remarks Therapeutic value Current treatment options Therapeutic value No estimate possible yet This assessment does not indicate any potential inclusion in the package. Substantiation Duration of treatment Frequency of administration 2 times a day Dosage per administration 10-70 mg References NCT02435173 Additional remarks Expected patient volume per year Patient volume Market share is generally not included unless otherwise stated. Maximum patient volume for sluice References Additional remarks Expected cost per patient per year Cost References Additional remarks Potential total cost per year Total cost Total cost for sluice Additional remarks Off label use Off label use Unknown Indications off label use References Additional remarks Indication extension Indication extensions Unknown Indication extensions References Additional remarks Other information Additional remarks