Apremilast

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
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	September 2024
Orphan drug	Yes
Registration phase	Registration application pending
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
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.

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

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

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.

Leniolisib