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Lumacaftor / ivacaftor

Orkambi

Extended indication Extension of indication to include treatment of cystic fibrosis for children aged 1 to less than 2 y
Therapeutic value No estimate possible yet
Registration phase Registered and reimbursed

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

Registration route Centralised (EMA)
Type of trajectory Normal trajectory
ATMP No
Submission date June 2022
Expected Registration July 2023
Orphan drug Yes
Registration phase Registered and reimbursed
Additional remarks Positieve CHMP-opinie april 2023

Therapeutic value

Therapeutic value No estimate possible yet

Expected patient volume per year

Patient volume

25

Market share is generally not included unless otherwise stated.
References Expertopinie (1);
Additional remarks 50% van de patiënten met cystische fibrose (CF) is homozygoot. Er worden 50 kinderen met CF geboren per jaar. Dit betekent dat er ongeveer 25 nieuwe patiënten in aanmerking zullen komen per jaar (1).

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.