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 patiƫnten 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.