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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 comments 
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.