Ivacaftor

Active substance Ivacaftor
Domain Lung diseases
Reason of inclusion Indication extension
Main indication Cystic fibrosis
Extended indication Extension of indication to extend the indication of Kalydeco (ivacaftor) tablets in combination regimen with Kaftrio (ivacaftor/tezacaftor/elexacaftor) tablets for the treatment of adults and adolescents aged 12 years and older with cystic fibrosis (CF) who have at least one F508del mutation in the CFTR gene.

Product

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
Submission date September 2020
Expected Registration July 2021
Orphan drug Yes
Registration phase Registration application pending

Therapeutic value

Therapeutic value No judgement
Duration of treatment Not found

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.