Nilotinib Medicine 09 January 2018 Extended indication Paediatric patients with newly diagnosed Philadelphia chromosome positive chronic myelogenous leukaemia (CML) in the chronic phase and paediatric patients with chronic phase Philadelphia chromosome positive CML with resistance or intolerance to prior therapy including imatinib. Therapeutic value No judgement Total cost € 240,000.00 Registration phase Unknown Product Active substance Nilotinib Domain Oncology and Hematology Reason of inclusion Main indication Leukemia Extended indication Paediatric patients with newly diagnosed Philadelphia chromosome positive chronic myelogenous leukaemia (CML) in the chronic phase and paediatric patients with chronic phase Philadelphia chromosome positive CML with resistance or intolerance to prior therapy including imatinib. Current proprietary name Already available biosimilars / generics Proprietary name Tasigna Manufacturer Novartis Portfolio holder Mechanism of action Unknown Route of administration Oral Therapeutical formulation Capsule Budgetting framework Intermural (MSZ) Centre of expertise Additional remarks Registration Registration route Centralised (EMA) Type of trajectory Unknown Particularity Unknown ATMP Unknown Submission date Expected Registration September 2017 Orphan drug No Registration phase Unknown Reimbursement Medicine sluice Additional remarks Geregistreerd in november 2017. Therapeutic value Current treatment options Therapeutic value No judgement This assessment does not indicate any potential inclusion in the package. Substantiation Duration of treatment Average 1 year / years Frequency of administration 2 times a day Dosage per administration 400 mg References Additional remarks De dosering bij pediatrische patiënten is gepersonaliseerd en gebaseerd op de lichaamsoppervlakte (mg/m2). De aanbevolen dosis van nilotinib is 230 mg/m2 tweemaal daags, afgerond naar de dichtstbijzijnde 50 mg dosis (tot een maximale enkelvoudige dosis van 400 mg). Gemiddelde behandelduur bedraagt 52,19 weken. Expected patient volume per year Patient volume 5 - 10 Market share is generally not included unless otherwise stated. Maximum patient volume for sluice References NKR Additional remarks NKR: Chronische myeloproliferatieve aandoeningen bij kinderen: 56. Van alle myeloproliferatieve aandoeningen betreft 18 % CML. Maximale volume daarom geschat op: 10. Realistisch volume gescaht op: 5. Expected cost per patient per year Cost € 10,000.00 - 50,000.00 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 Fabrikant Additional remarks Huidige AIP is €1010,93 voor Tasigna 150 mg (5x8) en €1296,80 voor Tasigna 200 mg (5x8). Potential total cost per year Total cost € 240,000.00 Total cost for sluice Additional remarks Off label use Off label use Unknown Indications off label use References Additional remarks Indication extension Indication extensions Unknown Indication extensions References Additional remarks Other information Additional remarks