Extended indication Kaftrio is indicated in a combination regimen with ivacaftor for the treatment of cystic fibrosis (C
Therapeutic value Possible added value
Total cost 24,500,000.00
Registration phase Registered and reimbursed

Product

Active substance Elexacaftor / tezacaftor / ivacaftor
Domain Lung diseases
Reason of inclusion Indication extension
Main indication Cystic fibrosis
Extended indication Kaftrio is indicated in a combination regimen with ivacaftor for the treatment of cystic fibrosis (CF) in patients aged 6 years and older who have at least one F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene
Proprietary name Kaftrio
Manufacturer Vertex
Mechanism of action Protein chaperone
Route of administration Oral
Therapeutical formulation Tablet
Budgetting framework Extramural (GVS)

Registration

Registration route Centralised (EMA)
Type of trajectory Normal trajectory
ATMP No
Submission date December 2020
Expected Registration January 2022
Orphan drug Yes
Registration phase Registered and reimbursed
Additional comments Positieve CHMP opinie ontvangen november 2021. Publicatie advies aan de minister wordt verwacht in juni/juli 2022.

Therapeutic value

Therapeutic value Possible added value
Substantiation Aangezien CF een progressieve ziekte betreft, heeft behandeling op jonge leeftijd extra meerwaarde gezien schade dan (gedeeltelijk) voorkomen kan worden.

Expected patient volume per year

Patient volume

175

Market share is generally not included unless otherwise stated.

References Data CF registratie 2019
Additional comments Alle kinderen met de F508del mutatie zullen Kaftrio voorgeschreven krijgen. Er zijn in deze leeftijdsgroep 121 kinderen homozygoot, 42 heterozygoot en 12 kinderen met residual function. Dit betekent dat er 175 kinderen in totaal in aanmerking komen.

Expected cost per patient per year

Cost < 140,000.00
References Medicijnkosten.nl
Additional comments De prijs voor een tablet Kafrtio bedraagt €191,12. Bij een doorlopende behandling van 2 tabletten per dag komt dit per jaar neer op €139.517,60. Voor ivacaftor/tezacaftor/elexacaftor (Kaftrio) is een financieel arrangement afgesloten tot en met 31 december 2025.

Potential total cost per year

Total cost

24,500,000.00

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.