Extended indication Uitbreiding van Kaftrio in combinatie met ivacaftor voor patienten met tenminste één F508del mutatie
Therapeutic value No estimate possible yet
Registration phase Clinical trials

Product

Active substance Elexacaftor / tezacaftor / ivacaftor
Domain Lung diseases
Reason of inclusion New medicine (specialité)
Main indication Cystic fibrosis
Extended indication Uitbreiding van Kaftrio in combinatie met ivacaftor voor patienten met tenminste één F508del mutatie naar de leeftijd vanaf 6 jaar (uitbreiding voor 6 tot 11 jarigen)
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 2021
Expected Registration October 2022
Orphan drug Yes
Registration phase Clinical trials
Additional comments Fabrikant verwacht indiening eind 2021.

Therapeutic value

Therapeutic value No estimate possible yet

Expected patient volume per year

There is currently nothing known about the expected patient volume.

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.