Ivacaftor / Tezacaftor / Elexacaftor Medicine 04 June 2026 Extended indication Treatment of cystic fibrosis (CF) in paediatric patients aged 1 to less than 6 years who have at least one non-Class I mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene). Therapeutic value Total cost Registration phase Registration application pending Product Active substance Ivacaftor / Tezacaftor / Elexacaftor Domain Lung diseases Reason of inclusion Indication extension IND Main indication Cystic fibrosis Extended indication Treatment of cystic fibrosis (CF) in paediatric patients aged 1 to less than 6 years who have at least one non-Class I mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene). Current proprietary name Already available biosimilars / generics Proprietary name Kaftrio Manufacturer Vertex Portfolio holder Vertex Mechanism of action Protein chaperone Route of administration Oral Therapeutical formulation Granulate Budgetting framework Extramural (GVS) Centre of expertise Additional remarks Registration Registration route Centralised (EMA) Type of trajectory Particularity ATMP Submission date April 2026 Expected Registration March 2027 Orphan drug Registration phase Registration application pending Reimbursement Medicine sluice Additional remarks Therapeutic value Current treatment options Therapeutic value Substantiation Duration of treatment Frequency of administration Dosage per administration 45 mg/30 mg/60 mg granules 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 Indications off label use References Additional remarks Indication extension Indication extensions Indication extensions References Additional remarks Other information Additional remarks