Ivacaftor / Tezacaftor / Elexacaftor Medicine 04 June 2024 Extended indication Extension of the indication for Kaftrio (ivacaftor/tezacaftor/elexacaftor) and Kalydeco (ivacaftor) in a combination regimen to include the treatment of patients with cystic fibrosis (CF) aged 2 years and older who do not carry any F508del mutations and have at least one ivacaftor/tezacaftor/elexacaftor-responsive mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene Therapeutic value Possible added 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 Extension of the indication for Kaftrio (ivacaftor/tezacaftor/elexacaftor) and Kalydeco (ivacaftor) in a combination regimen to include the treatment of patients with cystic fibrosis (CF) aged 2 years and older who do not carry any F508del mutations and have at least one ivacaftor/tezacaftor/elexacaftor-responsive 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 Intermural (MSZ) Centre of expertise Additional remarks Registration Registration route Centralised (EMA) Type of trajectory Normal trajectory Particularity Unknown ATMP No Submission date November 2023 Expected Registration January 2025 Orphan drug Yes Registration phase Registration application pending Reimbursement Medicine sluice Additional remarks Therapeutic value Current treatment options Therapeutic value Possible added value This assessment does not indicate any potential inclusion in the package. Substantiation Het veld geeft aan hier al een tijdje op te wachten. Het is nu geregistreerd vanaf 6 jaar en bij mensen met tenminste één dF508 mutatie. Deze uitbreiding betreft zowel leeftijd als mutaties. Dit geneesmiddel wordt een gamechanger genoemd. De uitbreiding zal leiden tot meer beschikbaarheid van dit geneesmiddel (momenteel is het beschikbaar voor circa 90% van de CF populatie, met deze uitbreiding komen de jongere kinderen erbij en waarschijnlijk naar schatting enkele procenten van de gehele CF populatie). De therapeutische meerwaarde is ondiscutabel. Er wordt verwacht dat er minder longschade op jonge leeftijd plaatsvindt en daardoor nog meer ziektewinst en minder opnames. Duration of treatment Frequency of administration Dosage per administration References NCT05274269; NCT05331183 Additional remarks Expected patient volume per year Patient volume Market share is generally not included unless otherwise stated. Maximum patient volume for sluice References 1. Expertopinie; Additional remarks Qua aantal patiënten zal het in Nederland meevallen aangezien al 90% in aanmerking komt voor dit geneesmiddel. Expected cost per patient per year Cost € < 99,363.00 This amount gives an indication of the total cost. It is the result of the average expected patient volume times the average cost per patient. both per year. References GIPdatabank Additional remarks De vergoeding per gebruiker van Kaftrio was €99.363 in 2023. Voor Kaftrio is een financieel arrangement afgesloten tot en met 31 december 2025. 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