Ivacaftor Medicine 13 December 2018 Extended indication Kalydeco tablets are indicated in a combination regimen with tezacaftor 100 mg/ivacaftor 150 mg tablets for the treatment of patients with cystic fibrosis (CF) aged 12 years and older who are homozygous for the F508del mutation or who are heterozygous for the F508del mutation and have one of the following mutations in the CFTR gene: P67L, R117C, L206W, R352Q, A455E, D579G, 711+3A→G, S945L, S977F, R1070W, D1152H, 2789+5G→A, 3272-26A→G, and 3849+10kbC→T. Therapeutic value No judgement Total cost € 34,720,000.00 Registration phase Registered and reimbursed Product Active substance Ivacaftor Domain Lung diseases Reason of inclusion New medicine (specialité) Main indication Cystic fibrosis Extended indication Kalydeco tablets are indicated in a combination regimen with tezacaftor 100 mg/ivacaftor 150 mg tablets for the treatment of patients with cystic fibrosis (CF) aged 12 years and older who are homozygous for the F508del mutation or who are heterozygous for the F508del mutation and have one of the following mutations in the CFTR gene: P67L, R117C, L206W, R352Q, A455E, D579G, 711+3A→G, S945L, S977F, R1070W, D1152H, 2789+5G→A, 3272-26A→G, and 3849+10kbC→T. Current proprietary name Already available biosimilars / generics Proprietary name Kalydeco 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 Unknown Particularity New therapeutical formulation ATMP Unknown Submission date Expected Registration September 2018 Orphan drug Yes Registration phase Registered and reimbursed Reimbursement Medicine sluice Additional remarks Avonddosering bij symkevi regime. Positieve CHMP-opinie in juli 2018. Geregistreerd in september 2018. Therapeutic value Current treatment options Therapeutic value No judgement This assessment does not indicate any potential inclusion in the package. Substantiation Duration of treatment Frequency of administration 2 times a day Dosage per administration 150 mg References SmPC Additional remarks De aanbevolen dosis voor volwassenen en adolescenten van 12 jaar en ouder is één tezacaftor 100 mg/ivacaftor 150 mg tablet ’s ochtends en één Kalydeco 150 mg tablet ’s avonds in te nemen, met een tussentijd van ongeveer 12 uur samen met vetbevattend voedsel (zie ‘Wijze van toediening’). Expected patient volume per year Patient volume < 140 Market share is generally not included unless otherwise stated. Maximum patient volume for sluice References Nederlandse Cystic Fibrosis Stichting. Additional remarks Symkevi (tezacaftor/ivacaftor) is geïndiceerd voor patiënten van 12 jaar en ouder, met een dubbele F508del-mutatie of één F508del plus één van de volgende 14 mutaties: P67L, R117C, L206W, R352Q, A455E, D579G, 711+3A→G, S945L, S977F, R1070W, D1152H, 2789+5G→A, 3272-26A→G, 3849+10kbC→T. In Nederland gaat het om een groep van 140 mensen, naast de groep met een dubbele F508del-mutatie die op dit moment Orkambi gebruikt. Een avonddosering van ivacaftor wordt hier aan toegevoegd. Expected cost per patient per year Cost € 248,000.00 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; SmPC Additional remarks Tweemaal daags 150 mg Kalydeco kost per jaar €248.099,63. Potential total cost per year Total cost € 34,720,000.00 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