Tezacaftor / ivacaftor Medicine 09 January 2018 Extended indication Cystische fibrose voor patiënten van 12 jaar of ouder met specifieke mutaties in het CFTR gen Therapeutic value No judgement Total cost € 84,660,000.00 Registration phase Registration application pending Product Active substance Tezacaftor / ivacaftor Domain Lung diseases Reason of inclusion Main indication Cystic fibrosis Extended indication Cystische fibrose voor patiënten van 12 jaar of ouder met specifieke mutaties in het CFTR gen Current proprietary name Already available biosimilars / generics Proprietary name Symkevi Manufacturer Vertex Portfolio holder Mechanism of action Unknown Route of administration Oral Therapeutical formulation Tablet Budgetting framework Extramural (GVS) Centre of expertise Additional remarks In patienten met F508del mutatie. Registration Registration route Centralised (EMA) Type of trajectory Normal trajectory Particularity New medicine ATMP Unknown Submission date September 2017 Expected Registration August 2018 Orphan drug Yes Registration phase Registration application pending Reimbursement Medicine sluice Additional remarks Therapeutic value Current treatment options Orkambi Therapeutic value No judgement This assessment does not indicate any potential inclusion in the package. Substantiation Duration of treatment Frequency of administration Dosage per administration 100 mg tezacaftro en 150 mg ivacaftor References Additional remarks Expected patient volume per year Patient volume < 498 Market share is generally not included unless otherwise stated. Maximum patient volume for sluice References Additional remarks 1500 personen met CF. Totale aantal patiënten dat jaarlijks voor lumacaftor/ivacaftor in aanmerking komt betreft 498 (BIA Orkambi), dit betreft dan ook het maximale aantal personen die voor tezacaftor / ivacaftor in aanmerking komen. Expected cost per patient per year Cost € 170,000.00 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 Medicijnkosten.nl Additional remarks Uitgaande van oorspronkelijke kosten Orkambi. Potential total cost per year Total cost € 84,660,000.00 Total cost for sluice Additional remarks Off label use Off label use Unknown Indications off label use References Additional remarks Indication extension Indication extensions Yes Indication extensions Cystische fibrose voor patiënten van 6 tot en met 11 jaar met specifieke mutaties in het CFTR gen (verwachting Q4 2019). References Fabrikant Additional remarks Other information Additional remarks