Gilteritinib Medicine 12 June 2018 Extended indication Monotherapy for the treatment of adults with relapsed or refractory acute myeloid leukemia (AML) who are FLT3 mutation-positive. Therapeutic value No judgement Total cost € 1,110,000.00 Registration phase Registration application pending Product Active substance Gilteritinib Domain Oncology and Hematology Reason of inclusion New medicine (specialité) Main indication AML / MDS Extended indication Monotherapy for the treatment of adults with relapsed or refractory acute myeloid leukemia (AML) who are FLT3 mutation-positive. Current proprietary name Already available biosimilars / generics Proprietary name Manufacturer Astellas Portfolio holder Mechanism of action Tyrosine kinase inhibitor 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 Accelerated assessment Particularity Unknown ATMP Unknown Submission date April 2019 Expected Registration December 2019 Orphan drug Yes Registration phase Registration application pending 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 Median 4.1 month / months Frequency of administration 1 times a day Dosage per administration 120 mg References NCT02421939; Hovon; Perl et al. Lancet Oncol. 2017 Aug; 18(8): 1061–1075. Fase III studie ADMIRAL. Additional remarks Fase 1/2 studie: 100 (40%) van de 249 patienten lieten een respons zien, met 19 (8%) volledige remissies, 10 (4%) complete remissies met onvolledig herstel van de plaatjes, 46 (18%) complete remissies met onvolledig hematologisch herstel en 25 (10%) onvolledige remissies; gaat in toekomstige HOVON studie vergeleken worden met imatinib. Expected patient volume per year Patient volume 10 - 20 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. Nagel et al. Ann Hematol. 2017 Dec;96(12):1993-2003 Additional remarks Incidentie AML patienten in Nederland is 774 (IKNL, 2017) waarvan ongeveer 25% FLT3 gemuteerd (194) . Bij ongeveer de helft van het aantal patiënten komt AML terug (97). Quizartinib zal eveneens voor deze indicatie geregistreerd worden waardoor het verwachte marktaandeel uitkomt op 50% (49). In de praktijk zal het waarschijnlijk om beperkte hoeveelheid patiënten gaan die gebridged worden naar allo HCT, inschatting expert opinie: 10-20 patiënten. Expected cost per patient per year Cost € < 74,000.00 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 Potential total cost per year Total cost € 1,110,000.00 Total cost for sluice Additional remarks Off label use Off label use Unknown Indications off label use References Additional remarks Indication extension Indication extensions 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