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Elexacaftor / tezacaftor / ivacaftor

Kaftrio

Extended indication Kaftrio is indicated in a combination regimen with ivacaftor for the treatment of cystic fibrosis (C
Therapeutic value Possible added value
Registration phase Positive CHMP opinion

Product

Active substance Elexacaftor / tezacaftor / ivacaftor
Domain Lung diseases
Reason of inclusion New medicine (specialité)
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 Positive CHMP opinion
Additional remarks Positieve CHMP opinie ontvangen november 2021.

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 remarks 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

There is currently nothing known about the expected cost.

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

There is currently nothing known about indication extensions.

Other information

There is currently no futher information available.