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
Route of administration Oral
Therapeutical formulation Capsule
Budgetting framework Extramural (GVS)

Registration

Registration route Centralised (EMA)
Type of trajectory Normal trajectory
ATMP No
Submission date November 2022
Expected Registration February 2024
Registration phase Registration application pending
Additional remarks Eerder in behandeling genomen als versnelde beoordeling, inmiddels als normaal traject.

Therapeutic value

Current treatment options Immunosuppressiva. Een voorbeeld hiervan is rapamycine (sirolimus), dat bij APDS met succes wordt gebruikt.
Therapeutic value No estimate possible yet
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 continuous
Frequency of administration 2 times a day
Dosage per administration 10-70 mg
References NCT02435173

Expected patient volume per year

Patient volume

< 17

Market share is generally not included unless otherwise stated.

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

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

Indication extension No

Other information

There is currently no futher information available.