Lumacaftor / ivacaftor Medicine 07 December 2022 Extended indication Extension of indication to include treatment of cystic fibrosis for children aged 1 to less than 2 years old of age who are homozygous for the F508del mutation in the CFTR gene. Therapeutic value No estimate possible yet Total cost Registration phase Registered Product Active substance Lumacaftor / ivacaftor Domain Lung diseases Reason of inclusion Indication extension IND Main indication Cystic fibrosis Extended indication Extension of indication to include treatment of cystic fibrosis for children aged 1 to less than 2 years old of age who are homozygous for the F508del mutation in the CFTR gene. Current proprietary name Already available biosimilars / generics Proprietary name Orkambi Manufacturer Vertex Portfolio holder Mechanism of action Unknown 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 2022 Expected Registration July 2023 Orphan drug Yes Registration phase Registered Reimbursement Medicine sluice Additional remarks Positieve CHMP-opinie april 2023 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 25 Market share is generally not included unless otherwise stated. Maximum patient volume for sluice References Expertopinie (1); Additional remarks 50% van de patiënten met cystische fibrose (CF) is homozygoot. Er worden 50 kinderen met CF geboren per jaar. Dit betekent dat er ongeveer 25 nieuwe patiënten in aanmerking zullen komen per jaar (1). 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