You are here:
Kaftrio
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
Possible added value
Positive CHMP opinion
Elexacaftor / tezacaftor / ivacaftor
Lung diseases
New medicine (specialité)
Cystic fibrosis
Vertex
Protein chaperone
Oral
Tablet
Extramural (GVS)
Centralised (EMA)
Normal trajectory
No
December 2020
January 2022
Yes
Positieve CHMP opinie ontvangen november 2021.
Aangezien CF een progressieve ziekte betreft, heeft behandeling op jonge leeftijd extra meerwaarde gezien schade dan (gedeeltelijk) voorkomen kan worden.
175
Market share is generally not included unless otherwise stated.
Data CF registratie 2019
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.
There is currently nothing known about the expected cost.
There is currently nothing known about the possible total cost.
There is currently nothing known about off label use.
There is currently nothing known about indication extensions.
There is currently no futher information available.
Understanding of expected market entry of innovative medicines