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Ivacaftor

Kalydeco

Extended indication Kalydeco tablets are indicated in a combination regimen with ivacaftor /tezacaftor /elexacaftor tabl
Therapeutic value No estimate possible yet
Registration phase Positive CHMP opinion

Product

Active substance Ivacaftor
Domain Lung diseases
Reason of inclusion Indication extension
Main indication Cystic fibrosis
Extended indication Kalydeco tablets are indicated in a combination regimen with ivacaftor /tezacaftor /elexacaftor tablets for the treatment of adults, and adolescents and children aged 6 years and older with cystic fibrosis (CF) who have at least one F508del mutation in the CFTR gene
Proprietary name Kalydeco
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 June 2021
Expected Registration January 2022
Orphan drug Yes
Registration phase Positive CHMP opinion

Therapeutic value

Therapeutic value No estimate possible yet

Expected patient volume per year

Patient volume

< 107

Market share is generally not included unless otherwise stated.
References Data CF registratie 2019
Additional remarks Er zullen 107 patiënten in aanmerking komen in deze leeftijdsgroep.

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.