Ivacaftor Medicine 07 December 2021 Extended indication Kalydeco tablets are indicated in a combination regimen with ivacaftor /tezacaftor /elexacaftor tablets for the treatment of adults, and adolescents and children aged 6 years and older with cystic fibrosis (CF) who have at least one F508del mutation in the CFTR gene Therapeutic value No estimate possible yet Total cost Registration phase Positive CHMP opinion Product Active substance Ivacaftor Domain Lung diseases Reason of inclusion Indication extension IND Main indication Cystic fibrosis Extended indication Kalydeco tablets are indicated in a combination regimen with ivacaftor /tezacaftor /elexacaftor tablets for the treatment of adults, and adolescents and children aged 6 years and older with cystic fibrosis (CF) who have at least one F508del mutation in the CFTR gene Current proprietary name Already available biosimilars / generics Proprietary name Kalydeco Manufacturer Vertex Portfolio holder Mechanism of action Protein chaperone Route of administration Oral Therapeutical formulation Tablet Budgetting framework Extramural (GVS) Centre of expertise Additional remarks Registration Registration route Centralised (EMA) Type of trajectory Normal trajectory Particularity Unknown ATMP No Submission date June 2021 Expected Registration January 2022 Orphan drug Yes Registration phase Positive CHMP opinion 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 < 107 Market share is generally not included unless otherwise stated. Maximum patient volume for sluice References Data CF registratie 2019 Additional remarks Er zullen 107 patiënten in aanmerking komen in deze leeftijdsgroep. 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