Vanzacaftor / tezacaftor / deutivacaftor

Extended indication	Vanzacaftor/tezacaftor/deutivacaftor monotherapy for treatment of cystic fibrosis in adolescents and adults who have at least one F508del mutation or a mutation responsive to triple combination CFTR modulators (CFTRm)
Therapeutic value	Possible benefit in ease of use
Total cost	314,344,800.00
Registration phase	Registration application pending

Product

Active substance	Vanzacaftor / tezacaftor / deutivacaftor
Domain	Lung diseases
Reason of inclusion	Indication extension
Main indication	Cystic fibrosis
Extended indication	Vanzacaftor/tezacaftor/deutivacaftor monotherapy for treatment of cystic fibrosis in adolescents and adults who have at least one F508del mutation or a mutation responsive to triple combination CFTR modulators (CFTRm)
Manufacturer	Vertex
Portfolio holder	Vertex
Route of administration	Oral
Therapeutical formulation	Tablet
Budgetting framework	Extramural (GVS)
Additional remarks	CFTR modulation

Registration

Registration route	Centralised (EMA)
Type of trajectory	Normal trajectory
ATMP	No
Submission date	May 2024
Expected Registration	June 2025
Orphan drug	Yes
Registration phase	Registration application pending

Therapeutic value

Current treatment options	CFTR-modulatoren
Therapeutic value	Possible benefit in ease of use
Substantiation	Er lijkt een voordeel te zijn met een dosering van een keer per dag in plaats van twee keer per dag. Vooralsnog lijkt de effectiviteit even groot te zijn als ivacaftor/tezacaftor/elexacaftor.
Duration of treatment	continuous
Frequency of administration	1 times a day
References	NCT05033080; NCT05444257; NCT05076149; Expertopinie

Expected patient volume per year

Patient volume	1,580 - 1,660 Market share is generally not included unless otherwise stated.
References	Nederlandse Cystic Fibrosis Stichting. 2024 (1); Expertopinie (2)
Additional remarks	In Nederland zijn zo'n 1.750 CF-patiënten (1). Er wordt verwacht dat alle patiënten die onder ivacaftor/tezacaftor/elexacaftor ook voor dit middel in aanmerking komen. Dat komt neer op 90-95% van de Nederlandse CF-populatie (2).

Patient volume

1,580 - 1,660

Market share is generally not included unless otherwise stated.

References

Nederlandse Cystic Fibrosis Stichting. 2024 (1); Expertopinie (2)

Additional remarks

In Nederland zijn zo'n 1.750 CF-patiënten (1). Er wordt verwacht dat alle patiënten die onder ivacaftor/tezacaftor/elexacaftor ook voor dit middel in aanmerking komen. Dat komt neer op 90-95% van de Nederlandse CF-populatie (2).

Expected cost per patient per year

Cost	194,040.00
References	Zorginstituut
Additional remarks	Kaftrio kost per patiënt per jaar 194.040 euro. Het is waarschijnlijk dat de kosten van vanzacaftor/tezacaftor/deutivacaftor in dezelfde orde van grootte gaan vallen.

Potential total cost per year

Total cost	314,344,800.00 This amount gives an indication of the total cost. It is the result of the average expected patient volume times the average cost per patient. both per year.

Total cost

314,344,800.00

This amount gives an indication of the total cost. It is the result of the average expected patient volume times the average cost per patient. both per year.

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.