Elexacaftor / tezacaftor / ivacaftor

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
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.

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.