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.