Vanzacaftor Medicine 04 June 2024 Extended indication Cystic Fibrosis Participants 1 Year of Age and Older Therapeutic value No estimate possible yet Total cost 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 Current proprietary name Already available biosimilars / generics Proprietary name Manufacturer Vertex Portfolio holder Vertex Mechanism of action Unknown Route of administration Oral Therapeutical formulation Tablet Budgetting framework Extramural (GVS) Centre of expertise Additional remarks CFTR modulation Registration Registration route Centralised (EMA) Type of trajectory Unknown Particularity Unknown ATMP No Submission date 2024 Expected Registration 2025 Orphan drug Yes Registration phase Clinical trials Reimbursement Medicine sluice Additional remarks Therapeutic value Current treatment options Therapeutic value No estimate possible yet This assessment does not indicate any potential inclusion in the package. Substantiation Duration of treatment Frequency of administration Dosage per administration References Additional remarks Expected patient volume per year Patient volume Market share is generally not included unless otherwise stated. Maximum patient volume for sluice References Additional remarks Expected cost per patient per year Cost References Additional remarks Potential total cost per year Total cost Total cost for sluice Additional remarks Off label use Off label use Unknown Indications off label use References Additional remarks Indication extension Indication extensions Unknown Indication extensions References Additional remarks Other information Additional remarks