Elexacaftor / tezacaftor / ivacaftor Medicine 08 June 2021 Extended indication Kaftrio is indicated in a combination regimen with ivacaftor for the treatment of cystic fibrosis (CF) in patients aged 6 years and older who have at least one F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene Therapeutic value Possible added value Total cost Registration phase Positive CHMP opinion Product Active substance Elexacaftor / tezacaftor / ivacaftor Domain Lung diseases Reason of inclusion New medicine (specialité) Main indication Cystic fibrosis Extended indication Kaftrio is indicated in a combination regimen with ivacaftor for the treatment of cystic fibrosis (CF) in patients aged 6 years and older who have at least one F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene Current proprietary name Already available biosimilars / generics Proprietary name Kaftrio 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 December 2020 Expected Registration January 2022 Orphan drug Yes Registration phase Positive CHMP opinion Reimbursement Medicine sluice Additional remarks Positieve CHMP opinie ontvangen november 2021. Therapeutic value Current treatment options Therapeutic value Possible added value This assessment does not indicate any potential inclusion in the package. Substantiation Aangezien CF een progressieve ziekte betreft, heeft behandeling op jonge leeftijd extra meerwaarde gezien schade dan (gedeeltelijk) voorkomen kan worden. Duration of treatment Frequency of administration Dosage per administration References Additional remarks Expected patient volume per year Patient volume 175 Market share is generally not included unless otherwise stated. Maximum patient volume for sluice References Data CF registratie 2019 Additional remarks Alle kinderen met de F508del mutatie zullen Kaftrio voorgeschreven krijgen. Er zijn in deze leeftijdsgroep 121 kinderen homozygoot, 42 heterozygoot en 12 kinderen met residual function. Dit betekent dat er 175 kinderen in totaal in aanmerking komen. 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