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 Mechanism of action Unknown Route of administration Oral Therapeutical formulation Capsule Budgetting framework Extramural (GVS) Centre of expertise Additional remarks Registration Registration route Centralised (EMA) Type of trajectory Normal trajectory Particularity Unknown ATMP No Submission date November 2022 Expected Registration September 2024 Orphan drug Yes Registration phase Registration application pending Reimbursement Medicine sluice Additional remarks Eerder in behandeling genomen als versnelde beoordeling, inmiddels als normaal traject. Therapeutic value Current treatment options Regarding the current treatment options, there is no licensed treatment currently available for APDS patients in the Netherlands. Current treatment options include immunoglobulin replacement therapy (IRT), antimicrobials, immunosuppressive therapies, mTOR inhibitors, and HSCT is the only potential curative treatment with significant morbidity and mortality risks. Therapeutic value No estimate possible yet This assessment does not indicate any potential inclusion in the package. Substantiation Het lijkt er op dat leniolisib betere resultaten heeft dan sirolimus. In de fase 2 en 3 studie werden de eindpunten op de afname van de omvang van lymfeklieren en de toename van naïeve B-lymfocyten gehaald. 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 < 17 Market share is generally not included unless otherwise stated. Maximum patient volume for sluice References Orphanet; ESID registrer (2) Additional remarks De prevalentie is kleiner dan 1 op de miljoen mensen. In het Europese register van 2021 zien we de volgende aantallen: 175 patiënten met een APDS1/2 mutatie in het ESID register, level 1. Expected cost per patient per year Cost References Drugs.com Additional remarks De kosten in de Verenigde Staten voor een orale tablet 70 mg bedragen ongeveer $ 49.732 voor 60 tabletten. De prijs in de Verenigde Staten ligt vaak hoger dan in Nederland. 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 No Indication extensions References Additional remarks Other information Additional remarks