Vanzacaftor / tezacaftor / deutivacaftor Medicine 04 June 2024 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 IND 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) Current proprietary name Already available biosimilars / generics Proprietary name Manufacturer Vertex Portfolio holder Vertex Mechanism of action Unknown Route of administration Oral Therapeutical formulation Tablet Budgetting framework Extramural (GVS) Centre of expertise Additional remarks CFTR modulation Registration Registration route Centralised (EMA) Type of trajectory Normal trajectory Particularity Unknown ATMP No Submission date May 2024 Expected Registration June 2025 Orphan drug Yes Registration phase Registration application pending Reimbursement Medicine sluice Additional remarks Therapeutic value Current treatment options CFTR-modulatoren Therapeutic value Possible benefit in ease of use This assessment does not indicate any potential inclusion in the package. 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 Frequency of administration 1 times a day Dosage per administration References NCT05033080; NCT05444257; NCT05076149; Expertopinie Additional remarks Expected patient volume per year Patient volume 1,580 - 1,660 Market share is generally not included unless otherwise stated. Maximum patient volume for sluice 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 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. 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 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