Gilteritinib Medicine 12 June 2018 Extended indication Relapse/Refractory FLT3 gemuteerde AML Therapeutic value No judgement Total cost Registration phase Clinical trials Product Active substance Gilteritinib Domain Oncology and Hematology Reason of inclusion New medicine (specialité) Main indication Leukemia Extended indication Relapse/Refractory FLT3 gemuteerde AML Current proprietary name Already available biosimilars / generics Proprietary name Manufacturer Astellas Portfolio holder Mechanism of action Unknown Route of administration Oral Therapeutical formulation Tablet Budgetting framework Intermural (MSZ) Centre of expertise Additional remarks FLT3 inhibitor Registration Registration route Centralised (EMA) Type of trajectory Normal trajectory Particularity Unknown ATMP Unknown Submission date March 2019 Expected Registration April 2020 Orphan drug Yes Registration phase Clinical trials Reimbursement Medicine sluice Additional remarks Therapeutic value Current treatment options Chemotherapie Therapeutic value No judgement This assessment does not indicate any potential inclusion in the package. Substantiation Dit geneesmiddel zal waarschijnlijk ook de competitie aangaan met midostaurin en andere flt3 remmers Duration of treatment Frequency of administration 1 times a day Dosage per administration 120 mg References NCT02421939; Hovon; Perl et al. Blood 2016 128:1069. Additional remarks phase 1/2 study: 100 (40%) of 249 patients in the full analysis set achieved a response, with 19 (8%) achieving complete remission, ten (4%) complete remission with incomplete platelet recovery, 46 (18%) complete remission with incomplete haematological recovery, and 25 (10%) partial remission; gaat in toekomstige HOVON studie vergeleken worden met imatinib. Expected patient volume per year Patient volume 58 - 70 Market share is generally not included unless otherwise stated. Maximum patient volume for sluice References Fabrikant; NKR; Levis. Hematology Am Soc Hematol Educ Program. 2013; 2013: 220–226; kanker.nl. Additional remarks Incidentie van AML patiënten in Nederland is 774 (IKNL 2017) waarvan 30% FLT3 gemuteerd is (232) en waarvan 50%-60% relapse/refractory is (116-139). De fabrikant geeft aan dat het een marktaandeel van 50% verwacht (58-70). 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 Yes Indication extensions FLT3 gemuteerde AML: onderhoudstherapie (na inductie/consolidatie chemotherapie / na allogene stamceltransplantatie) en nieuw gediagnosticeerde patienten die geen standaard chemo kunnen ontvangen. References Fabrikant; NCT02927262; NCT02997202; NCT02752035. Additional remarks Other information Additional remarks