Lumacaftor / ivacaftor

Extended indication	Extension of indication to include treatment of cystic fibrosis for children aged 1 to less than 2 years old of age who are homozygous for the F508del mutation in the CFTR gene.
Therapeutic value	No estimate possible yet
Registration phase	Registration application pending

Product

Active substance	Lumacaftor / ivacaftor
Domain	Lung diseases
Reason of inclusion	Indication extension
Main indication	Cystic fibrosis
Extended indication	Extension of indication to include treatment of cystic fibrosis for children aged 1 to less than 2 years old of age who are homozygous for the F508del mutation in the CFTR gene.
Proprietary name	Orkambi
Manufacturer	Vertex
Route of administration	Oral
Therapeutical formulation	Tablet
Budgetting framework	Extramural (GVS)

Registration route	Centralised (EMA)
Type of trajectory	Normal trajectory
ATMP	No
Submission date	June 2022
Expected Registration	April 2023
Orphan drug	Yes
Registration phase	Registration application pending

Therapeutic value	No estimate possible yet

There is currently nothing known about the expected patient volume.

There is currently nothing known about the expected cost.

There is currently nothing known about the possible total cost.

There is currently nothing known about off label use.

There is currently nothing known about indication extensions.

There is currently no futher information available.