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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 comments 
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 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

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.