Tezacaftor / ivacaftor Medicine 09 January 2018 Extended indication Taaislijmziekte Therapeutic value No judgement Total cost € 187,000,000.00 Registration phase Unknown Product Active substance Tezacaftor / ivacaftor Domain Lung diseases Reason of inclusion Main indication Cystic fibrosis Extended indication Taaislijmziekte Current proprietary name Already available biosimilars / generics Proprietary name 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 Unknown ATMP Unknown Submission date September 2017 Expected Registration July 2018 Orphan drug Yes Registration phase Unknown 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 700 - 1,500 Market share is generally not included unless otherwise stated. Maximum patient volume for sluice References Additional remarks 1500 personen met CF, de helft daarvan wordt behandeld met Orkambi. 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 € 187,000,000.00 Total cost for sluice Additional remarks Off label use Off label use Unknown Indications off label use References Additional remarks Indication extension Indication extensions No Indication extensions References Additional remarks Other information Additional remarks