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 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 New medicine ATMP Unknown Submission date June 2019 Expected Registration June 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 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 < 100 Market share is generally not included unless otherwise stated. Maximum patient volume for sluice References NKR; Levis. Hematology Am Soc Hematol Educ Program. 2013; 2013: 220–226; kanker.nl. Additional remarks 674 diagnoses AML in 2015. Ongeveer 30% heeft een FLT3 mutatie. Bij ongeveer de helft van het aantal patiënten komt AML terug. 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