Elexacaftor / tezacaftor / ivacaftor

Extended indication	An Extension of indication for Sarclisa to add combination with carfilzomib and dexamethasone, for the treatment of patients with multiple myeloma who have received at least one prior therapy.
Therapeutic value	No judgement
Registration phase	Registration application pending

Product

Active substance	Elexacaftor / tezacaftor / ivacaftor
Domain	Lung diseases
Reason of inclusion	Indication extension
Main indication	Cystic fibrosis
Extended indication	An Extension of indication for Sarclisa to add combination with carfilzomib and dexamethasone, for the treatment of patients with multiple myeloma who have received at least one prior therapy.
Proprietary name	Kaftrio
Manufacturer	Vertex
Mechanism of action	Protein chaperone
Route of administration	Oral
Therapeutical formulation	Tablet
Budgetting framework	Extramural (GVS)

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	No judgement

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.