Elexacaftor / tezacaftor / ivacaftor Medicine 08 June 2021 Extended indication Uitbreiding van Kaftrio in combinatie met ivacaftor voor patienten met tenminste één F508del mutatie naar de leeftijd vanaf 6 jaar (uitbreiding voor 6 tot 11 jarigen) Therapeutic value No estimate possible yet Total cost Registration phase Clinical trials Product Active substance Elexacaftor / tezacaftor / ivacaftor Domain Lung diseases Reason of inclusion New medicine (specialité) Main indication Cystic fibrosis Extended indication Uitbreiding van Kaftrio in combinatie met ivacaftor voor patienten met tenminste één F508del mutatie naar de leeftijd vanaf 6 jaar (uitbreiding voor 6 tot 11 jarigen) 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 2021 Expected Registration October 2022 Orphan drug Yes Registration phase Clinical trials Reimbursement Medicine sluice Additional remarks Fabrikant verwacht indiening eind 2021. 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