Extended indication

Cystic Fibrosis Participants 1 Year of Age and Older

Therapeutic value

No estimate possible yet

Registration phase

Clinical trials

Product

Active substance

Vanzacaftor

Domain

Lung diseases

Reason of inclusion

New medicine (specialité)

Main indication

Cystic fibrosis

Extended indication

Cystic Fibrosis Participants 1 Year of Age and Older

Manufacturer

Vertex

Portfolio holder

Vertex

Route of administration

Oral

Therapeutical formulation

Tablet

Budgetting framework

Extramural (GVS)

Additional remarks
CFTR modulation

Registration

Registration route

Centralised (EMA)

ATMP

No

Submission date

2024

Expected Registration

2025

Orphan drug

Yes

Registration phase

Clinical trials

Therapeutic value

Therapeutic value

No estimate possible yet

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.